Trial Outcomes & Findings for Efficacy, Safety, Tolerability, Immunogenicity and Pharmacokinetic Evaluation of HYQVIA in Pediatric PIDD Subjects (NCT NCT03277313)

Results Overview

The rate of ASBI was defined as the mean number of ASBI per participant-year based on the United States (U.S.) Food and drugs Administration (FDA) guidance for industry to support marketing of human immune globulin intravenous (IGIV) as replacement therapy for primary humoral immunodeficiency and the European Medicines Agency (EMA) guideline on the clinical investigation of human normal immunoglobulin for SC and /or intramuscular administration. ASBI included bacteremia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, and visceral abscess, diagnosed according to the Diagnostic Criteria for Acute Serious Bacterial Infections.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

44 participants

Primary outcome timeframe

From first dose of study drug up to end of Study Epoch 2 (up to approximately 37.2 months)

Results posted on

2023-10-23

Participant Flow

Participants took part in the study at 19 investigative sites in the United States from 25 September 2017 to 20 July 2022. Pediatric participants with a diagnosis of primary immunodeficiency diseases (PIDD) were enrolled in this study.

Pediatric participants who received intravenous(IV) or non-HYQVIA subcutaneous(SC) immunoglobulin therapy prior to enrollment received ramp-up doses of HYQVIA in Epoch 1 and at 3- or 4-week intervals in Epoch 2. Epoch 3 was planned for safety follow-up if needed, however no participants continued in Epoch 3. Data is reported only for Epoch 1 and 2.

Participant milestones

Participant milestones
Measure	Epoch 1 Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Epoch 1 (Six-week Ramp-up Period) STARTED	44	0
Epoch 1 (Six-week Ramp-up Period) COMPLETED	43	0
Epoch 1 (Six-week Ramp-up Period) NOT COMPLETED	1	0
Epoch 2 (36 Months After Epoch 1) STARTED	0	43
Epoch 2 (36 Months After Epoch 1) COMPLETED	0	34
Epoch 2 (36 Months After Epoch 1) NOT COMPLETED	0	9

Reasons for withdrawal

Reasons for withdrawal
Measure	Epoch 1 Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Epoch 1 (Six-week Ramp-up Period) Adverse Event	1	0
Epoch 2 (36 Months After Epoch 1) Adverse Event	0	1
Epoch 2 (36 Months After Epoch 1) Physician Decision	0	1
Epoch 2 (36 Months After Epoch 1) Withdrawal by Subject	0	6
Epoch 2 (36 Months After Epoch 1) Reason not Specified	0	1

Baseline Characteristics

Efficacy, Safety, Tolerability, Immunogenicity and Pharmacokinetic Evaluation of HYQVIA in Pediatric PIDD Subjects

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Epoch 1 n=44 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks.
Age, Continuous	9.0 years STANDARD_DEVIATION 3.63 • n=5 Participants
Sex: Female, Male Female	18 Participants n=5 Participants
Sex: Female, Male Male	26 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	39 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants
Race (NIH/OMB) White	40 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Region of Enrollment United States	44 Participants n=5 Participants
Height	133.60 cm STANDARD_DEVIATION 24.024 • n=5 Participants
Weight	37.78 kg STANDARD_DEVIATION 19.858 • n=5 Participants

PRIMARY outcome

Timeframe: From first dose of study drug up to end of Study Epoch 2 (up to approximately 37.2 months)

Population: FAS included all participants who provided informed consent, and met enrollment eligibility.

The rate of ASBI was defined as the mean number of ASBI per participant-year based on the United States (U.S.) Food and drugs Administration (FDA) guidance for industry to support marketing of human immune globulin intravenous (IGIV) as replacement therapy for primary humoral immunodeficiency and the European Medicines Agency (EMA) guideline on the clinical investigation of human normal immunoglobulin for SC and /or intramuscular administration. ASBI included bacteremia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, and visceral abscess, diagnosed according to the Diagnostic Criteria for Acute Serious Bacterial Infections.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=44 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Rate Represented as Mean Number of Acute Serious Bacterial Infections (ASBI) Per Participant-year	0.04 ASBI per participant-year Standard Error 0.027	—

SECONDARY outcome

Timeframe: From first dose of study drug up to end of Study Epoch 2 (up to approximately 37.2 months)

Population: FAS included all participants who provided informed consent, and met enrollment eligibility.

The rate of all infections was defined as the mean number of all infections per participant-year. Number of all infections was calculated as number of infections per participant-year.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=44 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Rate Represented as Mean Number of All Infections Per Participant-year	3.12 infections per participant-year Standard Error 0.450	—

SECONDARY outcome

Timeframe: Study Epoch 2, Year 1: Months 0, 6, and 12; Year 2: Months 18, 24 and 36

Population: FAS=all participants who provided informed consent, and met enrollment eligibility. Only FAS participants in Epoch 2 were analyzed for this outcome measure. Overall number analyzed=number of participants with data available for analyses. Number analyzed=number of participants with data available for analysis at specified timepoint.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=40 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses IgG Total: Month 0	10.381 grams per liter (g/L) Standard Deviation 2.9191	—
Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses IgG Total: Month 6	9.199 grams per liter (g/L) Standard Deviation 1.9577	—
Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses IgG Total: Month 12	9.214 grams per liter (g/L) Standard Deviation 1.9800	—
Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses IgG Total: Month 18	13.80 grams per liter (g/L) Standard Deviation NA Standard deviation (SD) was not estimable for 1 participant.	—
Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses IgG Total: Month 24	10.80 grams per liter (g/L) Standard Deviation NA SD was not estimable for 1 participant.	—
Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses IgG Total: Month 36	13.50 grams per liter (g/L) Standard Deviation NA SD was not estimable for 1 participant.	—
Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses IgG Subclass 1: Month 0	5.888 grams per liter (g/L) Standard Deviation 2.7193	—
Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses IgG Subclass 1: Month 6	5.317 grams per liter (g/L) Standard Deviation 1.4887	—
Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses IgG Subclass 1: Month 12	5.284 grams per liter (g/L) Standard Deviation 1.2103	—
Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses IgG Subclass 1: Month 18	7.590 grams per liter (g/L) Standard Deviation NA SD was not estimable for 1 participant.	—
Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses IgG Subclass 1: Month 24	7.710 grams per liter (g/L) Standard Deviation NA SD was not estimable for 1 participant.	—
Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses IgG Subclass 2: Month 0	3.311 grams per liter (g/L) Standard Deviation 0.6833	—
Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses IgG Subclass 2: Month 6	3.156 grams per liter (g/L) Standard Deviation 0.6694	—
Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses IgG Subclass 2: Month 12	3.106 grams per liter (g/L) Standard Deviation 0.5839	—
Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses IgG Subclass 2: Month 18	3.480 grams per liter (g/L) Standard Deviation NA SD was not estimable for 1 participant.	—
Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses IgG Subclass 2: Month 24	3.510 grams per liter (g/L) Standard Deviation NA SD was not estimable for 1 participant.	—
Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses IgG Subclass 3: Month 0	0.534 grams per liter (g/L) Standard Deviation 0.2838	—
Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses IgG Subclass 3: Month 6	0.508 grams per liter (g/L) Standard Deviation 0.2545	—
Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses IgG Subclass 3: Month 12	0.507 grams per liter (g/L) Standard Deviation 0.2799	—
Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses IgG Subclass 3: Month 18	0.360 grams per liter (g/L) Standard Deviation NA SD was not estimable for 1 participant.	—
Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses IgG Subclass 3: Month 24	0.430 grams per liter (g/L) Standard Deviation NA SD was not estimable for 1 participant.	—
Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses IgG Subclass 4: Month 0	0.2998 grams per liter (g/L) Standard Deviation 0.26802	—
Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses IgG Subclass 4: Month 6	0.3161 grams per liter (g/L) Standard Deviation 0.32899	—
Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses IgG Subclass 4: Month 12	0.3118 grams per liter (g/L) Standard Deviation 0.32790	—
Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses IgG Subclass 4: Month 18	0.9380 grams per liter (g/L) Standard Deviation NA SD was not estimable for 1 participant.	—
Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses IgG Subclass 4: Month 24	1.4560 grams per liter (g/L) Standard Deviation NA SD was not estimable for 1 participant.	—

SECONDARY outcome

Timeframe: Study Epoch 2, Year 2: Months 6, 24, and 36

Population: FAS=all participants who provided informed consent, and met enrollment eligibility. Only FAS participants in Epoch 2 were analyzed for this outcome measure. Overall number analyzed=number of participants with data available for analyses. Number analyzed=number of participants with data available for analysis at specified timepoint.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=38 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Epoch 2: Trough Levels of Specific Antibodies to Clinically Relevant Pathogens Categorized as Clostridium Tetani Toxoid and Hepatitis B Virus Clostridium Tetani Toxoid Antibody: Month 6	1.685 international unit per milliliter(IU/mL) Standard Deviation 0.6793	—
Epoch 2: Trough Levels of Specific Antibodies to Clinically Relevant Pathogens Categorized as Clostridium Tetani Toxoid and Hepatitis B Virus Clostridium Tetani Toxoid Antibody: Month 24	2.450 international unit per milliliter(IU/mL) Standard Deviation NA SD was not estimable for one participant.	—
Epoch 2: Trough Levels of Specific Antibodies to Clinically Relevant Pathogens Categorized as Clostridium Tetani Toxoid and Hepatitis B Virus Clostridium Tetani Toxoid Antibody: Completion/ Termination (Month 36)	1.598 international unit per milliliter(IU/mL) Standard Deviation 0.5889	—
Epoch 2: Trough Levels of Specific Antibodies to Clinically Relevant Pathogens Categorized as Clostridium Tetani Toxoid and Hepatitis B Virus Hepatitis B Virus Antibody: Month 6	199.6 international unit per milliliter(IU/mL) Standard Deviation 122.97	—
Epoch 2: Trough Levels of Specific Antibodies to Clinically Relevant Pathogens Categorized as Clostridium Tetani Toxoid and Hepatitis B Virus Hepatitis B Virus Antibody: Month 24	146.0 international unit per milliliter(IU/mL) Standard Deviation NA SD was not estimable for 1 participant.	—
Epoch 2: Trough Levels of Specific Antibodies to Clinically Relevant Pathogens Categorized as Clostridium Tetani Toxoid and Hepatitis B Virus Hepatitis B Virus Antibody: Completion/ Termination (Month 36)	256.7 international unit per milliliter(IU/mL) Standard Deviation 219.53	—

SECONDARY outcome

Timeframe: Study Epoch 2, Year 2: Months 6, 24, and 36

Population: FAS=all participants who provided informed consent, and met enrollment eligibility. Only FAS participants in Epoch 2 were analyzed for this outcome measure. Overall number analyzed=number of participants with data available for analyses. Number analyzed=number of participants with data available for analysis at specified timepoint.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=39 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Epoch 2: Trough Levels of Specific Antibodies to Clinically Relevant Pathogen Haemophilus Influenzae B Month 6	1.839 milligrams per liter (mg/L) Standard Deviation 1.5222	—
Epoch 2: Trough Levels of Specific Antibodies to Clinically Relevant Pathogen Haemophilus Influenzae B Month 24	1.810 milligrams per liter (mg/L) Standard Deviation NA SD was not estimable for 1 participant.	—
Epoch 2: Trough Levels of Specific Antibodies to Clinically Relevant Pathogen Haemophilus Influenzae B Completion/Termination (Month 36)	1.678 milligrams per liter (mg/L) Standard Deviation 0.8009	—

SECONDARY outcome

Timeframe: Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion

Population: Pharmacokinetic analysis set (PKAS) included all participants in the Safety Analysis Set who have at least 1 evaluable post-dose concentration data for PK assessments without major protocol deviations or events affecting the PK results. Overall number analyzed is the number of participants with data available for analyses. As pre-specified in statistical analysis plan (SAP), this outcome measure was planned only for Epoch 2.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=37 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Epoch 2: Area Under the Curve Normalized for Week (AUCweek)	74.57 gramsday per liter (gday/L) Geometric Coefficient of Variation 19.4	—

SECONDARY outcome

Timeframe: Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion

Population: PKAS included all participants in the Safety Analysis Set who have at least 1 evaluable post-dose concentration data for PK assessments without major protocol deviations or events affecting the PK results. Overall number analyzed is the number of participants with data available for analyses. As pre-specified in SAP, this outcome measure was planned only for Epoch 2.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=37 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Epoch 2: Area Under the Curve Over the Infusion Interval (AUCTau)	288.8 g*day/L Geometric Coefficient of Variation 21.7	—

SECONDARY outcome

Timeframe: Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion

Population: PKAS included all participants in the Safety Analysis Set who have at least 1 evaluable post-dose concentration data for PK assessments without major protocol deviations or events affecting the PK results. Overall number analyzed is the number of participants with data available for analyses. As pre-specified in SAP, this outcome measure was planned only for Epoch 2.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=37 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Epoch 2: Apparent Clearance (CL/F)	56.45 milliliters per day (mL/day) Geometric Coefficient of Variation 61.4	—

SECONDARY outcome

Timeframe: Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion

Population: PKAS included all participants in the Safety Analysis Set who have at least 1 evaluable post-dose concentration data for PK assessments without major protocol deviations or events affecting the PK results. Overall number analyzed is the number of participants with data available for analyses. As pre-specified in SAP, this outcome measure was planned only for Epoch 2.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=38 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Epoch 2: Maximum Concentration (Cmax)	12.94 g/L Geometric Coefficient of Variation 17.4	—

SECONDARY outcome

Timeframe: Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion

Population: PKAS included all participants in the Safety Analysis Set who have at least 1 evaluable post-dose concentration data for PK assessments without major protocol deviations or events affecting the PK results. Overall number analyzed is the number of participants with data available for analyses. As pre-specified in SAP, this outcome measure was planned only for Epoch 2.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=38 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Epoch 2: Minimum Concentration (Cmin)	8.571 g/L Geometric Coefficient of Variation 25.6	—

SECONDARY outcome

Timeframe: Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion

Population: PKAS included all participants in the Safety Analysis Set who have at least 1 evaluable post-dose concentration data for PK assessments without major protocol deviations or events affecting the PK results. Overall number analyzed is the number of participants with data available for analyses. As pre-specified in SAP, this outcome measure was planned only for Epoch 2.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=38 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Epoch 2: Time to Maximum Concentration (Tmax)	4.20 days Interval 0.0 to 26.9	—

SECONDARY outcome

Timeframe: Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion

Population: PKAS included all participants in the Safety Analysis Set who have at least 1 evaluable post-dose concentration data for PK assessments without major protocol deviations or events affecting the PK results. Overall number analyzed is the number of participants with data available for analyses. As pre-specified in SAP, this outcome measure was planned only for Epoch 2.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=19 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Epoch 2: Terminal Half-life (T 1/2)	44.98 days Geometric Coefficient of Variation 45.9	—

SECONDARY outcome

Timeframe: From first dose of study drug up to EOS (up to 4 years 9 months)

Population: Safety Analysis Set (SAS) included all participants who received at least one dose of HyQvia.

An SAE is defined as an untoward medical occurrence that at any dose is fatal, life-threatening, requires inpatient hospitalization or results in prolongation of an existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, is a medically important event. Number of participants who experienced SAEs, related or not related, was reported.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=44 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 n=43 Participants Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Number of Participants With Serious Adverse Events (SAEs) Excluding Infections, Related and Not Related SAE: Related	0 Participants	0 Participants
Number of Participants With Serious Adverse Events (SAEs) Excluding Infections, Related and Not Related SAE: Not Related	0 Participants	1 Participants

SECONDARY outcome

Timeframe: From first dose of study drug up to EOS (up to 4 years 9 months)

Population: SAS included all participants who received at least one dose of HyQvia.

Rate of SAEs per infusion was calculated as number of serious adverse events/total number of infusions administered to participants in the analysis set. Rate of SAEs per infusion (excluding infections) was reported.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=126 Infusions Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 n=633 Infusions Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Rate of SAEs Excluding Infections, Related and Not Related, Per Infusion SAE: Related	0.0 SAEs per infusion	0.0 SAEs per infusion
Rate of SAEs Excluding Infections, Related and Not Related, Per Infusion SAE: Not Related	0.0 SAEs per infusion	0.002 SAEs per infusion

SECONDARY outcome

Timeframe: From first dose of study drug up to EOS (up to 4 years 9 months)

Population: SAS included all participants who received at least one dose of HyQvia.

An Adverse Event (AE) is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. A TEAE is defined as an adverse event with an onset that occurs after receiving study drug. Number of participants who experienced TEAEs, related or not related, was reported.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=44 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 n=43 Participants Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Number of Participants With All Treatment Emergent Adverse Events (TEAEs) Excluding Infections, Related and Not Related TEAE: Related	25 Participants	31 Participants
Number of Participants With All Treatment Emergent Adverse Events (TEAEs) Excluding Infections, Related and Not Related TEAE: Not Related	16 Participants	36 Participants

SECONDARY outcome

Timeframe: From first dose of study drug up to EOS (up to 4 years 9 months)

Population: SAS included all participants who received at least one dose of HyQvia.

Rate of all TEAEs per infusion was calculated as number of any adverse reaction events/total number of infusions administered to participants in the analysis set. Rate of any adverse reactions per infusion (excluding infections) was reported.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=126 Infusion Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 n=633 Infusion Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Rate of All TEAEs Excluding Infections, Related and Not Related, Per Infusion TEAE: Related	0.675 adverse reaction event per infusion	0.397 adverse reaction event per infusion
Rate of All TEAEs Excluding Infections, Related and Not Related, Per Infusion TEAE: Not Related	0.270 adverse reaction event per infusion	0.262 adverse reaction event per infusion

SECONDARY outcome

Timeframe: From first dose of study drug up to EOS (up to 4 years 9 months)

Population: SAS included all participants who received at least one dose of HyQvia.

An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. A TEAE is defined as an adverse event with an onset that occurs after receiving study drug. Number of participants who experienced local TEAEs, related or not related, was reported.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=44 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 n=43 Participants Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Number of Participants With Local TEAEs Excluding Infections, Related and Not Related Local TEAE: Related	22 Participants	28 Participants
Number of Participants With Local TEAEs Excluding Infections, Related and Not Related Local TEAE: Not Related	2 Participants	6 Participants

SECONDARY outcome

Timeframe: From first dose of study drug up to EOS (up to 4 years 9 months)

Population: SAS included all participants who received at least one dose of HyQvia.

Rate of local TEAEs per infusion was calculated as number of local TEAEs/total number of infusions administered to participants in the analysis set. Rate of local TEAEs per infusion (excluding infections) was reported.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=126 Infusion Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 n=633 Infusion Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Rate of Local TEAEs Excluding Infections, Related and Not Related, Per Infusion Local TEAE: Related	0.460 local TEAEs per infusion	0.212 local TEAEs per infusion
Rate of Local TEAEs Excluding Infections, Related and Not Related, Per Infusion Local TEAE: Not Related	0.016 local TEAEs per infusion	0.013 local TEAEs per infusion

SECONDARY outcome

Timeframe: From first dose of study drug up to EOS (up to 4 years 9 months)

Population: SAS included all participants who received at least one dose of HyQvia.

An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. A TEAE is defined as an adverse event with an onset that occurs after receiving study drug. Number of participants who experienced systemic TEAEs, related or not related, was reported.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=44 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 n=43 Participants Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Number of Participants With Systemic TEAEs Excluding Infections, Related and Not Related Systemic TEAE: Related	12 Participants	20 Participants
Number of Participants With Systemic TEAEs Excluding Infections, Related and Not Related Systemic TEAE: Not Related	16 Participants	33 Participants

SECONDARY outcome

Timeframe: From first dose of study drug up to EOS (up to 4 years 9 months)

Population: SAS included all participants who received at least one dose of HyQvia.

Rate of systemic TEAEs per infusion was calculated as number of systemic TEAEs/total number of infusions administered to participants in the analysis set. Rate of systemic TEAEs per infusion (excluding infections) was reported.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=126 Infusion Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 n=633 Infusion Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Rate of Systemic TEAEs Excluding Infections, Related and Not Related, Per Infusion Systemic TEAE: Related	0.214 systemic TEAEs per infusion	0.185 systemic TEAEs per infusion
Rate of Systemic TEAEs Excluding Infections, Related and Not Related, Per Infusion Systemic TEAE: Not Related	0.254 systemic TEAEs per infusion	0.250 systemic TEAEs per infusion

SECONDARY outcome

Timeframe: From beginning of infusion up to 72 hours post infusion

Population: SAS included all participants who received at least one dose of HyQvia.

An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. TEAE is defined as an adverse event with an onset that occurs after receiving study drug. Number of participants who experienced all temporally associated TEAEs, related or not related, was reported.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=44 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 n=43 Participants Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Number of Participants With All Temporally Associated TEAEs Excluding Infections	27 Participants	35 Participants

SECONDARY outcome

Timeframe: From beginning of infusion up to 72 hours post infusion

Population: SAS included all participants who received at least one dose of HyQvia.

Rate of all temporally associated TEAEs per infusion was calculated as number of all temporally associated TEAEs/total number of infusions administered to participants in the analysis set. Rate of all temporally associated TEAEs per infusion (excluding infections) was reported.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=126 Infusion Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 n=633 Infusion Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Rate of All Temporally Associated TEAEs Excluding Infections Per Infusion	0.722 temporally associated TEAEs per infusion	0.460 temporally associated TEAEs per infusion

SECONDARY outcome

Timeframe: From beginning of infusion up to 72 hours post infusion

Population: SAS included all participants who received at least one dose of HyQvia.

An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. A TEAE is defined as an adverse event with an onset that occurs after receiving study drug. Number of participants with any related (causally) and/or temporally associated TEAEs (excluding infections) was reported. Temporally-associated TEAEs were defined as TEAEs which begin during infusion of IP or within 72 hours following the end of IP infusion.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=44 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 n=43 Participants Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Number of Participants With All Related (Causally) and/or Temporally Associated TEAEs Excluding Infections	27 Participants	35 Participants

SECONDARY outcome

Timeframe: From beginning of infusion up to 72 hours post infusion

Population: SAS included all participants who received at least one dose of HyQvia.

Rate of any related (causally) and/or temporally associated TEAEs per infusion was calculated as number of related and/or temporally associated adverse events/ total number of infusions administered to participants in the analysis set. TEAEs recorded in the study database as "possibly related" or "probably related" to HYQVIA are considered HYQVIA-related adverse events. Temporally-associated TEAEs were defined as TEAEs which begin during infusion of IP or within 72 hours following the end of IP infusion. Rate of any related (causally) and/or temporally associated TEAEs per infusion (excluding infections) was reported.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=126 Infusion Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 n=633 Infusion Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Rate of Participants With All Related (Causally) and/or Temporally Associated TEAEs Excluding Infections Per Infusion	0.762 related temporally TEAEs per infusion	0.482 related temporally TEAEs per infusion

SECONDARY outcome

Timeframe: From first dose of study drug up to EOS (up to 4 years 9 months)

Population: SAS included all participants who received at least one dose of HyQvia.

An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. A TEAE is defined as an adverse event with an onset that occurs after receiving study drug. Percentages are rounded off to whole number at the nearest decimal.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=44 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 n=43 Participants Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Percentage of Participants With Any TEAEs Excluding Infections	65.9 percentage of participants	93.0 percentage of participants

SECONDARY outcome

Timeframe: From first dose of study drug up to EOS (up to 4 years 9 months)

Population: FAS included all participants who provided informed consent, and met enrollment eligibility.

Number of participants who developed positive titer (rHuPH20 titer ≥160) of binding antibodies to rHuPH20 was reported. Neutralizing antibodies were only tested if the participant had a titer of ≥160 of binding antibodies. Participants with multiple assessments of titer of ≥160 of binding antibodies are counted only once.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=44 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Number of Participants Who Developed Positive Titer (≥160) of Binding or Neutralizing Antibodies to rHuPH20	1 Participants	—

SECONDARY outcome

Timeframe: From first dose of study drug up to EOS (up to 4 years 9 months)

Population: FAS included all participants who provided informed consent, and met enrollment eligibility.

Percentage of participants who developed positive titer (rHuPH20 titer ≥160) of binding antibodies to rHuPH20 was reported. Neutralizing antibodies were only tested if the participant had a titer of ≥160 of binding antibodies. Participants with multiple assessments of titer of ≥160 of binding antibodies are counted only once. Percentages are rounded off to whole number at the nearest decimal.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=44 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Percentage of Participants Who Developed Positive Titer (≥160) of Binding or Neutralizing Antibodies to rHuPH20	2.3 percentage of participants	—

SECONDARY outcome

Timeframe: Study Epoch 2: Up to 36 months

Population: SAS included all participants who received at least one dose of HyQvia. As pre-specified in SAP, this outcome measure was planned only for Epoch 2.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=43 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Epoch 2: Number of Infusions Per Month	1.10 infusions per month Interval 1.0 to 1.5	—

SECONDARY outcome

Timeframe: Study Epoch 2: Up to 36 months

Population: SAS included all participants who received at least one dose of HyQvia. As pre-specified in SAP, this outcome measure was planned only for Epoch 2.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=633 Infusion Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Epoch 2: Number of Infusion Sites (Needle Sticks) Per Infusion	1.83 infusion sites per infusion Standard Deviation 0.366	—

SECONDARY outcome

Timeframe: Study Epoch 2: Up to 36 months

Population: SAS included all participants who received at least one dose of HyQvia. As pre-specified in SAP, this outcome measure was planned only for Epoch 2.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=43 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Epoch 2: Number of Infusion Sites (Needle Sticks) Per Month	2.17 infusion sites per month Interval 1.1 to 2.9	—

SECONDARY outcome

Timeframe: Study Epoch 2: Up to 36 months

Population: SAS included all participants who received at least one dose of HyQvia. As pre-specified in SAP, this outcome measure was planned only for Epoch 2.

Duration of infusion is calculated as (stop time of infusion - start time of infusion) (in Epoch 2). Duration of infusion is the time from the start of rHuPH20 infusion until the stop time of immunoglobulin infusion.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=632 Infusion Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Epoch 2: Duration of Infusion	85.0 minutes (mins) Interval 45.0 to 215.0	—

SECONDARY outcome

Timeframe: Study Epoch 2: Up to 36 months

Population: SAS included all participants who received at least one dose of HyQvia. As pre-specified in SAP, this outcome measure was planned only for Epoch 2.

HYQVIA treatment infusions in Epoch 2 were given at a rate of 10 milliliters per hour per site (mL/h/site) to 160 ml/h/site (body weight \[BW\] of \<40 kg) and 10 mL/h/site to 300 mL/h/site (BW of ≥40 kg).

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=633 Infusion Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Epoch 2: Maximum Infusion Rate Per Site	173.5 milliliters per hour per site(mL/h/site) Standard Deviation 81.32	—

SECONDARY outcome

Timeframe: Study Epoch 2: Up to 36 months

Population: SAS included all participants who received at least one dose of HyQvia. As pre-specified in SAP, this outcome measure was planned only for Epoch 2.

Infusion volume per site is calculated as (actual IgG volume (mL) / total number of infusion sites used).

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=633 Infusion Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Epoch 2: Infusion Volume Per Site	101.3 mL/site Standard Deviation 51.69	—

SECONDARY outcome

Timeframe: Study Epoch 2: Up to 36 months

Population: SAS included all participants who received at least one dose of HyQvia. As pre-specified in SAP, this outcome measure was planned only for Epoch 2.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=633 Infusion Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Epoch 2: Infusions Which Were Discontinued, Slowed, or Interrupted Due to an AE Infusions That Were Discontinued	0 infusions	—
Epoch 2: Infusions Which Were Discontinued, Slowed, or Interrupted Due to an AE Infusions That Were Slowed	0 infusions	—
Epoch 2: Infusions Which Were Discontinued, Slowed, or Interrupted Due to an AE Infusions That Were Interrupted	17 infusions	—

SECONDARY outcome

Timeframe: Study Epoch 2: Up to 36 months

Population: SAS included all participants who received at least one dose of HyQvia. As pre-specified in SAP, this outcome measure was planned only for Epoch 2.

Percentages are rounded off to whole number at the nearest decimal.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=633 Infusion Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Epoch 2: Percentage of Infusions Which Were Discontinued, Slowed, or Interrupted Due to an AE Infusions That Were Discontinued	0 percentage of infusions	—
Epoch 2: Percentage of Infusions Which Were Discontinued, Slowed, or Interrupted Due to an AE Infusions That Were Slowed	0 percentage of infusions	—
Epoch 2: Percentage of Infusions Which Were Discontinued, Slowed, or Interrupted Due to an AE Infusions That Were Interrupted	2.69 percentage of infusions	—

SECONDARY outcome

Timeframe: Epoch 1 (up to 6 weeks)

Population: SAS included all participants who received at least one dose of HyQvia. Overall number analyzed is the number of participants with data available for analyses. As pre-specified in SAP, this outcome measure was planned only for Epoch 1.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=43 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Epoch 1: Number of Weeks to Reach Final Dose Interval	6.14 weeks Interval 3.0 to 6.6	—

SECONDARY outcome

Timeframe: Study Epoch 2: Up to 36 months

Population: SAS included all participants who received at least one dose of HyQvia. As pre-specified in SAP, this outcome measure was planned only for Epoch 2.

Percentages are rounded off to whole number at the nearest decimal. Percentages may sum up to more than 100% as assigned treatment interval could be changed during the study.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=43 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Epoch 2: Percentage of Participants Who Achieved a Treatment Interval of Three or Four Weeks in Epoch 2 Every 3 Weeks	18.6 percentage of participants	—
Epoch 2: Percentage of Participants Who Achieved a Treatment Interval of Three or Four Weeks in Epoch 2 Every 4 Weeks	83.7 percentage of participants	—

SECONDARY outcome

Timeframe: Study Epoch 2: Up to 12 months

Population: SAS included all participants who received at least one dose of HyQvia. As pre-specified in SAP, this outcome measure was planned only for Epoch 2.

Percentages are rounded off to whole number at the nearest decimal.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=43 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Epoch 2: Percentage of Participants Who Maintained a Treatment Interval of Three or Four Weeks in Epoch 2 for 12 Months	74.4 percentage of participants	—

SECONDARY outcome

Timeframe: Epoch 1: Baseline (First Infusion); Study Epoch 2: Up to Month 36

Population: SAS included all participants who received at least one dose of HyQvia. Overall number analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analysis at a specified timepoint.

Peds-QL=generic questionnaire developed and validated to measure HRQoL among pediatric population. It included assessment of 4 dimensions: physical functioning (8 items), emotional functioning (8 items), social functioning (8 items), and school functioning (Age groups: Toddler (2-4years),Young child (5-7years),Child (8-12years), and Teens (13-\<16years). Depending on the participants age, questionnaire may be completed by participant or parent/caregiver as appropriate. Items were scored on a 5-point Likert scale: 0=never a problem; 1=almost never a problem; 2=sometimes a problem; 3=often a problem; and 4=almost always a problem). All Scores were transformed on a total scale from 0-100 where, 0=100, 1=75, 2=50, 3=25, and 4=0. Higher scores indicating better health status. End of Epoch 2 summarizes all participant's data for their last epoch 2 visit (so not including the participant that discontinued in epoch 1).

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=18 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Health-related Quality of Life (HRQoL): Change From Baseline in Pediatric Quality of Life Inventory (Peds-QL) Score Baseline: 2-4 years	72.67 score on a scale Standard Deviation 12.969	—
Health-related Quality of Life (HRQoL): Change From Baseline in Pediatric Quality of Life Inventory (Peds-QL) Score Baseline: 5-7 years	77.29 score on a scale Standard Deviation 18.908	—
Health-related Quality of Life (HRQoL): Change From Baseline in Pediatric Quality of Life Inventory (Peds-QL) Score Baseline: 8-12 years	75.42 score on a scale Standard Deviation 17.227	—
Health-related Quality of Life (HRQoL): Change From Baseline in Pediatric Quality of Life Inventory (Peds-QL) Score Baseline: 13-<16 years	76.09 score on a scale Standard Deviation 7.894	—
Health-related Quality of Life (HRQoL): Change From Baseline in Pediatric Quality of Life Inventory (Peds-QL) Score Change From Baseline at Epoch 2 Month 12: 2-4 years	4.57 score on a scale Standard Deviation 17.134	—
Health-related Quality of Life (HRQoL): Change From Baseline in Pediatric Quality of Life Inventory (Peds-QL) Score Change From Baseline at Epoch 2 Month 12: 5-7 years	-3.74 score on a scale Standard Deviation 15.170	—
Health-related Quality of Life (HRQoL): Change From Baseline in Pediatric Quality of Life Inventory (Peds-QL) Score Change From Baseline at Epoch 2 Month 12: 8-12 years	0.84 score on a scale Standard Deviation 21.050	—
Health-related Quality of Life (HRQoL): Change From Baseline in Pediatric Quality of Life Inventory (Peds-QL) Score Change From Baseline at Epoch 2 Month 12: 13-<16 years	-6.09 score on a scale Standard Deviation 8.750	—
Health-related Quality of Life (HRQoL): Change From Baseline in Pediatric Quality of Life Inventory (Peds-QL) Score Change From Baseline at Completion/ termination (Month 36): 2-4 years	8.73 score on a scale Standard Deviation 18.799	—
Health-related Quality of Life (HRQoL): Change From Baseline in Pediatric Quality of Life Inventory (Peds-QL) Score Change From Baseline at Completion/ termination (Month 36): 5-7 years	-26.09 score on a scale Standard Deviation NA SD was not estimable for 1 participant.	—
Health-related Quality of Life (HRQoL): Change From Baseline in Pediatric Quality of Life Inventory (Peds-QL) Score Change From Baseline at Completion/ termination (Month 36): 8-12 years	-5.16 score on a scale Standard Deviation 19.735	—
Health-related Quality of Life (HRQoL): Change From Baseline in Pediatric Quality of Life Inventory (Peds-QL) Score Change From Baseline at Completion/ termination (Month 36): 13-<16 years	3.62 score on a scale Standard Deviation 5.471	—
Health-related Quality of Life (HRQoL): Change From Baseline in Pediatric Quality of Life Inventory (Peds-QL) Score Change From Baseline at End of Epoch 2: 2-4 years	8.16 score on a scale Standard Deviation 17.730	—
Health-related Quality of Life (HRQoL): Change From Baseline in Pediatric Quality of Life Inventory (Peds-QL) Score Change From Baseline at End of Epoch 2: 5-7 years	-4.59 score on a scale Standard Deviation 16.266	—
Health-related Quality of Life (HRQoL): Change From Baseline in Pediatric Quality of Life Inventory (Peds-QL) Score Change From Baseline at End of Epoch 2: 8-12 years	-1.98 score on a scale Standard Deviation 14.027	—
Health-related Quality of Life (HRQoL): Change From Baseline in Pediatric Quality of Life Inventory (Peds-QL) Score Change From Baseline at End of Epoch 2: 13-<16 years	-2.45 score on a scale Standard Deviation 8.807	—

SECONDARY outcome

Timeframe: Epoch 1: Baseline (First Infusion); Study Epoch 2: Up to Month 36

Population: SAS included all participants who received at least one dose of HyQvia. Overall number analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analysis at a specified timepoint.

The EQ-5D is a validated, self-administered assessment of overall health consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). Participants were asked to describe their health state that day by choosing 1 of 3 responses that reflect the levels of severity for each of the five dimensions: no problems, some or moderate problems, or extreme problems. The domain scores are calculated with higher scores indicating worsening health status. The EQ-5D also includes a standard vertical 20-cm visual analogue scale (similar to a thermometer) for recording a participant's rating of their current HRQoL state, which ranged from 0 to 100, where 0 indicated worst imaginable health state and 100 was best imaginable health state. End of Epoch 2 summarizes all participant's data for their last epoch 2 visit (so not including the participant that discontinued in epoch 1).

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=43 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
HRQoL: Change From Baseline in EuroQol Five Dimensions Questionnaire (EQ-5D) Score Baseline: Visual Analogue Scale (VAS) Score	82.84 score on a scale Standard Deviation 16.806	—
HRQoL: Change From Baseline in EuroQol Five Dimensions Questionnaire (EQ-5D) Score Baseline: Self-care	1.16 score on a scale Standard Deviation 0.485	—
HRQoL: Change From Baseline in EuroQol Five Dimensions Questionnaire (EQ-5D) Score Baseline: Usual Activities	1.14 score on a scale Standard Deviation 0.413	—
HRQoL: Change From Baseline in EuroQol Five Dimensions Questionnaire (EQ-5D) Score Baseline: Pain/ Discomfort	1.28 score on a scale Standard Deviation 0.454	—
HRQoL: Change From Baseline in EuroQol Five Dimensions Questionnaire (EQ-5D) Score Baseline: Anxiety/ Depression	1.28 score on a scale Standard Deviation 0.549	—
HRQoL: Change From Baseline in EuroQol Five Dimensions Questionnaire (EQ-5D) Score Change From Baseline at Epoch 2 Month 12: VAS Score	0.64 score on a scale Standard Deviation 23.504	—
HRQoL: Change From Baseline in EuroQol Five Dimensions Questionnaire (EQ-5D) Score Change From Baseline at Epoch 2 Month 12: Mobility	-0.09 score on a scale Standard Deviation 0.292	—
HRQoL: Change From Baseline in EuroQol Five Dimensions Questionnaire (EQ-5D) Score Change From Baseline at Epoch 2 Month 12: Self-care	-0.12 score on a scale Standard Deviation 0.415	—
HRQoL: Change From Baseline in EuroQol Five Dimensions Questionnaire (EQ-5D) Score Change From Baseline at Epoch 2 Month 12: Usual Activities	0.03 score on a scale Standard Deviation 0.394	—
HRQoL: Change From Baseline in EuroQol Five Dimensions Questionnaire (EQ-5D) Score Change From Baseline at Epoch 2 Month 12: Pain/ Discomfort	0.18 score on a scale Standard Deviation 0.528	—
HRQoL: Change From Baseline in EuroQol Five Dimensions Questionnaire (EQ-5D) Score Change From Baseline at Epoch 2 Month 12: Anxiety/ Depression	-0.06 score on a scale Standard Deviation 0.556	—
HRQoL: Change From Baseline in EuroQol Five Dimensions Questionnaire (EQ-5D) Score Change From Baseline at Completion/ Termination (Month 36): VAS Score	-8.93 score on a scale Standard Deviation 26.993	—
HRQoL: Change From Baseline in EuroQol Five Dimensions Questionnaire (EQ-5D) Score Change From Baseline at Completion/ Termination (Month 36): Mobility	0.00 score on a scale Standard Deviation 0.000	—
HRQoL: Change From Baseline in EuroQol Five Dimensions Questionnaire (EQ-5D) Score Change From Baseline at Completion/ Termination (Month 36): Self-care	0.00 score on a scale Standard Deviation 0.000	—
HRQoL: Change From Baseline in EuroQol Five Dimensions Questionnaire (EQ-5D) Score Change From Baseline at Completion/ Termination (Month 36): Usual Activities	0.00 score on a scale Standard Deviation 0.535	—
HRQoL: Change From Baseline in EuroQol Five Dimensions Questionnaire (EQ-5D) Score Change From Baseline at Completion/ Termination (Month 36): Pain/ Discomfort	-0.07 score on a scale Standard Deviation 0.594	—
HRQoL: Change From Baseline in EuroQol Five Dimensions Questionnaire (EQ-5D) Score Change From Baseline at Completion/ Termination (Month 36): Anxiety/ Depression	-0.27 score on a scale Standard Deviation 0.594	—
HRQoL: Change From Baseline in EuroQol Five Dimensions Questionnaire (EQ-5D) Score Change From Baseline at End of Epoch 2: VAS Score	-2.83 score on a scale Standard Deviation 24.895	—
HRQoL: Change From Baseline in EuroQol Five Dimensions Questionnaire (EQ-5D) Score Change From Baseline at End of Epoch 2: Mobility	-0.07 score on a scale Standard Deviation 0.264	—
HRQoL: Change From Baseline in EuroQol Five Dimensions Questionnaire (EQ-5D) Score Change From Baseline at End of Epoch 2: Self-care	-0.10 score on a scale Standard Deviation 0.374	—
HRQoL: Change From Baseline in EuroQol Five Dimensions Questionnaire (EQ-5D) Score Change From Baseline at End of Epoch 2: Usual Activities	0.00 score on a scale Standard Deviation 0.387	—
HRQoL: Change From Baseline in EuroQol Five Dimensions Questionnaire (EQ-5D) Score Change From Baseline at End of Epoch 2: Pain/ Discomfort	0.07 score on a scale Standard Deviation 0.565	—
HRQoL: Change From Baseline in EuroQol Five Dimensions Questionnaire (EQ-5D) Score Change From Baseline at End of Epoch 2: Anxiety/ Depression	-0.10 score on a scale Standard Deviation 0.539	—
HRQoL: Change From Baseline in EuroQol Five Dimensions Questionnaire (EQ-5D) Score Baseline: Mobility	1.07 score on a scale Standard Deviation 0.258	—

SECONDARY outcome

Timeframe: Epoch 1: Baseline (First Infusion); Study Epoch 2: Up to Month 36

Population: SAS included all participants who received at least one dose of HyQvia. Overall number analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analysis at a specified timepoint.

The LQI is a validated questionnaire assessing participant perceptions of their HRQoL and their treatment specifically among participants who use IgG therapy. This questionnaire covers 4 domains: treatment interferences, therapy-related problems, therapy setting, and treatment costs. The LQI domains are scored from 0 to 100, with higher scores associated with better IgG treatment satisfaction. End of Epoch 2 summarizes all participant's data for their last epoch 2 visit (so not including the participant that discontinued in epoch 1).

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=43 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Life Quality Index (LQI) Score Baseline: Treatment Interferences	69.77 score on a scale Standard Deviation 17.518	—
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Life Quality Index (LQI) Score Baseline: Therapy-related Problems	65.21 score on a scale Standard Deviation 17.080	—
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Life Quality Index (LQI) Score Baseline: Therapy Setting	85.66 score on a scale Standard Deviation 14.610	—
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Life Quality Index (LQI) Score Baseline: Treatment Costs	60.85 score on a scale Standard Deviation 27.310	—
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Life Quality Index (LQI) Score Change From Baseline at Epoch 2 Month 12: Treatment Interferences	4.71 score on a scale Standard Deviation 18.419	—
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Life Quality Index (LQI) Score Change From Baseline at Epoch 2 Month 12: Therapy-related Problems	4.67 score on a scale Standard Deviation 17.670	—
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Life Quality Index (LQI) Score Change From Baseline at Epoch 2 Month 12: Therapy Setting	-6.23 score on a scale Standard Deviation 23.189	—
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Life Quality Index (LQI) Score Change From Baseline at Epoch 2 Month 12: Treatment Costs	4.55 score on a scale Standard Deviation 36.330	—
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Life Quality Index (LQI) Score Change From Baseline at Completion/Termination (Month 36): Treatment Interferences	-10.52 score on a scale Standard Deviation 23.850	—
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Life Quality Index (LQI) Score Change From Baseline at Completion/Termination (Month 36): Therapy-related Problems	-13.39 score on a scale Standard Deviation 31.656	—
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Life Quality Index (LQI) Score Change From Baseline at Completion/Termination (Month 36): Therapy Setting	-15.87 score on a scale Standard Deviation 27.808	—
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Life Quality Index (LQI) Score Change From Baseline at Completion/Termination (Month 36): Treatment Costs	-1.19 score on a scale Standard Deviation 32.826	—
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Life Quality Index (LQI) Score Change From Baseline at End of Epoch 2: Treatment Interferences	1.49 score on a scale Standard Deviation 20.908	—
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Life Quality Index (LQI) Score Change From Baseline at End of Epoch 2: Therapy-related Problems	0.41 score on a scale Standard Deviation 24.399	—
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Life Quality Index (LQI) Score Change From Baseline at End of Epoch 2: Therapy Setting	-10.16 score on a scale Standard Deviation 24.718	—
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Life Quality Index (LQI) Score Change From Baseline at End of Epoch 2: Treatment Costs	4.27 score on a scale Standard Deviation 35.164	—

SECONDARY outcome

Timeframe: Epoch 1: Baseline (First Infusion); Study Epoch 2: Up to Month 36

Population: SAS included all participants who received at least one dose of HyQvia. Overall number analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analysis at a specified timepoint.

The TSQM-9 is a 9-item, validated, self-administered instrument to assess participant satisfaction with medication, which assesses 3 domains: effectiveness, convenience, and global satisfaction. The TSQM-9 domain scores range from 0 to 100 with higher scores representing higher satisfaction. End of Epoch 2 summarizes all participant's data for their last epoch 2 visit (so not including the participant that discontinued in epoch 1).

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=43 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Treatment Satisfaction and Medication Questionnaire (TSQM-9) Score Baseline: Effectiveness	74.81 score on a scale Standard Deviation 15.494	—
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Treatment Satisfaction and Medication Questionnaire (TSQM-9) Score Baseline: Convenience	66.15 score on a scale Standard Deviation 16.436	—
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Treatment Satisfaction and Medication Questionnaire (TSQM-9) Score Baseline: Global Satisfaction	79.07 score on a scale Standard Deviation 16.525	—
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Treatment Satisfaction and Medication Questionnaire (TSQM-9) Score Change From Baseline at Epoch 2, Month 12: Effectiveness	6.40 score on a scale Standard Deviation 16.204	—
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Treatment Satisfaction and Medication Questionnaire (TSQM-9) Score Change From Baseline at Epoch 2, Month 12: Convenience	2.19 score on a scale Standard Deviation 19.834	—
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Treatment Satisfaction and Medication Questionnaire (TSQM-9) Score Change From Baseline at Epoch 2, Month 12: Global Satisfaction	4.76 score on a scale Standard Deviation 18.529	—
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Treatment Satisfaction and Medication Questionnaire (TSQM-9) Score Change From Baseline at Completion/ termination (Month 36): Effectiveness	-12.70 score on a scale Standard Deviation 33.577	—
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Treatment Satisfaction and Medication Questionnaire (TSQM-9) Score Change From Baseline at Completion/ termination (Month 36): Convenience	-7.14 score on a scale Standard Deviation 20.375	—
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Treatment Satisfaction and Medication Questionnaire (TSQM-9) Score Change From Baseline at Completion/ termination (Month 36): Global Satisfaction	-17.35 score on a scale Standard Deviation 36.283	—
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Treatment Satisfaction and Medication Questionnaire (TSQM-9) Score Change From Baseline at end of Epoch 2: Effectiveness	3.52 score on a scale Standard Deviation 20.591	—
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Treatment Satisfaction and Medication Questionnaire (TSQM-9) Score Change From Baseline at end of Epoch 2: Convenience	-0.81 score on a scale Standard Deviation 20.244	—
Treatment Preference and Satisfaction: Change From Baseline in Assessment of Treatment Satisfaction and Medication Questionnaire (TSQM-9) Score Change From Baseline at end of Epoch 2: Global Satisfaction	-0.17 score on a scale Standard Deviation 24.406	—

SECONDARY outcome

Timeframe: Study Epoch 2: Up to Month 36

Population: SAS included all participants who received at least one dose of HyQvia. As pre-specified in SAP, this outcome measure was planned only for Epoch 2.

The treatment preference questionnaire, internally developed by the study sponsor, is a self-administered, non-validated scale assessing participant preference for various attributes of immunoglobulin G (IgG) therapy.End of Epoch 2 summarizes all participant's data for their last epoch 2 visit (so not including the participant that discontinued in epoch 1).

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=43 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Treatment Preference and Satisfaction: Number of Participants Who Completed Treatment Preference Questionnaire Month 12	34 Participants	—
Treatment Preference and Satisfaction: Number of Participants Who Completed Treatment Preference Questionnaire Completion/ Termination (Month 36)	14 Participants	—
Treatment Preference and Satisfaction: Number of Participants Who Completed Treatment Preference Questionnaire End of Epoch 2	42 Participants	—

SECONDARY outcome

Timeframe: From first dose of study drug up to EOS (up to 4 years 9 months)

Population: SAS included all participants who received at least one dose of HyQvia.

Days not able to go to school or work, or to perform normal daily activities due to infection or other illnesses were calculated as days not able to go to school or work, or to perform normal daily activities due to infection or other illnesses per participant-year. Per participant-years = number or days reported / total number of years of study duration, i.e., the sum of study duration for all participants in the analysis set, divided by 365.25.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=52 Years Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Health Resource Utilization: Days Not Able to go to School or Work, or to Perform Normal Daily Activities	4.28 days per participant-year	—

SECONDARY outcome

Timeframe: From first dose of study drug up to EOS (up to 4 years 9 months)

Population: SAS included all participants who received at least one dose of HyQvia.

Days on antibiotics were calculated as days on antibiotics per participant-year. Per participant-years = number or days reported / total number of years of study duration, i.e., the sum of study duration for all participants in the analysis set, divided by 365.25.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=52 Infusion Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Health Resource Utilization: Days on Antibiotics	26.77 days per participant-year	—

SECONDARY outcome

Timeframe: From first dose of study drug up to EOS (up to 4 years 9 months)

Population: SAS included all participants who received at least one dose of HyQvia.

Number of hospitalizations, indication for the hospitalization (infection or non-infection) were calculated as number of hospitalizations, indication for the hospitalization (infection or non-infection) per participant-year. Per participant-years = number or days reported / total number of years of study duration, i.e., the sum of study duration for all participants in the analysis set, divided by 365.25.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=44 Participants Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Health Resource Utilization: Number of Hospitalizations	0.08 hospitalizations per participant-year	—

SECONDARY outcome

Timeframe: From first dose of study drug up to EOS (up to 4 years 9 months)

Population: SAS included all participants who received at least one dose of HyQvia.

Number of days hospitalized were calculated as number of days hospitalized per participant-year. Per participant-years = number or days reported / total number of years of study duration, i.e., the sum of study duration for all participants in the analysis set, divided by 365.25.

Outcome measures

Outcome measures
Measure	Epoch 1 + Epoch 2 n=52 Participant-Years Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks. Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.	Epoch 2 Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Health Resource Utilization: Number of Days Hospitalized Per Participant-Year	0.21 days per participant-year	—

Adverse Events

Epoch 1

Serious events: 1 serious events

Other events: 28 other events

Deaths: 0 deaths

Epoch 2

Serious events: 3 serious events

Other events: 40 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Epoch 1 n=44 participants at risk Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks.	Epoch 2 n=43 participants at risk Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Infections and infestations Adenovirus infection	0.00% 0/44 • From first dose of study drug up to EOS (up to 4 years 9 months) At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant via the participant diary or observed by the investigator was recorded, irrespective of the relation to study treatment. SAS included all participants who received at least one dose of HYQVIA.	2.3% 1/43 • From first dose of study drug up to EOS (up to 4 years 9 months) At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant via the participant diary or observed by the investigator was recorded, irrespective of the relation to study treatment. SAS included all participants who received at least one dose of HYQVIA.
Infections and infestations Clostridium difficile colitis	0.00% 0/44 • From first dose of study drug up to EOS (up to 4 years 9 months) At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant via the participant diary or observed by the investigator was recorded, irrespective of the relation to study treatment. SAS included all participants who received at least one dose of HYQVIA.	2.3% 1/43 • From first dose of study drug up to EOS (up to 4 years 9 months) At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant via the participant diary or observed by the investigator was recorded, irrespective of the relation to study treatment. SAS included all participants who received at least one dose of HYQVIA.
Infections and infestations Pneumonia	2.3% 1/44 • From first dose of study drug up to EOS (up to 4 years 9 months) At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant via the participant diary or observed by the investigator was recorded, irrespective of the relation to study treatment. SAS included all participants who received at least one dose of HYQVIA.	0.00% 0/43 • From first dose of study drug up to EOS (up to 4 years 9 months) At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant via the participant diary or observed by the investigator was recorded, irrespective of the relation to study treatment. SAS included all participants who received at least one dose of HYQVIA.
Respiratory, thoracic and mediastinal disorders Tonsillar hypertrophy	0.00% 0/44 • From first dose of study drug up to EOS (up to 4 years 9 months) At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant via the participant diary or observed by the investigator was recorded, irrespective of the relation to study treatment. SAS included all participants who received at least one dose of HYQVIA.	2.3% 1/43 • From first dose of study drug up to EOS (up to 4 years 9 months) At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant via the participant diary or observed by the investigator was recorded, irrespective of the relation to study treatment. SAS included all participants who received at least one dose of HYQVIA.

Other adverse events

Additional Information

Study Director

Takeda

Phone: +1 866 842 5335

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The first study related publication will be a multi-center publication submitted within 24 months after conclusion or termination of a study at all sites. After such multi site publication, all proposed site publications and presentations will be submitted to sponsor for review 60 days in advance of publication. Site will remove Sponsor confidential information unrelated to study results. Sponsor can delay a proposed publication for another 60 days to preserve intellectual property.
Publication restrictions are in place

Restriction type: OTHER