Trial Outcomes & Findings for Octaplas Pediatric Plasma Exchange Trial (NCT NCT01938378)

NCT ID: NCT01938378

Last Updated: 2020-03-24

Results Overview

Monitoring of adverse drug reactions caused by the octaplas™used for plasma exchange.

• Recruitment status: COMPLETED
• Study phase: PHASE4
• Target enrollment: 41 participants
• Primary outcome timeframe: up to 8 days including the 24 hour follow-up from treatment
• Results posted on: 2020-03-24

Participant Flow

Participant milestones

Measure	Pediatric Patients Undergoing TPE octaplas™ Octaplas™: octaplas™ infusion solution for IV administration, ABO compatibile. Recommended dose for a plasma exchange is 40 to 60 ml/kg.
Overall Study STARTED	41
Overall Study COMPLETED	41
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Octaplas Pediatric Plasma Exchange Trial

Baseline characteristics by cohort

Measure	Pediatric Patients Undergoing TPE Age Group 1 n=15 Participants Ages 2-\<12	Pediatric Patients Undergoing TPE Age Group 2 n=13 Participants Ages 12-\<17	Pediatric Patients Undergoing TPE Age Group 3 n=13 Participants Ages ≥17	Total n=41 Participants Total of all reporting groups
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Continuous	6.1 years STANDARD_DEVIATION 2.34 • n=5 Participants	13.8 years STANDARD_DEVIATION 1.52 • n=7 Participants	18.1 years STANDARD_DEVIATION 1.04 • n=5 Participants	12.3 years STANDARD_DEVIATION 5.40 • n=4 Participants
Sex: Female, Male Female	12 Participants n=5 Participants	7 Participants n=7 Participants	4 Participants n=5 Participants	23 Participants n=4 Participants
Sex: Female, Male Male	3 Participants n=5 Participants	6 Participants n=7 Participants	9 Participants n=5 Participants	18 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants	1 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	15 Participants n=5 Participants	12 Participants n=7 Participants	12 Participants n=5 Participants	39 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	2 Participants n=7 Participants	0 Participants n=5 Participants	2 Participants n=4 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants	7 Participants n=4 Participants
Race (NIH/OMB) White	13 Participants n=5 Participants	9 Participants n=7 Participants	10 Participants n=5 Participants	32 Participants n=4 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants

PRIMARY outcome

Timeframe: up to 8 days including the 24 hour follow-up from treatment

Monitoring of adverse drug reactions caused by the octaplas™used for plasma exchange.

Outcome measures

Measure	Pediatric Patients Undergoing TPE Age Group 1 n=15 Participants Ages 2-\<12	Pediatric Patients Undergoing TPE Age Group 2 n=13 Participants Ages 12 - \<17	Pediatric Patients Undergoing TPE Age Group 3 n=13 Participants Ages ≥17	All Patients n=41 Participants All ages	Change From Pre-TPE to Follow-Up Mean change in level from Pre-TPE to Follow-Up
Monitoring of Adverse Drug Reactions Caused by the Octaplas™Used for Plasma Exchange.	0 event	5 event	3 event	8 event	—

PRIMARY outcome

Timeframe: up to 8 days including the 24 hour follow-up from treatment

Monitoring of Thrombotic Events (TEs) and Thromboembolic Events (TEEs) caused by the octaplas™used for plasma exchange.

Outcome measures

Measure	Pediatric Patients Undergoing TPE Age Group 1 n=15 Participants Ages 2-\<12	Pediatric Patients Undergoing TPE Age Group 2 n=13 Participants Ages 12 - \<17	Pediatric Patients Undergoing TPE Age Group 3 n=13 Participants Ages ≥17	All Patients n=41 Participants All ages	Change From Pre-TPE to Follow-Up Mean change in level from Pre-TPE to Follow-Up
Monitoring of TEs and TEEs Caused by the Octaplas™Used for Plasma Exchange.	0 event	0 event	0 event	0 event	—

SECONDARY outcome

Timeframe: up to 8 days including the 24 hour follow-up

Population: Not all patients received blood draws/multiple TPEs

Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end.

Outcome measures

Measure	Pediatric Patients Undergoing TPE Age Group 1 n=41 Participants Ages 2-\<12	Pediatric Patients Undergoing TPE Age Group 2 n=41 Participants Ages 12 - \<17	Pediatric Patients Undergoing TPE Age Group 3 n=41 Participants Ages ≥17	All Patients All ages	Change From Pre-TPE to Follow-Up Mean change in level from Pre-TPE to Follow-Up
Assessment of Blood Urea Nitrogen Levels TPE 1	26.4 mg/dL Standard Deviation 18.69	25.2 mg/dL Standard Deviation 18.22	-1.4 mg/dL Standard Deviation 6.56	—	—
Assessment of Blood Urea Nitrogen Levels TPE 2	27.3 mg/dL Standard Deviation 19.31	27.6 mg/dL Standard Deviation 18.67	1.2 mg/dL Standard Deviation 4.69	—	—
Assessment of Blood Urea Nitrogen Levels TPE 3	32.5 mg/dL Standard Deviation 25.47	26.4 mg/dL Standard Deviation 25.29	1.1 mg/dL Standard Deviation 3.21	—	—
Assessment of Blood Urea Nitrogen Levels TPE 4	28.3 mg/dL Standard Deviation 14.38	28.6 mg/dL Standard Deviation 16.21	1.5 mg/dL Standard Deviation 2.67	—	—
Assessment of Blood Urea Nitrogen Levels TPE 5	31.2 mg/dL Standard Deviation 18.95	35.5 mg/dL Standard Deviation 25.12	-0.5 mg/dL Standard Deviation 4.36	—	—
Assessment of Blood Urea Nitrogen Levels TPE 6	21.0 mg/dL Standard Deviation 0	20.0 mg/dL Standard Deviation 0	-1.0 mg/dL Standard Deviation 0	—	—

SECONDARY outcome

Timeframe: up to 8 days including the 24 hour follow-up

Population: Not all patients received blood draws/multiple TPEs

Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end.

Outcome measures

Measure	Pediatric Patients Undergoing TPE Age Group 1 n=41 Participants Ages 2-\<12	Pediatric Patients Undergoing TPE Age Group 2 n=41 Participants Ages 12 - \<17	Pediatric Patients Undergoing TPE Age Group 3 n=41 Participants Ages ≥17	All Patients All ages	Change From Pre-TPE to Follow-Up Mean change in level from Pre-TPE to Follow-Up
Assessment of Carbon Dioxide Levels TPE 1	25.0 mmol/L Standard Deviation 4.04	26.9 mmol/L Standard Deviation 4.62	2.2 mmol/L Standard Deviation 3.03	—	—
Assessment of Carbon Dioxide Levels TPE 3	26.2 mmol/L Standard Deviation 3.13	26.7 mmol/L Standard Deviation 2.33	1.2 mmol/L Standard Deviation 1.72	—	—
Assessment of Carbon Dioxide Levels TPE 4	26.3 mmol/L Standard Deviation 3.13	26.6 mmol/L Standard Deviation 4.87	1.0 mmol/L Standard Deviation 2.98	—	—
Assessment of Carbon Dioxide Levels TPE 5	27.3 mmol/L Standard Deviation 4.32	27.0 mmol/L Standard Deviation 7.75	0.3 mmol/L Standard Deviation 5.50	—	—
Assessment of Carbon Dioxide Levels TPE 6	29.0 mmol/L Standard Deviation 0	32.0 mmol/L Standard Deviation 0	3.0 mmol/L Standard Deviation 0	—	—
Assessment of Carbon Dioxide Levels TPE 2	27.3 mmol/L Standard Deviation 3.58	28.2 mmol/L Standard Deviation 3.96	0.5 mmol/L Standard Deviation 2.79	—	—

SECONDARY outcome

Timeframe: up to 8 days including the 24 hour follow-up

Population: Not all patients received blood draws/multiple TPEs

Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end.

Outcome measures

Measure	Pediatric Patients Undergoing TPE Age Group 1 n=41 Participants Ages 2-\<12	Pediatric Patients Undergoing TPE Age Group 2 n=41 Participants Ages 12 - \<17	Pediatric Patients Undergoing TPE Age Group 3 n=41 Participants Ages ≥17	All Patients All ages	Change From Pre-TPE to Follow-Up Mean change in level from Pre-TPE to Follow-Up
Assessment of Chloride Levels TPE 1	105.0 mmol/L Standard Deviation 4.24	105.2 mmol/L Standard Deviation 5.15	0.5 mmol/L Standard Deviation 3.31	—	—
Assessment of Chloride Levels TPE 2	105.5 mmol/L Standard Deviation 4.44	105.4 mmol/L Standard Deviation 5.04	0.6 mmol/L Standard Deviation 2.72	—	—
Assessment of Chloride Levels TPE 3	106.7 mmol/L Standard Deviation 5.28	107.5 mmol/L Standard Deviation 6.15	-0.3 mmol/L Standard Deviation 2.37	—	—
Assessment of Chloride Levels TPE 4	106.4 mmol/L Standard Deviation 7.06	106.8 mmol/L Standard Deviation 6.98	-0.9 mmol/L Standard Deviation 2.59	—	—
Assessment of Chloride Levels TPE 5	103.0 mmol/L Standard Deviation 5.22	104.8 mmol/L Standard Deviation 7.93	1.0 mmol/L Standard Deviation 6.78	—	—
Assessment of Chloride Levels TPE 6	111.0 mmol/L Standard Deviation 0	108.0 mmol/L Standard Deviation 0	-3.0 mmol/L Standard Deviation 0	—	—

SECONDARY outcome

Timeframe: up to 8 days including the 24 hour follow-up

Population: Not all patients received blood draws/multiple TPEs

Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end.

Outcome measures

Measure	Pediatric Patients Undergoing TPE Age Group 1 n=41 Participants Ages 2-\<12	Pediatric Patients Undergoing TPE Age Group 2 n=41 Participants Ages 12 - \<17	Pediatric Patients Undergoing TPE Age Group 3 n=41 Participants Ages ≥17	All Patients All ages	Change From Pre-TPE to Follow-Up Mean change in level from Pre-TPE to Follow-Up
Assessment of Creatinine Levels TPE 1	1.7 mg/dl Standard Deviation 2.24	1.3 mg/dl Standard Deviation 1.45	-0.4 mg/dl Standard Deviation 1.10	—	—
Assessment of Creatinine Levels TPE 2	1.3 mg/dl Standard Deviation 1.00	1.2 mg/dl Standard Deviation 0.95	-0.1 mg/dl Standard Deviation 0.31	—	—
Assessment of Creatinine Levels TPE 3	1.1 mg/dl Standard Deviation 0.78	0.9 mg/dl Standard Deviation 0.84	-0.1 mg/dl Standard Deviation 0.22	—	—
Assessment of Creatinine Levels TPE 5	1.1 mg/dl Standard Deviation 0.66	1.2 mg/dl Standard Deviation 0.87	0.1 mg/dl Standard Deviation 0.09	—	—
Assessment of Creatinine Levels TPE 6	0.8 mg/dl Standard Deviation 0	0.8 mg/dl Standard Deviation 0	0.0 mg/dl Standard Deviation 0.0	—	—
Assessment of Creatinine Levels TPE 4	1.0 mg/dl Standard Deviation 0.51	0.9 mg/dl Standard Deviation 0.59	0.0 mg/dl Standard Deviation 0.09	—	—

SECONDARY outcome

Timeframe: up to 8 days including the 24 hour follow-up

Population: Not all patients received blood draws/multiple TPEs

Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end.

Outcome measures

Measure	Pediatric Patients Undergoing TPE Age Group 1 n=41 Participants Ages 2-\<12	Pediatric Patients Undergoing TPE Age Group 2 n=41 Participants Ages 12 - \<17	Pediatric Patients Undergoing TPE Age Group 3 n=41 Participants Ages ≥17	All Patients All ages	Change From Pre-TPE to Follow-Up Mean change in level from Pre-TPE to Follow-Up
Assessment of Glucose Levels TPE 1	144.7 mg/dl Standard Deviation 70.10	135.2 mg/dl Standard Deviation 41.89	-12.0 mg/dl Standard Deviation 65.83	—	—
Assessment of Glucose Levels TPE 2	138.2 mg/dl Standard Deviation 61.92	142.7 mg/dl Standard Deviation 75.86	12.5 mg/dl Standard Deviation 44.76	—	—
Assessment of Glucose Levels TPE 3	141.9 mg/dl Standard Deviation 65.12	156.9 mg/dl Standard Deviation 57.67	22.6 mg/dl Standard Deviation 45.69	—	—
Assessment of Glucose Levels TPE 4	191.0 mg/dl Standard Deviation 142.81	142.1 mg/dl Standard Deviation 59.04	-44.3 mg/dl Standard Deviation 158.61	—	—
Assessment of Glucose Levels TPE 5	147.2 mg/dl Standard Deviation 85.33	140.8 mg/dl Standard Deviation 61.70	15.8 mg/dl Standard Deviation 38.59	—	—
Assessment of Glucose Levels TPE 6	77.0 mg/dl Standard Deviation 0	75.0 mg/dl Standard Deviation 0	-2.0 mg/dl Standard Deviation 0	—	—

SECONDARY outcome

Timeframe: up to 8 days including the 24 hour follow-up

Population: Not all patients received blood draws/multiple TPEs

Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end.

Outcome measures

Measure	Pediatric Patients Undergoing TPE Age Group 1 n=41 Participants Ages 2-\<12	Pediatric Patients Undergoing TPE Age Group 2 n=41 Participants Ages 12 - \<17	Pediatric Patients Undergoing TPE Age Group 3 n=41 Participants Ages ≥17	All Patients All ages	Change From Pre-TPE to Follow-Up Mean change in level from Pre-TPE to Follow-Up
Assessment of Potassium Levels TPE 1	3.9 mmol/L Standard Deviation 0.57	3.8 mmol/L Standard Deviation 0.58	-0.1 mmol/L Standard Deviation 0.54	—	—
Assessment of Potassium Levels TPE 2	3.9 mmol/L Standard Deviation 0.43	4.0 mmol/L Standard Deviation 0.47	0.1 mmol/L Standard Deviation 0.26	—	—
Assessment of Potassium Levels TPE 3	3.7 mmol/L Standard Deviation 0.57	3.9 mmol/L Standard Deviation 0.36	0.1 mmol/L Standard Deviation 0.49	—	—
Assessment of Potassium Levels TPE 4	4.1 mmol/L Standard Deviation 0.62	4.0 mmol/L Standard Deviation 0.64	-0.2 mmol/L Standard Deviation 0.65	—	—
Assessment of Potassium Levels TPE 5	3.9 mmol/L Standard Deviation 0.30	3.8 mmol/L Standard Deviation 0.37	-0.1 mmol/L Standard Deviation 0.61	—	—
Assessment of Potassium Levels TPE 6	4.2 mmol/L Standard Deviation 0	3.5 mmol/L Standard Deviation 0	-0.7 mmol/L Standard Deviation 0	—	—

SECONDARY outcome

Timeframe: up to 8 days including the 24 hour follow-up

Population: Not all patients received blood draws/multiple TPEs

Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end.

Outcome measures

Measure	Pediatric Patients Undergoing TPE Age Group 1 n=41 Participants Ages 2-\<12	Pediatric Patients Undergoing TPE Age Group 2 n=41 Participants Ages 12 - \<17	Pediatric Patients Undergoing TPE Age Group 3 n=41 Participants Ages ≥17	All Patients All ages	Change From Pre-TPE to Follow-Up Mean change in level from Pre-TPE to Follow-Up
Assessment of Sodium Levels TPE 1	140.0 mmol/L Standard Deviation 3.66	141.6 mmol/L Standard Deviation 3.23	1.6 mmol/L Standard Deviation 2.41	—	—
Assessment of Sodium Levels TPE 2	141.2 mmol/L Standard Deviation 3.28	142.3 mmol/L Standard Deviation 4.41	1.5 mmol/L Standard Deviation 1.84	—	—
Assessment of Sodium Levels TPE 3	141.2 mmol/L Standard Deviation 3.87	142.9 mmol/L Standard Deviation 5.07	1.5 mmol/L Standard Deviation 2.84	—	—
Assessment of Sodium Levels TPE 4	140.7 mmol/L Standard Deviation 4.64	142.4 mmol/L Standard Deviation 5.13	0.8 mmol/L Standard Deviation 1.75	—	—
Assessment of Sodium Levels TPE 5	138.8 mmol/L Standard Deviation 3.19	141.3 mmol/L Standard Deviation 3.86	1.0 mmol/L Standard Deviation 2.16	—	—
Assessment of Sodium Levels TPE 6	142.0 mmol/L Standard Deviation 0	144.0 mmol/L Standard Deviation 0	2.0 mmol/L Standard Deviation 0	—	—

SECONDARY outcome

Timeframe: up to 8 days including the 24 hour follow-up

Population: Not all patients received blood draws/multiple TPEs

Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end.

Outcome measures

Measure	Pediatric Patients Undergoing TPE Age Group 1 n=41 Participants Ages 2-\<12	Pediatric Patients Undergoing TPE Age Group 2 n=41 Participants Ages 12 - \<17	Pediatric Patients Undergoing TPE Age Group 3 n=41 Participants Ages ≥17	All Patients All ages	Change From Pre-TPE to Follow-Up Mean change in level from Pre-TPE to Follow-Up
Assessment of Leukocyte Levels TPE 1	12.8 10^9 cells/L Standard Deviation 6.84	13.6 10^9 cells/L Standard Deviation 7.45	0.9 10^9 cells/L Standard Deviation 3.80	—	—
Assessment of Leukocyte Levels TPE 2	14.9 10^9 cells/L Standard Deviation 7.45	15.3 10^9 cells/L Standard Deviation 6.43	1.2 10^9 cells/L Standard Deviation 3.93	—	—
Assessment of Leukocyte Levels TPE 3	13.2 10^9 cells/L Standard Deviation 6.49	13.9 10^9 cells/L Standard Deviation 7.20	-0.5 10^9 cells/L Standard Deviation 2.44	—	—
Assessment of Leukocyte Levels TPE 4	10.8 10^9 cells/L Standard Deviation 6.32	12.7 10^9 cells/L Standard Deviation 6.40	1.2 10^9 cells/L Standard Deviation 1.14	—	—
Assessment of Leukocyte Levels TPE 5	8.7 10^9 cells/L Standard Deviation 3.17	10.7 10^9 cells/L Standard Deviation 3.65	1.6 10^9 cells/L Standard Deviation 1.55	—	—
Assessment of Leukocyte Levels TPE 6	3.7 10^9 cells/L Standard Deviation 0	7.0 10^9 cells/L Standard Deviation 0	3.3 10^9 cells/L Standard Deviation 0	—	—

SECONDARY outcome

Timeframe: up to 8 days including the 24 hour follow-up

Population: Not all patients received blood draws/multiple TPEs

Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end.

Outcome measures

Measure	Pediatric Patients Undergoing TPE Age Group 1 n=41 Participants Ages 2-\<12	Pediatric Patients Undergoing TPE Age Group 2 n=41 Participants Ages 12 - \<17	Pediatric Patients Undergoing TPE Age Group 3 n=41 Participants Ages ≥17	All Patients All ages	Change From Pre-TPE to Follow-Up Mean change in level from Pre-TPE to Follow-Up
Assessment of Erythrocyte Levels TPE 1	4.0 10^12 cells/L Standard Deviation 0.70	4.1 10^12 cells/L Standard Deviation 0.81	0.1 10^12 cells/L Standard Deviation 0.26	—	—
Assessment of Erythrocyte Levels TPE 2	3.6 10^12 cells/L Standard Deviation 0.60	3.7 10^12 cells/L Standard Deviation 0.68	0.1 10^12 cells/L Standard Deviation 0.27	—	—
Assessment of Erythrocyte Levels TPE 3	3.6 10^12 cells/L Standard Deviation 0.70	3.8 10^12 cells/L Standard Deviation 0.60	0.1 10^12 cells/L Standard Deviation 0.26	—	—
Assessment of Erythrocyte Levels TPE 4	3.7 10^12 cells/L Standard Deviation 0.67	3.4 10^12 cells/L Standard Deviation 0.71	0.1 10^12 cells/L Standard Deviation 0.17	—	—
Assessment of Erythrocyte Levels TPE 5	3.9 10^12 cells/L Standard Deviation 0.96	3.2 10^12 cells/L Standard Deviation 0.35	-0.1 10^12 cells/L Standard Deviation 0.49	—	—
Assessment of Erythrocyte Levels TPE 6	2.9 10^12 cells/L Standard Deviation 0	2.8 10^12 cells/L Standard Deviation 0	-0.1 10^12 cells/L Standard Deviation 0	—	—

SECONDARY outcome

Timeframe: up to 8 days including the 24 hour follow-up

Population: Not all patients received blood draws/multiple TPEs

Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end.

Outcome measures

Measure	Pediatric Patients Undergoing TPE Age Group 1 n=41 Participants Ages 2-\<12	Pediatric Patients Undergoing TPE Age Group 2 n=41 Participants Ages 12 - \<17	Pediatric Patients Undergoing TPE Age Group 3 n=41 Participants Ages ≥17	All Patients All ages	Change From Pre-TPE to Follow-Up Mean change in level from Pre-TPE to Follow-Up
Assessment of Hemoglobin Levels TPE 1	11.5 g/dL Standard Deviation 1.94	12.1 g/dL Standard Deviation 2.22	0.4 g/dL Standard Deviation 0.74	—	—
Assessment of Hemoglobin Levels TPE 2	10.7 g/dL Standard Deviation 1.79	11.0 g/dL Standard Deviation 2.03	0.3 g/dL Standard Deviation 0.74	—	—
Assessment of Hemoglobin Levels TPE 3	10.4 g/dL Standard Deviation 1.90	11.2 g/dL Standard Deviation 2.03	0.3 g/dL Standard Deviation 0.70	—	—
Assessment of Hemoglobin Levels TPE 4	10.6 g/dL Standard Deviation 1.61	9.9 g/dL Standard Deviation 1.94	-0.1 g/dL Standard Deviation 0.67	—	—
Assessment of Hemoglobin Levels TPE 5	10.9 g/dL Standard Deviation 2.13	9.4 g/dL Standard Deviation 1.06	-0.5 g/dL Standard Deviation 1.44	—	—
Assessment of Hemoglobin Levels TPE 6	8.7 g/dL Standard Deviation 0	8.3 g/dL Standard Deviation 0	-0.4 g/dL Standard Deviation 0	—	—

SECONDARY outcome

Timeframe: up to 8 days including the 24 hour follow-up

Population: Not all patients received blood draws/multiple TPEs

Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end.

Outcome measures

Measure	Pediatric Patients Undergoing TPE Age Group 1 n=41 Participants Ages 2-\<12	Pediatric Patients Undergoing TPE Age Group 2 n=41 Participants Ages 12 - \<17	Pediatric Patients Undergoing TPE Age Group 3 n=41 Participants Ages ≥17	All Patients All ages	Change From Pre-TPE to Follow-Up Mean change in level from Pre-TPE to Follow-Up
Assessment of Hematocrit Levels TPE 1	34.1 % of the blood that is red blood cells Standard Deviation 5.31	35.6 % of the blood that is red blood cells Standard Deviation 6.44	1.1 % of the blood that is red blood cells Standard Deviation 2.12	—	—
Assessment of Hematocrit Levels TPE 2	31.7 % of the blood that is red blood cells Standard Deviation 4.75	32.8 % of the blood that is red blood cells Standard Deviation 5.24	0.8 % of the blood that is red blood cells Standard Deviation 2.39	—	—
Assessment of Hematocrit Levels TPE 3	31.0 % of the blood that is red blood cells Standard Deviation 4.98	33.3 % of the blood that is red blood cells Standard Deviation 5.00	1.0 % of the blood that is red blood cells Standard Deviation 2.37	—	—
Assessment of Hematocrit Levels TPE 4	31.7 % of the blood that is red blood cells Standard Deviation 4.29	30.2 % of the blood that is red blood cells Standard Deviation 5.50	0.6 % of the blood that is red blood cells Standard Deviation 1.25	—	—
Assessment of Hematocrit Levels TPE 5	32.9 % of the blood that is red blood cells Standard Deviation 5.73	28.8 % of the blood that is red blood cells Standard Deviation 2.95	-1.2 % of the blood that is red blood cells Standard Deviation 4.03	—	—
Assessment of Hematocrit Levels TPE 6	27.7 % of the blood that is red blood cells Standard Deviation 0	26.1 % of the blood that is red blood cells Standard Deviation 0	-1.6 % of the blood that is red blood cells Standard Deviation 0	—	—

SECONDARY outcome

Timeframe: up to 8 days including the 24 hour follow-up

Population: Not all patients received blood draws/multiple TPEs

Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end.

Outcome measures

Measure	Pediatric Patients Undergoing TPE Age Group 1 n=41 Participants Ages 2-\<12	Pediatric Patients Undergoing TPE Age Group 2 n=41 Participants Ages 12 - \<17	Pediatric Patients Undergoing TPE Age Group 3 n=41 Participants Ages ≥17	All Patients All ages	Change From Pre-TPE to Follow-Up Mean change in level from Pre-TPE to Follow-Up
Assessment of Mean Corpuscular Volume Levels TPE 1	86.4 fL Standard Deviation 6.96	86.8 fL Standard Deviation 6.68	0.2 fL Standard Deviation 1.21	—	—
Assessment of Mean Corpuscular Volume Levels TPE 2	88.1 fL Standard Deviation 7.46	90.0 fL Standard Deviation 6.91	0.3 fL Standard Deviation 1.55	—	—
Assessment of Mean Corpuscular Volume Levels TPE 3	88.2 fL Standard Deviation 11.38	87.8 fL Standard Deviation 3.57	-0.1 fL Standard Deviation 1.55	—	—
Assessment of Mean Corpuscular Volume Levels TPE 4	87.0 fL Standard Deviation 7.90	89.9 fL Standard Deviation 6.78	-0.3 fL Standard Deviation 2.01	—	—
Assessment of Mean Corpuscular Volume Levels TPE 5	85.9 fL Standard Deviation 9.30	90.2 fL Standard Deviation 7.02	-0.1 fL Standard Deviation 2.13	—	—
Assessment of Mean Corpuscular Volume Levels TPE 6	95.0 fL Standard Deviation 0	93.0 fL Standard Deviation 0	-2.0 fL Standard Deviation 0	—	—

SECONDARY outcome

Timeframe: up to 8 days including the 24 hour follow-up

Population: Not all patients received blood draws/multiple TPEs

Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end.

Outcome measures

Measure	Pediatric Patients Undergoing TPE Age Group 1 n=41 Participants Ages 2-\<12	Pediatric Patients Undergoing TPE Age Group 2 n=41 Participants Ages 12 - \<17	Pediatric Patients Undergoing TPE Age Group 3 n=41 Participants Ages ≥17	All Patients All ages	Change From Pre-TPE to Follow-Up Mean change in level from Pre-TPE to Follow-Up
Assessment of Mean Corpuscular Hemoglobin Levels TPE 1	29.0 pg Standard Deviation 2.54	29.4 pg Standard Deviation 2.18	0.1 pg Standard Deviation 0.70	—	—
Assessment of Mean Corpuscular Hemoglobin Levels TPE 2	29.6 pg Standard Deviation 2.47	30.2 pg Standard Deviation 2.48	0.1 pg Standard Deviation 0.77	—	—
Assessment of Mean Corpuscular Hemoglobin Levels TPE 3	29.2 pg Standard Deviation 2.19	29.4 pg Standard Deviation 1.31	-0.2 pg Standard Deviation 0.58	—	—
Assessment of Mean Corpuscular Hemoglobin Levels TPE 4	28.9 pg Standard Deviation 2.41	29.2 pg Standard Deviation 1.50	-0.8 pg Standard Deviation 1.18	—	—
Assessment of Mean Corpuscular Hemoglobin Levels TPE 5	28.3 pg Standard Deviation 2.74	29.4 pg Standard Deviation 1.75	-0.4 pg Standard Deviation 0.31	—	—
Assessment of Mean Corpuscular Hemoglobin Levels TPE 6	29.8 pg Standard Deviation 0	29.6 pg Standard Deviation 0	-0.2 pg Standard Deviation 0	—	—

SECONDARY outcome

Timeframe: up to 8 days including the 24 hour follow-up

Population: Not all patients received blood draws/multiple TPEs

Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end.

Outcome measures

Measure	Pediatric Patients Undergoing TPE Age Group 1 n=41 Participants Ages 2-\<12	Pediatric Patients Undergoing TPE Age Group 2 n=41 Participants Ages 12 - \<17	Pediatric Patients Undergoing TPE Age Group 3 n=41 Participants Ages ≥17	All Patients All ages	Change From Pre-TPE to Follow-Up Mean change in level from Pre-TPE to Follow-Up
Assessment of Mean Corpuscular Hemoglobin Concentration Levels TPE 1	33.6 g/dL Standard Deviation 1.38	33.9 g/dL Standard Deviation 1.20	0.1 g/dL Standard Deviation 0.75	—	—
Assessment of Mean Corpuscular Hemoglobin Concentration Levels TPE 2	33.6 g/dL Standard Deviation 1.35	33.6 g/dL Standard Deviation 1.58	0 g/dL Standard Deviation 0.97	—	—
Assessment of Mean Corpuscular Hemoglobin Concentration Levels TPE 3	33.4 g/dL Standard Deviation 2.17	33.5 g/dL Standard Deviation 1.43	-0.2 g/dL Standard Deviation 0.91	—	—
Assessment of Mean Corpuscular Hemoglobin Concentration Levels TPE 4	33.2 g/dL Standard Deviation 1.16	32.6 g/dL Standard Deviation 1.19	-0.8 g/dL Standard Deviation 1.15	—	—
Assessment of Mean Corpuscular Hemoglobin Concentration Levels TPE 5	33.1 g/dL Standard Deviation 1.75	32.6 g/dL Standard Deviation 1.21	-0.5 g/dL Standard Deviation 0.62	—	—
Assessment of Mean Corpuscular Hemoglobin Concentration Levels TPE 6	31.4 g/dL Standard Deviation 0	31.8 g/dL Standard Deviation 0	0.4 g/dL Standard Deviation 0	—	—

SECONDARY outcome

Timeframe: up to 8 days including the 24 hour follow-up

Population: Not all patients received blood draws/multiple TPEs

Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end.

Outcome measures

Measure	Pediatric Patients Undergoing TPE Age Group 1 n=41 Participants Ages 2-\<12	Pediatric Patients Undergoing TPE Age Group 2 n=41 Participants Ages 12 - \<17	Pediatric Patients Undergoing TPE Age Group 3 n=41 Participants Ages ≥17	All Patients All ages	Change From Pre-TPE to Follow-Up Mean change in level from Pre-TPE to Follow-Up
Assessment of Mean Red Cell Distribution Width Levels TPE 6	18.2 % red blood cell variation volume/size Standard Deviation 0	18.0 % red blood cell variation volume/size Standard Deviation 0	-0.2 % red blood cell variation volume/size Standard Deviation 0	—	—
Assessment of Mean Red Cell Distribution Width Levels TPE 1	14.4 % red blood cell variation volume/size Standard Deviation 2.66	13.9 % red blood cell variation volume/size Standard Deviation 1.94	0.0 % red blood cell variation volume/size Standard Deviation 0.26	—	—
Assessment of Mean Red Cell Distribution Width Levels TPE 2	14.6 % red blood cell variation volume/size Standard Deviation 2.83	15.0 % red blood cell variation volume/size Standard Deviation 3.02	0.1 % red blood cell variation volume/size Standard Deviation 0.37	—	—
Assessment of Mean Red Cell Distribution Width Levels TPE 3	15.2 % red blood cell variation volume/size Standard Deviation 3.18	15.0 % red blood cell variation volume/size Standard Deviation 2.31	-0.1 % red blood cell variation volume/size Standard Deviation 0.33	—	—
Assessment of Mean Red Cell Distribution Width Levels TPE 4	16.2 % red blood cell variation volume/size Standard Deviation 3.10	16.5 % red blood cell variation volume/size Standard Deviation 3.10	0.0 % red blood cell variation volume/size Standard Deviation 0.25	—	—
Assessment of Mean Red Cell Distribution Width Levels TPE 5	17.1 % red blood cell variation volume/size Standard Deviation 3.04	18.6 % red blood cell variation volume/size Standard Deviation 3.24	0.3 % red blood cell variation volume/size Standard Deviation 0.52	—	—

SECONDARY outcome

Timeframe: up to 8 days including the 24 hour follow-up

Population: Not all patients received blood draws/multiple TPEs

Blood samples compare levels before each TPE (therapeutic plasma exchange), during each TPE, and after each TPE. Pre-TPE is within 24 hours before TPE start; Follow-Up is 24 (+/-2) hours after TPE end.

Outcome measures

Measure	Pediatric Patients Undergoing TPE Age Group 1 n=41 Participants Ages 2-\<12	Pediatric Patients Undergoing TPE Age Group 2 n=41 Participants Ages 12 - \<17	Pediatric Patients Undergoing TPE Age Group 3 n=41 Participants Ages ≥17	All Patients n=41 Participants All ages	Change From Pre-TPE to Follow-Up n=41 Participants Mean change in level from Pre-TPE to Follow-Up
Assessment of Mean Ionized Calcium Levels TPE 1	1.225 mmol/L Standard Deviation 0.0731	1.129 mmol/L Standard Deviation 0.1541	-0.093 mmol/L Standard Deviation 0.1583	1.194 mmol/L Standard Deviation 0.0852	-0.030 mmol/L Standard Deviation 0.0927
Assessment of Mean Ionized Calcium Levels TPE 2	1.212 mmol/L Standard Deviation 0.0678	1.192 mmol/L Standard Deviation 0.1224	-0.001 mmol/L Standard Deviation 0.0967	1.204 mmol/L Standard Deviation 0.0832	0.026 mmol/L Standard Deviation 0.1203
Assessment of Mean Ionized Calcium Levels TPE 3	1.165 mmol/L Standard Deviation 0.2119	1.181 mmol/L Standard Deviation 0.1062	0.020 mmol/L Standard Deviation 0.1696	1.224 mmol/L Standard Deviation 0.0926	0.026 mmol/L Standard Deviation 0.0684
Assessment of Mean Ionized Calcium Levels TPE 4	1.205 mmol/L Standard Deviation 0.0763	1.265 mmol/L Standard Deviation 0.1060	0.062 mmol/L Standard Deviation 0.0805	1.204 mmol/L Standard Deviation 0.0666	0.011 mmol/L Standard Deviation 0.0995
Assessment of Mean Ionized Calcium Levels TPE 5	1.200 mmol/L Standard Deviation 0.0452	1.240 mmol/L Standard Deviation 0.1039	0.040 mmol/L Standard Deviation 0.0640	1.168 mmol/L Standard Deviation 0.0948	-0.032 mmol/L Standard Deviation 0.1089
Assessment of Mean Ionized Calcium Levels TPE 6	1.248 mmol/L Standard Deviation 0	1.148 mmol/L Standard Deviation 0	-0.100 mmol/L Standard Deviation 0	1.173 mmol/L Standard Deviation 0	-0.075 mmol/L Standard Deviation 0

SECONDARY outcome

Timeframe: up to 8 days including the 24 hour follow-up

Population: Not all patients received multiple TPEs

Excellent: defined as the treatment was well tolerated by the patient; Moderate: defined as Adverse Drug Reaction (ADR(s)) were observed, but easily resolved or not clinically significant; Poor: defined as ADR(s) were observed requiring significant medical intervention

Outcome measures

Measure	Pediatric Patients Undergoing TPE Age Group 1 n=41 Participants Ages 2-\<12	Pediatric Patients Undergoing TPE Age Group 2 Ages 12 - \<17	Pediatric Patients Undergoing TPE Age Group 3 Ages ≥17	All Patients All ages	Change From Pre-TPE to Follow-Up Mean change in level from Pre-TPE to Follow-Up
Investigator's Assessment of Overall Safety Patients Who Underwent TPE 1 · Excellent	37 Participants	—	—	—	—
Investigator's Assessment of Overall Safety Patients Who Underwent TPE 1 · Moderate	4 Participants	—	—	—	—
Investigator's Assessment of Overall Safety Patients Who Underwent TPE 1 · Poor	0 Participants	—	—	—	—
Investigator's Assessment of Overall Safety Patients Who Underwent TPE 2 · Excellent	25 Participants	—	—	—	—
Investigator's Assessment of Overall Safety Patients Who Underwent TPE 2 · Moderate	1 Participants	—	—	—	—
Investigator's Assessment of Overall Safety Patients Who Underwent TPE 2 · Poor	0 Participants	—	—	—	—
Investigator's Assessment of Overall Safety Patients Who Underwent TPE 3 · Excellent	18 Participants	—	—	—	—
Investigator's Assessment of Overall Safety Patients Who Underwent TPE 3 · Moderate	0 Participants	—	—	—	—
Investigator's Assessment of Overall Safety Patients Who Underwent TPE 3 · Poor	0 Participants	—	—	—	—
Investigator's Assessment of Overall Safety Patients Who Underwent TPE 4 · Excellent	9 Participants	—	—	—	—
Investigator's Assessment of Overall Safety Patients Who Underwent TPE 4 · Moderate	1 Participants	—	—	—	—
Investigator's Assessment of Overall Safety Patients Who Underwent TPE 4 · Poor	0 Participants	—	—	—	—
Investigator's Assessment of Overall Safety Patients Who Underwent TPE 5 · Excellent	6 Participants	—	—	—	—
Investigator's Assessment of Overall Safety Patients Who Underwent TPE 5 · Moderate	0 Participants	—	—	—	—
Investigator's Assessment of Overall Safety Patients Who Underwent TPE 5 · Poor	0 Participants	—	—	—	—
Investigator's Assessment of Overall Safety Patients Who Underwent TPE 6 · Excellent	1 Participants	—	—	—	—
Investigator's Assessment of Overall Safety Patients Who Underwent TPE 6 · Poor	0 Participants	—	—	—	—
Investigator's Assessment of Overall Safety Patients Who Underwent TPE 6 · Moderate	0 Participants	—	—	—	—

Adverse Events

Pediatric Patients Undergoing TPE Age Group 1

Serious events: 1 serious events

Other events: 0 other events

Deaths: 1 deaths

Pediatric Patients Undergoing TPE Age Group 2

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Pediatric Patients Undergoing TPE Age Group 3

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

All Patients

Serious events: 1 serious events

Other events: 4 other events

Deaths: 1 deaths

Serious adverse events

Measure	Pediatric Patients Undergoing TPE Age Group 1 n=15 participants at risk Ages 2-\<12	Pediatric Patients Undergoing TPE Age Group 2 n=13 participants at risk Ages 12 - \<17	Pediatric Patients Undergoing TPE Age Group 3 n=13 participants at risk Ages ≥17	All Patients n=41 participants at risk All ages
General disorders Multi-organ failure	6.7% 1/15 • Number of events 1 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse	0.00% 0/13 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse	0.00% 0/13 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse	2.4% 1/41 • Number of events 1 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse

Other adverse events

Measure	Pediatric Patients Undergoing TPE Age Group 1 n=15 participants at risk Ages 2-\<12	Pediatric Patients Undergoing TPE Age Group 2 n=13 participants at risk Ages 12 - \<17	Pediatric Patients Undergoing TPE Age Group 3 n=13 participants at risk Ages ≥17	All Patients n=41 participants at risk All ages
Injury, poisoning and procedural complications Citrate toxicity	0.00% 0/15 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse	15.4% 2/13 • Number of events 2 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse	0.00% 0/13 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse	4.9% 2/41 • Number of events 2 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse
General disorders Pyrexia	0.00% 0/15 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse	7.7% 1/13 • Number of events 1 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse	0.00% 0/13 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse	2.4% 1/41 • Number of events 1 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse
Investigations Inflammatory marker increased	0.00% 0/15 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse	0.00% 0/13 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse	7.7% 1/13 • Number of events 1 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse	2.4% 1/41 • Number of events 1 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse
Musculoskeletal and connective tissue disorders Myalgia	0.00% 0/15 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse	0.00% 0/13 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse	7.7% 1/13 • Number of events 1 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse	2.4% 1/41 • Number of events 1 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse
Nervous system disorders Headache	0.00% 0/15 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse	7.7% 1/13 • Number of events 1 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse	0.00% 0/13 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse	2.4% 1/41 • Number of events 1 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse
Skin and subcutaneous tissue disorders Urticaria	0.00% 0/15 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse	7.7% 1/13 • Number of events 1 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse	0.00% 0/13 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse	2.4% 1/41 • Number of events 1 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse
Gastrointestinal disorders Nausea	0.00% 0/15 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse	0.00% 0/13 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse	7.7% 1/13 • Number of events 1 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse	2.4% 1/41 • Number of events 1 • Up to 8 days ADRs will be elicited using a standard non-leading question such as "How have you been since the last evaluation?" If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient's guardian and/or Investigator or study nurse

Additional Information

Michael Eppolito

Octapharma

Phone: 201-604-1155

Email: michael.eppolito@octapharma.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place