Trial Outcomes & Findings for An Open-Label, Multi-Center Clinical Trial of Eculizumab in Pediatric Patients With Atypical Hemolytic-Uremic Syndrome (NCT NCT01193348)

NCT ID: NCT01193348

Last Updated: 2015-04-29

Results Overview

Proportion of Patients with Complete TMA response was determined and defined by normalization of hematological parameters (platelet count and LDH) and ≥ 25% improvement in serum creatinine from baseline which was sustained for at least two consecutive measurements obtained at least four weeks apart).

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 22 participants
• Primary outcome timeframe: Through 26 weeks
• Results posted on: 2015-04-29

Participant Flow

A total of 27 patients diagnosed with aHUS signed the informed consent and of these, 22 patients were treated. Five patients were excluded from the study due to failed screening procedure and did not receive eculizumab.

At screening, patients had to have a platelet count < lower limit of normal range (<LLN) and serum creatinine level > 97 percentile for age; and had to exhibit signs or symptoms of hemolysis at the start of the current aHUS event with fragmented RBC and a negative Coombs test.

Participant milestones

Measure	Eculizumab Eculizumab: Fixed dosing is based on body weight cohorts. Adjustment of dose to accommodate patient growth is possible.
Screening Period STARTED	27
Screening Period COMPLETED	22
Screening Period NOT COMPLETED	5
Treatment Period (26 Weeks) STARTED	22
Treatment Period (26 Weeks) COMPLETED	19
Treatment Period (26 Weeks) NOT COMPLETED	3
Extension Treatment Period STARTED	17
Extension Treatment Period COMPLETED	16
Extension Treatment Period NOT COMPLETED	1
Post Treatment Period (Patients Who Disc STARTED	10
Post Treatment Period (Patients Who Disc COMPLETED	8
Post Treatment Period (Patients Who Disc NOT COMPLETED	2

Reasons for withdrawal

Measure	Eculizumab Eculizumab: Fixed dosing is based on body weight cohorts. Adjustment of dose to accommodate patient growth is possible.
Screening Period Screen Failure	5
Treatment Period (26 Weeks) Positive Shiga-Toxin Result vial Local	1
Treatment Period (26 Weeks) Serious Adverse Event	1
Treatment Period (26 Weeks) Patient Requested to Withdraw	1
Extension Treatment Period Physician Decision	1
Post Treatment Period (Patients Who Disc Patient did not return for visit	2

Baseline Characteristics

An Open-Label, Multi-Center Clinical Trial of Eculizumab in Pediatric Patients With Atypical Hemolytic-Uremic Syndrome

Baseline characteristics by cohort

Measure	Eculizumab n=22 Participants Eculizumab: Fixed dosing is based on body weight cohorts. Adjustment of dose to accommodate patient growth is possible.
Age, Continuous	6.6 Years STANDARD_DEVIATION 6.06 • n=5 Participants
Sex: Female, Male Female	10 Participants n=5 Participants
Sex: Female, Male Male	12 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	2 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	0 Participants n=5 Participants
Race (NIH/OMB) White	18 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants

PRIMARY outcome

Timeframe: Through 26 weeks

Population: The tabulations of the proportions of patients with Complete TMA Response through 26 weeks were performed for the ITT population. Exact binomial two-sided 95% CI using the Clopper-Pearson method for the responder rate was presented.

Proportion of Patients with Complete TMA response was determined and defined by normalization of hematological parameters (platelet count and LDH) and ≥ 25% improvement in serum creatinine from baseline which was sustained for at least two consecutive measurements obtained at least four weeks apart).

Outcome measures

Measure	Eculizumab n=22 Participants Eculizumab: Fixed dosing is based on body weight cohorts. Adjustment of dose to accommodate patient growth is possible.
Proportion of Patients With Complete TMA Response	63.6 Percentage of Participants Interval 40.7 to 82.8

SECONDARY outcome

Timeframe: Through 26 weeks

Population: The tabulations of the proportions of patients with Complete Hematologic Response through 26 weeks were performed for the ITT population. The number of patients with response and responder rate, along with an exact two-sided 95% CI were summarized.

Proportion of Patients with Complete Hematologic response through 26 weeks of treatment was determined and defined by normalization of platelet count and LDH sustained for at least two consecutive measurements obtained at least four weeks apart.

Outcome measures

Measure	Eculizumab n=22 Participants Eculizumab: Fixed dosing is based on body weight cohorts. Adjustment of dose to accommodate patient growth is possible.
Proportion of Patients With Complete Hematologic Response	81.8 Percentage of Participants Interval 59.7 to 94.8

SECONDARY outcome

Timeframe: Through 26 weeks

Population: The tabulations of the proportions of patients with Platelet Count Normalization through 26 weeks were performed for the ITT population. The number of patients with response and responder rate, along with an exact two-sided 95% CI were summarized.

Proportion of Patients with Platelet Count Normalization through 26 weeks of treatment was determined and defined as the platelet count observed to be ≥ 150 x 109/L on at least two consecutive measurements which span a period of at least four weeks.

Outcome measures

Measure	Eculizumab n=22 Participants Eculizumab: Fixed dosing is based on body weight cohorts. Adjustment of dose to accommodate patient growth is possible.
Proportion of Patients With Platelet Count Normalization	95.5 Percentage of Participants Interval 77.2 to 99.9

SECONDARY outcome

Timeframe: Through 26 weeks

Population: The tabulations of the proportions of patients with Estimated Glomerular Filtration Rate (eGFR) Improvement through 26 weeks were performed for the ITT population. The number of patients with response and responder rate, along with an exact two-sided 95% CI were summarized.

Proportion of Patients with Estimated Glomerular Filtration Rate (eGFR) Improvement was determined and defined as an increase in eGFR by ≥ 15 mL/min/1.73m2 from baseline, sustained for at least two consecutive measurements obtained at least four weeks apart.

Outcome measures

Measure	Eculizumab n=22 Participants Eculizumab: Fixed dosing is based on body weight cohorts. Adjustment of dose to accommodate patient growth is possible.
Proportion of Patients With Estimated Glomerular Filtration Rate (eGFR) Improvement	86.4 Percentage of Participants Interval 65.1 to 97.1

SECONDARY outcome

Timeframe: Through 26 weeks

Population: Change from baseline platelet counts were analyzed for the ITT population using a repeated measures ANOVA model. The estimated LS means of change from baseline at each post-baseline visit alongside with 95% CIs and P-values were calculated, as were the parameter estimates for the covariates and their associated P-values.

Outcome measures

Measure	Eculizumab n=22 Participants Eculizumab: Fixed dosing is based on body weight cohorts. Adjustment of dose to accommodate patient growth is possible.
Platelet Count Change From Baseline to 26 Weeks	204.96 10^9 cells/L Interval 164.44 to 245.49

SECONDARY outcome

Timeframe: Through End of Study, Median Exposure 55 Weeks

Population: The tabulations of the proportions of patients with Complete TMA Response through end of study were performed for the ITT population. Exact binomial two-sided 95% CI using the Clopper-Pearson method for the responder rate was presented.

Proportion of Patients with Complete TMA response was determined and defined by normalization of hematological parameters (platelet count and LDH) and ≥ 25% improvement in serum creatinine from baseline which was sustained for at least two consecutive measurements obtained at least four weeks apart).

Outcome measures

Measure	Eculizumab n=22 Participants Eculizumab: Fixed dosing is based on body weight cohorts. Adjustment of dose to accommodate patient growth is possible.
Proportion of Patients With Complete TMA Response	68.2 Percentage of Participants Interval 45.1 to 86.1

SECONDARY outcome

Timeframe: Through End of Study, Median Exposure 55 Weeks

Population: The tabulations of the proportions of patients with Complete Hematologic Response through end of study were performed for the ITT population. The number of patients with response and responder rate, along with an exact two-sided 95% CI were summarized.

Proportion of Patients with Complete Hematologic response through end of study was determined and defined by normalization of platelet count and LDH sustained for at least two consecutive measurements obtained at least four weeks apart.

Outcome measures

Measure	Eculizumab n=22 Participants Eculizumab: Fixed dosing is based on body weight cohorts. Adjustment of dose to accommodate patient growth is possible.
Proportion of Patients With Complete Hematologic Response	90.9 Percentage of Participants Interval 70.8 to 98.9

SECONDARY outcome

Timeframe: Through End of Study, Median Exposure 55 Weeks

Population: The tabulations of the proportions of patients with Platelet Count Normalization through end of study were performed for the ITT population. The number of patients with response and responder rate, along with an exact two-sided 95% CI were summarized.

Proportion of Patients with Platelet Count Normalization through end of study was determined and defined as the platelet count observed to be ≥ 150 x 109/L on at least two consecutive measurements which span a period of at least four weeks

Outcome measures

Measure	Eculizumab n=22 Participants Eculizumab: Fixed dosing is based on body weight cohorts. Adjustment of dose to accommodate patient growth is possible.
Proportion of Patients With Platelet Count Normalization	95.5 Percentage of Participants Interval 77.2 to 99.9

SECONDARY outcome

Timeframe: Through End of Study, Median Exposure 55 Weeks

Population: The tabulations of the proportions of patients with eGFR Improvement through end of study were performed for the ITT population. The number of patients with response and responder rate, along with an exact two-sided 95% CI were summarized.

Proportion of Patients with Estimated Glomerular Filtration Rate (eGFR) Improvement was determined and defined as an increase in eGFR by ≥ 15 mL/min/1.73m2 from baseline, sustained for at least two consecutive measurements obtained at least four weeks apart.

Outcome measures

Measure	Eculizumab n=22 Participants Eculizumab: Fixed dosing is based on body weight cohorts. Adjustment of dose to accommodate patient growth is possible.
Proportion of Patients With Estimated Glomerular Filtration Rate (eGFR) Improvement	86.4 Percentage of Participants Interval 65.1 to 97.1

SECONDARY outcome

Timeframe: Through 52 Weeks

Population: Change from baseline platelet counts were analyzed for the ITT population using a repeated measures ANOVA model. The estimated LS means of change from baseline at each post-baseline visit alongside with 95% CIs and P-values were calculated, as were the parameter estimates for the covariates and their associated P-values.

Outcome measures

Measure	Eculizumab n=22 Participants Eculizumab: Fixed dosing is based on body weight cohorts. Adjustment of dose to accommodate patient growth is possible.
Platelet Count Change From Baseline to 52 Weeks	165.43 10^9 cells/L Interval 98.43 to 232.46

SECONDARY outcome

Timeframe: Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort.Maintenance Phase was started 1 week after induction phase and dosing of eculizumab administration was every 2 weeks or every 3 weeks depending on patient weight cohort

Population: PK parameters Cmin and Cmax were estimated using a population PK model developed from the observed PK concentration data

Outcome measures

Measure	Eculizumab n=3 Participants Eculizumab: Fixed dosing is based on body weight cohorts. Adjustment of dose to accommodate patient growth is possible.
Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 5 - <10kg) N=3 Min Concentration during induction period	223.2 micrograms/mL Standard Deviation 19.0
Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 5 - <10kg) N=3 Max concentration during induction period	312.8 micrograms/mL Standard Deviation 51.2
Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 5 - <10kg) N=3 Min concentration during maintenance	187.2 micrograms/mL Standard Deviation 42.0
Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 5 - <10kg) N=3 Max concentration during maintenance	499.7 micrograms/mL Standard Deviation 27.8

SECONDARY outcome

Timeframe: Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort.Maintenance Phase was started 1 week after induction phase and dosing of eculizumab administration was every 2 weeks or every 3 weeks depending on patient weight cohort

Population: PK parameters Cmin and Cmax were estimated using a population PK model developed from the observed PK concentration data

Outcome measures

Measure	Eculizumab n=7 Participants Eculizumab: Fixed dosing is based on body weight cohorts. Adjustment of dose to accommodate patient growth is possible.
Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 10 - <20kg) N=7 Min Concentration during induction period	297.2 micrograms/mL Standard Deviation 74.7
Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 10 - <20kg) N=7 Max concentration during induction period	436.3 micrograms/mL Standard Deviation 128.3
Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 10 - <20kg) N=7 Min concentration during maintenance	241.8 micrograms/mL Standard Deviation 106.0
Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 10 - <20kg) N=7 Max concentration during maintenance	459.5 micrograms/mL Standard Deviation 105.5

SECONDARY outcome

Timeframe: Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort. Maintenance Phase was started either 2 weeks or 3 weeks after induction phase depending on patient weight cohort

Population: PK parameters Cmin and Cmax were estimated using a population PK model developed from the observed PK concentration data

Outcome measures

Measure	Eculizumab n=6 Participants Eculizumab: Fixed dosing is based on body weight cohorts. Adjustment of dose to accommodate patient growth is possible.
Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 20 - <30kg) Min Concentration during induction period	185.1 micrograms/mL Standard Deviation 19.5
Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 20 - <30kg) Max concentration during induction period	242.5 micrograms/mL Standard Deviation 24.5
Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 20 - <30kg) Min concentration during maintenance	337.6 micrograms/mL Standard Deviation 43.4
Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 20 - <30kg) Max concentration during maintenance	579.5 micrograms/mL Standard Deviation 66.5

SECONDARY outcome

Timeframe: Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort. Maintenance Phase was started either 2 weeks or 3 weeks after induction phase depending on patient weight cohort

Population: PK parameters Cmin and Cmax were estimated using a population PK model developed from the observed PK concentration data. N=0 in the maintenance phase because the patient changed to ≥40kg body weight category

Outcome measures

Measure	Eculizumab n=1 Participants Eculizumab: Fixed dosing is based on body weight cohorts. Adjustment of dose to accommodate patient growth is possible.
Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 30 - <40kg) N=1 Min Concentration during induction period	196.8 micrograms/mL
Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 30 - <40kg) N=1 Max concentration during induction period	282.2 micrograms/mL
Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 30 - <40kg) N=1 Min concentration during Maintenance	NA micrograms/mL Standard Deviation NA N=6 in the maintenance phase because the patient in the 30 - \<40kg cohort group changed to the ≥40kg body weight category
Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 30 - <40kg) N=1 Max concentration during Maintenance	NA micrograms/mL Standard Deviation NA N=6 in the maintenance phase because the patient in the 30 - \<40kg cohort group changed to the ≥40kg body weight category

SECONDARY outcome

Timeframe: Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort. Maintenance Phase was started either 2 weeks or 3 weeks after induction phase depending on patient weight cohort

Population: PK parameters Cmin and Cmax were estimated using a population PK model developed from the observed PK concentration data.

Outcome measures

Measure	Eculizumab n=5 Participants Eculizumab: Fixed dosing is based on body weight cohorts. Adjustment of dose to accommodate patient growth is possible.
Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort ≥40kg) N=5 Max concentration during induction period	180.9 micrograms/mL Standard Deviation 42.6
Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort ≥40kg) N=5 Min concentration during maintenance	278.4 micrograms/mL Standard Deviation 130.3
Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort ≥40kg) N=5 Max concentration during maintenance	572.8 micrograms/mL Standard Deviation 267.7
Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort ≥40kg) N=5 Min Concentration during induction period	125.5 micrograms/mL Standard Deviation 26.3

Adverse Events

Eculizumab

Serious events: 13 serious events

Other events: 20 other events

Deaths: 0 deaths

Serious adverse events

Measure	Eculizumab n=22 participants at risk Eculizumab: Fixed dosing is based on body weight cohorts. Adjustment of dose to accommodate patient growth is possible.
Blood and lymphatic system disorders Anaemia	9.1% 2/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Blood and lymphatic system disorders Bone marrow failure	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Gastrointestinal disorders Abdominal pain	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
General disorders Device malfunction	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
General disorders Medical device complication	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
General disorders Pyrexia	9.1% 2/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Hepatobiliary disorders Cholecystitis	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations Device related sepsis	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations Enterocolitis viral	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations Gastroenteritis viral	9.1% 2/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations Infection	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations Parainfluenzae virus infection	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations Peritonitis	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations Pharyngitis	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations Pyelonephritis	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations Respiratory syncytial virus infection	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations Tonsillitis	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations Upper respiratory tract infection	9.1% 2/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations Urinary tract infection	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations Viral upper respiratory tract infection	9.1% 2/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Injury, poisoning and procedural complications Overdose	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Injury, poisoning and procedural complications Wrist fracture	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Metabolism and nutrition disorders Metabolic disorder	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Psychiatric disorders Agitation	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Renal and urinary disorders Renal impairment	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Reproductive system and breast disorders Uterine polyp	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Respiratory, thoracic and mediastinal disorders Acute respiratory failure	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Respiratory, thoracic and mediastinal disorders Tonsillar haemorrhage	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Vascular disorders Hypertension	9.1% 2/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)

Other adverse events

Measure	Eculizumab n=22 participants at risk Eculizumab: Fixed dosing is based on body weight cohorts. Adjustment of dose to accommodate patient growth is possible.
Blood and lymphatic system disorders ANAEMIA	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Blood and lymphatic system disorders LEUKOPENIA	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Blood and lymphatic system disorders LYMPHOPENIA	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Blood and lymphatic system disorders NEUTROPENIA	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Blood and lymphatic system disorders LYMPHADENOPATHY	9.1% 2/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Blood and lymphatic system disorders THROMBOCYTOPENIA	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Cardiac disorders BUNDLE BRANCH BLOCK RIGHT	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Cardiac disorders PERICARDIAL EFFUSION	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Ear and labyrinth disorders EAR PAIN	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Endocrine disorders HYPOTHYROIDISM	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Eye disorders EYE DISCHARGE	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Eye disorders ULCERATIVE KERATITIS	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Eye disorders VISION BLURRED	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Gastrointestinal disorders ABDOMINAL DISCOMFORT	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Gastrointestinal disorders ABDOMINAL PAIN	31.8% 7/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Gastrointestinal disorders ABDOMINAL PAIN UPPER	9.1% 2/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Gastrointestinal disorders CONSTIPATION	9.1% 2/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Gastrointestinal disorders DIARRHOEA	36.4% 8/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Gastrointestinal disorders ABDOMINAL DISTENSION	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Gastrointestinal disorders DYSPEPSIA	13.6% 3/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Gastrointestinal disorders DYSPHAGIA	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Gastrointestinal disorders GASTRITIS	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Gastrointestinal disorders VOMITING	31.8% 7/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
General disorders ASTHENIA	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
General disorders APPLICATION SITE ECZEMA	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
General disorders CHEST PAIN	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
General disorders DRUG WITHDRAWAL SYNDROME	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
General disorders FATIGUE	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
General disorders INJECTION SITE RASH	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
General disorders INJECTION SITE HAEMATOMA	9.1% 2/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
General disorders MALAISE	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
General disorders PAIN	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
General disorders OEDEMA PERIPHERAL	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
General disorders PYREXIA	50.0% 11/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations ACUTE TONSILLITIS	9.1% 2/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations BETA HAEMOLYTIC STREPTOCOCCAL INFECTION	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations BRONCHITIS	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations CANDIDURIA	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations CATHETER SITE INFECTION	13.6% 3/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations CONJUNCTIVITIS VIRAL	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations EAR INFECTION	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations ESHERICHIA URINARY TRACT INFECTION	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations FUNGAL INFECTION	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations GASTROENTERITIS	9.1% 2/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations GENITAL CANDIDIASIS	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations INFLUENZA	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations NASOPHARYNGITIS	31.8% 7/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations ORAL FUNGAL INFECTION	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations OTITIS MEDIA	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations PHARYNGITIS	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations PATHOGEN RESISTANCE	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations PYELONEPHRITIS	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations RESPIRATORY TRACT INFECTION VIRAL	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations RHINITIS	22.7% 5/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations SINUSITIS	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations SKIN INFECTION	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations UPPER RESPIRATORY TRACT INFECTION	31.8% 7/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations URINARY TRACT INFECTION	18.2% 4/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations VARICELLA	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations VIRAL INFECTION	9.1% 2/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Injury, poisoning and procedural complications ANIMAL BITE	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Injury, poisoning and procedural complications CONTUSION	9.1% 2/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Injury, poisoning and procedural complications INFUSION RELATED REACTION	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Injury, poisoning and procedural complications JOINT DISLOCATION	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Injury, poisoning and procedural complications SCRATCH	9.1% 2/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Investigations BLOOD BICARBONATE DECREASED	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Investigations ELECTROCARDIOGRAM T WAVE INVERSION	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Investigations ELECTROENCEPHALOGRAM ABNORMAL	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Investigations WEIGHT INCREASED	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Metabolism and nutrition disorders HYPOKALAEMIA	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Metabolism and nutrition disorders IRON DEFICIENCY	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Metabolism and nutrition disorders METABOLIC DISORDER	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Metabolism and nutrition disorders VITAMIN D DEFICIENCY	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Musculoskeletal and connective tissue disorders ARTHRALGIA	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Musculoskeletal and connective tissue disorders BACK PAIN	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Musculoskeletal and connective tissue disorders MUSCLE SPASMS	13.6% 3/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Musculoskeletal and connective tissue disorders MUSCULAR WEAKNESS	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Musculoskeletal and connective tissue disorders MYALGIA	13.6% 3/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Musculoskeletal and connective tissue disorders NECK PAIN	9.1% 2/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Musculoskeletal and connective tissue disorders PAIN IN EXTREMITY	9.1% 2/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Neoplasms benign, malignant and unspecified (incl cysts and polyps) SKIN PAPILLOMA	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Nervous system disorders DEMYELINATING POLYNEUROPATHY	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Nervous system disorders HEADACHE	22.7% 5/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Nervous system disorders SINUS HEADACHE	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Psychiatric disorders FOOD AVERSION	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Psychiatric disorders MOOD ALTERED	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Renal and urinary disorders CALCULUS URINARY	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Renal and urinary disorders DYSURIA	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Renal and urinary disorders PROTEINURIA	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Renal and urinary disorders GLYCOSURIA	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Renal and urinary disorders MICROALBUMINURIA	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Renal and urinary disorders POLYURIA	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Reproductive system and breast disorders VAGINAL HAEMORRHAGE	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Respiratory, thoracic and mediastinal disorders COUGH	40.9% 9/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Respiratory, thoracic and mediastinal disorders DYSPNOEA	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Respiratory, thoracic and mediastinal disorders EPISTAXIS	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Respiratory, thoracic and mediastinal disorders OROPHARYNGEAL PAIN	13.6% 3/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Respiratory, thoracic and mediastinal disorders PLEURAL EFFUSION	9.1% 2/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Respiratory, thoracic and mediastinal disorders RHINORRHOEA	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Respiratory, thoracic and mediastinal disorders TONSILLAR HYPERTROPHY	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Skin and subcutaneous tissue disorders ALOPECIA	13.6% 3/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Skin and subcutaneous tissue disorders DERMATITIS DIAPER	9.1% 2/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Skin and subcutaneous tissue disorders ECZEMA	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Skin and subcutaneous tissue disorders INCREASED TENDENCY TO BRUISE	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Skin and subcutaneous tissue disorders PRURITUS	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Skin and subcutaneous tissue disorders PURPURA	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Skin and subcutaneous tissue disorders RASH	18.2% 4/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Skin and subcutaneous tissue disorders RASH ERYTHEMATOUS	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Skin and subcutaneous tissue disorders RASH PAPULAR	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Skin and subcutaneous tissue disorders SKIN LESION	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Vascular disorders HAEMATOMA	9.1% 2/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Vascular disorders HYPERTENSION	18.2% 4/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Vascular disorders PALLOR	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Immune system disorders DRUG HYPERSENSITIVITY	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations PHARYNGITIS STREPTOCOCCAL	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Infections and infestations VIRAL UPPER RESPIRATORY TRACT INFECTION	9.1% 2/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Injury, poisoning and procedural complications NAIL INJURY	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Injury, poisoning and procedural complications PROCEDURAL PAIN	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Surgical and medical procedures CENTRAL VENOUS CATHETER REMOVAL	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)
Hepatobiliary disorders CHOLECYSTITIS	4.5% 1/22 • Through end of study; exposure to eculizumab in this study extended for a median duration of 12.6 months and ranged from 1 dose to 24.5 months. At every visit, patients were asked a standard non-leading question to elicit any changes in their medical well-being including inquiry about any hospitalization, accidents and new or changed concomitant medication regimens. AEs were also documented from any data collected (e.g. laboratory values, physical examination findings, ECG changes, etc.)

Additional Information

Alexion Pharmaceuticals, Inc.

Alexion Pharmaceuticals, Inc.

Phone: clinicaltrials@alxn.com

Email: clinicaltrials@alxn.com

Results disclosure agreements

• Principal investigator is a sponsor employee Participation in this multicenter study involved a commitment to publish the data from the study in a cooperative publication prior to release of study results on an individual basis.
• Publication restrictions are in place

Restriction type: OTHER