Trial Outcomes & Findings for Acute Graft-versus-Host Disease Treatment (BMT CTN 0802) (NCT NCT01002742)

Last Updated: 2023-01-04

Results Overview

Success is defined as alive and free of GVHD at day 56 after randomization, all others are considered to be a study failure.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

236 participants

Primary outcome timeframe

Day 56

Results posted on

2023-01-04

Participant Flow

Participants were enrolled from February 17, 2010 to November 11, 2011 from 36 different transplant centers.

Participant milestones

Participant milestones
Measure	Placebo Corticosteroids with placebo	Mycophenolate Mofetil Corticosteroids with Mycophenolate Mofetil
Overall Study STARTED	119	117
Overall Study COMPLETED	119	116
Overall Study NOT COMPLETED	0	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Placebo Corticosteroids with placebo	Mycophenolate Mofetil Corticosteroids with Mycophenolate Mofetil
Overall Study Determined to be ineligible	0	1

Baseline Characteristics

Acute Graft-versus-Host Disease Treatment (BMT CTN 0802)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Placebo n=119 Participants Corticosteroids with placebo	Mycophenolate Mofetil n=116 Participants Corticosteroids with Mycophenolate Mofetil	Total n=235 Participants Total of all reporting groups
Age, Customized Median Age	52.9 years n=5 Participants	54 years n=7 Participants	53.8 years n=5 Participants
Sex: Female, Male Female	41 Participants n=5 Participants	45 Participants n=7 Participants	86 Participants n=5 Participants
Sex: Female, Male Male	78 Participants n=5 Participants	71 Participants n=7 Participants	149 Participants n=5 Participants
Primary Disease Acute myeloid leukemia	47 participants n=5 Participants	41 participants n=7 Participants	88 participants n=5 Participants
Primary Disease Acute lymphoblastic leukemia	14 participants n=5 Participants	16 participants n=7 Participants	30 participants n=5 Participants
Primary Disease Chronic myeloid leukemia	5 participants n=5 Participants	3 participants n=7 Participants	8 participants n=5 Participants
Primary Disease Myelodysplastic syndrome	17 participants n=5 Participants	20 participants n=7 Participants	37 participants n=5 Participants
Primary Disease Lymphoma	17 participants n=5 Participants	17 participants n=7 Participants	34 participants n=5 Participants
Primary Disease Other	19 participants n=5 Participants	19 participants n=7 Participants	38 participants n=5 Participants
Unrelated Donor	72 participants n=5 Participants	66 participants n=7 Participants	138 participants n=5 Participants
Graft Source Bone marrow	16 participants n=5 Participants	23 participants n=7 Participants	39 participants n=5 Participants
Graft Source Peripheral blood stem cell	102 participants n=5 Participants	91 participants n=7 Participants	193 participants n=5 Participants
Graft Source Cord blood	1 participants n=5 Participants	2 participants n=7 Participants	3 participants n=5 Participants
Myeloablative Conditioning	74 participants n=5 Participants	74 participants n=7 Participants	148 participants n=5 Participants
Grade of acute Graft-Vs-Host-Disease (GVHD) at Diagnosis Grade I	16 participants n=5 Participants	11 participants n=7 Participants	27 participants n=5 Participants
Grade of acute Graft-Vs-Host-Disease (GVHD) at Diagnosis Grade II	62 participants n=5 Participants	68 participants n=7 Participants	130 participants n=5 Participants
Grade of acute Graft-Vs-Host-Disease (GVHD) at Diagnosis Grade III	34 participants n=5 Participants	30 participants n=7 Participants	64 participants n=5 Participants
Grade of acute Graft-Vs-Host-Disease (GVHD) at Diagnosis Grade IV	7 participants n=5 Participants	7 participants n=7 Participants	14 participants n=5 Participants
Cutaneous involvement at onset No Rash	36 participants n=5 Participants	31 participants n=7 Participants	67 participants n=5 Participants
Cutaneous involvement at onset Maculopapular Rash, <25% of Body Surface	14 participants n=5 Participants	11 participants n=7 Participants	25 participants n=5 Participants
Cutaneous involvement at onset Maculopapular Rash, 25-50% of Body Surface	21 participants n=5 Participants	27 participants n=7 Participants	48 participants n=5 Participants
Cutaneous involvement at onset Generalized Erythroderma	44 participants n=5 Participants	46 participants n=7 Participants	90 participants n=5 Participants
Cutaneous involvement at onset Erythroderma and Bullae Formation/Desquamation	4 participants n=5 Participants	1 participants n=7 Participants	5 participants n=5 Participants
Upper GI Abnormalities at Diagnosis No Protracted Nausea and Vomiting	82 participants n=5 Participants	85 participants n=7 Participants	167 participants n=5 Participants
Upper GI Abnormalities at Diagnosis Persistent Nausea, Vomiting or Anorexia	37 participants n=5 Participants	31 participants n=7 Participants	68 participants n=5 Participants
Lower GI Abnormalities at Diagnosis No Diarrhea	66 participants n=5 Participants	57 participants n=7 Participants	123 participants n=5 Participants
Lower GI Abnormalities at Diagnosis Diarrhea Less ≤ 500 mL/day	19 participants n=5 Participants	21 participants n=7 Participants	40 participants n=5 Participants
Lower GI Abnormalities at Diagnosis Diarrhea >500 but ≤ 1000 mL/day	14 participants n=5 Participants	21 participants n=7 Participants	35 participants n=5 Participants
Lower GI Abnormalities at Diagnosis Diarrhea >1000 but ≤ 1500 mL/day	9 participants n=5 Participants	8 participants n=7 Participants	17 participants n=5 Participants
Lower GI Abnormalities at Diagnosis Diarrhea >1500 mL/day	11 participants n=5 Participants	7 participants n=7 Participants	18 participants n=5 Participants
Lower GI Abnormalities at Diagnosis Stool with Frank Blood or Melena	0 participants n=5 Participants	2 participants n=7 Participants	2 participants n=5 Participants
Liver Abnormalities at Diagnosis Bilirubin <2 mg/dL	110 participants n=5 Participants	106 participants n=7 Participants	216 participants n=5 Participants
Liver Abnormalities at Diagnosis Bilirubin 2-3 mg/dL	5 participants n=5 Participants	1 participants n=7 Participants	6 participants n=5 Participants
Liver Abnormalities at Diagnosis Bilirubin 3.1-6 mg/dL	4 participants n=5 Participants	6 participants n=7 Participants	10 participants n=5 Participants
Liver Abnormalities at Diagnosis Bilirubin 6.1-15 mg/dL	0 participants n=5 Participants	2 participants n=7 Participants	2 participants n=5 Participants
Liver Abnormalities at Diagnosis Bilirubin >15 mg/dL	0 participants n=5 Participants	1 participants n=7 Participants	1 participants n=5 Participants

PRIMARY outcome

Timeframe: Day 56

Success is defined as alive and free of GVHD at day 56 after randomization, all others are considered to be a study failure.

Outcome measures

Outcome measures
Measure	Placebo n=119 Participants Corticosteroids with placebo	Mycophenolate Mofetil n=116 Participants Corticosteroids with Mycophenolate Mofetil
GVHD-free Survival GVHD free	60 participants	69 participants
GVHD-free Survival Study Failure	59 participants	47 participants

SECONDARY outcome

Timeframe: Days 14, 28, and 56

CR is defined as a score of 0 for the GVHD grading in all evaluable organs.

Outcome measures

Outcome measures
Measure	Placebo n=119 Participants Corticosteroids with placebo	Mycophenolate Mofetil n=116 Participants Corticosteroids with Mycophenolate Mofetil
Percentage of Surviving Participants With Complete Response (CR) Day 14	49.6 percentage of participants	44 percentage of participants
Percentage of Surviving Participants With Complete Response (CR) Day 28	44.5 percentage of participants	46.6 percentage of participants
Percentage of Surviving Participants With Complete Response (CR) Day 56	53.8 percentage of participants	60.3 percentage of participants

SECONDARY outcome

Timeframe: Day 90

Flares are defined as any progression of acute GVHD after an initial response (i.e., earlier CR or PR) that requires re-escalation of steroid dosing, or initiation of additional topical or systemic therapy.

Outcome measures

Outcome measures
Measure	Placebo n=119 Participants Corticosteroids with placebo	Mycophenolate Mofetil n=116 Participants Corticosteroids with Mycophenolate Mofetil
Incidence of GVHD Flares Requiring Increased Therapy	16 participants	8 participants

SECONDARY outcome

Timeframe: Day 56, Day 180 and Day 360 post-treatment

Population: No data collected

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Days 28 and 56

The cumulative steroid dose for each patient will be calculated by adding the doses (end of each week's dose) for each of the first four weeks of treatment, divided by the number of days of survival during this interval. The cumulative steroid dose was calculated for all patients per treatment arm and compared.

Outcome measures

Outcome measures
Measure	Placebo n=119 Participants Corticosteroids with placebo	Mycophenolate Mofetil n=116 Participants Corticosteroids with Mycophenolate Mofetil
Cumulative Steroid Dose Day 28	0.63 mg/kg	0.60 mg/kg
Cumulative Steroid Dose Day 56	0.20 mg/kg	0.17 mg/kg

SECONDARY outcome

Timeframe: Day 56

Outcome measures

Outcome measures
Measure	Placebo n=119 Participants Corticosteroids with placebo	Mycophenolate Mofetil n=116 Participants Corticosteroids with Mycophenolate Mofetil
Incidence of Topical/Non-absorbable Therapy	81 participants	77 participants

SECONDARY outcome

Timeframe: Months 6 and 12

Outcome measures

Outcome measures
Measure	Placebo n=119 Participants Corticosteroids with placebo	Mycophenolate Mofetil n=116 Participants Corticosteroids with Mycophenolate Mofetil
Overall GVHD-free Survival Post-randomization 6 Months	73.4 percentage of participants Interval 64.4 to 80.5	72.0 percentage of participants Interval 62.8 to 79.3
Overall GVHD-free Survival Post-randomization 12 Months	64.7 percentage of participants Interval 55.2 to 72.6	57.8 percentage of participants Interval 48.2 to 66.3

SECONDARY outcome

Timeframe: 12 months post-randomization

Outcome measures

Outcome measures
Measure	Placebo n=119 Participants Corticosteroids with placebo	Mycophenolate Mofetil n=116 Participants Corticosteroids with Mycophenolate Mofetil
Incidence of Chronic GVHD	43.3 percentage of participants Interval 34.2 to 52.4	41.5 percentage of participants Interval 32.3 to 50.7

SECONDARY outcome

Timeframe: 6 Months

Number of participants that experienced at least one infection.

Outcome measures

Outcome measures
Measure	Placebo n=119 Participants Corticosteroids with placebo	Mycophenolate Mofetil n=116 Participants Corticosteroids with Mycophenolate Mofetil
Incidence of Systemic Infections	77 participants	81 participants

SECONDARY outcome

Timeframe: 12 months

Outcome measures

Outcome measures
Measure	Placebo n=119 Participants Corticosteroids with placebo	Mycophenolate Mofetil n=116 Participants Corticosteroids with Mycophenolate Mofetil
Incidence of Epstein-Barr Virus (EBV)-Associated Lymphoma	4 participants	6 participants

SECONDARY outcome

Timeframe: Year 1

Outcome measures

Outcome measures
Measure	Placebo n=119 Participants Corticosteroids with placebo	Mycophenolate Mofetil n=116 Participants Corticosteroids with Mycophenolate Mofetil
Incidence of Cytomegalovirus (CMV) Reactivation	39 percentage of participants	44 percentage of participants

SECONDARY outcome

Timeframe: Year 1

Outcome measures

Outcome measures
Measure	Placebo n=119 Participants Corticosteroids with placebo	Mycophenolate Mofetil n=116 Participants Corticosteroids with Mycophenolate Mofetil
Cumulative Incidence of a Severe/Life-threatening/Fatal Infections	42.9 percentage of participants Interval 33.9 to 52.0	44.5 percentage of participants Interval 35.3 to 53.6

SECONDARY outcome

Timeframe: Year 1

DFS includes death or progression/relapse of malignancy

Outcome measures

Outcome measures
Measure	Placebo n=119 Participants Corticosteroids with placebo	Mycophenolate Mofetil n=116 Participants Corticosteroids with Mycophenolate Mofetil
Disease-Free Survival (DFS) Post-Randomization	63 percentage of participants Interval 53.6 to 71.1	53.9 percentage of participants Interval 44.4 to 62.5

SECONDARY outcome

Timeframe: Year 1

Outcome measures

Outcome measures
Measure	Placebo n=119 Participants Corticosteroids with placebo	Mycophenolate Mofetil n=116 Participants Corticosteroids with Mycophenolate Mofetil
Treatment Related Mortality (TRM)	21.5 percentage of participants Interval 14.0 to 29.0	21.8 percentage of participants Interval 14.2 to 29.4

SECONDARY outcome

Timeframe: Day 56

Population: No data collected

Outcome measures

Outcome data not reported

Adverse Events

Placebo

Serious events: 13 serious events

Other events: 0 other events

Deaths: 0 deaths

Mycophenolate Mofetil

Serious events: 13 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Adverse event data not reported

Additional Information

Adam Mendizabal, PhD

The Emmes Corporation

Phone: 301-251-1161

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Placebo n=119 participants at risk Corticosteroids with placebo	Mycophenolate Mofetil n=116 participants at risk Corticosteroids with Mycophenolate Mofetil
Gastrointestinal disorders Vomiting	0.00% 0/119 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.	0.86% 1/116 • Number of events 1 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Vascular disorders Orthostatic hypotension	0.00% 0/119 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.	0.86% 1/116 • Number of events 1 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Gastrointestinal disorders Diarrhoea	0.00% 0/119 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.	0.86% 1/116 • Number of events 1 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Nervous system disorders Cerebellar ataxia	0.00% 0/119 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.	0.86% 1/116 • Number of events 1 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	0.00% 0/119 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.	0.86% 1/116 • Number of events 1 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Nervous system disorders Syncope	0.00% 0/119 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.	0.86% 1/116 • Number of events 1 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Gastrointestinal disorders Gastrointestinal haemorrhage	0.00% 0/119 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.	0.86% 1/116 • Number of events 1 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Skin and subcutaneous tissue disorders Stevens-Johnson syndrome	0.00% 0/119 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.	0.86% 1/116 • Number of events 1 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Renal and urinary disorders Renal failure	0.00% 0/119 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.	0.86% 1/116 • Number of events 1 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Infections and infestations Sepsis	0.00% 0/119 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.	0.86% 1/116 • Number of events 1 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Investigations Transaminases increased	0.00% 0/119 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.	0.86% 1/116 • Number of events 1 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Lymphoproliferative disorder	0.00% 0/119 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.	0.86% 1/116 • Number of events 1 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Cardiac disorders Atrial fibrillation	0.00% 0/119 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.	0.86% 1/116 • Number of events 1 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Nervous system disorders Reversible posterior leukoencephalopathy syndrome	0.84% 1/119 • Number of events 1 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.	0.00% 0/116 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Injury, poisoning and procedural complications Hip fracture	0.84% 1/119 • Number of events 1 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.	0.00% 0/116 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Infections and infestations Septic shock	1.7% 2/119 • Number of events 2 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.	0.00% 0/116 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Renal and urinary disorders Haematuria	2.5% 3/119 • Number of events 3 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.	0.00% 0/116 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Respiratory, thoracic and mediastinal disorders Respiratory failure	0.84% 1/119 • Number of events 1 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.	0.00% 0/116 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Musculoskeletal and connective tissue disorders Fistula	0.84% 1/119 • Number of events 1 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.	0.00% 0/116 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Acute myeloid leukaemia	0.84% 1/119 • Number of events 1 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.	0.00% 0/116 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Hepatobiliary disorders Hepatic failure	0.84% 1/119 • Number of events 1 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.	0.00% 0/116 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Surgical and medical procedures Vertebroplasty	0.84% 1/119 • Number of events 1 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.	0.00% 0/116 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Blood and lymphatic system disorders Thrombotic thrombocytopenic purpura	0.84% 1/119 • Number of events 1 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.	0.00% 0/116 • 1 year following initiation of therapy. Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.