Trial Outcomes & Findings for Stem Cell Transplantation in Individuals With Multiple Myeloma (BMT CTN 0102) (NCT NCT00075829)

NCT ID: NCT00075829

Last Updated: 2021-11-01

Results Overview

Patients are considered a failure for this endpoint if they die or if they progress or relapse.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 710 participants
• Primary outcome timeframe: Year 3
• Results posted on: 2021-11-01

Participant Flow

Participants were enrolled between December 2003 and March 2007 from 37 different transplant centers.

Participant milestones

Measure	Auto-Auto Standard Risk Tandem autologous transplant plus thalidomide/dexamethasone (Thal-Dex) or observation (Obs) for standard risk patients PFS and OS did not differ between the Thal-Dex and the Obs arms and were thus pooled for analysis.	Auto-Allo Standard Risk Autologous transplant plus non-myeloablative allogeneic transplant for standard risk patients	Auto-Auto High Risk Tandem autologous transplant plus thalidomide/dexamethasone (Thal-Dex) or observation (Obs) for high risk patients PFS and OS did not differ between the Thal-Dex and the Obs arms and were thus pooled for analysis.	Auto-Allo High Risk Autologous transplant plus non-myeloablative allogeneic transplant for high risk patients
Overall Study STARTED	436	189	48	37
Overall Study COMPLETED	178	71	14	13
Overall Study NOT COMPLETED	258	118	34	24

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Stem Cell Transplantation in Individuals With Multiple Myeloma (BMT CTN 0102)

Baseline characteristics by cohort

Measure	Auto-Auto Standard Risk n=436 Participants Tandem autologous transplant plus thalidomide/dexamethasone (Thal-Dex) or observation (Obs) for standard risk patients PFS and OS did not differ between the Thal-Dex and the Obs arms and were thus pooled for analysis.	Auto-Allo Standard Risk n=189 Participants Autologous transplant plus non-myeloablative allogeneic transplant for standard risk patients	Auto-Auto High Risk n=48 Participants Tandem autologous transplant plus thalidomide/dexamethasone (Thal-Dex) or observation (Obs) for high risk patients PFS and OS did not differ between the Thal-Dex and the Obs arms and were thus pooled for analysis.	Auto-Allo High Risk n=37 Participants Autologous transplant plus non-myeloablative allogeneic transplant for high risk patients	Total n=710 Participants Total of all reporting groups
Sex: Female, Male Male	260 Participants n=5 Participants	111 Participants n=7 Participants	27 Participants n=5 Participants	21 Participants n=4 Participants	419 Participants n=21 Participants
Race/Ethnicity, Customized African American	77 participants n=5 Participants	18 participants n=7 Participants	8 participants n=5 Participants	3 participants n=4 Participants	106 participants n=21 Participants
Age, Continuous	55 years n=5 Participants	53 years n=7 Participants	57 years n=5 Participants	51 years n=4 Participants	53.2 years n=21 Participants
Sex: Female, Male Female	176 Participants n=5 Participants	78 Participants n=7 Participants	21 Participants n=5 Participants	16 Participants n=4 Participants	291 Participants n=21 Participants
Race/Ethnicity, Customized Caucasian	335 participants n=5 Participants	161 participants n=7 Participants	38 participants n=5 Participants	33 participants n=4 Participants	567 participants n=21 Participants
Race/Ethnicity, Customized Other	24 participants n=5 Participants	10 participants n=7 Participants	2 participants n=5 Participants	1 participants n=4 Participants	37 participants n=21 Participants
Karnofsky Performance score 100 - 90	344 participants n=5 Participants	148 participants n=7 Participants	27 participants n=5 Participants	26 participants n=4 Participants	545 participants n=21 Participants
Karnofsky Performance score < 90	92 participants n=5 Participants	41 participants n=7 Participants	21 participants n=5 Participants	11 participants n=4 Participants	165 participants n=21 Participants
β-2 Microglobulin	2.0 mg/L n=5 Participants	2.0 mg/L n=7 Participants	4.4 mg/L n=5 Participants	3.7 mg/L n=4 Participants	3.025 mg/L n=21 Participants
Durie-Salmon Stages I-II	142 participants n=5 Participants	59 participants n=7 Participants	10 participants n=5 Participants	9 participants n=4 Participants	220 participants n=21 Participants
Durie-Salmon Stage III	294 participants n=5 Participants	130 participants n=7 Participants	38 participants n=5 Participants	28 participants n=4 Participants	490 participants n=21 Participants
Interval from diagnosis to transplantation	7 months n=5 Participants	7 months n=7 Participants	7 months n=5 Participants	7 months n=4 Participants	7 months n=21 Participants
Disease status CR	41 participants n=5 Participants	24 participants n=7 Participants	1 participants n=5 Participants	3 participants n=4 Participants	69 participants n=21 Participants
Disease status Near CR	65 participants n=5 Participants	22 participants n=7 Participants	1 participants n=5 Participants	2 participants n=4 Participants	90 participants n=21 Participants
Disease status Very Good PR	79 participants n=5 Participants	32 participants n=7 Participants	1 participants n=5 Participants	7 participants n=4 Participants	119 participants n=21 Participants
Disease status PR	158 participants n=5 Participants	76 participants n=7 Participants	21 participants n=5 Participants	14 participants n=4 Participants	269 participants n=21 Participants
Disease status MR	31 participants n=5 Participants	17 participants n=7 Participants	10 participants n=5 Participants	3 participants n=4 Participants	61 participants n=21 Participants
Disease status SD	21 participants n=5 Participants	6 participants n=7 Participants	6 participants n=5 Participants	4 participants n=4 Participants	37 participants n=21 Participants
Disease status Not Evaluable	30 participants n=5 Participants	12 participants n=7 Participants	8 participants n=5 Participants	4 participants n=4 Participants	54 participants n=21 Participants
Disease status Unknown	11 participants n=5 Participants	0 participants n=7 Participants	0 participants n=5 Participants	0 participants n=4 Participants	11 participants n=21 Participants

PRIMARY outcome

Timeframe: Year 3

Population: Patients that completed second transplant

Patients are considered a failure for this endpoint if they die or if they progress or relapse.

Outcome measures

Measure	Auto-Auto Standard Risk n=366 Participants Tandem autologous transplant plus thalidomide/dexamethasone or observation for standard risk patients	Auto-Allo Standard Risk n=156 Participants Autologous transplant plus non-myeloablative allogeneic transplant for standard risk patients	Auto-Auto High Risk n=31 Participants Tandem autologous transplant plus thalidomide/dexamethasone or observation for high risk patients	Auto-Allo High Risk n=29 Participants Autologous transplant plus non-myeloablative allogeneic transplant for high risk patients
Progression-Free Survival (PFS)	46 percentage of patients Interval 42.0 to 51.0	43 percentage of patients Interval 36.0 to 51.0	33 percentage of patients Interval 22.0 to 50.0	40 percentage of patients Interval 27.0 to 60.0

SECONDARY outcome

Timeframe: Years 1, 2, and 3

Population: Patients that completed second transplant

The event is death from any cause, patients alive at the time of last observation are considered censored.

Outcome measures

Measure	Auto-Auto Standard Risk n=366 Participants Tandem autologous transplant plus thalidomide/dexamethasone or observation for standard risk patients	Auto-Allo Standard Risk n=156 Participants Autologous transplant plus non-myeloablative allogeneic transplant for standard risk patients	Auto-Auto High Risk Tandem autologous transplant plus thalidomide/dexamethasone or observation for high risk patients	Auto-Allo High Risk Autologous transplant plus non-myeloablative allogeneic transplant for high risk patients
Overall Survival (OS) for Standard Risk 1 year	95 percentage of patients Interval 93.0 to 97.0	91 percentage of patients Interval 87.0 to 95.0	—	—
Overall Survival (OS) for Standard Risk 2 years	89 percentage of patients Interval 86.0 to 92.0	85 percentage of patients Interval 80.0 to 91.0	—	—
Overall Survival (OS) for Standard Risk 3 years	80 percentage of patients Interval 77.0 to 84.0	77 percentage of patients Interval 72.0 to 84.0	—	—

SECONDARY outcome

Timeframe: Year 3

Population: Patients that completed second transplant

The event is death from any cause, patients alive at the time of last observation are considered censored.

Outcome measures

Measure	Auto-Auto Standard Risk n=31 Participants Tandem autologous transplant plus thalidomide/dexamethasone or observation for standard risk patients	Auto-Allo Standard Risk n=29 Participants Autologous transplant plus non-myeloablative allogeneic transplant for standard risk patients	Auto-Auto High Risk Tandem autologous transplant plus thalidomide/dexamethasone or observation for high risk patients	Auto-Allo High Risk Autologous transplant plus non-myeloablative allogeneic transplant for high risk patients
Overall Survival (OS) for High Risk	67 percentage of participants Interval 54.0 to 82.0	59 percentage of participants Interval 45.0 to 78.0	—	—

SECONDARY outcome

Timeframe: Year 3

Population: Patients that completed second transplant

Patients are considered experiencing an event when they progress. Deaths without progression are considered as a competing risk. Patients initiating non-protocol anti-myeloma therapy are considered to have progressed on this protocol.

Outcome measures

Measure	Auto-Auto Standard Risk n=366 Participants Tandem autologous transplant plus thalidomide/dexamethasone or observation for standard risk patients	Auto-Allo Standard Risk n=156 Participants Autologous transplant plus non-myeloablative allogeneic transplant for standard risk patients	Auto-Auto High Risk n=31 Participants Tandem autologous transplant plus thalidomide/dexamethasone or observation for high risk patients	Auto-Allo High Risk n=29 Participants Autologous transplant plus non-myeloablative allogeneic transplant for high risk patients
Cumulative Incidence of Progression/Relapse	50 percentage of patients Interval 46.0 to 55.0	46 percentage of patients Interval 39.0 to 54.0	57 percentage of patients Interval 42.0 to 71.0	38 percentage of patients Interval 22.0 to 54.0

SECONDARY outcome

Timeframe: Year 3

Population: Patients that completed second transplant

TRM is defined as death occurring in a patient from causes other than relapse or progression.

Outcome measures

Measure	Auto-Auto Standard Risk n=366 Participants Tandem autologous transplant plus thalidomide/dexamethasone or observation for standard risk patients	Auto-Allo Standard Risk n=156 Participants Autologous transplant plus non-myeloablative allogeneic transplant for standard risk patients	Auto-Auto High Risk n=31 Participants Tandem autologous transplant plus thalidomide/dexamethasone or observation for high risk patients	Auto-Allo High Risk n=29 Participants Autologous transplant plus non-myeloablative allogeneic transplant for high risk patients
Cumulative Incidence of Treatment Related Mortality (TRM)	4 percentage of participants Interval 2.0 to 5.0	11 percentage of participants Interval 7.0 to 16.0	11 percentage of participants Interval 2.0 to 19.0	22 percentage of participants Interval 8.0 to 35.0

SECONDARY outcome

Timeframe: Year 1

Population: Patients that completed second transplant

Upon recovery from the first autograft, but at least 60 days (preferably between 60-120 days) after the first autograft, patients will receive a second transplant according to treatment assignments.

Outcome measures

Measure	Auto-Auto Standard Risk n=366 Participants Tandem autologous transplant plus thalidomide/dexamethasone or observation for standard risk patients	Auto-Allo Standard Risk n=156 Participants Autologous transplant plus non-myeloablative allogeneic transplant for standard risk patients	Auto-Auto High Risk n=31 Participants Tandem autologous transplant plus thalidomide/dexamethasone or observation for high risk patients	Auto-Allo High Risk n=29 Participants Autologous transplant plus non-myeloablative allogeneic transplant for high risk patients
Interval From First to Second Transplantation	98 days Interval 58.0 to 193.0	105 days Interval 61.0 to 262.0	101 days Interval 63.0 to 155.0	111 days Interval 61.0 to 155.0

SECONDARY outcome

Timeframe: Day 100

Population: GVHD was only assessed on the Auto-Allo arm for standard risk patients that completed second transplant

Incidence and severity of GVHD will be scored according to the BMT clinical trials network Manual of Procedures.

Outcome measures

Measure	Auto-Auto Standard Risk n=156 Participants Tandem autologous transplant plus thalidomide/dexamethasone or observation for standard risk patients	Auto-Allo Standard Risk Autologous transplant plus non-myeloablative allogeneic transplant for standard risk patients	Auto-Auto High Risk Tandem autologous transplant plus thalidomide/dexamethasone or observation for high risk patients	Auto-Allo High Risk Autologous transplant plus non-myeloablative allogeneic transplant for high risk patients
Incidences of Graft Versus Host Disease (GVHD) Grades II-IV	26 percentage of patients Interval 19.0 to 33.0	—	—	—
Incidences of Graft Versus Host Disease (GVHD) Grades III-IV	9 percentage of patients Interval 4.0 to 14.0	—	—	—

SECONDARY outcome

Timeframe: Years 1 and 2

Population: GVHD was only assessed on the Auto-Allo arm for standard risk patients that completed second transplant

Incidence and severity of chronic GVHD will be scored according to the BMT clinical trials network Manual of Procedures.

Outcome measures

Measure	Auto-Auto Standard Risk n=156 Participants Tandem autologous transplant plus thalidomide/dexamethasone or observation for standard risk patients	Auto-Allo Standard Risk Autologous transplant plus non-myeloablative allogeneic transplant for standard risk patients	Auto-Auto High Risk Tandem autologous transplant plus thalidomide/dexamethasone or observation for high risk patients	Auto-Allo High Risk Autologous transplant plus non-myeloablative allogeneic transplant for high risk patients
Incidences of Chronic GVHD 1 year	47 percentage of patients Interval 39.0 to 56.0	—	—	—
Incidences of Chronic GVHD 2 years	54 percentage of patients Interval 46.0 to 63.0	—	—	—

Adverse Events

Auto-Auto Standard Risk

Serious events: 15 serious events

Other events: 1 other events

Deaths: 0 deaths

Auto-Allo Standard Risk

Serious events: 10 serious events

Other events: 1 other events

Deaths: 0 deaths

Auto-Auto High Risk

Serious events: 2 serious events

Other events: 0 other events

Deaths: 0 deaths

Auto-Allo High Risk

Serious events: 5 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	Auto-Auto Standard Risk n=436 participants at risk Tandem autologous transplant plus thalidomide/dexamethasone or observation for standard risk patients	Auto-Allo Standard Risk n=189 participants at risk Autologous transplant plus non-myeloablative allogeneic transplant for standard risk patients	Auto-Auto High Risk n=48 participants at risk Tandem autologous transplant plus thalidomide/dexamethasone or observation for high risk patients	Auto-Allo High Risk n=37 participants at risk Autologous transplant plus non-myeloablative allogeneic transplant for high risk patients
Blood and lymphatic system disorders Thrombocytopenic purpura	0.00% 0/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	2.7% 1/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Cardiac disorders Myocardial infarction	0.23% 1/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Ear and labyrinth disorders Deafness	0.23% 1/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Eye disorders Diplopia	0.00% 0/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.53% 1/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Gastrointestinal disorders Diverticulum	0.00% 0/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.53% 1/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
General disorders Accidental death	0.23% 1/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
General disorders Mucosal inflammation	0.00% 0/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	2.7% 1/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
General disorders Non-cardiac chest pain	0.00% 0/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.53% 1/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Hepatobiliary disorders Cholecystitis	0.23% 1/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Immune system disorders Anaphylactic reaction	0.00% 0/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.53% 1/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Immune system disorders Engraftment syndrome	0.23% 1/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Immune system disorders Graft versus host disease	0.23% 1/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	2.7% 1/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Infections and infestations Appendicitis	0.00% 0/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.53% 1/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Infections and infestations Respiratory syncytial virus infection	0.23% 1/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Infections and infestations Viral cardiomyopathy	0.23% 1/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Injury, poisoning and procedural complications Delayed engraftment	0.00% 0/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.53% 1/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Investigations Fever	0.00% 0/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	2.7% 1/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Investigations Liver function test abnormal	0.00% 0/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.53% 1/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Musculoskeletal and connective tissue disorders Muscle haemorrhage	0.00% 0/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	2.1% 1/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Adenocarcinoma	0.23% 1/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Lymphoproliferative disorder	0.23% 1/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Metastasis	0.23% 1/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Myelodysplastic syndrome	0.23% 1/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Nervous system disorders Cerebrovascular accident	0.00% 0/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	2.7% 1/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Nervous system disorders Convulsion	0.00% 0/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	2.7% 1/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Psychiatric disorders Completed suicide	0.23% 1/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Renal and urinary disorders Renal mass	0.23% 1/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Respiratory, thoracic and mediastinal disorders Acute respiratory distress syndrome	0.00% 0/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.53% 1/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	2.1% 1/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Respiratory, thoracic and mediastinal disorders Organising pneumonia	0.23% 1/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Respiratory, thoracic and mediastinal disorders Pneumonitis	0.00% 0/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.53% 1/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	0.23% 1/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Respiratory, thoracic and mediastinal disorders Respiratory failure	0.00% 0/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.53% 1/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Surgical and medical procedures Cholecystectomy	0.00% 0/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	2.7% 1/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Surgical and medical procedures Gallbladder operation	0.00% 0/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.53% 1/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.

Other adverse events

Measure	Auto-Auto Standard Risk n=436 participants at risk Tandem autologous transplant plus thalidomide/dexamethasone or observation for standard risk patients	Auto-Allo Standard Risk n=189 participants at risk Autologous transplant plus non-myeloablative allogeneic transplant for standard risk patients	Auto-Auto High Risk n=48 participants at risk Tandem autologous transplant plus thalidomide/dexamethasone or observation for high risk patients	Auto-Allo High Risk n=37 participants at risk Autologous transplant plus non-myeloablative allogeneic transplant for high risk patients
Blood and lymphatic system disorders Thrombocytopenia	0.23% 1/436 • Number of events 1 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/189 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Angiosarcoma	0.00% 0/436 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.53% 1/189 • Number of events 1 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/48 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.	0.00% 0/37 • 3-years post-first transplant 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. Only unexpected adverse events were required to be reported through the adverse event system per protocol.

Additional Information

Adam Mendizabal

The EMMES Corporation

Phone: 301-251-1161

Email: amendizabal@EMMES.com

Results disclosure agreements

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