Trial Outcomes & Findings for A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia (NCT NCT03207009)

NCT ID: NCT03207009

Last Updated: 2024-03-07

Results Overview

TI was defined as a weighted average hemoglobin (Hb) \>= 9 grams per deciliter (g/dL) without any packed red blood cell (pRBC) transfusions for a continuous period of \>= 12 months at any time during the study after drug product infusion.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 19 participants
• Primary outcome timeframe: From 12 to 24 months post-transplant
• Results posted on: 2024-03-07

Participant Flow

The study was conducted at 9 study centers in France, Germany, Greece, Italy, United Kingdom, and United States, of which 8 active centers had enrolled participants from 08 June 2017 to 15 November 2022.

A total of 19 participants were enrolled, of which 18 participants aged <=50 years were treated with LentiGlobin BB305 Drug Product.

Participant milestones

Measure	LentiGlobin BB305 Drug Product Participants \<=50 years of age received a single intravenous (IV) infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kilogram (kg) body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Overall Study STARTED	19
Overall Study Intent-to-Treat (ITT) Population	19
Overall Study Transplant Population (TP)	18
Overall Study COMPLETED	18
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Measure	LentiGlobin BB305 Drug Product Participants \<=50 years of age received a single intravenous (IV) infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kilogram (kg) body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Overall Study Withdrew consent prior to conditioning	1

Baseline Characteristics

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia

Baseline characteristics by cohort

Measure	LentiGlobin BB305 Drug Product n=19 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Age, Continuous	14.8 Years STANDARD_DEVIATION 8.77 • n=5 Participants
Sex: Female, Male Female	9 Participants n=5 Participants
Sex: Female, Male Male	10 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	18 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	8 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	10 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: From 12 to 24 months post-transplant

Population: TP included all participants who received beti-cel. Participant evaluable for TI are defined as participant who have achieved TI, have not achieved TI in their parent study, or completed their parent study.

TI was defined as a weighted average hemoglobin (Hb) >= 9 grams per deciliter (g/dL) without any packed red blood cell (pRBC) transfusions for a continuous period of >= 12 months at any time during the study after drug product infusion.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=18 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Percentage of Participants Who Have Achieved Transfusion Independence (TI)	88.9 Percentage of participants Interval 65.3 to 98.6

SECONDARY outcome

Timeframe: At Month 24 post-transplant

Population: TP included all participants who received beti-cel. Participants evaluable for TI are defined as participants who have achieved TI, have not achieved TI in their parent study, or completed their parent study.

TI was defined as a weighted average hemoglobin (Hb) >= 9 grams per deciliter (g/dL) without any packed red blood cell (pRBC) transfusions for a continuous period of >= 12 months at any time during the study after drug product infusion.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=18 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Percentage of Participants Who Have Achieved Transfusion Independence (TI) at Month 24	88.9 Percentage of participants Interval 65.3 to 98.6

SECONDARY outcome

Timeframe: From start of TI up to Month 24 (actual maximum time frame of up to approximately 25 months due to visit window)

Population: TP included all participants who received beti-cel. Participants evaluable for TI are defined as participants who have achieved TI, have not achieved TI in their parent study, or completed their parent study. Here, "overall number of participants analyzed" signifies those participants who achieved TI. Data are presented through the Month 24 Visit based on calendar dates and including visit windows.

Duration of TI was calculated as the time from the start of TI (i.e. first Hb >=9 with no transfusions in the preceding 60 days) up to the last available Hb at which the TI criteria are still met using Kaplan-Meier methodology.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=16 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Duration of Transfusion Independence (TI)	20.86 Months Interval 13.1 to 25.4

SECONDARY outcome

Timeframe: From drug product infusion to start of TI (up to Month 24 [actual maximum time frame of up to approximately 25 months due to visit window])

Population: TP included all participants who received beti-cel. Participants evaluable for TI are defined as participants who have achieved TI, have not achieved TI in their parent study, or completed their parent study. Here, "overall number of participants analyzed" signifies those participants who achieved TI. Data are presented through the Month 24 Visit based on calendar dates and including visit windows.

Time from drug product infusion to achievement of TI was calculated as the time from drug product infusion to the first hemoglobin at which a participant can be declared as TI (that is to 'start of TI + >= 12 months', dependent on Hb lab schedule).

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=16 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Time From Drug Product Infusion to Achievement of Transfusion Independence (TI)	15.67 Months Interval 14.8 to 24.5

SECONDARY outcome

Timeframe: Timeframe varied by subject. For a given subject, the endpoint was calculated from 60 days after the last pRBC transfusion (so transfused blood did not confound the weighted average calculation) through the Month 24 Visit for that subject.

Population: TP included all participants who received beti-cel. Participants evaluable for TI are defined as participants who have achieved TI, have not achieved TI in their parent study, or completed their parent study. Here, "overall number of participants analyzed" signifies those participants who achieved TI.

Weighted average Hb was defined as the weighted average of Hb values without any pRBC transfusions in the proceeding 60 days for a given subject. Ratio of the time between two Hb values and the time between the first and the last Hb values was used as the weight for calculation.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=16 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Weighted Average Hemoglobin (Hb) During Transfusion Independence (TI)	10.817 gram per deciliter (g/dL) Standard Deviation 1.2535

SECONDARY outcome

Timeframe: From 12 to 24 months post-transplant

Population: TP included all participants who received beti-cel. Participants evaluable for TI are defined as participants who have achieved TI, have not achieved TI in their parent study, or completed their parent study. Here, "overall number of participants analyzed" signifies those participants who were evaluable for this outcome.

TR was defined as demonstration of a 60 percent (%) reduction in the annualized volume of pRBC transfusion requirements (in milliliter per kilogram [mL/kg]) in the post-treatment time period from 12 Months post-drug product infusion through Month 24 compared to the annualized mL/kg pRBC transfusion requirement during the 24 months prior to study enrollment.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=18 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Percentage of Participants Who Meet the Definition of Transfusion Reduction (TR)	94.4 Percentage of participants Interval 72.7 to 99.9

SECONDARY outcome

Timeframe: 12 months post-drug product infusion through Month 24

Population: TP included all participants who received beti-cel.

Percentage of participants with a reduction in the annualized mL/kg pRBCs transfused from 12 months post-drug product infusion through Month 24 (approximately a 12-month period) of at least 50%, 60%, 75%, 90% or 100% compared to the annualized mL/kg pRBC transfusion requirement during the 24 months prior to enrollment.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=18 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Percentage of Participants Who Had a Reduction of At Least 50%, 60%, 75%, 90% or 100% in the Annualized pRBCs Transfusion Volume Reduction at >= 50%	94.4 Percentage of participants Interval 72.7 to 99.9
Percentage of Participants Who Had a Reduction of At Least 50%, 60%, 75%, 90% or 100% in the Annualized pRBCs Transfusion Volume Reduction at >= 60%	94.4 Percentage of participants Interval 72.7 to 99.9
Percentage of Participants Who Had a Reduction of At Least 50%, 60%, 75%, 90% or 100% in the Annualized pRBCs Transfusion Volume Reduction at >= 75%	94.4 Percentage of participants Interval 72.7 to 99.9
Percentage of Participants Who Had a Reduction of At Least 50%, 60%, 75%, 90% or 100% in the Annualized pRBCs Transfusion Volume Reduction at >= 90%	94.4 Percentage of participants Interval 72.7 to 99.9
Percentage of Participants Who Had a Reduction of At Least 50%, 60%, 75%, 90% or 100% in the Annualized pRBCs Transfusion Volume Reduction at 100%	88.9 Percentage of participants Interval 65.3 to 98.6

SECONDARY outcome

Timeframe: From 12 months post-drug product infusion through Month 24

Population: TP included all participants who received beti-cel.

Annualized number of pRBC transfusions from 12 months post-drug product infusion through Month 24 was reported.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=18 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Annualized Number of pRBC Transfusions	0.68 pRBC transfusions per year Standard Deviation 2.701

SECONDARY outcome

Timeframe: From 12 to 24 months post-transplant

Population: TP included all participants who received beti-cel.

Annualized volume of pRBC transfusions from 12 months post-drug product infusion through Month 24 compared to the annualized volume of transfusions during the 24 months prior to enrollment.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=18 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Annualized Volume of pRBC Transfusions	11.589 milliliter/kilogram/year (mL/kg/year) Standard Deviation 47.4080

SECONDARY outcome

Timeframe: From start of drug product infusion up to Month 24

Population: TP included all participants who received beti-cel.

Time from drug product infusion to last pRBC transfusion was reported.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=18 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Time From Drug Product Infusion to Last pRBC Transfusion	0.986 months Interval 0.0 to 23.36

SECONDARY outcome

Timeframe: From last pRBC Transfusion up to Month 24 (actual maximum time frame of up to approximately 27 months due to visit window)

Population: TP included all participants who received beti-cel. Data are presented through the Month 24 Visit based on calendar dates and including visit windows.

Time From Last pRBC Transfusion to the Month 24 was reported.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=18 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Time From Last pRBC Transfusion to 24 Months	23.211 Months Interval 0.16 to 27.47

SECONDARY outcome

Timeframe: 12 months post-drug product infusion through Month 24

Population: TP included all participant who received beti-cel.

The weighted average nadir Hb was defined as the most recent Hb prior to each pRBC transfusion, on the day of transfusion or within 3 days and, if there was a period of more than 60 days without transfusion, all Hb records between Day 61 and last follow-up or next transfusion (inclusive) was included. The weighted average nadir Hb during the period of 12 months post-drug product infusion to Month 24 was compared to the weighted average nadir Hb during the 24 months prior to enrollment.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=18 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Weighted Average Nadir Hemoglobin (Hb)	10.653 g/dL Standard Deviation 1.5096

SECONDARY outcome

Timeframe: At Month 6, 9, 12, 18 and 24

Population: TP included all participant who received beti-cel. Here, "overall number of participants analyzed" signifies those participants who had a least 1 unsupported total Hb measurement during Study HGB-212 and "number analyzed" signifies those participants who had an unsupported total Hb measurement at the specific timepoint.

Unsupported total Hb level was defined as the total Hb measurement level without any acute or chronic pRBC transfusions within 60 days prior to the measurement date.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=17 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Unsupported Total Hb Levels at Month 6, 9, 12, 18 and 24 At Month 6	10.41 g/dL Standard Deviation 1.253
Unsupported Total Hb Levels at Month 6, 9, 12, 18 and 24 At Month 9	10.61 g/dL Standard Deviation 1.338
Unsupported Total Hb Levels at Month 6, 9, 12, 18 and 24 At Month 12	10.65 g/dL Standard Deviation 1.619
Unsupported Total Hb Levels at Month 6, 9, 12, 18 and 24 At Month 18	11.00 g/dL Standard Deviation 1.483
Unsupported Total Hb Levels at Month 6, 9, 12, 18 and 24 At Month 24	10.82 g/dL Standard Deviation 1.580

SECONDARY outcome

Timeframe: At Months 6, 9, 12, 18 and 24

Population: TP included all participants who received beti-cel. Here, "Overall number of participants analyzed" signifies those participants who had a least 1 unsupported total Hb measurement during Study HGB-212 and "number analyzed" signifies those participants who had an unsupported total Hb measurement at the specific timepoint.

Number of participants with unsupported total Hb levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) meeting the thresholds were reported at Months 6, 9, 12, 18 and 24. Participants were evaluable if they had an unsupported total Hb measurement at the specific timepoint, where unsupported total Hb level is defined as the total Hb measurement level without any acute or chronic pRBC transfusions within 60 days prior to the measurement date.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=17 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 12 (>=12 g/dL)	3 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 18 (>=12 g/dL)	3 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 18 (>=13 g/dL)	3 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 18 (>=14 g/dL)	0 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 24 (>=10 g/dL)	10 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 6 (>=10 g/dL)	11 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 6 (>=11 g/dL)	4 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 6 (>=12 g/dL)	3 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 6 (>=13 g/dL)	1 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 6 (>=14 g/dL)	0 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 9 (>=10 g/dL)	13 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 9 (>=11 g/dL)	5 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 9 (>=12 g/dL)	3 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 9 (>=13 g/dL)	1 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 9 (>=14 g/dL)	0 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 12 (>=10 g/dL)	12 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 12 (>=11 g/dL)	6 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 12 (>=13 g/dL)	2 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 12 (>=14 g/dL)	1 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 18 (>=10 g/dL)	13 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 18 (>=11 g/dL)	5 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 24 (>=11 g/dL)	7 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 24 (>=12 g/dL)	3 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 24 (>=13 g/dL)	3 Participants
Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 At Month 24 (>=14 g/dL)	1 Participants

SECONDARY outcome

Timeframe: From 6 to 24 months

Population: TP included all participant who received beti-cel.

Percentage of participants who have not received chelation therapy for at least 6 months following drug product infusion were reported.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=18 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Percentage of Participants Who Have Not Received Chelation Therapy for At Least 6 Months Following Drug Product Infusion	61.1 Percentage of participants

SECONDARY outcome

Timeframe: From last Iron Chelation up to Month 24 (actual maximum time frame of up to approximately 29 months due to visit window)

Population: TP included all participants who received beti-cel. Participants were evaluable for this endpoint if they had not received iron chelation therapy for at least 6 months following drug product infusion. Here, "Overall number of participants analyzed" signifies those participants who were evaluable for this outcome. Data are presented through the Month 24 Visit based on calendar dates and including visit windows.

Time from last iron chelation use to last follow-up to 24 months was reported.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=13 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Time From Last Iron Chelation Use to Last Follow-up	17.81 Months Interval 0.3 to 28.9

SECONDARY outcome

Timeframe: From drug product infusion through Month 24

Population: TP included all participants who received beti-cel.

Therapeutic phlebotomy could be used in lieu of chelation in participants who had Hb consistently >= 11 g/dL and who were no longer receiving regular transfusions, at the discretion of the investigator. Number of participants who used therapeutic phlebotomy post Drug Product infusion for up to Month 24 were reported.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=18 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Number of Participants Who Used Therapeutic Phlebotomy Post Drug Product Infusion	2 Participants

SECONDARY outcome

Timeframe: From drug product infusion through Month 24

Population: TP included all participants who received beti-cel. Here, "Overall number of participants analyzed" signifies those participants who received therapeutic phlebotomy.

Annualized phlebotomy therapy usage (number of procedures per year, calculated from DP infusion through last follow-up) were reported.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=2 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Annualized Phlebotomy Therapy Usage Following Drug Product Infusion	4.22 Number of procedures per year Standard Deviation 3.192

SECONDARY outcome

Timeframe: Baseline, Month 12 and 24

Population: TP included all participants who received beti-cel. Participants were evaluable for TI if they had completed the study (i.e., completed the Month 24 Visit), achieved TI, or did not achieve TI during the study. Here, "number analyzed" signifies those participants who had LIC data available at Baseline, Months 12 and 24.

Change From Baseline in Liver Iron Content by MRI at Months 12 and 24 were reported. Baseline is defined as value closest to but prior to conditioning.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=18 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Change From Baseline in Liver Iron Content by Magnetic Resonance Imaging (MRI) Change at Month 12	3.171 milligram per gram (mg/g) Standard Deviation 5.4134
Change From Baseline in Liver Iron Content by Magnetic Resonance Imaging (MRI) Change at Month 24	1.033 milligram per gram (mg/g) Standard Deviation 4.4407

SECONDARY outcome

Timeframe: Baseline, Months 12 and 24

Population: TP included all participants who received beti-cel. Participants were evaluable for TI if they had completed the study (i.e., completed the Month 24 Visit), achieved TI, or did not achieve TI during the study. Here, "number analyzed" signifies those participants who were evaluable at specific timepoint.

Change From Baseline in Cardiac T2* on MRI from Baseline, Month 12 and 24 was reported.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=18 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Change From Baseline in Cardiac T2* on MRI Change at Month 12	-0.2 milliseconds Interval -27.0 to 7.0
Change From Baseline in Cardiac T2* on MRI Change at Month 24	0.2 milliseconds Interval -33.0 to 12.0

SECONDARY outcome

Timeframe: Baseline, Month 12 and 24

Population: TP included all participants who received beti-cel. Participants are evaluable for TI if they had completed the study (i.e., completed the Month 24 Visit), achieved TI, or did not achieve TI during the study. Here, "number analyzed" signifies those participants who were evaluable at specific timepoint.

Serum ferritin was commonly used for an indirect estimation of body iron stores. Although sensitive, it is not specific for iron overload as it can be elevated in a variety of infectious and inflammatory states, and in the presence of cytolysis. Change from baseline in serum ferritin at Months 12 and 24 was reported.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=18 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Change From Baseline in Serum Ferritin Change at Month 12	87.3 picomole per liter (pmol/L) Standard Deviation 1302.76
Change From Baseline in Serum Ferritin Change at Month 24	-605.2 picomole per liter (pmol/L) Standard Deviation 1716.90

SECONDARY outcome

Timeframe: Baseline, Month 12 and 24

Population: TP included all participants who received beti-cel. Here, "overall number of participants analyzed" signifies those participants who were evaluable for this outcome and "number analyzed" signifies those participants who were evaluable at specific timepoint.

PedsQL GCS designed to measure health-related quality of life in pediatric and adolescents (2-18 years). It encompassed 4 dimensions of functioning (physical [8 items], emotional [5 items], social [5 items], school [3 items]). Age groups: Toddler (2-4 years), Young pediatric (5-7 years), Pediatric (8-12 years), Teens (13-18 years). The questionnaire was also completed by parent/caregiver to assess parents' perceptions of their children's quality of life. The Toddler group consisted of 21 items, using a 5-point Likert scale (0 to 4); all other groups consisted of 23 items, with a 3-point Likert scale (0, 2, 4) for young pediatric, a 5-point Likert scale for pediatric and teens groups. All reported scores were transformed on a scale from 0 to 100 for each domain where 0=100, 1=75, 2=50, 3=25, and 4=0. Higher scores correspond with higher quality of life.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=13 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Total Scores at Months 12 and 24 Parent total score: Change at Month 12	-6.20 Score on a scale Standard Deviation 12.948
Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Total Scores at Months 12 and 24 Parent total score: Change at Month 24	-3.49 Score on a scale Standard Deviation 19.621
Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Total Scores at Months 12 and 24 Patient total score: Change at Month 12	2.96 Score on a scale Standard Deviation 14.857
Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Total Scores at Months 12 and 24 Patient total score: Change at Month 24	4.84 Score on a scale Standard Deviation 11.321

SECONDARY outcome

Timeframe: Baseline, Months 12 and 24

Population: TP included all participants who received beti-cel. Here, "overall number of participants analyzed" signifies those participants who were evaluable for this outcome.

EQ-5D is a validated, standardized, generic instrument that was most widely used preference based health related quality of life questionnaire in cost effectiveness and health technologies assessment. EQ-5D-Y was a version of instrument specifically developed and validated for use by youths aged 12 through 17 years. The EQ-5D-Y visual analog scale (VAS) consisted of a 20-cm vertical VAS, with anchors of 0 (worst imaginable health state) and 100 (best imaginable health state). Respondents were asked to rate their own health state today by drawing a line from a box containing these words to the point on the scale that they felt most accurately reflected their current health state. The VAS was reported (raw data) on a scale of 0-100 where 0= death and 100= perfect health. Higher scores corresponded with higher quality of life.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=7 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Change From Baseline in EuroQol Quality of Life 5-Dimension Youth Scale (EQ-5D-Y) VAS Health Status at Months 12 and 24 Health State: Change at Month 12	0.3 Score on a scale Standard Deviation 11.91
Change From Baseline in EuroQol Quality of Life 5-Dimension Youth Scale (EQ-5D-Y) VAS Health Status at Months 12 and 24 Health State: Change at Month 24	2.0 Score on a scale Standard Deviation 12.33

SECONDARY outcome

Timeframe: Baseline, Months 12 and 24

Population: TP included all participants who received beti-cel. Here, "overall number of participants analyzed" signifies those participants who were evaluable for this outcome.

EQ-5D is a validated, standardized, generic instrument that was most widely used preference based health related quality of life (HRQoL) questionnaire in cost effectiveness and health technologies assessment. Participants age >=18 at time of informed consent were eligible to complete the EQ-5D-3L visual analog scale (VAS) which consisted of a 20-cm vertical VAS, with anchors of 0 (worst imaginable health state) and 100 (best imaginable health state). Respondents were asked to rate their own health state today by drawing a line from a box containing these words to the point on the scale that they felt most accurately reflected their current health state. The VAS was reported (raw data) on a scale of 0-100 where 0= death and 100= perfect health. Negative change in score indicated decrease in quality of life (as measured by EQ5D VAS) from baseline.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=5 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Change From Baseline in EuroQol Quality of Life 5-Dimension Adult Scale (EQ-5D-3L) VAS Heath Status Score at Months 12 and 24 Health State: Change at Month 12	-3.6 Score on a scale Standard Deviation 13.13
Change From Baseline in EuroQol Quality of Life 5-Dimension Adult Scale (EQ-5D-3L) VAS Heath Status Score at Months 12 and 24 Health State: Change at Month 24	-2.4 Score on a scale Standard Deviation 10.50

SECONDARY outcome

Timeframe: Baseline, Months 12 and 24

Population: TP included all participants who received beti-cel. Here, "overall number of participants analyzed" signifies those participants who were evaluable for this outcome.

FACT-BMT is assessed bone marrow transplant related quality of life in adults. Total score was sum of sub-scale scores for 5 domains: Physical Well-Being, Social/Family Well-Being, Emotional Well-Being, Functional Well-Being, and Bone Marrow Transplantation Subscale. Each item scored on a 5-point Likert scale based on participant agreement with each statement: 0 for "not at all," 1 for "a little bit," 2 for "somewhat," 3 for "quite a bit," and 4 for "very much. Reported scores were transformed as follows: After taking into account reverse scores for questions constructed in negative form, subscale score for each domain was calculated by multiplying sum of item scores by number of items in subscale, then dividing by number of items answered. Total score was sum of subscale total added together and ranges from 0-148. Higher scores corresponded with higher quality of life.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=4 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Change From Baseline in Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) Questionnaire Total Score Total Score: Change at Month 12	0.33 Score on a scale Standard Deviation 6.716
Change From Baseline in Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) Questionnaire Total Score Total Score: Change at Month 24	2.58 Score on a scale Standard Deviation 4.246

SECONDARY outcome

Timeframe: Baseline, Months 12 and 24

Population: TP included all participants who received beti-cel. Here, "overall number of participants analyzed" signifies those participants who were evaluable for this outcome.

SF-36 was designed to measure health-related quality of life in adults. The instrument consisted of 36 items that were aggregated into 8 multi-item scales (physical functioning [1=yes, limited a lot, to 3=no, not limited at all], role-physical [1=all of time, to 5=none of time], bodily pain [1=very severe, to 6=none], general health [1=poor, to 5=excellent], vitality [1=none of time, to 5=all of time], social functioning [1=all of time, to 5=none of time], role emotional [1=all of time, to 5=none of time] and mental health [1=all of time, to 5=none of the time]). The 8 scales were summarized as Physical Component Summary (PCS) score (physical functioning, role-physical, bodily pain, general health scales) and Mental Component Summary (MCS) score (vitality, social functioning, role-emotional, mental health scales). Reported summary scores were transformed on a scale from 0-100 (higher scores corresponded with higher quality of life), with change from baseline results being presented.

Outcome measures

Measure	LentiGlobin BB305 Drug Product n=5 Participants Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Change From Baseline in Short Form-36 Health Survey (SF-36), Version 2, Acute (Physical and Mental Component Summary Scores) at Months 12 and 24 Mental Component Summary: Change at Month 12	2.42 Score on a scale Standard Deviation 7.637
Change From Baseline in Short Form-36 Health Survey (SF-36), Version 2, Acute (Physical and Mental Component Summary Scores) at Months 12 and 24 Physical Component Summary: Change at Month 12	-0.89 Score on a scale Standard Deviation 10.289
Change From Baseline in Short Form-36 Health Survey (SF-36), Version 2, Acute (Physical and Mental Component Summary Scores) at Months 12 and 24 Physical Component Summary: Change at Month 24	1.09 Score on a scale Standard Deviation 8.167
Change From Baseline in Short Form-36 Health Survey (SF-36), Version 2, Acute (Physical and Mental Component Summary Scores) at Months 12 and 24 Mental Component Summary: Change at Month 24	2.08 Score on a scale Standard Deviation 7.281

Adverse Events

LentiGlobin BB305 Drug Product

Serious events: 6 serious events

Other events: 19 other events

Deaths: 0 deaths

Serious adverse events

Measure	LentiGlobin BB305 Drug Product n=19 participants at risk Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Cardiac disorders Cardiac failure congestive	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Nervous system disorders Headache	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
General disorders Pyrexia	15.8% 3/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Blood and lymphatic system disorders Neutropenia	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Blood and lymphatic system disorders Febrile neutropenia	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Blood and lymphatic system disorders Thrombocytopenia	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Gastrointestinal disorders Gastric ulcer perforation	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Gastrointestinal disorders Stomatitis	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Reproductive system and breast disorders Bartholin's cyst	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Infections and infestations Soft tissue infection	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Infections and infestations Device related infection	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.

Other adverse events

Measure	LentiGlobin BB305 Drug Product n=19 participants at risk Participants \<=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of \>=5.0 × 10\^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population).
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Focal nodular hyperplasia	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Vascular disorders Hot flush	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Vascular disorders Hypertension	21.1% 4/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Vascular disorders Hypotension	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
General disorders Adverse drug reaction	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
General disorders Application site pain	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
General disorders Application site swelling	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
General disorders Catheter site pain	21.1% 4/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
General disorders Fatigue	15.8% 3/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
General disorders Feeling cold	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
General disorders Injection site reaction	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
General disorders Mucosal inflammation	36.8% 7/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
General disorders Non-cardiac chest pain	10.5% 2/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
General disorders Pain	15.8% 3/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
General disorders Puncture site pain	10.5% 2/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
General disorders Pyrexia	36.8% 7/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Immune system disorders Hypersensitivity	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Reproductive system and breast disorders Vaginal haemorrhage	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Respiratory, thoracic and mediastinal disorders Cough	21.1% 4/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Respiratory, thoracic and mediastinal disorders Dyspnoea	15.8% 3/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Respiratory, thoracic and mediastinal disorders Epistaxis	36.8% 7/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Respiratory, thoracic and mediastinal disorders Hypoxia	21.1% 4/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Respiratory, thoracic and mediastinal disorders Laryngeal pain	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Respiratory, thoracic and mediastinal disorders Pharyngeal inflammation	21.1% 4/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Respiratory, thoracic and mediastinal disorders Pulmonary mass	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	10.5% 2/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Respiratory, thoracic and mediastinal disorders Upper respiratory tract inflammation	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Psychiatric disorders Anxiety	21.1% 4/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Psychiatric disorders Insomnia	10.5% 2/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Investigations Alanine aminotransferase increased	42.1% 8/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Investigations Aspartate aminotransferase increased	36.8% 7/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Investigations Blood bilirubin increased	15.8% 3/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Investigations Blood creatinine increased	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Investigations Blood testosterone decreased	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Investigations C-reactive protein increased	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Investigations Coombs direct test positive	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Investigations Gamma-glutamyltransferase increased	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Investigations Pulmonary function test decreased	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Investigations Transaminases increased	10.5% 2/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Investigations Weight decreased	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Injury, poisoning and procedural complications Contusion	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Injury, poisoning and procedural complications Incision site pain	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Injury, poisoning and procedural complications Post procedural discomfort	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Injury, poisoning and procedural complications Procedural pain	47.4% 9/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Injury, poisoning and procedural complications Skin abrasion	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Injury, poisoning and procedural complications Transfusion reaction	10.5% 2/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Cardiac disorders Cardiac failure congestive	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Cardiac disorders Tachycardia	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Nervous system disorders Ageusia	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Nervous system disorders Dizziness	15.8% 3/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Nervous system disorders Headache	36.8% 7/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Nervous system disorders Paraesthesia	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Nervous system disorders Syncope	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Blood and lymphatic system disorders Anaemia	78.9% 15/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Blood and lymphatic system disorders Febrile neutropenia	57.9% 11/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Blood and lymphatic system disorders Immune thrombocytopenia	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Blood and lymphatic system disorders Leukopenia	31.6% 6/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Blood and lymphatic system disorders Lymphopenia	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Blood and lymphatic system disorders Neutropenia	84.2% 16/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Blood and lymphatic system disorders Neutrophilia	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Blood and lymphatic system disorders Pancytopenia	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Blood and lymphatic system disorders Thrombocytopenia	89.5% 17/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Ear and labyrinth disorders Ear pain	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Eye disorders Chalazion	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Eye disorders Conjunctival haemorrhage	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Eye disorders Dry eye	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Gastrointestinal disorders Abdominal pain	42.1% 8/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Gastrointestinal disorders Abdominal pain upper	15.8% 3/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Gastrointestinal disorders Anal inflammation	10.5% 2/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Gastrointestinal disorders Constipation	21.1% 4/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Gastrointestinal disorders Diarrhoea	26.3% 5/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Gastrointestinal disorders Dyspepsia	10.5% 2/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Gastrointestinal disorders Gingival bleeding	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Gastrointestinal disorders Lip dry	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Gastrointestinal disorders Mouth haemorrhage	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Gastrointestinal disorders Nausea	47.4% 9/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Gastrointestinal disorders Oral pain	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Gastrointestinal disorders Palatal ulcer	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Gastrointestinal disorders Stomatitis	57.9% 11/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Gastrointestinal disorders Vomiting	63.2% 12/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Hepatobiliary disorders Cholelithiasis	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Hepatobiliary disorders Hepatic mass	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Hepatobiliary disorders Hyperbilirubinaemia	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Hepatobiliary disorders Jaundice	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Skin and subcutaneous tissue disorders Alopecia	63.2% 12/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Skin and subcutaneous tissue disorders Dermatitis contact	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Skin and subcutaneous tissue disorders Dry skin	15.8% 3/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Skin and subcutaneous tissue disorders Ecchymosis	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Skin and subcutaneous tissue disorders Erythema	15.8% 3/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Skin and subcutaneous tissue disorders Hidradenitis	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Skin and subcutaneous tissue disorders Intertrigo	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Skin and subcutaneous tissue disorders Petechiae	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Skin and subcutaneous tissue disorders Pruritus	15.8% 3/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Skin and subcutaneous tissue disorders Rash	21.1% 4/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Skin and subcutaneous tissue disorders Rash macular	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Skin and subcutaneous tissue disorders Rash maculo-papular	10.5% 2/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Skin and subcutaneous tissue disorders Skin exfoliation	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Skin and subcutaneous tissue disorders Skin hyperpigmentation	26.3% 5/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Skin and subcutaneous tissue disorders Skin hypopigmentation	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Skin and subcutaneous tissue disorders Urticaria	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Renal and urinary disorders Dysuria	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Renal and urinary disorders Haematuria	10.5% 2/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Renal and urinary disorders Pollakiuria	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Renal and urinary disorders Proteinuria	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Endocrine disorders Delayed puberty	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Endocrine disorders Hypogonadism	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Endocrine disorders Secondary hypogonadism	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Musculoskeletal and connective tissue disorders Arthralgia	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Musculoskeletal and connective tissue disorders Back pain	21.1% 4/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Musculoskeletal and connective tissue disorders Bone pain	26.3% 5/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Musculoskeletal and connective tissue disorders Flank pain	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Musculoskeletal and connective tissue disorders Muscular weakness	10.5% 2/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Musculoskeletal and connective tissue disorders Musculoskeletal pain	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Musculoskeletal and connective tissue disorders Myalgia	10.5% 2/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Musculoskeletal and connective tissue disorders Osteopenia	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Musculoskeletal and connective tissue disorders Pain in extremity	15.8% 3/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Infections and infestations Bronchitis	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Infections and infestations COVID-19	15.8% 3/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Infections and infestations Cystitis	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Infections and infestations Herpes zoster	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Infections and infestations Infection	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Infections and infestations Lice infestation	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Infections and infestations Mucosal infection	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Infections and infestations Nasopharyngitis	21.1% 4/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Infections and infestations Oral herpes	15.8% 3/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Infections and infestations Pyelonephritis	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Infections and infestations Rhinovirus infection	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Infections and infestations Sinusitis	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Infections and infestations Staphylococcal infection	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Infections and infestations Systemic infection	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Infections and infestations Upper respiratory tract infection	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Infections and infestations Varicella zoster virus infection	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Infections and infestations Viral infection	10.5% 2/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Infections and infestations Vulvovaginal candidiasis	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Metabolism and nutrition disorders Decreased appetite	26.3% 5/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Metabolism and nutrition disorders Hyperchloraemia	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Metabolism and nutrition disorders Hyperphosphataemia	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Metabolism and nutrition disorders Hypocalcaemia	42.1% 8/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Metabolism and nutrition disorders Hypokalaemia	10.5% 2/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Metabolism and nutrition disorders Hypomagnesaemia	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Metabolism and nutrition disorders Hyponatraemia	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Metabolism and nutrition disorders Hypophosphataemia	10.5% 2/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Metabolism and nutrition disorders Metabolic acidosis	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Metabolism and nutrition disorders Vitamin K deficiency	5.3% 1/19 • From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects). ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.

Additional Information

Study Medical Director

bluebird bio, Inc

Phone: +1-833-999-6378

Email: clinicaltrials@bluebirdbio.com

Results disclosure agreements

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