Trial Outcomes & Findings for High-Dose Immunosuppression and Autologous Transplantation for Multiple Sclerosis (HALT MS) Study (NCT NCT00288626)

Last Updated: 2017-09-19

Results Overview

Event-free survival (EFS) is survival without death or disease activity from any one of the following criteria: 1) loss of neurological function, defined as a change in pretransplant Extended Disability Status Scale (EDSS) of \> 0.5. 2) Relapse, defined as the development of a new neurological sign and corresponding symptom, or worsening of an existing neurological sign and symptom, localized to central nervous system white matter, resulting in neurological deficit/disability, and lasting over 48 hours. 3) New lesions on magnetic resonance imaging (MRI), defined as presence of 2 or more independent multiple sclerosis brain lesions detected on MRI 1 year or more after stem cell transplant. Kaplan-Meier estimates of survival probability, with 90% confidence interval based on Greenwood's formula for standard error.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

25 participants

Primary outcome timeframe

5 years

Results posted on

2017-09-19

Participant Flow

36 participants were screened and 25 of those participants were enrolled at 3 sites in the US between August 2006 and August 2009. 24 participants were transplanted with the cellular product between February 2007 and April 2010

Participant milestones

Participant milestones
Measure	HDIT and HCT Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥ 2.0 x 10\^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m\^2, etoposide 200 mg/m\^2 cytarabine 200 mg/m\^2, and melphalan 140 mg/m\^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to \> 500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome.
Overall Study STARTED	24
Overall Study COMPLETED	17
Overall Study NOT COMPLETED	7

Reasons for withdrawal

Reasons for withdrawal
Measure	HDIT and HCT Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥ 2.0 x 10\^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m\^2, etoposide 200 mg/m\^2 cytarabine 200 mg/m\^2, and melphalan 140 mg/m\^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to \> 500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome.
Overall Study Death	3
Overall Study Withdrawal by Subject	4

Baseline Characteristics

High-Dose Immunosuppression and Autologous Transplantation for Multiple Sclerosis (HALT MS) Study

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	HDIT and HCT n=24 Participants Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10\^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m\^2, etoposide 200 mg/m\^2 cytarabine 200 mg/m\^2, and melphalan 140 mg/m\^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to \> 500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome.
Age, Continuous	36.5 years STANDARD_DEVIATION 7.7 • n=5 Participants
Sex: Female, Male Female	16 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	23 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	0 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	23 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	24 participants n=5 Participants
Expanded Disability Status Scale (EDSS) at Baseline	4.4 units on a scale STANDARD_DEVIATION .64 • n=5 Participants
Number of Gadolinium-Enhanced Lesions at Baseline	2.3 Lesions per scan STANDARD_DEVIATION 5.8 • n=5 Participants
T1 Lesion Volume at Baseline	1.2 milliliters STANDARD_DEVIATION 2.7 • n=5 Participants
T2 Lesion Volume at Baseline	11.1 milliliters STANDARD_DEVIATION 13.7 • n=5 Participants
Normalized Brain Volume at Screening	1.6 liters STANDARD_DEVIATION 0.1 • n=5 Participants

PRIMARY outcome

Timeframe: 5 years

Population: Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT).

Outcome measures

Outcome measures
Measure	HDIT and HCT n=24 Participants Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10\^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m\^2, etoposide 200 mg/m\^2 cytarabine 200 mg/m\^2, and melphalan 140 mg/m\^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to \>500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome.
Event-Free Survival Probability During the 5 Years After Transplant	0.692 Probability Interval 0.502 to 0.821

SECONDARY outcome

Timeframe: 3 years

Population: Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT).

Outcome measures

Outcome measures
Measure	HDIT and HCT n=24 Participants Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10\^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m\^2, etoposide 200 mg/m\^2 cytarabine 200 mg/m\^2, and melphalan 140 mg/m\^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to \>500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome.
Event-Free Survival Probability During the 3 Years After Transplant	0.784 Probability Interval 0.601 to 0.89

SECONDARY outcome

Timeframe: From study entry to death, loss to follow-up, or the end of the study, whichever came first, up to 6 years

Population: Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT).

The probability that a participant did not experienced a treatment-related death estimated at 1, 2, 3, 4, and 5 years following transplant via the Kaplan-Meier Method. Greenwood's formula for standard error was used to calculate 90% confidence intervals. Participants that did not experience a treatment-related death were censored at the time of last follow-up. A treatment-related death was defined as death that occurred at any time after study entry and that was possibly, probably, or definitely related to the cellular product or possibly, probably, or definitely related to mobilization of autologous peripheral blood hematopoietic progenitor cells with G-CSF and prednisone or to the high-dose immunosuppressive therapy. There were no treatment-related mortality events in the study.

Outcome measures

Outcome measures
Measure	HDIT and HCT n=24 Participants Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10\^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m\^2, etoposide 200 mg/m\^2 cytarabine 200 mg/m\^2, and melphalan 140 mg/m\^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to \>500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome.
Survival From Treatment-Related Mortality 1 Year Post Transplant	1.0 Probability Interval 1.0 to 1.0
Survival From Treatment-Related Mortality 2 Years Post Transplant	1.0 Probability Interval 1.0 to 1.0
Survival From Treatment-Related Mortality 3 Years Post Transplant	1.0 Probability Interval 1.0 to 1.0
Survival From Treatment-Related Mortality 4 Years Post Transplant	1.0 Probability Interval 1.0 to 1.0
Survival From Treatment-Related Mortality 5 Years Post Transplant	1.0 Probability Interval 1.0 to 1.0

SECONDARY outcome

Timeframe: From study entry to death, loss to follow-up, or the end of the study, whichever came first, up to 6 years

Population: Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT)

The probability that a participant did not experienced a death estimated at 1, 2, 3, 4, and 5 years following transplant via the Kaplan-Meier Method. Greenwood's formula for standard error was used to calculate 90% confidence intervals. Participants that did not die were censored at the time of last follow-up.

Outcome measures

Outcome measures
Measure	HDIT and HCT n=24 Participants Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10\^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m\^2, etoposide 200 mg/m\^2 cytarabine 200 mg/m\^2, and melphalan 140 mg/m\^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to \>500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome.
Overall Survival 1 Year Post Transplant	1.0 Probability Interval 1.0 to 1.0
Overall Survival 2 Years Post Transplant	1.0 Probability Interval 1.0 to 1.0
Overall Survival 3 Years Post Transplant	0.957 Probability Interval 0.794 to 0.991
Overall Survival 4 Years Post Transplant	0.911 Probability Interval 0.742 to 0.971
Overall Survival 5 Years Post Transplant	0.863 Probability Interval 0.683 to 0.945

SECONDARY outcome

Timeframe: From study entry to death, loss to follow-up, or the end of the study, whichever came first, up to 6 years

Population: Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT)

The probability that a participant did not experienced a MS-related death estimated at 1, 2, 3, 4, and 5 years following transplant via the Kaplan-Meier Method. Greenwood's formula for standard error was used to calculate 90% confidence intervals. Participants that did not experience a MS-related death were censored at the time of last follow-up. A MS-related death was defined as death that occurred at any time after study entry and that was possibly, probably, or definitely related to disease progression.

Outcome measures

Outcome measures
Measure	HDIT and HCT n=24 Participants Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10\^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m\^2, etoposide 200 mg/m\^2 cytarabine 200 mg/m\^2, and melphalan 140 mg/m\^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to \>500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome.
Survival From MS-Related Mortality 1 Year Post Transplant	1.0 Probability Interval 1.0 to 1.0
Survival From MS-Related Mortality 2 Years Post Transplant	1.0 Probability Interval 1.0 to 1.0
Survival From MS-Related Mortality 3 Years Post Transplant	0.957 Probability Interval 0.794 to 0.991
Survival From MS-Related Mortality 4 Years Post Transplant	0.957 Probability Interval 0.794 to 0.991
Survival From MS-Related Mortality 5 Years Post Transplant	0.906 Probability Interval 0.728 to 0.97

SECONDARY outcome

Timeframe: From the time of enrollment until completion of the 5-year follow-up, an average of 6 years.

Population: Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT)

Morbidity is the occurrence of NCI Common Terminology Criteria for Adverse Events (CTCAE) v3.0 adverse event grade 3 or higher.

Outcome measures

Outcome measures
Measure	HDIT and HCT n=24 Participants Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10\^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m\^2, etoposide 200 mg/m\^2 cytarabine 200 mg/m\^2, and melphalan 140 mg/m\^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to \>500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome.
Percent of Participants Who Experienced All-Cause Morbidity	100 percentage of participants

SECONDARY outcome

Timeframe: From the time of Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT) to 1 year after HCT.

Population: Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT).

Morbidity is the occurrence of NCI Common Terminology Criteria for Adverse Events (CTCAE) v3.0 adverse event grade 3 or higher.

Outcome measures

Outcome measures
Measure	HDIT and HCT n=24 Participants Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10\^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m\^2, etoposide 200 mg/m\^2 cytarabine 200 mg/m\^2, and melphalan 140 mg/m\^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to \>500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome.
Percent of Participants Who Experienced All-Cause Morbidity Within 12 Months of Post-HCT	95.8 Percentage of Participants

SECONDARY outcome

Timeframe: From time of graft infusion to time of engraftment, up to 6 years

Population: Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT)

Neutrophil engraftment, or neutrophil count recovery, is defined as an Absolute Neutrophil Count (ANC) \> 500/ μL for 2 consecutive measurements on different days. Normal range is 1500 to 8000/μL. Reference: http://www.medicinenet.com

Outcome measures

Outcome measures
Measure	HDIT and HCT n=24 Participants Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10\^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m\^2, etoposide 200 mg/m\^2 cytarabine 200 mg/m\^2, and melphalan 140 mg/m\^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to \>500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome.
Time to Neutrophil Engraftment	10.8 Days Standard Deviation 1.1

SECONDARY outcome

Timeframe: From time of graft infusion to time of engraftment, up to 6 years

Population: Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT)

Platelet engraftment, or platelet count recovery, is defined as Platelets \> 20,000/μL for two consecutive measurements on different days with no platelet transfusions in the preceding 7 days. Normal range is 150,000-450,000/μL. Reference: http://www.hopkinsmedicine.org/heart\_vascular\_institute/clinical\_services/centers\_excellence/womens\_cardiovascular\_health\_center/patient\_information/health\_topics/platelets.html.

Outcome measures

Outcome measures
Measure	HDIT and HCT n=24 Participants Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10\^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m\^2, etoposide 200 mg/m\^2 cytarabine 200 mg/m\^2, and melphalan 140 mg/m\^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to \>500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome.
Time to Platelet Engraftment	18.5 Days Standard Deviation 3.4

SECONDARY outcome

Timeframe: 1, 2, and 4 years after HCT

Population: Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT)

Outcome measures

Outcome measures
Measure	HDIT and HCT n=24 Participants Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10\^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m\^2, etoposide 200 mg/m\^2 cytarabine 200 mg/m\^2, and melphalan 140 mg/m\^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to \>500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome.
Event-Free Survival Probability After Transplant 1 Year Post Transplant	0.958 Probability Interval 0.802 to 0.992
Event-Free Survival Probability After Transplant 2 Years Post Transplant	0.828 Probability Interval 0.65 to 0.92
Event-Free Survival Probability After Transplant 4 Years Post Transplant	0.738 Probability Interval 0.55 to 0.857

SECONDARY outcome

Timeframe: 1 to 5 years after HCT

Population: Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT)

MS progression is measured as number of days from transplant to first Kurtzke's Expanded Disability Status Scale (EDSS) increase of more than 0.5 relative to the baseline measurement. EDSS assesses disability in Multiple Sclerosis patients. Eight functional systems are evaluated: visual, brain stem, pyramidal, cerebellar, sensory, bowel and bladder, cerebral, and ambulation. The overall score ranges from 0 (normal neurological exam) to 10 (death due to MS). Kaplan-Meier estimates of survival probability, with 90% confidence intervals based on Greenwood's formula for standard error.

Outcome measures

Outcome measures
Measure	HDIT and HCT n=24 Participants Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10\^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m\^2, etoposide 200 mg/m\^2 cytarabine 200 mg/m\^2, and melphalan 140 mg/m\^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to \>500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome.
MS Progression-Free Survival Probability After Transplant 1 Year Post Transplant	1.0 Probability Interval 1.0 to 1.0
MS Progression-Free Survival Probability After Transplant 2 Years Post Transplant	0.913 Probability Interval 0.747 to 0.972
MS Progression-Free Survival Probability After Transplant 3 Years Post Transplant	0.913 Probability Interval 0.747 to 0.972
MS Progression-Free Survival Probability After Transplant 4 Years Post Transplant	0.913 Probability Interval 0.747 to 0.972
MS Progression-Free Survival Probability After Transplant 5 Years Post Transplant	0.913 Probability Interval 0.747 to 0.972

SECONDARY outcome

Timeframe: 1 to 5 years after HCT

Population: Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT)

MS disease activity is measured as days from transplant to first occurrence of \>= 2 new MS lesions on Magnetic resonance imaging (MRI) relative to baseline. Kaplan-Meier estimates of survival probability, with 90% confidence intervals based on Greenwood's formula for standard error.

Outcome measures

Outcome measures
Measure	HDIT and HCT n=24 Participants Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10\^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m\^2, etoposide 200 mg/m\^2 cytarabine 200 mg/m\^2, and melphalan 140 mg/m\^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to \>500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome.
MRI Activity-Free Survival Probability After Transplant 1 Year Post Transplant	0.958 Probability Interval 0.802 to 0.992
MRI Activity-Free Survival Probability After Transplant 2 Years Post Transplant	0.958 Probability Interval 0.802 to 0.992
MRI Activity-Free Survival Probability After Transplant 3 Years Post Transplant	0.958 Probability Interval 0.802 to 0.992
MRI Activity-Free Survival Probability After Transplant 4 Years Post Transplant	0.910 Probability Interval 0.739 to 0.971
MRI Activity-Free Survival Probability After Transplant 5 Years Post Transplant	0.863 Probability Interval 0.681 to 0.945

SECONDARY outcome

Timeframe: 1 to 5 years after HCT

Population: Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT)

MS clinical relapse is defined as the development of a new neurological sign and corresponding symptom, or worsening of an existing neurological sign and symptom, localized to central nervous system white matter, resulting in neurological deficit or disability, and lasting over 48 hours. Clinical relapse was determined by the participant's neurologist and was measured as days from transplant to new or worsening neurological symptom relative to baseline. Kaplan-Meier estimates of survival probability, with 90% confidence interval based on Greenwood's formula for standard error.

Outcome measures

Outcome measures
Measure	HDIT and HCT n=24 Participants Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10\^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m\^2, etoposide 200 mg/m\^2 cytarabine 200 mg/m\^2, and melphalan 140 mg/m\^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to \>500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome.
MS Relapse-Free Survival Probability After Transplant 1 Year Post Transplant	0.958 Probability Interval 0.802 to 0.992
MS Relapse-Free Survival Probability After Transplant 2 Years Post Transplant	0.915 Probability Interval 0.752 to 0.973
MS Relapse-Free Survival Probability After Transplant 3 Years Post Transplant	0.869 Probability Interval 0.695 to 0.947
MS Relapse-Free Survival Probability After Transplant 4 Years Post Transplant	0.869 Probability Interval 0.695 to 0.947
MS Relapse-Free Survival Probability After Transplant 5 Years Post Transplant	0.869 Probability Interval 0.695 to 0.947

SECONDARY outcome

Timeframe: 1 to 5 years after HCT

Population: Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT)

Treatment with disease-modifying therapy was measured by the number of days from transplant to the first treatment with an additional disease-modifying therapy. Examples of therapy include interferon beta-1a, glatiramer acetate, natalizumab, alemtuzumab, other immunosuppressive medications, or experimental therapies directed against MS activity. Kaplan-Meier estimates of survival probability, with 90% confidence interval based on Greenwood's formula for standard error.

Outcome measures

Outcome measures
Measure	HDIT and HCT n=24 Participants Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10\^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m\^2, etoposide 200 mg/m\^2 cytarabine 200 mg/m\^2, and melphalan 140 mg/m\^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to \>500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome.
Disease-Modifying Therapy Survival Probability After Transplant 1 Year Post Transplant	1.0 Probability Interval 1.0 to 1.0
Disease-Modifying Therapy Survival Probability After Transplant 2 Years Post Transplant	1.0 Probability Interval 1.0 to 1.0
Disease-Modifying Therapy Survival Probability After Transplant 3 Years Post Transplant	1.0 Probability Interval 1.0 to 1.0
Disease-Modifying Therapy Survival Probability After Transplant 4 Years Post Transplant	1.0 Probability Interval 1.0 to 1.0
Disease-Modifying Therapy Survival Probability After Transplant 5 Years Post Transplant	0.950 Probability Interval 0.767 to 0.99

SECONDARY outcome

Timeframe: 6 months to 5 years after HCT

Population: Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT)

Kurtzke's Expanded Disability Status Scale (EDSS) assesses disability in Multiple Sclerosis patients. Eight functional systems are evaluated: visual, brain stem, pyramidal, cerebellar, sensory, bowel and bladder, cerebral, and ambulation. The overall score ranges from 0 (normal neurological exam) to 10 (death due to MS). Change from baseline was computed as the value at the time point minus the baseline value. A negative value in change from baseline indicates an improvement and a positive value indicates worsening. A change of \> 0.5 in EDSS was a treatment-failure criterion.

Outcome measures

Outcome measures
Measure	HDIT and HCT n=24 Participants Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10\^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m\^2, etoposide 200 mg/m\^2 cytarabine 200 mg/m\^2, and melphalan 140 mg/m\^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to \>500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome.
Change From Baseline in Extended Disability Status Scale (EDSS) 6 Months Post Transplant	-0.3 units on a scale Standard Deviation 1.0
Change From Baseline in Extended Disability Status Scale (EDSS) 1 Year Post Transplant	-0.7 units on a scale Standard Deviation 0.9
Change From Baseline in Extended Disability Status Scale (EDSS) 2 Years Post Transplant	-0.8 units on a scale Standard Deviation 1.1
Change From Baseline in Extended Disability Status Scale (EDSS) 3 Years Post Transplant	-0.8 units on a scale Standard Deviation 1.1
Change From Baseline in Extended Disability Status Scale (EDSS) 4 Years Post Transplant	-0.6 units on a scale Standard Deviation 1.2
Change From Baseline in Extended Disability Status Scale (EDSS) 5 Years Post Transplant	-0.9 units on a scale Standard Deviation 1.0

SECONDARY outcome

Timeframe: 8 weeks to 5 years after HCT

Population: Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT)

Multiple sclerosis disease-related lesions were assessed by gadolinium-enhanced magnetic resonance imaging (MRI). Change from baseline was computed as the value at the time point minus the baseline value. A negative value in change from baseline indicates an improvement and a positive value indicates worsening.

Outcome measures

Outcome measures
Measure	HDIT and HCT n=24 Participants Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10\^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m\^2, etoposide 200 mg/m\^2 cytarabine 200 mg/m\^2, and melphalan 140 mg/m\^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to \>500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome.
Change From Baseline in Number of Gadolinium-Enhanced Lesions 8 Weeks Post Transplant	-2.1 Lesions per scan Standard Deviation 4.9
Change From Baseline in Number of Gadolinium-Enhanced Lesions 6 Months Post Transplant	-2.3 Lesions per scan Standard Deviation 6.0
Change From Baseline in Number of Gadolinium-Enhanced Lesions 1 Year Post Transplant	-2.5 Lesions per scan Standard Deviation 6.1
Change From Baseline in Number of Gadolinium-Enhanced Lesions 2 Years Post Transplant	-2.5 Lesions per scan Standard Deviation 6.1
Change From Baseline in Number of Gadolinium-Enhanced Lesions 3 Years Post Transplant	-1.3 Lesions per scan Standard Deviation 2.2
Change From Baseline in Number of Gadolinium-Enhanced Lesions 4 Years Post Transplant	-2.2 Lesions per scan Standard Deviation 6.7
Change From Baseline in Number of Gadolinium-Enhanced Lesions 5 Years Post Transplant	-2.7 Lesions per scan Standard Deviation 7.0

SECONDARY outcome

Timeframe: 6 Months to 5 years after HCT

Population: Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT)

A T2-weighted magnetic resonance imaging (MRI) scan was used to determine the number of new T2 lesions in the brain relative to Baseline. A value of 0 means that the participant didn't worsen. Values greater than 0 indicate an increase in disease activity from baseline.

Outcome measures

Outcome measures
Measure	HDIT and HCT n=24 Participants Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10\^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m\^2, etoposide 200 mg/m\^2 cytarabine 200 mg/m\^2, and melphalan 140 mg/m\^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to \>500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome.
Number of New T2-Weighted Lesions From Baseline 6 Months Post Transplant	0.2 Lesions per scan Standard Deviation 0.5
Number of New T2-Weighted Lesions From Baseline 1 Year Post Transplant	0.2 Lesions per scan Standard Deviation 0.5
Number of New T2-Weighted Lesions From Baseline 2 Years Post Transplant	0.2 Lesions per scan Standard Deviation 0.5
Number of New T2-Weighted Lesions From Baseline 3 Years Post Transplant	0.1 Lesions per scan Standard Deviation 0.3
Number of New T2-Weighted Lesions From Baseline 4 Years Post Transplant	0.4 Lesions per scan Standard Deviation 1.2
Number of New T2-Weighted Lesions From Baseline 5 Years Post Transplant	0.1 Lesions per scan Standard Deviation 0.3

SECONDARY outcome

Timeframe: 8 weeks to 5 years after HCT

Population: Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT)

A T2-weighted magnetic resonance imaging (MRI) scan was used to assess the volume of T2 lesions in the brain. Change from baseline was computed as the value at the time point minus the baseline value.

Outcome measures

Outcome measures
Measure	HDIT and HCT n=24 Participants Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10\^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m\^2, etoposide 200 mg/m\^2 cytarabine 200 mg/m\^2, and melphalan 140 mg/m\^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to \>500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome.
Change From Baseline in T2-Weighted Lesion Volume 8 Weeks Post Transplant	-0.8 milliliters Standard Deviation 1.9
Change From Baseline in T2-Weighted Lesion Volume 6 Months Post Transplant	-0.7 milliliters Standard Deviation 1.5
Change From Baseline in T2-Weighted Lesion Volume 1 Year Post Transplant	-1.0 milliliters Standard Deviation 1.6
Change From Baseline in T2-Weighted Lesion Volume 2 Years Post Transplant	-1.6 milliliters Standard Deviation 3.0
Change From Baseline in T2-Weighted Lesion Volume 3 Years Post Transplant	-1.9 milliliters Standard Deviation 3.1
Change From Baseline in T2-Weighted Lesion Volume 4 Years Post Transplant	-1.9 milliliters Standard Deviation 3.2
Change From Baseline in T2-Weighted Lesion Volume 5 Years Post Transplant	-2.3 milliliters Standard Deviation 3.6

SECONDARY outcome

Timeframe: 8 weeks to 5 years after HCT

Population: Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT)

A T1-weighted magnetic resonance imaging (MRI) scan was used to assess the volume of T1 lesions in the brain. Change from baseline was computed as the value at the time point minus the baseline value.

Outcome measures

Outcome measures
Measure	HDIT and HCT n=24 Participants Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10\^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m\^2, etoposide 200 mg/m\^2 cytarabine 200 mg/m\^2, and melphalan 140 mg/m\^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to \>500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome.
Change From Baseline in T1-Weighted Lesion Volume 8 Weeks Post Transplant	-0.1 milliliter Standard Deviation 0.4
Change From Baseline in T1-Weighted Lesion Volume 6 Months Post Transplant	0.0 milliliter Standard Deviation 0.4
Change From Baseline in T1-Weighted Lesion Volume 1 Year Post Transplant	0.2 milliliter Standard Deviation 0.4
Change From Baseline in T1-Weighted Lesion Volume 2 Years Post Transplant	0.3 milliliter Standard Deviation 0.7
Change From Baseline in T1-Weighted Lesion Volume 3 Years Post Transplant	0.4 milliliter Standard Deviation 0.6
Change From Baseline in T1-Weighted Lesion Volume 4 Years Post Transplant	0.4 milliliter Standard Deviation 0.7
Change From Baseline in T1-Weighted Lesion Volume 5 Years Post Transplant	0.3 milliliter Standard Deviation 0.7

SECONDARY outcome

Timeframe: 8 weeks to 5 years after HCT

Population: Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT)

Magnetic resonance imaging (MRI) scan techniques measured ventricular volumes and grey and white matter brain volumes. Change from screening was computed as the value at the time point minus the screening value.

Outcome measures

Outcome measures
Measure	HDIT and HCT n=24 Participants Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10\^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m\^2, etoposide 200 mg/m\^2 cytarabine 200 mg/m\^2, and melphalan 140 mg/m\^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to \>500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome.
Percent Change From Screening in Brain Volume 8 Weeks Post Transplant	-0.8 Percent Change Standard Deviation 0.8
Percent Change From Screening in Brain Volume 6 Months Post Transplant	-1.1 Percent Change Standard Deviation 0.9
Percent Change From Screening in Brain Volume 1 Year Post Transplant	-1.2 Percent Change Standard Deviation 1.1
Percent Change From Screening in Brain Volume 2 Years Post Transplant	-1.6 Percent Change Standard Deviation 1.4
Percent Change From Screening in Brain Volume 3 Years Post Transplant	-2.2 Percent Change Standard Deviation 1.4
Percent Change From Screening in Brain Volume 4 Years Post Transplant	-2.0 Percent Change Standard Deviation 1.4
Percent Change From Screening in Brain Volume 5 Years Post Transplant	-2.3 Percent Change Standard Deviation 1.6

Adverse Events

HDIT and HCT

Serious events: 16 serious events

Other events: 25 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	HDIT and HCT n=25 participants at risk Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until \> 2.0 x 10\^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m\^2, etoposide 200 mg/m\^2 cytarabine 200 mg/m\^2, and melphalan 140 mg/m\^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to \> 500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome.
Blood and lymphatic system disorders Leukopenia	8.0% 2/25 • Number of events 3 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Blood and lymphatic system disorders Lymphopenia	12.0% 3/25 • Number of events 3 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Cardiac disorders Atrioventricular block	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Cardiac disorders Cardio-respiratory arrest	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Congenital, familial and genetic disorders Arteriovenous malformation	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Eye disorders Vision blurred	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Gastrointestinal disorders Constipation	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
General disorders Chest pain	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
General disorders Fatigue	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
General disorders Pyrexia	8.0% 2/25 • Number of events 4 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Hepatobiliary disorders Gallbladder obstruction	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Immune system disorders Engraftment syndrome	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Infections and infestations Acinetobacter infection	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Infections and infestations Bacteraemia	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Infections and infestations Cellulitis	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Infections and infestations Chronic sinusitis	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Infections and infestations Epstein-Barr virus infection	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Infections and infestations Meningitis aseptic	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Infections and infestations Pneumonia	4.0% 1/25 • Number of events 2 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Infections and infestations Sepsis	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Infections and infestations Urinary tract infection	4.0% 1/25 • Number of events 2 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Investigations Alanine aminotransferase increased	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Metabolism and nutrition disorders Dehydration	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Metabolism and nutrition disorders Hyperuricaemia	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Metabolism and nutrition disorders Hypokalaemia	8.0% 2/25 • Number of events 2 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Musculoskeletal and connective tissue disorders Back pain	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Musculoskeletal and connective tissue disorders Pain in extremity	8.0% 2/25 • Number of events 2 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Breast cancer in situ	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Nervous system disorders Anoxic encephalopathy	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Nervous system disorders Headache	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Nervous system disorders Hemiparesis	8.0% 2/25 • Number of events 2 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Nervous system disorders Motor dysfunction	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Nervous system disorders Multiple sclerosis	12.0% 3/25 • Number of events 3 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Nervous system disorders Neurological symptom	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Pregnancy, puerperium and perinatal conditions Pregnancy	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Psychiatric disorders Depression	4.0% 1/25 • Number of events 2 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Psychiatric disorders Mania	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Psychiatric disorders Suicide attempt	8.0% 2/25 • Number of events 2 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Respiratory, thoracic and mediastinal disorders Asthma	4.0% 1/25 • Number of events 3 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Respiratory, thoracic and mediastinal disorders Dyspnoea	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Respiratory, thoracic and mediastinal disorders Pneumonitis	4.0% 1/25 • Number of events 2 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	8.0% 2/25 • Number of events 2 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Respiratory, thoracic and mediastinal disorders Respiratory arrest	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Respiratory, thoracic and mediastinal disorders Respiratory failure	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Vascular disorders Deep vein thrombosis	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Vascular disorders Jugular vein thrombosis	4.0% 1/25 • Number of events 1 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.

Other adverse events

Other adverse events
Measure	HDIT and HCT n=25 participants at risk Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until \> 2.0 x 10\^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m\^2, etoposide 200 mg/m\^2 cytarabine 200 mg/m\^2, and melphalan 140 mg/m\^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to \> 500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome.
Blood and lymphatic system disorders Anaemia	16.0% 4/25 • Number of events 5 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Blood and lymphatic system disorders Febrile neutropenia	8.0% 2/25 • Number of events 2 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Blood and lymphatic system disorders Leukopenia	88.0% 22/25 • Number of events 31 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Blood and lymphatic system disorders Lymphopenia	100.0% 25/25 • Number of events 48 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Blood and lymphatic system disorders Neutropenia	92.0% 23/25 • Number of events 27 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Blood and lymphatic system disorders Thrombocytopenia	76.0% 19/25 • Number of events 25 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Endocrine disorders Hypothyroidism	8.0% 2/25 • Number of events 2 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Gastrointestinal disorders Abdominal pain	8.0% 2/25 • Number of events 2 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Gastrointestinal disorders Diarrhoea	20.0% 5/25 • Number of events 5 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Gastrointestinal disorders Gastritis	8.0% 2/25 • Number of events 2 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Gastrointestinal disorders Nausea	40.0% 10/25 • Number of events 10 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
General disorders Chest pain	8.0% 2/25 • Number of events 2 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Infections and infestations Bacteraemia	12.0% 3/25 • Number of events 5 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Infections and infestations Bronchitis	8.0% 2/25 • Number of events 2 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Infections and infestations Catheter site infection	8.0% 2/25 • Number of events 2 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Infections and infestations Cytomegalovirus infection	24.0% 6/25 • Number of events 6 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Infections and infestations Herpes zoster	28.0% 7/25 • Number of events 8 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Infections and infestations Infection	8.0% 2/25 • Number of events 2 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Infections and infestations Influenza	8.0% 2/25 • Number of events 2 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Infections and infestations Pharyngitis	8.0% 2/25 • Number of events 2 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Infections and infestations Sinusitis	20.0% 5/25 • Number of events 7 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Infections and infestations Upper respiratory tract infection	20.0% 5/25 • Number of events 9 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Infections and infestations Urinary tract infection	28.0% 7/25 • Number of events 12 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Injury, poisoning and procedural complications Radius fracture	8.0% 2/25 • Number of events 3 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Investigations Alanine aminotransferase increased	20.0% 5/25 • Number of events 6 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Metabolism and nutrition disorders Hyperglycaemia	16.0% 4/25 • Number of events 5 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Musculoskeletal and connective tissue disorders Neck pain	8.0% 2/25 • Number of events 2 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Musculoskeletal and connective tissue disorders Osteopenia	8.0% 2/25 • Number of events 2 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Nervous system disorders Headache	12.0% 3/25 • Number of events 6 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Nervous system disorders Multiple sclerosis	8.0% 2/25 • Number of events 2 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Nervous system disorders Peripheral sensory neuropathy	8.0% 2/25 • Number of events 2 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Skin and subcutaneous tissue disorders Rash	16.0% 4/25 • Number of events 4 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.
Vascular disorders Hypotension	8.0% 2/25 • Number of events 2 • From the time the participant signed informed consent until the participant completed the 5-year follow-up, an average of 6 years Adverse events were reported for all participants enrolled. One participant in the group did not receive a transplant and was followed for safety only, no other analyses were performed for this participant.

Additional Information

Director, Clinical Research Operations Program

DAIT/NIAID

Phone: 301-594-7669

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place