Trial Outcomes & Findings for Scleroderma Treatment With Autologous Transplant (STAT) Study (NCT NCT01413100)

Last Updated: 2025-01-07

Results Overview

Event Free Survival (EFS) was defined as survival without meeting the protocol defined endpoint of organ injury (kidney injury requiring renal replacement dialysis for \>6 months, sustained LVEF \<30%, or sustained decline of FVC \>20%).

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

21 participants

Primary outcome timeframe

At 5 years

Results posted on

2025-01-07

Participant Flow

Participant milestones

Participant milestones
Measure	Treatment (HDIT Autologous PBSCT) STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells \>= 2.5 x 10\^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or \*plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years. Anti-Thymocyte Globulin: Given IV Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT Cyclophosphamide: Given IV Filgrastim: Given SC Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given PO Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT Plerixafor: Given SC Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies
Overall Study STARTED	21
Overall Study COMPLETED	20
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Treatment (HDIT Autologous PBSCT) STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells \>= 2.5 x 10\^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or \*plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years. Anti-Thymocyte Globulin: Given IV Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT Cyclophosphamide: Given IV Filgrastim: Given SC Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given PO Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT Plerixafor: Given SC Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies
Overall Study Withdrawal by Subject	1

Baseline Characteristics

Scleroderma Treatment With Autologous Transplant (STAT) Study

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (HDIT Autologous PBSCT) n=21 Participants STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells \>= 2.5 x 10\^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or \*plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years. Anti-Thymocyte Globulin: Given IV Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT Cyclophosphamide: Given IV Filgrastim: Given SC Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given PO Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT Plerixafor: Given SC Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies
Age, Categorical <=18 years	1 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	20 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	16 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	19 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=5 Participants
Race (NIH/OMB) Asian	2 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants
Race (NIH/OMB) White	17 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	21 participants n=5 Participants

PRIMARY outcome

Timeframe: At 5 years

Population: 20 participants had data collected for this outcome measure.

Outcome measures

Outcome measures
Measure	Treatment (HDIT Autologous PBSCT) n=20 Participants STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells \>= 2.5 x 10\^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or \*plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years. Anti-Thymocyte Globulin: Given IV Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT Cyclophosphamide: Given IV Filgrastim: Given SC Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given PO Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT Plerixafor: Given SC Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies
EFS of Patients Undergoing Transplant	15 participants

SECONDARY outcome

Timeframe: At 5 years

Defined as any death.

Outcome measures

Outcome measures
Measure	Treatment (HDIT Autologous PBSCT) n=20 Participants STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells \>= 2.5 x 10\^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or \*plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years. Anti-Thymocyte Globulin: Given IV Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT Cyclophosphamide: Given IV Filgrastim: Given SC Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given PO Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT Plerixafor: Given SC Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies
All-cause Mortality	3 participants

SECONDARY outcome

Timeframe: From baseline to year 5

Population: 17 participants were able to be assessed for this outcome measure

By echocardiogram, left ventricle ejection fraction (LVEF) greater than or equal to 30% for greater than or equal to 3 months

Outcome measures

Outcome measures
Measure	Treatment (HDIT Autologous PBSCT) n=17 Participants STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells \>= 2.5 x 10\^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or \*plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years. Anti-Thymocyte Globulin: Given IV Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT Cyclophosphamide: Given IV Filgrastim: Given SC Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given PO Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT Plerixafor: Given SC Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies
The Number of Participants With Stable or Improved LVEF	14 participants

SECONDARY outcome

Timeframe: From baseline to year 5

Population: 17 participants who were alive post-transplant were assessed for this outcome measure

Measured by requiring chronic dialysis greater than or equal to 6 months after transplant or required kidney transplant.

Outcome measures

Outcome measures
Measure	Treatment (HDIT Autologous PBSCT) n=17 Participants STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells \>= 2.5 x 10\^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or \*plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years. Anti-Thymocyte Globulin: Given IV Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT Cyclophosphamide: Given IV Filgrastim: Given SC Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given PO Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT Plerixafor: Given SC Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies
Number of Participants Requiring Dialysis	2 participants

SECONDARY outcome

Timeframe: baseline to year 5

Population: 17 participants who were alive post-transplant were assessed for this outcome measure

Chronic renal dysfunction requiring dialysis greater than or equal to 6 months post transplant or requiring kidney transplant, sustained by echo LVEJF \<30% for at least 3 months post transplant, or sustained greater than or equal to 3 months decrease of FVC on pulmonary function test (PFT) \> 20% post transplant.

Outcome measures

Outcome measures
Measure	Treatment (HDIT Autologous PBSCT) n=17 Participants STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells \>= 2.5 x 10\^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or \*plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years. Anti-Thymocyte Globulin: Given IV Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT Cyclophosphamide: Given IV Filgrastim: Given SC Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given PO Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT Plerixafor: Given SC Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies
The Number of Participants With Disease Progression	5 participants

SECONDARY outcome

Timeframe: From baseline to 5 years

Population: Data were not collected.

UCSD healthcare utilization is a self-report instrument that asks the patient about outpatient and inpatient visits, prescription and non-prescription medications, any surgeries, and major medical expenses during the last 3 months.

Outcome measures

Outcome measures
Measure	Treatment (HDIT Autologous PBSCT) n=20 Participants STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells \>= 2.5 x 10\^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or \*plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years. Anti-Thymocyte Globulin: Given IV Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT Cyclophosphamide: Given IV Filgrastim: Given SC Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given PO Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT Plerixafor: Given SC Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies
The Number of Participants Who Completed ALL Health Care Utilization as Assessed by UCSD Healthcare Utilization Surveys	0 Participants

SECONDARY outcome

Timeframe: Baseline to year 4

Population: 17 participants who were alive post-transplant were assessed for this outcome measure

Scleroderma Health Assessment Questionnaire (SHAQ) is a self reported questionnaire with 8 domains including the following scales: pain, patient global assessment, vascular digital ulcers, lung involvement, and gastrointestinal involvement. The SHAQ is a quality of life measure. Each question is scored from 0 (defined as without difficulty), to 3 (defined as unable to do). Some domains are visual analog scales that are measured first and then changed to the 0-3 scale. The individual scores are combined and divided by 8. A higher score indicates worse functionality and changes in the SHAQ is measured as medium change from baseline. The reported medium change can range from -3 to 3. A negative medium change indicates a better outcome.

Outcome measures

Outcome measures
Measure	Treatment (HDIT Autologous PBSCT) n=17 Participants STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells \>= 2.5 x 10\^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or \*plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years. Anti-Thymocyte Globulin: Given IV Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT Cyclophosphamide: Given IV Filgrastim: Given SC Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given PO Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT Plerixafor: Given SC Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies
The Median SHAQ Score	-1.1 score on a scale Interval -1.6 to -0.6

SECONDARY outcome

Timeframe: From baseline to year 5

Population: All 17 participants who were alive post transplant were assessed for this outcome measure

Outcome measure was assessed as the number of participants who had greater than or equal to 3 months a \> 10% improvement in predicted FVC or a \> 15% improvement in DLCO.

Outcome measures

Outcome measures
Measure	Treatment (HDIT Autologous PBSCT) n=17 Participants STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells \>= 2.5 x 10\^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or \*plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years. Anti-Thymocyte Globulin: Given IV Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT Cyclophosphamide: Given IV Filgrastim: Given SC Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given PO Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT Plerixafor: Given SC Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies
The Number of Participants With Improvement in Pulmonary Function FVC improvement	8 participants
The Number of Participants With Improvement in Pulmonary Function DLCO improvement	7 participants

SECONDARY outcome

Timeframe: Mobilization to Day + 100 post transplant

Infections of grade 3 or above

Outcome measures

Outcome measures
Measure	Treatment (HDIT Autologous PBSCT) n=20 Participants STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells \>= 2.5 x 10\^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or \*plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years. Anti-Thymocyte Globulin: Given IV Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT Cyclophosphamide: Given IV Filgrastim: Given SC Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given PO Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT Plerixafor: Given SC Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies
The Number of Participants With Significant Infectious Complications	6 participants

SECONDARY outcome

Timeframe: Baseline to year 5

deaths without relapse of disease

Outcome measures

Outcome measures
Measure	Treatment (HDIT Autologous PBSCT) n=20 Participants STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells \>= 2.5 x 10\^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or \*plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years. Anti-Thymocyte Globulin: Given IV Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT Cyclophosphamide: Given IV Filgrastim: Given SC Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given PO Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT Plerixafor: Given SC Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies
The Number of Participants With Non-progression Mortality	2 participants

SECONDARY outcome

Timeframe: Baseline to year 5

Outcome measures

Outcome measures
Measure	Treatment (HDIT Autologous PBSCT) n=20 Participants STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells \>= 2.5 x 10\^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or \*plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years. Anti-Thymocyte Globulin: Given IV Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT Cyclophosphamide: Given IV Filgrastim: Given SC Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given PO Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT Plerixafor: Given SC Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies
The Number of Participants Who Survived	17 participants

SECONDARY outcome

Timeframe: Baseline to 1 year post-transplant, grade 3 or higher adverse events

Defined as adverse events (AEs) \>= Grade 3 and assessed by the investigator as 1 of the following: unrelated, unlikely, or possibly related to treatment; probably related to treatment; definitely related to treatment. The number of participants that experienced Grade 3 or higher adverse events is reported.

Outcome measures

Outcome measures
Measure	Treatment (HDIT Autologous PBSCT) n=20 Participants STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells \>= 2.5 x 10\^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or \*plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years. Anti-Thymocyte Globulin: Given IV Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT Cyclophosphamide: Given IV Filgrastim: Given SC Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given PO Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT Plerixafor: Given SC Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies
The Number of Participants Who Had Regimen-related Toxicities	15 participants

SECONDARY outcome

Timeframe: Transplant to year 5

The time in months from transplant to starting new therapy not MMF maintenance for relapse disease.

Outcome measures

Outcome measures
Measure	Treatment (HDIT Autologous PBSCT) n=20 Participants STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells \>= 2.5 x 10\^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or \*plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years. Anti-Thymocyte Globulin: Given IV Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT Cyclophosphamide: Given IV Filgrastim: Given SC Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given PO Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT Plerixafor: Given SC Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies
The Median Time of Initiation of Disease-modifying Antirheumatic Drugs (DMARDS) for Relapse After Transplant	13 months Interval 0.0 to 60.0

SECONDARY outcome

Timeframe: From transplant (day 0) to death or disease progression to year 5

Time to treatment failure is defined as death or initiation of disease modifying antirheumatic drug (DMARD).

Outcome measures

Outcome measures
Measure	Treatment (HDIT Autologous PBSCT) n=20 Participants STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells \>= 2.5 x 10\^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or \*plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years. Anti-Thymocyte Globulin: Given IV Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT Cyclophosphamide: Given IV Filgrastim: Given SC Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given PO Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT Plerixafor: Given SC Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies
Median Time to Treatment Failure	9 months Interval 1.2 to 53.0

SECONDARY outcome

Timeframe: Baseline to Day 90

Defined by death occurring at any time after start of mobilization procedure to day +90 after autologous HCT and definitely or probably resulting from treatment given in the study.

Outcome measures

Outcome measures
Measure	Treatment (HDIT Autologous PBSCT) n=20 Participants STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells \>= 2.5 x 10\^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or \*plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years. Anti-Thymocyte Globulin: Given IV Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT Cyclophosphamide: Given IV Filgrastim: Given SC Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given PO Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT Plerixafor: Given SC Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies
The Number of Participants With Treatment-related Mortality	2 participants

SECONDARY outcome

Timeframe: Baseline to year 5

Population: data were not collected

The first question assesses employment status, type of job for the employed (non-manual, manual or mixed manual/non-manual) and the status of those unemployed (homemaker, retired, student, unable to work due to SSc, unable to work due to non-SSc health problems, or other, i.e. volunteer). The next 3 questions apply only to employed patients and assess absenteeism (full days of work missed due to SSc), presenteeism (days with work productivity reduced by greater than or equal to 50%), and how much SSc interfered with work productivity on a scale of 0-10.

Outcome measures

Outcome measures
Measure	Treatment (HDIT Autologous PBSCT) n=20 Participants STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells \>= 2.5 x 10\^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or \*plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years. Anti-Thymocyte Globulin: Given IV Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT Cyclophosphamide: Given IV Filgrastim: Given SC Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given PO Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT Plerixafor: Given SC Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies
The Number of Participants Who Completed ALL Work Productivity Survey (WPS)	0 Participants

Adverse Events

Treatment (HDIT Autologous PBSCT)

Serious events: 3 serious events

Other events: 13 other events

Deaths: 3 deaths

Serious adverse events

Serious adverse events
Measure	Treatment (HDIT Autologous PBSCT) n=20 participants at risk STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells \>= 2.5 x 10\^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or \*plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years. Anti-Thymocyte Globulin: Given IV Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT Cyclophosphamide: Given IV Filgrastim: Given SC Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given PO Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT Plerixafor: Given SC Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies
Renal and urinary disorders Acute Kidney Injury	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Cardiac disorders Cardiac disorders	5.0% 1/20 • Number of events 2 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
General disorders Multi-system organ failure	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Respiratory, thoracic and mediastinal disorders Respiratory Failure	10.0% 2/20 • Number of events 2 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.

Other adverse events

Other adverse events
Measure	Treatment (HDIT Autologous PBSCT) n=20 participants at risk STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells \>= 2.5 x 10\^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or \*plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years. Anti-Thymocyte Globulin: Given IV Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT Cyclophosphamide: Given IV Filgrastim: Given SC Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given PO Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT Plerixafor: Given SC Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies
Skin and subcutaneous tissue disorders Rash	5.0% 1/20 • Number of events 2 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Skin and subcutaneous tissue disorders ulceration	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Vascular disorders hematoma	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Vascular disorders hypertension	10.0% 2/20 • Number of events 2 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Vascular disorders hypotension	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Vascular disorders pulmonary embolism	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Respiratory, thoracic and mediastinal disorders atelectasis	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Respiratory, thoracic and mediastinal disorders respiratory failure	15.0% 3/20 • Number of events 4 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Respiratory, thoracic and mediastinal disorders hypoxia	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Respiratory, thoracic and mediastinal disorders pulmonary edema	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Renal and urinary disorders Acute Kidney Injury (AKI)	10.0% 2/20 • Number of events 2 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Renal and urinary disorders hematuria	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
General disorders neoplasm, benign	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Nervous system disorders stroke	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Metabolism and nutrition disorders ALT increased	15.0% 3/20 • Number of events 3 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Metabolism and nutrition disorders anorexia	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Metabolism and nutrition disorders hypokalemia	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Investigations Elevated GGT	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Immune system disorders fever	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Gastrointestinal disorders gastric hemorrhage	10.0% 2/20 • Number of events 2 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Gastrointestinal disorders ileus	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Cardiac disorders ventricular tachycardia	10.0% 2/20 • Number of events 3 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Cardiac disorders cardiac arrhythmia's	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Cardiac disorders cardiomyopathy	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Immune system disorders Cytokine Rlease Syndrome	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
General disorders Sepsis	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
General disorders Respiratory Failure	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Respiratory, thoracic and mediastinal disorders Adult Respiratory Distress Syndrome	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
General disorders Cardiomyopathy	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
General disorders Acute KIdney Injury	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Infections and infestations Pneumonia	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Cardiac disorders atrial fibrillation	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Metabolism and nutrition disorders Alk Phos	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Metabolism and nutrition disorders hyperkalemia	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Metabolism and nutrition disorders acidosis	10.0% 2/20 • Number of events 2 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Metabolism and nutrition disorders AST increased	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Metabolism and nutrition disorders creatinine increased	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Metabolism and nutrition disorders hyponatremia	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Metabolism and nutrition disorders Hypophosphatemia	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Respiratory, thoracic and mediastinal disorders pneumonitis	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Vascular disorders renal artery aneurysm	5.0% 1/20 • Number of events 1 • All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant. AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.

Additional Information

Dr. Leona Holmberg

Fred Hutchinson Cancer Center

Phone: 206-667-6447

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place