Trial Outcomes & Findings for Allogeneic Hematopoietic Cell Transplantation for Severe Systemic Sclerosis (NCT NCT00622895)

Results Overview

The events will be defined as any one of the following: death; respiratory failure; renal failure, as defined by chronic dialysis \> or = 6 months or kidney transplantation; occurrence of cardiomyopathy, confirmed by clinical CHF (New York Class III or IV) or LVEF \< 30% by echocardiogram, sustained for at least 3 months despite therapy; organ dysfunction specific events must be documented on at least two occasions \> or = 3 months apart, or sustained for a 3-month period (documented from the first occurrence).

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

3 participants

Primary outcome timeframe

2 years

Results posted on

2018-06-04

Participant Flow

Participant milestones

Participant milestones
Measure	Treatment: Allogeneic HCT After Reduced Intensity Conditioning Conditioning regimen * Day -6: cyclophosphamide 50mg/kg. MESNA will be given for bladder prophylaxis . * Day -6, -5, -4: Horse ATG 30mg/kg IV x 3 days. * Days -6, -5, -4, -3 and -2: Fludarabine 40 mg/m2/day IV over one hour x 5 days. * Day -1: TBI 200 cGy at 6-7 cGy/min from a linear accelerator TBI. * Day 0: UCB infusion ( 2 units) Day -3: Commence tacrolimus at 0.03 mg/kg/day continuous IV infusion until the patient is tolerating oral intake then convert to oral PO b.i.d., continue to day +180 and taper to day +365. Day 0: After UCB transplant on day 0, mycophenolate mofetil will be given 1gm IV t.i.d. until the patient is tolerating oral intake and can convert to 1 gram PO T.I.D. Stop MMF at Day +30, if no acute GVHD, until day +100, and then taper 11% week over 8 weeks.
Overall Study STARTED	3
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Allogeneic Hematopoietic Cell Transplantation for Severe Systemic Sclerosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment: Allogeneic HCT After Reduced Intensity Conditioning n=3 Participants Conditioning regimen * Day -6: cyclophosphamide 50mg/kg. MESNA will be given for bladder prophylaxis . * Day -6, -5, -4: Horse ATG 30mg/kg IV x 3 days. * Days -6, -5, -4, -3 and -2: Fludarabine 40 mg/m2/day IV over one hour x 5 days. * Day -1: TBI 200 cGy at 6-7 cGy/min from a linear accelerator TBI. * Day 0: UCB infusion ( 2 units) Day -3: Commence tacrolimus at 0.03 mg/kg/day continuous IV infusion until the patient is tolerating oral intake then convert to oral PO b.i.d., continue to day +180 and taper to day +365. Day 0: After UCB transplant on day 0, mycophenolate mofetil will be given 1gm IV t.i.d. until the patient is tolerating oral intake and can convert to 1 gram PO T.I.D. Stop MMF at Day +30, if no acute GVHD, until day +100, and then taper 11% week over 8 weeks.
Age, Categorical <=18 years	0 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=93 Participants
Age, Categorical >=65 years	0 Participants n=93 Participants
Age, Continuous	44 years n=93 Participants
Sex: Female, Male Female	2 Participants n=93 Participants
Sex: Female, Male Male	1 Participants n=93 Participants
Region of Enrollment United States	3 participants n=93 Participants

PRIMARY outcome

Timeframe: 2 years

Population: UCB transplant recipients

The events will be defined as any one of the following: death; respiratory failure; renal failure, as defined by chronic dialysis \> or = 6 months or kidney transplantation; occurrence of cardiomyopathy, confirmed by clinical CHF (New York Class III or IV) or LVEF \< 30% by echocardiogram, sustained for at least 3 months despite therapy; organ dysfunction specific events must be documented on at least two occasions \> or = 3 months apart, or sustained for a 3-month period (documented from the first occurrence).

Outcome measures

Outcome measures
Measure	Treatment: Allogeneic HCT After Reduced Intensity Conditioning n=3 Participants Patients receive fludarabine phosphate IV on days -4, -3 and -2, cyclophosphamide IV over 1-2 hours on days -6, -5, 3, and 4, and undergo low-dose TBI on day -1. Patients receive bone marrow transplantation on day 0. Patients then receive cyclophosphamide IV on days +3 and +4, and beginning day +5 they start tacrolimus orally (PO) and mycophenolic acid. fludarabine phosphate: Given IV Mycophenolic Acid: Given PO tacrolimus: Given PO total-body irradiation: Undergo TBI bone marrow transplantation: Undergo transplantation reduced intensity allogeneic hematopoietic stem cell transplantation: Undergo transplantation quality-of-life assessment: Ancillary studies laboratory biomarker analysis: Correlative studies flow cytometry: Correlative studies biopsy: Punch biopsy of skin involved with scleroderma cyclophosphamide: Given IV
Event-free Survival (EFS)	1 Participants

SECONDARY outcome

Timeframe: 5 years

event-free survival after umbilical cord blood transplant

Outcome measures

Outcome measures
Measure	Treatment: Allogeneic HCT After Reduced Intensity Conditioning n=3 Participants Patients receive fludarabine phosphate IV on days -4, -3 and -2, cyclophosphamide IV over 1-2 hours on days -6, -5, 3, and 4, and undergo low-dose TBI on day -1. Patients receive bone marrow transplantation on day 0. Patients then receive cyclophosphamide IV on days +3 and +4, and beginning day +5 they start tacrolimus orally (PO) and mycophenolic acid. fludarabine phosphate: Given IV Mycophenolic Acid: Given PO tacrolimus: Given PO total-body irradiation: Undergo TBI bone marrow transplantation: Undergo transplantation reduced intensity allogeneic hematopoietic stem cell transplantation: Undergo transplantation quality-of-life assessment: Ancillary studies laboratory biomarker analysis: Correlative studies flow cytometry: Correlative studies biopsy: Punch biopsy of skin involved with scleroderma cyclophosphamide: Given IV
EFS	1 Participants

SECONDARY outcome

Timeframe: Up to 5 years

Population: survival of patients after UCB

Event is defined as death due to any cause.

Outcome measures

Outcome measures
Measure	Treatment: Allogeneic HCT After Reduced Intensity Conditioning n=3 Participants Patients receive fludarabine phosphate IV on days -4, -3 and -2, cyclophosphamide IV over 1-2 hours on days -6, -5, 3, and 4, and undergo low-dose TBI on day -1. Patients receive bone marrow transplantation on day 0. Patients then receive cyclophosphamide IV on days +3 and +4, and beginning day +5 they start tacrolimus orally (PO) and mycophenolic acid. fludarabine phosphate: Given IV Mycophenolic Acid: Given PO tacrolimus: Given PO total-body irradiation: Undergo TBI bone marrow transplantation: Undergo transplantation reduced intensity allogeneic hematopoietic stem cell transplantation: Undergo transplantation quality-of-life assessment: Ancillary studies laboratory biomarker analysis: Correlative studies flow cytometry: Correlative studies biopsy: Punch biopsy of skin involved with scleroderma cyclophosphamide: Given IV
Overall Survival	1 Participants

SECONDARY outcome

Timeframe: From time of transplant to 5 years

Defined as death occurring at any time after start of allogeneic HCT and definitely or probably resulting from treatment given in the study and not associated with disease progression.

Outcome measures

Outcome measures
Measure	Treatment: Allogeneic HCT After Reduced Intensity Conditioning n=3 Participants Patients receive fludarabine phosphate IV on days -4, -3 and -2, cyclophosphamide IV over 1-2 hours on days -6, -5, 3, and 4, and undergo low-dose TBI on day -1. Patients receive bone marrow transplantation on day 0. Patients then receive cyclophosphamide IV on days +3 and +4, and beginning day +5 they start tacrolimus orally (PO) and mycophenolic acid. fludarabine phosphate: Given IV Mycophenolic Acid: Given PO tacrolimus: Given PO total-body irradiation: Undergo TBI bone marrow transplantation: Undergo transplantation reduced intensity allogeneic hematopoietic stem cell transplantation: Undergo transplantation quality-of-life assessment: Ancillary studies laboratory biomarker analysis: Correlative studies flow cytometry: Correlative studies biopsy: Punch biopsy of skin involved with scleroderma cyclophosphamide: Given IV
Treatment-related Mortality	2 Participants

SECONDARY outcome

Timeframe: Up to 5 years

Population: 3 patients received umbilical cord blood (UCB) transplant.

Grades 3, 4 and 5 adverse events will be tracked from the start of mobilization or conditioning until day +100 after transplant or until patient departure from the center, whichever occurs first. Certain adverse events are usual and expected after transplant and will only be reported if they are \> Grade 4. Some Grade 4 events that are routinely expected (i.e. pancytopenia) will not be reported.

Outcome measures

Outcome measures
Measure	Treatment: Allogeneic HCT After Reduced Intensity Conditioning n=3 Participants Patients receive fludarabine phosphate IV on days -4, -3 and -2, cyclophosphamide IV over 1-2 hours on days -6, -5, 3, and 4, and undergo low-dose TBI on day -1. Patients receive bone marrow transplantation on day 0. Patients then receive cyclophosphamide IV on days +3 and +4, and beginning day +5 they start tacrolimus orally (PO) and mycophenolic acid. fludarabine phosphate: Given IV Mycophenolic Acid: Given PO tacrolimus: Given PO total-body irradiation: Undergo TBI bone marrow transplantation: Undergo transplantation reduced intensity allogeneic hematopoietic stem cell transplantation: Undergo transplantation quality-of-life assessment: Ancillary studies laboratory biomarker analysis: Correlative studies flow cytometry: Correlative studies biopsy: Punch biopsy of skin involved with scleroderma cyclophosphamide: Given IV
Regimen-related Toxicity (Greater Than or Equal to Grade III) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) Version 3.0 Renal failure	1 Participants
Regimen-related Toxicity (Greater Than or Equal to Grade III) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) Version 3.0 Diffuse alveolar hemorrhage	1 Participants
Regimen-related Toxicity (Greater Than or Equal to Grade III) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) Version 3.0 Pneumonitis	1 Participants

SECONDARY outcome

Timeframe: Up to 5 years

The percent of participants with definite and probable viral, fungal, and bacterial infections after transplant

Outcome measures

Outcome measures
Measure	Treatment: Allogeneic HCT After Reduced Intensity Conditioning n=3 Participants Patients receive fludarabine phosphate IV on days -4, -3 and -2, cyclophosphamide IV over 1-2 hours on days -6, -5, 3, and 4, and undergo low-dose TBI on day -1. Patients receive bone marrow transplantation on day 0. Patients then receive cyclophosphamide IV on days +3 and +4, and beginning day +5 they start tacrolimus orally (PO) and mycophenolic acid. fludarabine phosphate: Given IV Mycophenolic Acid: Given PO tacrolimus: Given PO total-body irradiation: Undergo TBI bone marrow transplantation: Undergo transplantation reduced intensity allogeneic hematopoietic stem cell transplantation: Undergo transplantation quality-of-life assessment: Ancillary studies laboratory biomarker analysis: Correlative studies flow cytometry: Correlative studies biopsy: Punch biopsy of skin involved with scleroderma cyclophosphamide: Given IV
The Percent of Participants With Definite and Probable Viral, Fungal, and Bacterial Infections	2 Participants

SECONDARY outcome

Timeframe: Up to 5 years

Population: One patient completed the pre-transplant and 5 year post transplant SHAQ (scleroderma health assessment questionnaire). Score range from 0 to 3. Minimum score: Score of 0 is excellent health. Maximum score: score of 3 is most severely impaired, poor quality of life.

The questionnaire includes measure of quality of life and measure of the scale of skin tightness, activity level and function specifically designed for patients with systemic sclerosis

Outcome measures

Outcome measures
Measure	Treatment: Allogeneic HCT After Reduced Intensity Conditioning n=1 Participants Patients receive fludarabine phosphate IV on days -4, -3 and -2, cyclophosphamide IV over 1-2 hours on days -6, -5, 3, and 4, and undergo low-dose TBI on day -1. Patients receive bone marrow transplantation on day 0. Patients then receive cyclophosphamide IV on days +3 and +4, and beginning day +5 they start tacrolimus orally (PO) and mycophenolic acid. fludarabine phosphate: Given IV Mycophenolic Acid: Given PO tacrolimus: Given PO total-body irradiation: Undergo TBI bone marrow transplantation: Undergo transplantation reduced intensity allogeneic hematopoietic stem cell transplantation: Undergo transplantation quality-of-life assessment: Ancillary studies laboratory biomarker analysis: Correlative studies flow cytometry: Correlative studies biopsy: Punch biopsy of skin involved with scleroderma cyclophosphamide: Given IV
Quality of Life as Assessed by the Modified Scleroderma Health Assessment Questionnaire (SHAQ) pre-transplant SHAQ	1.125 units on a scale
Quality of Life as Assessed by the Modified Scleroderma Health Assessment Questionnaire (SHAQ) 5 year post transplant	0.125 units on a scale
Quality of Life as Assessed by the Modified Scleroderma Health Assessment Questionnaire (SHAQ) pre-transplant Raynaud symptoms	3 units on a scale
Quality of Life as Assessed by the Modified Scleroderma Health Assessment Questionnaire (SHAQ) 5 year post transplant Raynaud symptoms	0.5 units on a scale
Quality of Life as Assessed by the Modified Scleroderma Health Assessment Questionnaire (SHAQ) pre-transplant Finger ulcer symptoms	3 units on a scale
Quality of Life as Assessed by the Modified Scleroderma Health Assessment Questionnaire (SHAQ) 5 year post-transplant Finger ulcer symptoms	0 units on a scale
Quality of Life as Assessed by the Modified Scleroderma Health Assessment Questionnaire (SHAQ) pre-transplant Overall health symptoms	2.5 units on a scale
Quality of Life as Assessed by the Modified Scleroderma Health Assessment Questionnaire (SHAQ) 5 year post-transplant overall health symptoms	0.2 units on a scale

SECONDARY outcome

Timeframe: Up to 5 years

Population: Pre-transplant (baseline) evaluation of physical functioning. Score from 0 to100 with 0 perfect health and 100 poor health. The pre-transplant (baseline) evaluation was completed by study participant. The participant did not complete the 5 year post transplant evaluation. The 5 year post-transplant SF32 form was not completed by the patient.

The Medical Outcome Short Form (36) Health Survey instrument (SF-36) is a general assessment of health quality of life with eight components: physical functioning, role limitations due to physical health, pain index, general health perceptions, vitality, social functioning, role limitations due to emotional problems and Mental Health Index. Each domain is positively scored, indicating that higher scores are associated with positive outcome.

Outcome measures

Outcome measures
Measure	Treatment: Allogeneic HCT After Reduced Intensity Conditioning n=1 Participants Patients receive fludarabine phosphate IV on days -4, -3 and -2, cyclophosphamide IV over 1-2 hours on days -6, -5, 3, and 4, and undergo low-dose TBI on day -1. Patients receive bone marrow transplantation on day 0. Patients then receive cyclophosphamide IV on days +3 and +4, and beginning day +5 they start tacrolimus orally (PO) and mycophenolic acid. fludarabine phosphate: Given IV Mycophenolic Acid: Given PO tacrolimus: Given PO total-body irradiation: Undergo TBI bone marrow transplantation: Undergo transplantation reduced intensity allogeneic hematopoietic stem cell transplantation: Undergo transplantation quality-of-life assessment: Ancillary studies laboratory biomarker analysis: Correlative studies flow cytometry: Correlative studies biopsy: Punch biopsy of skin involved with scleroderma cyclophosphamide: Given IV
Quality of Life as Assessed by the Medical Outcome Short Form (36) Health Survey Instrument (SF-36) SF-36 pretransplant overall score	49.3 units on a scale
Quality of Life as Assessed by the Medical Outcome Short Form (36) Health Survey Instrument (SF-36) pretransplant limitations due to physical health	50 units on a scale
Quality of Life as Assessed by the Medical Outcome Short Form (36) Health Survey Instrument (SF-36) pretransplant limitations due to emotional health	50 units on a scale

SECONDARY outcome

Timeframe: Up to 5 years post-transplant

Population: One patient was evaluated at baseline and at 5 years post-transplant. Skin score was evaluated using modified Rodnan skin score (mRSS).

The skin score measure is a scale: the name of the scale is the modified Rodnan skin score (mRSS). Total score of mRSS is from 0 to 51. Higher values represents worse skin score. Highest value is 51, represents very hidebound tight thick skin. Lowest value is 0, represent normal skin, no tightness.

Outcome measures

Outcome measures
Measure	Treatment: Allogeneic HCT After Reduced Intensity Conditioning n=1 Participants Patients receive fludarabine phosphate IV on days -4, -3 and -2, cyclophosphamide IV over 1-2 hours on days -6, -5, 3, and 4, and undergo low-dose TBI on day -1. Patients receive bone marrow transplantation on day 0. Patients then receive cyclophosphamide IV on days +3 and +4, and beginning day +5 they start tacrolimus orally (PO) and mycophenolic acid. fludarabine phosphate: Given IV Mycophenolic Acid: Given PO tacrolimus: Given PO total-body irradiation: Undergo TBI bone marrow transplantation: Undergo transplantation reduced intensity allogeneic hematopoietic stem cell transplantation: Undergo transplantation quality-of-life assessment: Ancillary studies laboratory biomarker analysis: Correlative studies flow cytometry: Correlative studies biopsy: Punch biopsy of skin involved with scleroderma cyclophosphamide: Given IV
Skin Score Pre-transplant (baseline) skin score	17 units on a scale (mRSS)
Skin Score 5 year post-transplant skin score	4 units on a scale (mRSS)

SECONDARY outcome

Timeframe: Up to day +56

Engraftment is defined as achieving \> 5% donor peripheral blood T cell chimerism by Day 56 after HCT. Primary graft failure is defined as a donor peripheral blood T cell chimerism peak of \< 5% by Day 56 post-HCT. Methodological requirements for chimerism are as defined by institutional standard of practice. Secondary Graft Failure is defined as documented engraftment followed by loss of the graft with donor peripheral blood T cell chimerism \< 5% as demonstrated by a chimerism assay

Outcome measures

Outcome measures
Measure	Treatment: Allogeneic HCT After Reduced Intensity Conditioning n=3 Participants Patients receive fludarabine phosphate IV on days -4, -3 and -2, cyclophosphamide IV over 1-2 hours on days -6, -5, 3, and 4, and undergo low-dose TBI on day -1. Patients receive bone marrow transplantation on day 0. Patients then receive cyclophosphamide IV on days +3 and +4, and beginning day +5 they start tacrolimus orally (PO) and mycophenolic acid. fludarabine phosphate: Given IV Mycophenolic Acid: Given PO tacrolimus: Given PO total-body irradiation: Undergo TBI bone marrow transplantation: Undergo transplantation reduced intensity allogeneic hematopoietic stem cell transplantation: Undergo transplantation quality-of-life assessment: Ancillary studies laboratory biomarker analysis: Correlative studies flow cytometry: Correlative studies biopsy: Punch biopsy of skin involved with scleroderma cyclophosphamide: Given IV
Incidence of Graft Rejection	0 Participants

SECONDARY outcome

Timeframe: Up to 5 years post-transplant

Population: 1 patient out of the 3 patients enrolled was evaluable for both acute and chronic GVHD. The grade of acute GVHD ranges from 0 to 4. Minimum score of 0 is normal or no GVHD. Maximum score of 4 is fatal GVHD. Chronic GVHD minimum score is 0 (none), maximum score 3: extensive and severe.

The grading of acute and chronic GVHD will follow previously published guidelines and according to institutional standard of practice but will also include capture of symptoms and characterization of alternative causes. The highest level of organ abnormalities, the etiologies contributing to the abnormalities and biopsy results pertaining to GVHD will be identified. Since both GVHD and SSc involve the skin and the gastrointestinal tract, all diagnostic biopsies of these organs will be centrally reviewed by a study pathologist.

Outcome measures

Outcome measures
Measure	Treatment: Allogeneic HCT After Reduced Intensity Conditioning n=1 Participants Patients receive fludarabine phosphate IV on days -4, -3 and -2, cyclophosphamide IV over 1-2 hours on days -6, -5, 3, and 4, and undergo low-dose TBI on day -1. Patients receive bone marrow transplantation on day 0. Patients then receive cyclophosphamide IV on days +3 and +4, and beginning day +5 they start tacrolimus orally (PO) and mycophenolic acid. fludarabine phosphate: Given IV Mycophenolic Acid: Given PO tacrolimus: Given PO total-body irradiation: Undergo TBI bone marrow transplantation: Undergo transplantation reduced intensity allogeneic hematopoietic stem cell transplantation: Undergo transplantation quality-of-life assessment: Ancillary studies laboratory biomarker analysis: Correlative studies flow cytometry: Correlative studies biopsy: Punch biopsy of skin involved with scleroderma cyclophosphamide: Given IV
Incidence and Severity of Graft-versus-host Disease (GVHD) acute GVHD severity maximum grade	2 units on a scale
Incidence and Severity of Graft-versus-host Disease (GVHD) chronic GVHD maximum grade	2 units on a scale

SECONDARY outcome

Timeframe: Up to 5 years post-transplant

Population: 3 patients enrolled were evaluable.

Percent of patients treated with DMARDS after allogeneic transplant in order to treat scleroderma disease signs and symptoms.

Outcome measures

Outcome measures
Measure	Treatment: Allogeneic HCT After Reduced Intensity Conditioning n=3 Participants Patients receive fludarabine phosphate IV on days -4, -3 and -2, cyclophosphamide IV over 1-2 hours on days -6, -5, 3, and 4, and undergo low-dose TBI on day -1. Patients receive bone marrow transplantation on day 0. Patients then receive cyclophosphamide IV on days +3 and +4, and beginning day +5 they start tacrolimus orally (PO) and mycophenolic acid. fludarabine phosphate: Given IV Mycophenolic Acid: Given PO tacrolimus: Given PO total-body irradiation: Undergo TBI bone marrow transplantation: Undergo transplantation reduced intensity allogeneic hematopoietic stem cell transplantation: Undergo transplantation quality-of-life assessment: Ancillary studies laboratory biomarker analysis: Correlative studies flow cytometry: Correlative studies biopsy: Punch biopsy of skin involved with scleroderma cyclophosphamide: Given IV
Incidence of Disease-modifying Antirheumatic Drugs (DMARDs) Initiated Post Transplant to Modify Disease participants requiring DMARDs	0 Participants
Incidence of Disease-modifying Antirheumatic Drugs (DMARDs) Initiated Post Transplant to Modify Disease participants evaluable	3 Participants

Adverse Events

Treatment: Allogeneic HCT After Reduced Intensity Conditioning

Serious events: 2 serious events

Other events: 1 other events

Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure	Treatment: Allogeneic HCT After Reduced Intensity Conditioning n=3 participants at risk Patients receive fludarabine phosphate IV on days -4, -3 and -2, cyclophosphamide IV over 1-2 hours on days -6, -5, 3, and 4, and undergo low-dose TBI on day -1. Patients receive bone marrow transplantation on day 0. Patients then receive cyclophosphamide IV on days +3 and +4, and beginning day +5 they start tacrolimus orally (PO) and mycophenolic acid. fludarabine phosphate: Given IV Mycophenolic Acid: Given PO tacrolimus: Given PO total-body irradiation: Undergo TBI bone marrow transplantation: Undergo transplantation reduced intensity allogeneic hematopoietic stem cell transplantation: Undergo transplantation quality-of-life assessment: Ancillary studies laboratory biomarker analysis: Correlative studies flow cytometry: Correlative studies biopsy: Punch biopsy of skin involved with scleroderma cyclophosphamide: Given IV
Respiratory, thoracic and mediastinal disorders Diffuse Alveolar Hemorrhage	33.3% 1/3 • Number of events 1
Respiratory, thoracic and mediastinal disorders Pneumonitis	33.3% 1/3 • Number of events 1
Renal and urinary disorders Renal failure	33.3% 1/3 • Number of events 1

Other adverse events

Other adverse events
Measure	Treatment: Allogeneic HCT After Reduced Intensity Conditioning n=3 participants at risk Patients receive fludarabine phosphate IV on days -4, -3 and -2, cyclophosphamide IV over 1-2 hours on days -6, -5, 3, and 4, and undergo low-dose TBI on day -1. Patients receive bone marrow transplantation on day 0. Patients then receive cyclophosphamide IV on days +3 and +4, and beginning day +5 they start tacrolimus orally (PO) and mycophenolic acid. fludarabine phosphate: Given IV Mycophenolic Acid: Given PO tacrolimus: Given PO total-body irradiation: Undergo TBI bone marrow transplantation: Undergo transplantation reduced intensity allogeneic hematopoietic stem cell transplantation: Undergo transplantation quality-of-life assessment: Ancillary studies laboratory biomarker analysis: Correlative studies flow cytometry: Correlative studies biopsy: Punch biopsy of skin involved with scleroderma cyclophosphamide: Given IV
Skin and subcutaneous tissue disorders GVHD	33.3% 1/3 • Number of events 3

Additional Information

George E. Georges MD

Fred Hutchinson Cancer Research Center

Phone: 206-667-6886

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place