Trial Outcomes & Findings for Reduced-Intensity Conditioning Followed By Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Multiple Myeloma (NCT NCT00054353)
NCT ID: NCT00054353
Last Updated: 2017-10-16
Results Overview
PFS will be calculated for all patients from the date of transplant until the time of progression, relapse, death, or the date the patient was last known to be in remission. Progressive disease is defined as greater than 25% increase in serum or urine M proteins compared to best response status after autologous transplant and/or appearance of new lytic bone lesions or plasmocytomas.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
16 participants
Primary outcome timeframe
At 1 year post-transplant
Results posted on
2017-10-16
Participant Flow
Participant milestones
| Measure |
Related Donor
PREPARATIVE REGIMEN: Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -3 and intermediate-dose melphalan IV over 15-20 minutes on day -2. Patients also undergo low-dose TBI on day 0.
TRANSPLANTATION: Patients undergo allogeneic PBSCT on day 0.
IMMUNOSUPPRESSION: Patients receive cyclosporine PO BID on days -3 to 80 with taper to day 180 (related donors) or on days -3 to 100 with taper to day 180 (unrelated donors). Patients also receive mycophenolate mofetil PO BID on days 0-27 (related donors) or TID on days 0-40 with taper to day 96 (unrelated donors).
fludarabine phosphate: Given IV melphalan: Given IV total-body irradiation: Undergo TBI mycophenolate mofetil: Given PO
cyclosporine: Given PO
nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
peripheral blood stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
laboratory biomarker analysis: Correlative s
|
Unrelated Donor
PREPARATIVE REGIMEN: Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -3 and intermediate-dose melphalan IV over 15-20 minutes on day -2. Patients also undergo low-dose TBI on day 0.
TRANSPLANTATION: Patients undergo allogeneic PBSCT on day 0.
IMMUNOSUPPRESSION: Patients receive cyclosporine PO BID on days -3 to 80 with taper to day 180 (related donors) or on days -3 to 100 with taper to day 180 (unrelated donors). Patients also receive mycophenolate mofetil PO BID on days 0-27 (related donors) or TID on days 0-40 with taper to day 96 (unrelated donors).
fludarabine phosphate: Given IV melphalan: Given IV total-body irradiation: Undergo TBI mycophenolate mofetil: Given PO
cyclosporine: Given PO
nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
peripheral blood stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
laboratory biomarker analysis: Correlative s
|
|---|---|---|
|
Overall Study
STARTED
|
12
|
4
|
|
Overall Study
COMPLETED
|
12
|
4
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Reduced-Intensity Conditioning Followed By Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Multiple Myeloma
Baseline characteristics by cohort
| Measure |
Related Donor
n=12 Participants
PREPARATIVE REGIMEN: Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -3 and intermediate-dose melphalan IV over 15-20 minutes on day -2. Patients also undergo low-dose TBI on day 0.
TRANSPLANTATION: Patients undergo allogeneic PBSCT on day 0.
IMMUNOSUPPRESSION: Patients receive cyclosporine PO BID on days -3 to 80 with taper to day 180 (related donors) or on days -3 to 100 with taper to day 180 (unrelated donors). Patients also receive mycophenolate mofetil PO BID on days 0-27 (related donors) or TID on days 0-40 with taper to day 96 (unrelated donors).
fludarabine phosphate: Given IV melphalan: Given IV
total-body irradiation: Undergo TBI
mycophenolate mofetil: Given PO
cyclosporine: Given PO
nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
peripheral blood stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
laboratory biomarker analysis: Correlative s
|
Unrelated Donor
n=4 Participants
PREPARATIVE REGIMEN: Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -3 and intermediate-dose melphalan IV over 15-20 minutes on day -2. Patients also undergo low-dose TBI on day 0.
TRANSPLANTATION: Patients undergo allogeneic PBSCT on day 0.
IMMUNOSUPPRESSION: Patients receive cyclosporine PO BID on days -3 to 80 with taper to day 180 (related donors) or on days -3 to 100 with taper to day 180 (unrelated donors). Patients also receive mycophenolate mofetil PO BID on days 0-27 (related donors) or TID on days 0-40 with taper to day 96 (unrelated donors).
fludarabine phosphate: Given IV melphalan: Given IV
total-body irradiation: Undergo TBI
mycophenolate mofetil: Given PO
cyclosporine: Given PO
nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
peripheral blood stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
laboratory biomarker analysis: Correlative s
|
Total
n=16 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
10 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
16 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
8 participants
n=5 Participants
|
4 participants
n=7 Participants
|
12 participants
n=5 Participants
|
|
Region of Enrollment
Italy
|
4 participants
n=5 Participants
|
0 participants
n=7 Participants
|
4 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: At 1 year post-transplantPopulation: There were 4 patients who, although they did not have progression/relapse, died before the 1 year mark, and thus are not included in the 1 year PFS count.
PFS will be calculated for all patients from the date of transplant until the time of progression, relapse, death, or the date the patient was last known to be in remission. Progressive disease is defined as greater than 25% increase in serum or urine M proteins compared to best response status after autologous transplant and/or appearance of new lytic bone lesions or plasmocytomas.
Outcome measures
| Measure |
Related Donor
n=9 Participants
PREPARATIVE REGIMEN: Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -3 and intermediate-dose melphalan IV over 15-20 minutes on day -2. Patients also undergo low-dose TBI on day 0.
TRANSPLANTATION: Patients undergo allogeneic PBSCT on day 0.
IMMUNOSUPPRESSION: Patients receive cyclosporine PO BID on days -3 to 80 with taper to day 180 (related donors) or on days -3 to 100 with taper to day 180 (unrelated donors). Patients also receive mycophenolate mofetil PO BID on days 0-27 (related donors) or TID on days 0-40 with taper to day 96 (unrelated donors).
fludarabine phosphate: Given IV melphalan: Given IV
total-body irradiation: Undergo TBI
mycophenolate mofetil: Given PO
cyclosporine: Given PO
nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
peripheral blood stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
laboratory biomarker analysis: Correlative s
|
Unrelated Donor
n=3 Participants
PREPARATIVE REGIMEN: Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -3 and intermediate-dose melphalan IV over 15-20 minutes on day -2. Patients also undergo low-dose TBI on day 0.
TRANSPLANTATION: Patients undergo allogeneic PBSCT on day 0.
IMMUNOSUPPRESSION: Patients receive cyclosporine PO BID on days -3 to 80 with taper to day 180 (related donors) or on days -3 to 100 with taper to day 180 (unrelated donors). Patients also receive mycophenolate mofetil PO BID on days 0-27 (related donors) or TID on days 0-40 with taper to day 96 (unrelated donors).
fludarabine phosphate: Given IV melphalan: Given IV
total-body irradiation: Undergo TBI
mycophenolate mofetil: Given PO
cyclosporine: Given PO
nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
peripheral blood stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
laboratory biomarker analysis: Correlative s
|
|---|---|---|
|
PFS
|
4 Participants
|
1 Participants
|
PRIMARY outcome
Timeframe: At day 100Early NRM will be monitored in a sequential fashion.
Outcome measures
| Measure |
Related Donor
n=12 Participants
PREPARATIVE REGIMEN: Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -3 and intermediate-dose melphalan IV over 15-20 minutes on day -2. Patients also undergo low-dose TBI on day 0.
TRANSPLANTATION: Patients undergo allogeneic PBSCT on day 0.
IMMUNOSUPPRESSION: Patients receive cyclosporine PO BID on days -3 to 80 with taper to day 180 (related donors) or on days -3 to 100 with taper to day 180 (unrelated donors). Patients also receive mycophenolate mofetil PO BID on days 0-27 (related donors) or TID on days 0-40 with taper to day 96 (unrelated donors).
fludarabine phosphate: Given IV melphalan: Given IV
total-body irradiation: Undergo TBI
mycophenolate mofetil: Given PO
cyclosporine: Given PO
nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
peripheral blood stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
laboratory biomarker analysis: Correlative s
|
Unrelated Donor
n=4 Participants
PREPARATIVE REGIMEN: Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -3 and intermediate-dose melphalan IV over 15-20 minutes on day -2. Patients also undergo low-dose TBI on day 0.
TRANSPLANTATION: Patients undergo allogeneic PBSCT on day 0.
IMMUNOSUPPRESSION: Patients receive cyclosporine PO BID on days -3 to 80 with taper to day 180 (related donors) or on days -3 to 100 with taper to day 180 (unrelated donors). Patients also receive mycophenolate mofetil PO BID on days 0-27 (related donors) or TID on days 0-40 with taper to day 96 (unrelated donors).
fludarabine phosphate: Given IV melphalan: Given IV
total-body irradiation: Undergo TBI
mycophenolate mofetil: Given PO
cyclosporine: Given PO
nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
peripheral blood stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
laboratory biomarker analysis: Correlative s
|
|---|---|---|
|
Non-relapse Mortality
|
1 Participants
|
1 Participants
|
PRIMARY outcome
Timeframe: Up to 5 yearsNumber of patients with Grade III-IV acute GVHD post-transplant. Severe GVHD will be monitored in a sequential fashion.
Outcome measures
| Measure |
Related Donor
n=12 Participants
PREPARATIVE REGIMEN: Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -3 and intermediate-dose melphalan IV over 15-20 minutes on day -2. Patients also undergo low-dose TBI on day 0.
TRANSPLANTATION: Patients undergo allogeneic PBSCT on day 0.
IMMUNOSUPPRESSION: Patients receive cyclosporine PO BID on days -3 to 80 with taper to day 180 (related donors) or on days -3 to 100 with taper to day 180 (unrelated donors). Patients also receive mycophenolate mofetil PO BID on days 0-27 (related donors) or TID on days 0-40 with taper to day 96 (unrelated donors).
fludarabine phosphate: Given IV melphalan: Given IV
total-body irradiation: Undergo TBI
mycophenolate mofetil: Given PO
cyclosporine: Given PO
nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
peripheral blood stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
laboratory biomarker analysis: Correlative s
|
Unrelated Donor
n=4 Participants
PREPARATIVE REGIMEN: Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -3 and intermediate-dose melphalan IV over 15-20 minutes on day -2. Patients also undergo low-dose TBI on day 0.
TRANSPLANTATION: Patients undergo allogeneic PBSCT on day 0.
IMMUNOSUPPRESSION: Patients receive cyclosporine PO BID on days -3 to 80 with taper to day 180 (related donors) or on days -3 to 100 with taper to day 180 (unrelated donors). Patients also receive mycophenolate mofetil PO BID on days 0-27 (related donors) or TID on days 0-40 with taper to day 96 (unrelated donors).
fludarabine phosphate: Given IV melphalan: Given IV
total-body irradiation: Undergo TBI
mycophenolate mofetil: Given PO
cyclosporine: Given PO
nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
peripheral blood stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
laboratory biomarker analysis: Correlative s
|
|---|---|---|
|
Incidence of Acute GVHD (Grades III-IV)
|
2 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: Up to 5 yearsNumber of subjects with chronic extensive GVHD post-transplant. Severe GVHD will be monitored in a sequential fashion.
Outcome measures
| Measure |
Related Donor
n=12 Participants
PREPARATIVE REGIMEN: Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -3 and intermediate-dose melphalan IV over 15-20 minutes on day -2. Patients also undergo low-dose TBI on day 0.
TRANSPLANTATION: Patients undergo allogeneic PBSCT on day 0.
IMMUNOSUPPRESSION: Patients receive cyclosporine PO BID on days -3 to 80 with taper to day 180 (related donors) or on days -3 to 100 with taper to day 180 (unrelated donors). Patients also receive mycophenolate mofetil PO BID on days 0-27 (related donors) or TID on days 0-40 with taper to day 96 (unrelated donors).
fludarabine phosphate: Given IV melphalan: Given IV
total-body irradiation: Undergo TBI
mycophenolate mofetil: Given PO
cyclosporine: Given PO
nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
peripheral blood stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
laboratory biomarker analysis: Correlative s
|
Unrelated Donor
n=4 Participants
PREPARATIVE REGIMEN: Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -3 and intermediate-dose melphalan IV over 15-20 minutes on day -2. Patients also undergo low-dose TBI on day 0.
TRANSPLANTATION: Patients undergo allogeneic PBSCT on day 0.
IMMUNOSUPPRESSION: Patients receive cyclosporine PO BID on days -3 to 80 with taper to day 180 (related donors) or on days -3 to 100 with taper to day 180 (unrelated donors). Patients also receive mycophenolate mofetil PO BID on days 0-27 (related donors) or TID on days 0-40 with taper to day 96 (unrelated donors).
fludarabine phosphate: Given IV melphalan: Given IV
total-body irradiation: Undergo TBI
mycophenolate mofetil: Given PO
cyclosporine: Given PO
nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
peripheral blood stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
laboratory biomarker analysis: Correlative s
|
|---|---|---|
|
Incidence of Chronic (Extensive) GVHD
|
4 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: At 6 months and then every year thereafter, up to 5 yearsNumber of subjects surviving. OS will be estimated by the method of Kaplan and Meier. Confidence intervals will be estimated.
Outcome measures
| Measure |
Related Donor
n=12 Participants
PREPARATIVE REGIMEN: Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -3 and intermediate-dose melphalan IV over 15-20 minutes on day -2. Patients also undergo low-dose TBI on day 0.
TRANSPLANTATION: Patients undergo allogeneic PBSCT on day 0.
IMMUNOSUPPRESSION: Patients receive cyclosporine PO BID on days -3 to 80 with taper to day 180 (related donors) or on days -3 to 100 with taper to day 180 (unrelated donors). Patients also receive mycophenolate mofetil PO BID on days 0-27 (related donors) or TID on days 0-40 with taper to day 96 (unrelated donors).
fludarabine phosphate: Given IV melphalan: Given IV
total-body irradiation: Undergo TBI
mycophenolate mofetil: Given PO
cyclosporine: Given PO
nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
peripheral blood stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
laboratory biomarker analysis: Correlative s
|
Unrelated Donor
n=4 Participants
PREPARATIVE REGIMEN: Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -3 and intermediate-dose melphalan IV over 15-20 minutes on day -2. Patients also undergo low-dose TBI on day 0.
TRANSPLANTATION: Patients undergo allogeneic PBSCT on day 0.
IMMUNOSUPPRESSION: Patients receive cyclosporine PO BID on days -3 to 80 with taper to day 180 (related donors) or on days -3 to 100 with taper to day 180 (unrelated donors). Patients also receive mycophenolate mofetil PO BID on days 0-27 (related donors) or TID on days 0-40 with taper to day 96 (unrelated donors).
fludarabine phosphate: Given IV melphalan: Given IV
total-body irradiation: Undergo TBI
mycophenolate mofetil: Given PO
cyclosporine: Given PO
nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
peripheral blood stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
laboratory biomarker analysis: Correlative s
|
|---|---|---|
|
OS
6 Months
|
8 Participants
|
3 Participants
|
|
OS
1 Year
|
5 Participants
|
2 Participants
|
|
OS
2 Years
|
4 Participants
|
0 Participants
|
|
OS
3 Years
|
3 Participants
|
0 Participants
|
|
OS
4 Years
|
3 Participants
|
0 Participants
|
|
OS
5 Years
|
3 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Up to 5 yearsNumber of subjects who engrafted post-transplant. Engraftment will be monitored in a sequential fashion.
Outcome measures
| Measure |
Related Donor
n=12 Participants
PREPARATIVE REGIMEN: Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -3 and intermediate-dose melphalan IV over 15-20 minutes on day -2. Patients also undergo low-dose TBI on day 0.
TRANSPLANTATION: Patients undergo allogeneic PBSCT on day 0.
IMMUNOSUPPRESSION: Patients receive cyclosporine PO BID on days -3 to 80 with taper to day 180 (related donors) or on days -3 to 100 with taper to day 180 (unrelated donors). Patients also receive mycophenolate mofetil PO BID on days 0-27 (related donors) or TID on days 0-40 with taper to day 96 (unrelated donors).
fludarabine phosphate: Given IV melphalan: Given IV
total-body irradiation: Undergo TBI
mycophenolate mofetil: Given PO
cyclosporine: Given PO
nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
peripheral blood stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
laboratory biomarker analysis: Correlative s
|
Unrelated Donor
n=4 Participants
PREPARATIVE REGIMEN: Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -3 and intermediate-dose melphalan IV over 15-20 minutes on day -2. Patients also undergo low-dose TBI on day 0.
TRANSPLANTATION: Patients undergo allogeneic PBSCT on day 0.
IMMUNOSUPPRESSION: Patients receive cyclosporine PO BID on days -3 to 80 with taper to day 180 (related donors) or on days -3 to 100 with taper to day 180 (unrelated donors). Patients also receive mycophenolate mofetil PO BID on days 0-27 (related donors) or TID on days 0-40 with taper to day 96 (unrelated donors).
fludarabine phosphate: Given IV melphalan: Given IV
total-body irradiation: Undergo TBI
mycophenolate mofetil: Given PO
cyclosporine: Given PO
nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
peripheral blood stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
laboratory biomarker analysis: Correlative s
|
|---|---|---|
|
Engraftment
|
12 Participants
|
4 Participants
|
SECONDARY outcome
Timeframe: Up to 5 yearsNumber of subjects who relapsed after achieving CR post-transplant. Relapse rate will be summarized using cumulative incidence estimates.
Outcome measures
| Measure |
Related Donor
n=12 Participants
PREPARATIVE REGIMEN: Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -3 and intermediate-dose melphalan IV over 15-20 minutes on day -2. Patients also undergo low-dose TBI on day 0.
TRANSPLANTATION: Patients undergo allogeneic PBSCT on day 0.
IMMUNOSUPPRESSION: Patients receive cyclosporine PO BID on days -3 to 80 with taper to day 180 (related donors) or on days -3 to 100 with taper to day 180 (unrelated donors). Patients also receive mycophenolate mofetil PO BID on days 0-27 (related donors) or TID on days 0-40 with taper to day 96 (unrelated donors).
fludarabine phosphate: Given IV melphalan: Given IV
total-body irradiation: Undergo TBI
mycophenolate mofetil: Given PO
cyclosporine: Given PO
nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
peripheral blood stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
laboratory biomarker analysis: Correlative s
|
Unrelated Donor
n=4 Participants
PREPARATIVE REGIMEN: Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -3 and intermediate-dose melphalan IV over 15-20 minutes on day -2. Patients also undergo low-dose TBI on day 0.
TRANSPLANTATION: Patients undergo allogeneic PBSCT on day 0.
IMMUNOSUPPRESSION: Patients receive cyclosporine PO BID on days -3 to 80 with taper to day 180 (related donors) or on days -3 to 100 with taper to day 180 (unrelated donors). Patients also receive mycophenolate mofetil PO BID on days 0-27 (related donors) or TID on days 0-40 with taper to day 96 (unrelated donors).
fludarabine phosphate: Given IV melphalan: Given IV
total-body irradiation: Undergo TBI
mycophenolate mofetil: Given PO
cyclosporine: Given PO
nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
peripheral blood stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
laboratory biomarker analysis: Correlative s
|
|---|---|---|
|
Relapse Rate
|
1 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Up to 5 yearsNumber of subjects who achieved CR post-transplant. Response rate will be summarized using cumulative incidence estimates.
Outcome measures
| Measure |
Related Donor
n=12 Participants
PREPARATIVE REGIMEN: Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -3 and intermediate-dose melphalan IV over 15-20 minutes on day -2. Patients also undergo low-dose TBI on day 0.
TRANSPLANTATION: Patients undergo allogeneic PBSCT on day 0.
IMMUNOSUPPRESSION: Patients receive cyclosporine PO BID on days -3 to 80 with taper to day 180 (related donors) or on days -3 to 100 with taper to day 180 (unrelated donors). Patients also receive mycophenolate mofetil PO BID on days 0-27 (related donors) or TID on days 0-40 with taper to day 96 (unrelated donors).
fludarabine phosphate: Given IV melphalan: Given IV
total-body irradiation: Undergo TBI
mycophenolate mofetil: Given PO
cyclosporine: Given PO
nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
peripheral blood stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
laboratory biomarker analysis: Correlative s
|
Unrelated Donor
n=4 Participants
PREPARATIVE REGIMEN: Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -3 and intermediate-dose melphalan IV over 15-20 minutes on day -2. Patients also undergo low-dose TBI on day 0.
TRANSPLANTATION: Patients undergo allogeneic PBSCT on day 0.
IMMUNOSUPPRESSION: Patients receive cyclosporine PO BID on days -3 to 80 with taper to day 180 (related donors) or on days -3 to 100 with taper to day 180 (unrelated donors). Patients also receive mycophenolate mofetil PO BID on days 0-27 (related donors) or TID on days 0-40 with taper to day 96 (unrelated donors).
fludarabine phosphate: Given IV melphalan: Given IV
total-body irradiation: Undergo TBI
mycophenolate mofetil: Given PO
cyclosporine: Given PO
nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
peripheral blood stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
laboratory biomarker analysis: Correlative s
|
|---|---|---|
|
Response Rate
|
2 Participants
|
1 Participants
|
Adverse Events
Related Donor
Serious events: 4 serious events
Other events: 9 other events
Deaths: 4 deaths
Unrelated Donor
Serious events: 1 serious events
Other events: 2 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Related Donor
n=12 participants at risk
PREPARATIVE REGIMEN: Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -3 and intermediate-dose melphalan IV over 15-20 minutes on day -2. Patients also undergo low-dose TBI on day 0.
TRANSPLANTATION: Patients undergo allogeneic PBSCT on day 0.
IMMUNOSUPPRESSION: Patients receive cyclosporine PO BID on days -3 to 80 with taper to day 180 (related donors) or on days -3 to 100 with taper to day 180 (unrelated donors). Patients also receive mycophenolate mofetil PO BID on days 0-27 (related donors) or TID on days 0-40 with taper to day 96 (unrelated donors).
fludarabine phosphate: Given IV melphalan: Given IV total-body irradiation: Undergo TBI mycophenolate mofetil: Given PO cyclosporine: Given PO nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
peripheral blood stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT laboratory biomarker analysis: Correlative s
|
Unrelated Donor
n=4 participants at risk
PREPARATIVE REGIMEN: Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -3 and intermediate-dose melphalan IV over 15-20 minutes on day -2. Patients also undergo low-dose TBI on day 0.
TRANSPLANTATION: Patients undergo allogeneic PBSCT on day 0.
IMMUNOSUPPRESSION: Patients receive cyclosporine PO BID on days -3 to 80 with taper to day 180 (related donors) or on days -3 to 100 with taper to day 180 (unrelated donors). Patients also receive mycophenolate mofetil PO BID on days 0-27 (related donors) or TID on days 0-40 with taper to day 96 (unrelated donors).
fludarabine phosphate: Given IV melphalan: Given IV total-body irradiation: Undergo TBI mycophenolate mofetil: Given PO cyclosporine: Given PO nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
peripheral blood stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT laboratory biomarker analysis: Correlative s
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Adult respiratory distress syndrome
|
16.7%
2/12 • Number of events 2 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/4 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Investigations
Blood bilirubin increased
|
8.3%
1/12 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/4 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
General disorders
Multi-organ failure
|
16.7%
2/12 • Number of events 2 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/4 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
0.00%
0/12 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
25.0%
1/4 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
Other adverse events
| Measure |
Related Donor
n=12 participants at risk
PREPARATIVE REGIMEN: Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -3 and intermediate-dose melphalan IV over 15-20 minutes on day -2. Patients also undergo low-dose TBI on day 0.
TRANSPLANTATION: Patients undergo allogeneic PBSCT on day 0.
IMMUNOSUPPRESSION: Patients receive cyclosporine PO BID on days -3 to 80 with taper to day 180 (related donors) or on days -3 to 100 with taper to day 180 (unrelated donors). Patients also receive mycophenolate mofetil PO BID on days 0-27 (related donors) or TID on days 0-40 with taper to day 96 (unrelated donors).
fludarabine phosphate: Given IV melphalan: Given IV total-body irradiation: Undergo TBI mycophenolate mofetil: Given PO cyclosporine: Given PO nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
peripheral blood stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT laboratory biomarker analysis: Correlative s
|
Unrelated Donor
n=4 participants at risk
PREPARATIVE REGIMEN: Patients receive fludarabine phosphate IV over 30 minutes on days -5 to -3 and intermediate-dose melphalan IV over 15-20 minutes on day -2. Patients also undergo low-dose TBI on day 0.
TRANSPLANTATION: Patients undergo allogeneic PBSCT on day 0.
IMMUNOSUPPRESSION: Patients receive cyclosporine PO BID on days -3 to 80 with taper to day 180 (related donors) or on days -3 to 100 with taper to day 180 (unrelated donors). Patients also receive mycophenolate mofetil PO BID on days 0-27 (related donors) or TID on days 0-40 with taper to day 96 (unrelated donors).
fludarabine phosphate: Given IV melphalan: Given IV total-body irradiation: Undergo TBI mycophenolate mofetil: Given PO cyclosporine: Given PO nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT
peripheral blood stem cell transplantation: Undergo reduced-intensity allogeneic PBSCT laboratory biomarker analysis: Correlative s
|
|---|---|---|
|
Renal and urinary disorders
Acute kidney injury
|
25.0%
3/12 • Number of events 3 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
25.0%
1/4 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Respiratory, thoracic and mediastinal disorders
Adult respiratory distress syndrome
|
8.3%
1/12 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/4 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Investigations
Alanine aminotransferase increased
|
8.3%
1/12 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/4 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Metabolism and nutrition disorders
Anorexia
|
8.3%
1/12 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/4 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Investigations
Blood bilirubin increased
|
8.3%
1/12 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
25.0%
1/4 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Blood and lymphatic system disorders
Bone marrow hypocellular
|
8.3%
1/12 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/4 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Investigations
Creatinine increased
|
8.3%
1/12 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
25.0%
1/4 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
General disorders
Fatigue
|
8.3%
1/12 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/4 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
General disorders
Fever
|
8.3%
1/12 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/4 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weaknes
|
8.3%
1/12 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/4 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Cardiac disorders
Heart failure
|
8.3%
1/12 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/4 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Vascular disorders
Hypotension
|
8.3%
1/12 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
25.0%
1/4 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
8.3%
1/12 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
25.0%
1/4 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Infections and infestations
Lung infection
|
8.3%
1/12 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/4 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Gastrointestinal disorders
Mucositis oral
|
8.3%
1/12 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/4 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
8.3%
1/12 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/4 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
0.00%
0/12 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
25.0%
1/4 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Cardiac disorders
Supraventricular tachycardia
|
0.00%
0/12 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
25.0%
1/4 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Nervous system disorders
Syncope
|
8.3%
1/12 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/4 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Blood and lymphatic system disorders
Thrombotic thrombocytopenic purpura
|
8.3%
1/12 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/4 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place