A Phase II trial of haploidentical transplantation with high dose, post-transplantation cyclophosphamide.
- Conditions
- ALL,AML,MDS,CML,NHL,MM
- Registration Number
- JPRN-UMIN000010316
- Lead Sponsor
- JSCT
- Brief Summary
Allogeneic hematopoietic stem cell transplantation (allo-SCT) using post-transplant cyclophosphamide (PTCy) is increasingly performed. We conducted a multicenter phase II study to evaluate the safety and efficacy of PTCy-based HLA-haploidentical peripheral blood stem cell transplantation (PTCy-haploPBSCT) after busulfan-containing reduced-intensity conditioning. Thirty-one patients were enrolled; 61% patients were not in remission and 42% patients had a history of prior allo-SCT. Neutrophil engraftment was achieved in 87% patients with a median of 19 days. The cumulative incidence of grades II to IV and III to IV acute graft-versushost disease (GVHD) and chronic GVHD at 1 year were 23%, 3%, and 15%, respectively. No patients developed severe chronic GVHD. Day 100 nonrelapse mortality (NRM) rate was 19.4%. Overall survival, relapse, and disease-free survival rates were 45%, 45%, and 34%, respectively, at 1 year. Subgroup analysis showed that patients who had a history of prior allo-SCT had lower engraftment, higher NRM, and lower overall survival than those not receiving a prior allo-SCT. Our results suggest that PTCy-haploPBSCT after busulfan-containing reduced-intensity conditioning achieved low incidences of acute and chronic GVHD and NRM and stable donor engraftment and low NRM, particularly in patients without a history of prior allo-SCT.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete: follow-up complete
- Sex
- All
- Target Recruitment
- 20
Not provided
1. Patients who are positive for HBs antigen, HCV antibody, or HIV antibody. 2. Patients with active other malignancies. 3. Patients with active infectious disease. 4. Women who are pregnant, of childbearing potential, or lactating. 5. Patients who experienced serious hypersensitivity or anaphylaxis to cyclophosphamide, fludarabine, tacrolimus, mycophenolate mofetil. 6. Positive anti-donor HLA antibody. 7. Patients who need chemotherapy within 13 days before transplantation. 8. Patients who are not eligible for this study at the discretion of the investigator.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Transplant-related mortality at 100 days.
- Secondary Outcome Measures
Name Time Method 1. Overall survival, progression-free survival, relapse at 100 days. 2. Overall survival, progression-free survival, Relapse, Transplant-related mortality at 1 year. 3. Neutrophil and platelet recovery. 4. Graft failure. 5. Full donor chimerism. 6. Acute Graft-versus-Host Disease. 7. Chronic Graft-versus-Host Disease. 8. Infection 9. Grade 3-4 non-hematologic toxicity within 100 days.