Comparison of Triple GVHD Prophylaxis Regimens for Nonmyeloablative or Reduced Intensity Conditioning Unrelated Mobilized Blood Cell Transplantation

Summary

This randomized phase II trial includes a blood stem cell transplant from an unrelated donor to treat blood cancer. The treatment also includes chemotherapy drugs, but in lower doses than conventional (standard) stem cell transplants. The researchers will compare two different drug combinations used to reduce the risk of a common but serious complication called "graft versus host disease" (GVHD) following the transplant. Two drugs, cyclosporine (CSP) and sirolimus (SIR), will be combined with either mycophenolate mofetil (MMF) or post-transplant cyclophosphamide (PTCy). This part of the transplant procedure is the main research focus of the study.

Timeline

Start

2017-09-11

Primary completion

2025-05-03

Completion

2025-05-03

Outcome

Met primary endpoint

Stopped (Enrollment): “Study was terminated prior to enrolling protocol target accrual goal due to declining accrual and Data Safety Monitoring Board recommendations.”

paper hazard ratio (HR) for CRFS failure of 0.46 (95% CI, 0.26 to 0.79; P = .005) for PTCy. PMID 41043099 ↗

Publications

• Ueda Oshima M, Vo PT, Boeckh M, Bouvier ME, Carpenter PA, Mielcarek M, Petersdorf EW, Storb R, Gooley T, Sandmaier BM. Sirolimus and Cyclosporine With Post-Transplant Cyclophosphamide or Mycophenolate Mofetil as Graft-Versus-Host Disease Prophylaxis in Unrelated Donor Hematopoietic Cell Transplantation. J Clin Oncol. 2025 Nov 20;43(33):3600-3609. doi: 10.1200/JCO-25-01238. Epub 2025 Oct 3.

Drugs

Indications