Phase II Study of Myeloablative Allogeneic Stem Cell Transplant Using Related or Unrelated Donors to Treat Blood Cancers and Disorders

What this Trial Is About

Researchers are evaluating a Phase II study of allogeneic hematopoietic stem cell transplantation HCT for treating various hematological diseases. This study uses a myeloablative preparative regimen that includes either total body irradiation TBI or a chemotherapy combination of fludarabine and busulfan for patients who cannot receive radiation. After the transplant, participants receive a regimen to prevent graft-versus-host disease GVHD consisting of post-transplant cyclophosphamide PTCy, tacrolimus, and mycophenolate mofetil MMF. Participants receive one of two preparative regimens the TBI regimen involves total body irradiation from day -5 to day -2, followed by the stem cell transplant on day 0. The non-TBI regimen consists of busulfan and fludarabine from day -5 to day -2, also followed by the transplant on day 0. Post-transplant medications start with cyclophosphamide on days 3 and 4, then tacrolimus from day 5 continuing until tapering around day 100 or day 60 for pediatric patients without GVHD. Mycophenolate mofetil is given from day 5 through day 35 or 7 days after engraftment if no acute GVHD is present. During the study, participants are monitored for outcomes such as chronic GVHD at 1 year, acute GVHD by day 100, relapse, overall survival, and treatment-related mortality over 2 years. Researchers also track hematologic recovery including neutrophil and platelet engraftment at various time points. Assessments include clinical evaluations and laboratory tests to monitor transplant success, immune response, and GVHD development. The study is sponsored by the Masonic Cancer Center at the University of Minnesota and runs until mid-2028.

Myeloablative Allo HSCT With Related or Unrelated Donor for Heme Disorders