High Dose Ruxolitinib with Stem Cell Transplant to Treat Myelofibrosis and Enlarged Spleen in Adults

What this Trial Is About

Researchers are evaluating whether giving ruxolitinib and busulfan before a stem cell transplant can reduce spleen size and improve transplant success for patients with myelofibrosis and splenomegaly. This Phase 2 study aims to compare patient survival and disease-free rates at 100 days after transplant against historical data, while also tracking various clinical outcomes such as graft function, relapse, and toxicity. Participants will receive high-dose ruxolitinib and busulfan prior to undergoing an allogeneic stem cell transplant. Additional medications like levetiracetam, eltrombopag, romiplostim, fludarabine phosphate, cyclophosphamide, mesna, and tacrolimus may also be given as part of the treatment. The treatment phase lasts about 100 days, followed by a one-year follow-up period to monitor patient progress and long-term outcomes. Throughout the study, participants will attend visits for tests and procedures to assess spleen size, blood counts, immune function, and adverse events. Researchers will measure safety and side effects over approximately one year. They will also observe engraftment success, graft-versus-host disease, transfusion needs, and survival rates to understand the treatment impact and transplant outcomes.

High Dose Ruxolitinib and Allogeneic Stem Cell Transplantation in Myelofibrosis Patients With Splenomegaly