Study of Stem Cell Transplant vs. Non-Transplant Therapies in High-Risk Myelofibrosis

Official Title

A Patient Preferences-Controlled Study of Allogeneic Hematopoietic Cell Transplantation Versus Best Available Non-Transplant Therapies in Patients With High-Risk Myelofibrosis (ALLO-BAT Study)

Summary:

The purpose of this research study is to see how effective hematopoietic stem cell transplantation (HCT) is compared to best available non-transplant therapies (BAT) in patients with high risk myelofibrosis. This will be done by asking participants to choose the treatment that they prefer to receive (HCT or BAT) and then comparing the outcomes of the participants in both treatment groups.

Trial Description

Primary Outcome:

• Number of patients allocated to hematopoietic stem cell transplantation (HCT)
• Number of patients allocated to best available non-transplant therapies (BAT)
• Overall survival rate of patients who receive hematopoietic stem cell transplantation (HCT)
• Overall survival rate of patients who receive best available non-transplant therapies (BAT)

Secondary Outcome:

• Median change in Patient Global Impression of Change (PGIC) score
• Median change in MPN Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)
• Median change in FACT-BMT Questionnaire
• Disease-free survival of patients who receive hematopoietic stem cell transplantation (HCT)
• Disease-free survival of patients who receive best available non-transplant therapies (BAT)
• Number of patients who receive hematopoietic stem cell transplantation (HCT) in remission (complete and partial)
• Number of patients who receive best available non-transplant therapies (BAT) in remission (complete and partial)

There is currently little information regarding which treatments are best for patients with myelofibrosis. On one hand, hematopoietic stem cell transplantation (HCT) is potentially curative treatment but is associated with significant risk of complications related to graft failure (the new donor cells does not grow properly after the transplant), side effects such as graft versus host disease (the patient's cells attack the new donor cells), and risk of infections. Non-transplant therapies such as ruxolitinib provide effective symptom control for few months to few years, but are not curative in nature. As such, this study will compare the effectiveness of HCT versus best available non-transplant therapies (BAT) in patients with high risk myelofibrosis. This is an observational study, meaning that participants will be followed to assess the effects of their treatment, but no intervention (treatments) will be given as a part of this study.

View this trial on ClinicalTrials.gov