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

Titre officiel

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)

Sommaire:

L’objectif de cette étude est de déterminer l’efficacité de la greffe de cellules souches hématopoïétiques (GCSH) par rapport aux meilleurs traitements possibles (MTP) sans greffe chez les patients atteints de myélofibrose à risque élevé. Pour ce faire, on demandera aux participants de choisir le traitement qu’ils préfèrent recevoir (GCSH ou MTP) et on comparera ensuite les résultats des participants dans les deux groupes de traitement.

Description de l'essai

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.

Voir cet essai sur ClinicalTrials.gov