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

  • STATUS
    Recruiting
  • End date
    Feb 5, 2026
  • participants needed
    90
  • sponsor
    University Health Network, Toronto
Updated on 24 February 2021
cancer
stem cell transplantation
hydroxyurea
ruxolitinib
essential thrombocythemia
thrombocytosis
polycythemia

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.

Description

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.

Details
Condition Myelosclerosis with myeloid metaplasia, Bone Marrow Neoplasms, Myelofibrosis, High-Risk Cancer, High-Risk Cancer, High-Risk Cancer, High-Risk Cancer, High-Risk Cancer
Treatment hydroxyurea, Ruxolitinib, Hematopoietic Stem Cell Transplant
Clinical Study IdentifierNCT04217356
SponsorUniversity Health Network, Toronto
Last Modified on24 February 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Recruitment Part
Documented diagnosis of pre-fibrotic primary myelofibrosis (pre-fibrotic PMF), overt PMF, post-polycythemia MF (PPV-MF) or post-essential thrombocythemia MF (PET-MF) confirmed by bone marrow biopsy
Have been tested or have results available for phenotypic driver mutations (JAK2/CALR/MPL) and high molecular risk (HMR) mutations using a broad myeloid malignancies targeted gene panel
Eastern Cooperative Oncology Group (ECOG) performance status 0-2
Able to provide informed consent
Adequate organ function
Donor search initiated or patient is agreeable to donor search
Meet the definition/criteria for high-risk myelofibrosis
Study Arm Allocation
Grade of fibrosis on bone marrow biopsy available according to World Health Organization (WHO) criteria
Results available for phenotypic driver mutations (JAK2/CALR/MPL) and targeted sequencing results using a broad myeloid malignancy panel with a minimal requirement to include results on High molecular risk (HMR) mutations such as ASXL1/EZH2/IDH1/IDH2/SRSF2/U2AF1/TP53
ECOG performance status 0-2
Adequate organ function
Information on donor search and donor type available

Exclusion Criteria

Recruitment Part
Blasts in peripheral blood or bone marrow 10%
For patients already on ruxolitinib at study entry, and meet the criteria of ruxolitinib failure
Previous history of transformation to blast phase or acute myeloid leukemia
Received allogeneic stem cell transplant for myeloproliferative neoplasm
Presence of an active uncontrolled infection
Myocardial infarction in the preceding 3 months
Active hepatitis A, B or C
Known human immunodeficiency virus (HIV) positive
History of active malignancy in the previous 2 years, except basal cell carcinoma or squamous cell carcinoma of skin or stage 0 cervical cancer
Any psychiatric illness or social circumstances or significant co-morbid conditions that will prevent patient from proceeding to allogeneic hematopoietic cell transplantation
Pregnant or breastfeeding women
Study Arm Allocation
Blasts in peripheral blood or bone marrow 10%
Meet the criteria of ruxolitinib failure
Presence of an active uncontrolled infection
Myocardial infarction in the preceding 3 months
Active hepatitis A, B or C
Known HIV positive
History of active malignancy in the previous 2 years, except basal cell carcinoma or squamous cell carcinoma of skin or stage 0 cervical cancer
Pregnant or breastfeeding women
Any psychiatric illness or social circumstances or significant co-morbid conditions that will prevent patient from proceeding to allogeneic hematopoietic cell transplantation
Time between registration and allocation of study arm >24 weeks
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