Hematopoietic stem cell transplant: Intravenous infusion of hematopoietic stem cells from a donor.
Ruxolitinib: Ruxolitinib is type of drug called a janus kinase (JAK) inhibitor. Ruxolitinib is taken orally (by mouth).
Hydroxyurea: Hydroxyurea is a type of drug called an antimetabolite. Hydroxyurea is taken orally (by mouth).
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.
Eligibility
Sex
ALL
Min age
18 Years
Max age
70 Years
Healthy volunteers
No
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
Primary outcome measure(s)
Number of patients allocated to hematopoietic stem cell transplantation (HCT) — 5 years
Number of patients allocated to best available non-transplant therapies (BAT) — 5 years
Overall survival rate of patients who receive hematopoietic stem cell transplantation (HCT) — 5 years Time from study allocation to death or last follow up.
Overall survival rate of patients who receive best available non-transplant therapies (BAT) — 5 years Time from study allocation to death or last follow up.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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