Assessing Hematopoietic Cell Transplantation for Myelofibrosis
Prospective Assessment of Allogeneic Hematopoietic Cell Transplantation in Patients With Myelofibrosis
This study is trying to see if patients with myelofibrosis who receive a stem cell transplant do better than those who didn't have the transplant.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 650 (estimated) |
| Ages | 55 Years and up |
| Sex | All |
| Sponsor | Center for International Blood and Marrow Transplant Research Research network |
| Locations | 1 site (Minneapolis, Minnesota) |
| Trial ID | NCT02934477 on ClinicalTrials.gov |
What this trial studies
This observational study aims to compare outcomes between patients with myelofibrosis who undergo allogeneic hematopoietic cell transplantation (HCT) and a cohort of historical non-HCT controls. Eligible patients include those with primary myelofibrosis, post-essential thrombocythemia myelofibrosis, or post-polycythemia vera myelofibrosis, aged 55 or older with intermediate-2 or high-risk disease. Data will be collected from multiple US academic centers, focusing on both HCT recipients and historical controls to evaluate the effectiveness of the treatment. The study will enroll approximately 650 patients receiving HCT and collect data from around 2,400 historical controls.
Who should consider this trial
Good fit: Ideal candidates are patients aged 55 or older with intermediate-2 or high-risk myelofibrosis.
Not a fit: Patients with low-risk myelofibrosis or those not eligible for HCT may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the effectiveness of HCT for patients with myelofibrosis, potentially improving treatment options.
How similar studies have performed: Previous studies have shown promising outcomes for HCT in similar patient populations, indicating a potential for success in this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria:
* Patients fulfilling the following criteria will be eligible for inclusion in the study:
* PMF, post-ET MF, or post-PV MF.
* Int-2 or high-risk disease as determined by the DIPSS.
* Age ≥55 at the time of DIPSS assessment.
* For the alloHCT arm:
* Donors must be a 6/6 HLA-matched related donors, defined by Class I (HLA-A and -B) intermediate resolution or high resolution DNA-based typing and Class II (HLA-DRBI) at high resolution DNA-based typing (but not monozygotic twins) OR an 8/8 HLA-A, -B, -C, and -DRB1 at high resolution DNA-based typing matched unrelated donor identified through the National Marrow Donor Program (NMDP)/Be The Match. Donors must meet institutional or NMDP/Be The Match selection criteria; there is no age restriction for sibling donors.
* Both peripheral blood stem cells and bone marrow grafts are allowed.
* All conditioning regimen intensities are allowed.
* All GVHD prophylaxis regimens are allowed.
* Haploidentical donors are allowed in the Haploidentical Donor Study
Exclusion Criteria:
* Patients with the following criteria will be ineligible for entry into the study:
* AlloHCT using umbilical cord blood unit(s) or HLA-mismatched adult donors (\< 6/6 HLA alleles for related and \< 8/8 HLA alleles for unrelated).
* Overlap syndromes.
Where this trial is running
Minneapolis, Minnesota
- Center for International Blood and Marrow Transplant Research — Minneapolis, Minnesota, United States (Recruiting)
Study contacts
- Study coordinator: Patricia Steinert, PhD
- Email: psteinert@mcw.edu
- Phone: 414-805-0700
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.