Myelofibrosis Clinical Trial
Official title:
Prospective Assessment of Allogeneic Hematopoietic Cell Transplantation in Patients With Myelofibrosis
This observational study will compare outcomes of a prospectively-enrolled cohort of Hematopoietic Stem Cell Transplant (HCT) recipients with outcomes of a cohort of age-matched historical non-HCT controls. Patients undergoing alloHCT will receive HCT in a US transplant center and be reported to the Center for International Blood and Marrow Transplant Research (CIBMTR) using well-established CIBMTR report forms and data collection procedures as well as a study-specific supplemental form. Data on the historical non-HCT controls will be collected at 14 US academic centers. These centers will provide data on all consecutive patients with PMF, post-ET MF, or post-PV MF referred to their institutions between 2000 and 2012.
Status | Recruiting |
Enrollment | 650 |
Est. completion date | October 2027 |
Est. primary completion date | November 2026 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 55 Years and older |
Eligibility | 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. |
Country | Name | City | State |
---|---|---|---|
United States | Center for International Blood and Marrow Transplant Research | Minneapolis | Minnesota |
Lead Sponsor | Collaborator |
---|---|
Center for International Blood and Marrow Transplant Research | National Cancer Institute (NCI), National Institutes of Health (NIH), National Marrow Donor Program |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Compare five year survival | Compare the five-year survival probabilities from DIPSS assessment between the two study arms: alloHCT recipients (arm 1) and non-HCT therapies (ruxolitinib / best supportive care) recipients (arm 2). | Five years post transplant | |
Secondary | Compare leukemia-free survival | Compare leukemia-free survival at five years from DIPSS assessment date to the date of progression to AML or death from any cause, whichever comes first. Two co-secondary analyses will be conducted, one for all alloHCT patients versus Arm 2 and one for the subset of patients receiving MAC prior to alloHCT versus Arm 2. Observation is censored at the date of last follow-up for patients known to be alive without leukemia. Progression to AML is defined as >20% leukemia blasts in bone marrow or in the peripheral blood. | Five years post transplant | |
Secondary | Cumulative incidences of chronic GVHD | Occurrence of symptoms in any organ system fulfilling the criteria of chronic GVHD. Patients are censored at last follow-up or second transplant. | Five years post transplant | |
Secondary | Cumulative incidences of acute GVHD | Occurrence of grade II, III, and/or IV skin, gastrointestinal, or liver abnormalities fulfilling the Consensus criteria of acute GVHD. Patients are censored at last follow-up or second transplant. | Five years post transplant | |
Secondary | Cumulative incidence of treatment related mortality | Death from any cause in the first 28 days post-transplantation, irrespective of relapse status. Death beyond day +28 will only be considered transplant-related if the disease is in remission. This event will be summarized as a cumulative incidence estimate with relapse/persistence as the competing risk. | Five years post transplant | |
Secondary | The impact of certain patient, disease and HCT related factors on survival in the alloHCT arm. | Evaluation of the impact of response to ruxolitinib therapy, patient age (<65 years vs. >= 65 years, disease duration and DIPSS on overall survival in the alloHCT arm. The time to event in the analyses will start at the time of transplant. | Five years post transplant | |
Secondary | The impact of certain patient, disease and HCT related factors on leukemia free survival in the alloHCT arm. | Evaluation of the impact of response to ruxolitinib therapy, patient age (<65 vs. >= 65 years), disease duration and DIPSS on leukemia free survival in the alloHCT arm. The time to event in the analyses will start at the time of transplant. | Five years post transplant | |
Secondary | The impact of certain patient, disease and HCT related factors on hematopoietic recovery in the alloHCT arm. | Evaluation of the impact of response to ruxolitinib therapy, patient age (<65 vs >=65 years), disease duration and DIPSS on hematopoietic recovery in the alloHCT arm. The time to event in the analyses will start at the time of transplant. | Five years post transplant | |
Secondary | The impact of certain patient, disease and HCT related factors on acute and chronic GVHD in the alloHCT arm. | Evaluation of the impact of response to ruxolitinib therapy, patient age (<65 vs >= 65 years), disease duration and DIPSS on acute and chronic GVHD in the alloHCT arm. The time to event in the analyses will start at the time of transplant. | Five years post transplant | |
Secondary | The impact of certain patient, disease and HCT related factors on treatment related mortality in the alloHCT arm. | Evaluation of the impact of response to ruxolitinib therapy, patient age (<65 vs >=65 years), disease duration, and DIPSS on treatment related mortality in the alloHCT arm. The time to event in the analyses will start at the time of transplant. | Five years post transplant. | |
Secondary | The impact of certain patient, disease and HCT related factors on relapse. | Evaluation of the impact of response to ruxolitinib therapy, patient age (65 vs >=65 years), disease duration and DIPSS on relapse in the alloHCT arm. The time to event in the analyses will start at the time of transplant. | Five years post transplant. |
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