Clinical trial NCT02934477: Prospective Assessment of Allogeneic Hematopoietic Cell Transplantation in Patients With Myelofibrosis
Official title: Prospective Assessment of Allogeneic Hematopoietic Cell Transplantation in Patients With Myelofibrosis
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Myelofibrosis
Keywords: Myelofibrosis
Hematopoietic Stem Cell Transplant
Medicare
Lead sponsor: Center for International Blood and Marrow Transplant Research
Sponsor class: NETWORK
Collaborators: name: National Marrow Donor Program
name: National Institutes of Health (NIH)
name: National Cancer Institute (NCI)
First posted: 2016-10-17
Enrollment: 650
Sex: ALL
Minimum age (years): 55
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with primary MF (PMF), post-essential thrombocythemia (ET) MF, or post-polycythemia vera (PV) MF, with intermediate-2 or high-risk disease as determined by the DIPSS, and aged ≥55 at the time of DIPSS assessment are eligible for this study. For the alloHCT arm of the HLA-Matched Donor HCT Study, donors must be either 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 donors; both peripheral blood stem cells and bone marrow grafts are allowed, and all conditioning regimen intensities and GVHD prophylaxis regimens are allowed. For the Haploidentical Donor Study, donors must be haploidentical.

Brief summary: 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.

Detailed description: Patients with primary MF (PMF), post-essential thrombocythemia (ET) MF, or post-polycythemia vera (PV) MF, with intermediate-2 or high-risk disease as determined by the DIPSS, and aged ≥55 at the time of DIPSS assessment are eligible for this study. For the allogeneic HCT arm of the HLA-Matched Donor HCT Study, donors must be either 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 donors; both peripheral blood stem cells and bone marrow grafts are allowed, and all conditioning regimen intensities and graph versus host disease (GVHD) prophylaxis regimens are allowed. For the Haploidentical Donor Study, donors must be haploidentical.

This study will target accrual of 650 patients receiving alloHCT, including approximately 225 receiving myeloablative conditioning. Participating centers are expected to provide data for approximately 2,400 patients to form the non-HCT historical control cohort.

Eligibility 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.

Primary outcomes:
measure: Compare five year survival
timeFrame: Five years post transplant
Secondary outcomes:
measure: Compare leukemia-free survival
timeFrame: Five years post transplant
measure: Cumulative incidences of chronic GVHD
timeFrame: Five years post transplant
measure: Cumulative incidences of acute GVHD
timeFrame: Five years post transplant
measure: Cumulative incidence of treatment related mortality
timeFrame: Five years post transplant
measure: The impact of certain patient, disease and HCT related factors on survival in the alloHCT arm.
timeFrame: Five years post transplant
measure: The impact of certain patient, disease and HCT related factors on leukemia free survival in the alloHCT arm.
timeFrame: Five years post transplant
measure: The impact of certain patient, disease and HCT related factors on hematopoietic recovery in the alloHCT arm.
timeFrame: Five years post transplant
measure: The impact of certain patient, disease and HCT related factors on acute and chronic GVHD in the alloHCT arm.
timeFrame: Five years post transplant
measure: The impact of certain patient, disease and HCT related factors on treatment related mortality in the alloHCT arm.
timeFrame: Five years post transplant.
measure: The impact of certain patient, disease and HCT related factors on relapse.
timeFrame: Five years post transplant.
Other outcomes:
Locations:
city: Minneapolis
country: United States
facility: Center for International Blood and Marrow Transplant Research
state: Minnesota
Study officials:
affiliation: Medical College of Wisconsin
name: Wael Saber, MD
role: STUDY_CHAIR
affiliation: Medical College of Wisconsin
name: Laura Michaelis, MD
role: STUDY_CHAIR
References: