Prospective Assessment of Allogeneic Hematopoietic Cell Transplantation in Patients With Myelofibrosis

NCT ID: NCT02934477

Last Updated: 2023-08-30

Basic Information

• Recruitment Status: RECRUITING
• Total Enrollment: 650 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2016-11-30
• Study Completion Date: 2027-10-31

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.

Conditions

Myelofibrosis

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Hematopoietic Stem Cell Transplant (HCT)

Patients undergoing alloHCT in a US transplant center and reported to the CIBMTR

Hematopoietic Stem Cell Transplant

Intervention Type: OTHER

This observational study will compare outcomes of prospectively enrolled HCT recipients with outcomes of a cohort of age-matched non-HCT controls.

Non-HCT

Historical non-transplant controls collected from 14 US academic centers. 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.

No interventions assigned to this group

Interventions

Hematopoietic Stem Cell Transplant

This observational study will compare outcomes of prospectively enrolled HCT recipients with outcomes of a cohort of age-matched non-HCT controls.

Intervention Type: OTHER

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.

• Minimum Eligible Age: 55 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Marrow Donor Program

OTHER

Sponsor Role: collaborator

National Institutes of Health (NIH)

NIH

Sponsor Role: collaborator

National Cancer Institute (NCI)

NIH

Sponsor Role: collaborator

Center for International Blood and Marrow Transplant Research

NETWORK

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Wael Saber, MD

Role: STUDY_CHAIR

Medical College of Wisconsin

Laura Michaelis, MD

Role: STUDY_CHAIR

Medical College of Wisconsin

Locations

Center for International Blood and Marrow Transplant Research

Minneapolis, Minnesota, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Patricia Steinert, PhD

Role: CONTACT

psteinert@mcw.edu

414-805-0700

Stephanie Farnia

Role: CONTACT

sfarnia@nmdp.org

763-406-8640

Facility Contacts

Michael Tierney

Role: primary

DatabaseIRB@NMDP.org

Related Links

https://www.cibmtr.org/pages/index.aspx

CIBMTR

Other Identifiers

U24CA076518

Identifier Type: NIH

Identifier Source: secondary_id

View Link

16-CMS-MF

Identifier Type: -

Identifier Source: org_study_id