Prospective Assessment of Allogeneic Hematopoietic Cell Transplantation in Patients With Myelofibrosis

NCT02934477 | Recruiting

• 2 other identifiers: 16-CMS-MFU24CA076518
• Study Type: observational
• Target: 650
• Locations: 1 country
• Sites: 1

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.

Conditions

Myelofibrosis

Keywords

Myelofibrosis; Hematopoietic Stem Cell Transplant; Medicare

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (10)

• Compare leukemia-free survival

  Five years post transplant

• Cumulative incidences of chronic GVHD

  Five years post transplant

• Cumulative incidences of acute GVHD

  Five years post transplant

• Cumulative incidence of treatment related mortality

  Five years post transplant

• The impact of certain patient, disease and HCT related factors on survival in the alloHCT arm.

  Five years post transplant

Study Arms (2)

Hematopoietic Stem Cell Transplant (HCT)

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

Other: Hematopoietic Stem Cell Transplant

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.

Interventions

Hematopoietic Stem Cell Transplant OTHER

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

Hematopoietic Stem Cell Transplant (HCT)

Eligibility Criteria

• Age: 55 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Center for International Blood and Marrow Transplant Research: lead
• National Marrow Donor Program: collaborator
• National Institutes of Health (NIH): collaborator
• National Cancer Institute (NCI): collaborator

Study Sites (1)

Center for International Blood and Marrow Transplant Research

Minneapolis, Minnesota, 55401, United States

RECRUITING

Related Links

• CIBMTR

MeSH Terms

Conditions

Primary Myelofibrosis

Interventions

Stem Cell Transplantation

Condition Hierarchy (Ancestors)

Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Cell Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Therapeutics; Transplantation; Surgical Procedures, Operative

Study Officials

• Wael Saber, MD

  Medical College of Wisconsin

  STUDY CHAIR

• Laura Michaelis, MD

  Medical College of Wisconsin

  STUDY CHAIR

Central Study Contacts

Patricia Steinert, PhD

CONTACT

414-805-0700; psteinert@mcw.edu

Stephanie Farnia

CONTACT

763-406-8640; sfarnia@nmdp.org

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: NETWORK
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 30, 2016
• First Posted: October 17, 2016
• Study Start: November 1, 2016
• Primary Completion (Estimated): November 1, 2026
• Study Completion (Estimated): October 1, 2027
• Last Updated: August 30, 2023

Record last verified: 2023-08

Locations

US (1)