Skip to main content
OpenTrials
Recruiting

NCT Number: NCT02934477

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.

Recruiting

Interested in participating?

Request Info

Key information

Conditions

Myelofibrosis Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Myeloproliferative Disorders Primary Myelofibrosis

Age range

55 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Center for International Blood and Marrow Transplant Research

Minneapolis, Minnesota, 55401, United States

Location status: Recruiting

Location contact

Michael Tierney

CONTACT

[email protected]

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

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.

Primary outcomes

  1. Compare five year survival

    Time frame: Five years post transplant

    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).

Secondary outcomes

  1. Compare leukemia-free survival

    Time frame: Five years post transplant

    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.

  2. Cumulative incidences of chronic GVHD

    Time frame: Five years post transplant

    Occurrence of symptoms in any organ system fulfilling the criteria of chronic GVHD. Patients are censored at last follow-up or second transplant.

  3. Cumulative incidences of acute GVHD

    Time frame: Five years post transplant

    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.

  4. Cumulative incidence of treatment related mortality

    Time frame: Five years post transplant

    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.

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

    Time frame: Five years post transplant

    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.

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

    Time frame: Five years post transplant

    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.

  7. The impact of certain patient, disease and HCT related factors on hematopoietic recovery in the alloHCT arm.

    Time frame: Five years post transplant

    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.

  8. The impact of certain patient, disease and HCT related factors on acute and chronic GVHD in the alloHCT arm.

    Time frame: Five years post transplant

    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.

  9. The impact of certain patient, disease and HCT related factors on treatment related mortality in the alloHCT arm.

    Time frame: Five years post transplant.

    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.

  10. The impact of certain patient, disease and HCT related factors on relapse.

    Time frame: Five years post transplant.

    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.

Study contacts

Contact information is provided by the study sponsor or research team.

Patricia Steinert, PhD

CONTACT

[email protected]

414-805-0700

Stephanie Farnia

CONTACT

[email protected]

763-406-8640

Sponsors and collaborators

Lead sponsor

Center for International Blood and Marrow Transplant Research

Network

Collaborators

  • National Cancer Institute (NCI)
  • National Institutes of Health (NIH)
  • National Marrow Donor Program

Registry information

Important dates

Study start
2016
Primary completion
2026
Study completion
2027
First posted
Oct 17, 2016
Registry last updated
Aug 30, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.