Prospective Assessment of Allogeneic Hematopoietic Cell Transplantation in Patients With Myelofibrosis
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 650
- 试验地点
- 1
- 主要终点
- Compare five year survival
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 55 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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.
结局指标
主要结局
Compare five year survival
时间窗: 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).
次要结局
- Cumulative incidence of treatment related mortality(Five years post transplant)
- The impact of certain patient, disease and HCT related factors on hematopoietic recovery in the alloHCT arm.(Five years post transplant)
- Cumulative incidences of acute GVHD(Five years post transplant)
- The impact of certain patient, disease and HCT related factors on acute and chronic GVHD in the alloHCT arm.(Five years post transplant)
- Compare leukemia-free survival(Five years post transplant)
- Cumulative incidences of chronic GVHD(Five years post transplant)
- The impact of certain patient, disease and HCT related factors on survival in the alloHCT arm.(Five years post transplant)
- The impact of certain patient, disease and HCT related factors on leukemia free survival in the alloHCT arm.(Five years post transplant)
- The impact of certain patient, disease and HCT related factors on treatment related mortality in the alloHCT arm.(Five years post transplant.)
- The impact of certain patient, disease and HCT related factors on relapse.(Five years post transplant.)