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Donor types and outcomes of transplantation in myelofibrosis: a CIBMTR study
Tania Jain1, Noel Estrada-Merly2, M Queralt Salas3
1Division of Hematological Malignancies and Bone Marrow Transplantation, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University, Baltimore, MD.
Insights
Hematopoietic cell transplantation (HCT) outcomes in myelofibrosis show matched sibling donors offer superior survival. Haploidentical donors are a viable alternative, particularly for ethnic minorities.
Area of Science:
- Hematology
- Oncology
- Transplantation Immunology
Background:
- Myelofibrosis is a serious bone marrow disorder.
- Hematopoietic cell transplantation (HCT) is a potential cure but donor availability can be a challenge.
- The role of different donor types in HCT for myelofibrosis requires ongoing evaluation.
Purpose of the Study:
- To evaluate the impact of various donor types on HCT outcomes in patients with myelofibrosis.
- To assess the increasing use of haploidentical donors and their outcomes compared to other donor types.
- To determine if haploidentical HCT is a viable option, especially for underrepresented ethnic minorities.
Main Methods:
- Retrospective analysis of 1597 patients undergoing HCT for myelofibrosis from 2013-2019 using CIBMTR registry data.
- Comparison of overall survival (OS), graft failure, relapse, and disease-free survival based on donor type: matched sibling donor (MSD), matched unrelated donor (MUD), mismatched unrelated donor (MMUD), and haploidentical donor.
- Focus on 1032 study-eligible patients receiving peripheral blood grafts for chronic-phase myelofibrosis.
Main Results:
- Haploidentical donor use increased significantly from 3% to 19% between 2013 and 2019.
- MSD-HCT was associated with superior OS in the first 3 months post-transplant compared to haploidentical, MUD, and MMUD.
- No significant difference in OS beyond 3 months, relapse, or disease-free survival was observed between haploidentical, MUD, and MMUD donors.
- Graft failure was more common in patients with advanced disease and those receiving nonmyeloablative conditioning.
Conclusions:
- While MSD-HCT offers superior outcomes, haploidentical HCT demonstrates comparable results to MUD and MMUD HCT in myelofibrosis.
- Haploidentical HCT, particularly with post-transplantation cyclophosphamide, is established as a feasible and effective treatment option.
- This approach significantly broadens HCT accessibility for ethnic minorities who are often underrepresented in donor registries.
Abstract:
We evaluate the impact of donor types on outcomes of hematopoietic cell transplantation (HCT) in myelofibrosis, using the Center for International Blood and Marrow Transplant Research registry data for HCTs done between 2013 and 2019. In all 1597 patients, the use of haploidentical donors increased from 3% in 2013 to 19% in 2019. In study-eligible 1032 patients who received peripheral blood grafts for chronic-phase myelofibrosis, 38% of recipients of haploidentical HCT were non-White/Caucasian. Matched sibling donor (MSD)-HCTs were associated with superior overall survival (OS) in the first 3 months (haploidentical hazard ratio [HR], 5.80 [95% confidence interval (CI), 2.52-13.35]; matched unrelated (MUD) HR, 4.50 [95% CI, 2.24-9.03]; mismatched unrelated HR, 5.13 [95% CI, 1.44-18.31]; P < .001). This difference in OS aligns with lower graft failure with MSD (haploidentical HR, 6.11 [95% CI, 2.98-12.54]; matched unrelated HR, 2.33 [95% CI, 1.20-4.51]; mismatched unrelated HR, 1.82 [95% CI, 0.58-5.72]). There was no significant difference in OS among haploidentical, MUD, and mismatched unrelated donor HCTs in the first 3 months. Donor type was not associated with differences in OS beyond 3 months after HCT, relapse, disease-free survival, or OS among patients who underwent HCT within 24 months of diagnosis. Patients who experienced graft failure had more advanced disease and commonly used nonmyeloablative conditioning. Although MSD-HCTs were superior, there is no significant difference in HCT outcomes from haploidentical and MUDs. These results establish haploidentical HCT with posttransplantation cyclophosphamide as a viable option in myelofibrosis, especially for ethnic minorities underrepresented in the donor registries.
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