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Abstract
Background. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the only proven curative therapy for chronic myeloid leukemia (CML), but lack of human leukocyte antigen (HLA)-matched sibling or unrelated donors has restricted its application. Recently, we developed an effective method for haploidentical allo-HSCT achieving comparable outcomes to HLA-identical transplantation.
Aim. To evaluate the outcomes of CML patients who underwent haploidentical allo-HSCT.
Methods. Ninety-three patients were treated with a modified busulfan(BU)/cyclophosphamide (CY)2 regimen, including antithymocyte globulin followed by unmanipulated blood and marrow transplantation.
Results. Our data showed that the cumulative incidence of acute graft-versus-host disease (GVHD) was 64.52%, and grade III–IV was 26.45%, 61.79% had chronic GVHD, and 28.93% had extensive chronic GVHD. Non-relapse mortality varied at 8.72% (100 days), 20.72% (1 year) and 20.72% (2 years). Probability of 1-year and 4-year leukemia-free survival was similar in chronic phase (CP) 1, CP2/CR2, accelerated phase, and blast crisis patients. Probability of 4-year overall survival varied as 76.5% (CP1), 85.7% (CP2/CR2), 73.3% (accelerated phase), and 61.5% (blast crisis). Multivariate analysis indicated that factors affecting transplantation outcomes were HLA-B+DR mismatches versus others for II–III acute GVHD and III–IV acute GVHD, the stage of disease at transplantation for relapse, and the time from diagnosis to transplantation for leukemia-free survival, overall survival, and transplantation-related mortality. In our protocol, survival of HSCT for advanced CML was similar to stable stage.
Conclusions. For patients lacking an HLA-identical related donor, haploidentical relatives are alternative HSCT donors.
Acknowledgements
This work is supported by New Century Excellent Talents in University (NECT-04-0011), Scientific Research Fund for Capital Medicine Development (grant no. 2006-1010), HI-tech research development program of China (grant no. 2006AA02Z4A0) and Program for Innovative Research Team in University (IRT0702). We would like to thank San Francisco Edit (www.sefedit.net) for their assistance in editing this manuscript.