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Bone Marrow Transplant. 2014 Apr;49(4):519-24. doi: 10.1038/bmt.2013.224. Epub 2014 Jan 27.

Long-term outcome of HLA-haploidentical hematopoietic SCT without in vitro T-cell depletion for adult severe aplastic anemia after modified conditioning and supportive therapy.

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Department of Hematology, Xinqiao Hospital, Third Military Medical University, Chongqing, China.
Department of Health Statistics, College of Military Preventive Medicine, Third Military Medical University, Chongqing, China.
Department of Hematology, General Hospital of Chengdu Military Region of PLA, Sichuan, China.
Department of Hematology, Sichuan Provincial Peoples Hospital, Sichuan, China.
Department of Hematology, General Hospital of Kunming Military Region of PLA, Yunnan, China.
Department of Hematology, Second Yunnan Provincial peoples hospital, Yunnan, China.


HLA-haploidentical hematopoietic SCT (HSCT) is an option for severe aplastic anemia (SAA) patients. Here, we evaluated the outcomes of 26 adult-SAA patients who received HLA-haploidentical HSCT in five transplant centers in southwestern China. Most of the patients in this study failed prior therapy and were transfused heavily before the transplantation. The patients received fludarabine+cyclophosphamide+antithymocyte globulin as conditioning regimens and then unmanipulated peripheral blood plus marrow transplantation. Micafungin, i.v. Ig and recombinant human TPO were used for post-grafting infection prevention and supportive care. Of 26 patients, 25 achieved engraftment at a median of 13 days (range, 11-19 days) after HSCT. One of 25 patients experienced graft rejection and did not achieve sustained engraftment after second HSCT. Therefore, the final engraftment rate was 92.3%. Three of 25 (12%) patients developed acute GVHD, 10 of 25 (40%) patients developed chronic GVHD (9 with limited whereas the other with extensive). The OS rate was 84.6% and the average follow-up time was 1313.2 (738-2005) days for surviving patients. This encouraging result suggests that HLA-haploidentical HSCT is an effective therapeutic option for adults with acquired SAA if an HLA-identical donor is not available.

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