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      Hematopoietic stem cell transplantation for homozygous β-thalassemia and β-thalassemia/hemoglobin E patients from haploidentical donors.

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          Abstract

          Thalassemia-free survival after allogeneic stem cell transplantation (SCT) is about 80-90% with either matched-related or -unrelated donors. We explored the use of a mismatched-related ('haplo- ') donor. All patients received two courses of pretransplant immunosuppressive therapy (PTIS) with fludarabine (Flu) and dexamethasone (Dxm). After two courses of PTIS, a conditioning regimen of rabbit antithymocyte globulin, Flu and IV busulfan (Bu) was given followed by T-cell-replete peripheral blood progenitor cells. GvHD prophylaxis consisted of cyclophosphamide (Cy) on days SCT +3 and +4 (post-Cy), and on day SCT +5 tacrolimus or sirolimus was started together with a short course of mycophenolate mofetil. Thirty-one patients underwent haplo-SCT. Their median age was 10 years (range, 2-20 years). Twenty-nine patients engrafted with 100% donor chimerism. Two patients suffered primary graft failure. Median time to neutrophil engraftment was 14 days (range, 11-18 days). Five patients developed mild to moderate, reversible veno-occlusive disease, while nine patients developed acute GvHD grade II. Only five patients developed limited-chronic GvHD. Projected overall and event-free survival rates at 2 years are 95% and 94%, respectively. The median follow up time is 12 months (range, 7-33 months).

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          Author and article information

          Journal
          Bone Marrow Transplant.
          Bone marrow transplantation
          Springer Nature
          1476-5365
          0268-3369
          Jun 2016
          : 51
          : 6
          Affiliations
          [1 ] Department of Pediatrics, Faculty of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
          [2 ] Department of Pediatrics, Chiangmai University Hospital, Chiangmai, Thailand.
          [3 ] Department of Pediatrics, Khonkaen University, Khonkaen, Thailand.
          [4 ] Department of Pediatrics, Siriraj Hospital, Mahidol University, Bangkok, Thailand.
          [5 ] Department of Pediatrics, Phramongkutklao Hospital, Bangkok, Thailand.
          [6 ] Queen Sirikit National Institute of Child Health, Bangkok, Thailand.
          [7 ] Sunpasitthiprasong Hospital, Ubon Ratchathani, Thailand.
          [8 ] Samitivej Hospital, Bangkok, Thailand.
          [9 ] Department of Pediatrics, Thammasat University, Pathum Thani, Thailand.
          [10 ] Department of Pharmacy Practice, Center of Pharmaceutical Outcomes Research, Naresuan University, Phitsanulok, Thailand.
          [11 ] Department of Child Health, Cipto Mangunkusumo Hospital, Universitas Indonesia, Jakarta, Indonesia.
          [12 ] Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
          [13 ] Department of Medicine, Siriraj Hospital, Mahidol University, Bangkok, Thailand.
          [14 ] Department of Stem Cell Transplantation and Cellular Therapy, University of Texas MD Anderson Cancer Center, Houston, TX, USA.
          Article
          bmt20167 NIHMS801491
          10.1038/bmt.2016.7
          4957689
          26878659
          68d70447-806b-4311-a9bc-4530977a9729
          History

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