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Bone Marrow Transplant. 2016 Jun;51(6):813-8. doi: 10.1038/bmt.2016.7. Epub 2016 Feb 15.

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

Author information

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.

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).

PMID:
26878659
PMCID:
PMC4957689
DOI:
10.1038/bmt.2016.7
[Indexed for MEDLINE]
Free PMC Article

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