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

U. Anurathapan, S. Hongeng, S. Pakakasama, N. Sirachainan, D. Songdej, A. Chuansumrit, P. Charoenkwan, A. Jetsrisuparb, K. Sanpakit, P. Rujkijyanont, A. Meekaewkunchorn, Y. Lektrakul, P. Iamsirirak, P. Surapolchai, W. Satayasai, S. Sirireung, R. Sruamsiri, Pustika Amalia Wahidiyat, A. Ungkanont, S. IssaragrisilB. S. Andersson

Research output: Contribution to journalArticlepeer-review

53 Citations (Scopus)

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).

Original languageEnglish
Pages (from-to)813-818
Number of pages6
JournalBone Marrow Transplantation
Volume51
Issue number6
DOIs
Publication statusPublished - 1 Jun 2016

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