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. 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

U Anurathapan  1 , S Hongeng  1 , S Pakakasama  1 , N Sirachainan  1 , D Songdej  1 , A Chuansumrit  1 , P Charoenkwan  2 , A Jetsrisuparb  3 , K Sanpakit  4 , P Rujkijyanont  5 , A Meekaewkunchorn  6 , Y Lektrakul  7 , P Iamsirirak  8 , P Surapolchai  9 , W Satayasai  9 , S Sirireung  1 , R Sruamsiri  10 , P A Wahidiyat  11 , A Ungkanont  12 , S Issaragrisil  13 , B S Andersson  14
Affiliations

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

U Anurathapan et al. Bone Marrow Transplant. 2016 Jun.

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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Conflict of interest statement

The authors declare no financial conflict(s) of interest.

Figures

Figure 1
Figure 1
Depiction of the transplant program with first pharmacological Preconditoning Therapy with ImmunoSuppressive drugs (PTIS), followed by reduced-toxicity conditioning with ATG, Fludarabine and IV Busulfan. For the haplo-identical donors we use post-Cy based GvHD prophylaxis and delayed calcineurin inhibitor-/ sirolimus therapy and short-course MMF, the latter two starting on day SCT +5. Please see Patients and Methods section for details.
Figure 2
Figure 2
Event free survival (EFS) and Overall survival (OS) of 31 thalassemia patients undergoing haploidentical hematopoietic stem cell transplantation (Haplo-SCT)
See this image and copyright information in PMC

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