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Inherited and Genetic Disorders

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

Bone Marrow Transplantation volume 51, pages 813–818 (2016)Cite this article

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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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Acknowledgements

This work was supported in part by grants from the Ramathibodi Foundation, the National Research University Grant, the Mahidol University Research Grant, the Office of the Higher Education Commission and Mahidol University under the National Research University Initiative, Thailand Research Fund, Research Chair Grant from the National Science and Technology Development Agency (NSTDA) and the National Institutes of Health of United States (CCSG CA16672).

Author contributions

SH and BSA designed the research. SH, SP and UA performed the research. SH, UA, RS and BSA analyzed the data and performed the statistical analyses. SH, SP, UA, RS and BSA wrote the manuscript. NS, DS, AC, PC, AJ, KS, PR, AM, YL, PI, PS, WS, SS, PA, AU and SI enrolled the patients.

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Authors and Affiliations

  1. Department of Pediatrics, Faculty of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand

    U Anurathapan, S Hongeng, S Pakakasama, N Sirachainan, D Songdej, A Chuansumrit & S Sirireung

  2. Department of Pediatrics, Chiangmai University Hospital, Chiangmai, Thailand

    P Charoenkwan

  3. Department of Pediatrics, Khonkaen University, Khonkaen, Thailand

    A Jetsrisuparb

  4. Department of Pediatrics, Siriraj Hospital, Mahidol University, Bangkok, Thailand

    K Sanpakit

  5. Department of Pediatrics, Phramongkutklao Hospital, Bangkok, Thailand

    P Rujkijyanont

  6. Queen Sirikit National Institute of Child Health, Bangkok, Thailand

    A Meekaewkunchorn

  7. Sunpasitthiprasong Hospital, Ubon Ratchathani, Thailand

    Y Lektrakul

  8. Samitivej Hospital, Bangkok, Thailand

    P Iamsirirak

  9. Department of Pediatrics, Thammasat University, Pathum Thani, Thailand

    P Surapolchai & W Satayasai

  10. Department of Pharmacy Practice, Center of Pharmaceutical Outcomes Research, Naresuan University, Phitsanulok, Thailand

    R Sruamsiri

  11. Department of Child Health, Cipto Mangunkusumo Hospital, Universitas Indonesia, Jakarta, Indonesia

    P A Wahidiyat

  12. Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand

    A Ungkanont

  13. Department of Medicine, Siriraj Hospital, Mahidol University, Bangkok, Thailand

    S Issaragrisil

  14. Department of Stem Cell Transplantation and Cellular Therapy, University of Texas MD Anderson Cancer Center, Houston, TX, USA

    B S Andersson

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Correspondence to S Hongeng or S Pakakasama.

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Anurathapan, U., Hongeng, S., Pakakasama, S. et al. Hematopoietic stem cell transplantation for homozygous β-thalassemia and β-thalassemia/hemoglobin E patients from haploidentical donors. Bone Marrow Transplant 51, 813–818 (2016). https://doi.org/10.1038/bmt.2016.7

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