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Allografting

Allogeneic stem cell transplantation from matched related and unrelated donors in thalassemia major patients using a reduced toxicity fludarabine-based regimen

Bone Marrow Transplantation volume 40, pages 957–964 (2007)Cite this article

Abstract

The only radical cure for thalassemia major patients today is the replacement of the defective hematopoietic system by allogeneic stem cell transplantation (allo-SCT). The major obstacles for the application of allo-SCT even from matched family members have been the transplant-related morbidity and mortality and graft failure that is usually associated with the recurrence of the thalassemia hematopoiesis. The outcome of allo-SCT from HLA-identical family donors is largely dependent on the age of the recipient as well as on pretransplant parameters reflecting the degree of organ damage from iron overload. In this study we report our experience of allo-SCT from matched related and unrelated donors, using a reduced toxicity conditioning consisting of fludarabine, busulfan or more recently busulfex and antithymocyte globulin, in a cohort of 20 patients with thalassemia major. The regimen-related toxicity was minimal, while the incidence of acute grade II–IV and chronic GVHD was 25 and 25%, respectively. With a median follow-up period of 39 months (range: 5–112 months) the overall survival was 100%, while thalassemia-free survival was 80%. Although the results of our study look promising, larger cohorts of patients and prospective clinical trials are required to confirm the benefits of our approach as a possible better alternative to the existing protocols.

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Acknowledgements

Work was carried out at the Danny Cunniff Leukemia Research Laboratory and we are thankful for the continuous support of our ongoing basic and clinical research. This research was partially supported by an unrestricted grant from ESP Pharma, Edison, NJ, USA.

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Authors and Affiliations

  1. Department of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah University Hospital, Jerusalem, Israel

    I B Resnick, M Aker, P Tsirigotis, M Y Shapira, A Abdul-Hai, M Bitan, B Gesundheit, A Amar, A Ackerstein, S Samuel, S Slavin & R Or

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  1. I B Resnick
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  2. M Aker
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  3. P Tsirigotis
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  4. M Y Shapira
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  5. A Abdul-Hai
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  11. S Slavin
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Correspondence to S Slavin.

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Resnick, I., Aker, M., Tsirigotis, P. et al. Allogeneic stem cell transplantation from matched related and unrelated donors in thalassemia major patients using a reduced toxicity fludarabine-based regimen. Bone Marrow Transplant 40, 957–964 (2007). https://doi.org/10.1038/sj.bmt.1705826

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