Treosulfan-based reduced toxicity hematopoietic stem cell transplantation in X-linked agammaglobulinemia: A cost-effective alternative to long-term immunoglobulin replacement in developing countries.
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| Abstract |
X-linked agammaglobulinemia (XLA) is a primary antibody disorder due to a mutation in the Bruton tyrosine kinase gene that requires lifelong immunoglobulin replacement resulting in a significant economic burden and treatment abandonment. Hematopoietic stem cell transplantation (HSCT) offers an alternative option for complete cure. In our series, two children with XLA underwent successful HSCT using a myeloablative conditioning with thiotepa, treosulfan, and fludarabine from a matched sibling donor. The second child had rejected his first graft following a busulfan-based regimen with resultant autologous reconstitution. At 6 months post-HSCT, serum IgG were normal, off IVIG, and had no infections. Both children after a median follow-up of 20 months have 100% chimerism. Treosulfan-based reduced toxicity myeloablative HSCT has encouraging results with a positive impact on the socioeconomics in developing countries.
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| Authors | Venkateswaran Vellaichamy Swaminathan, Ramya Uppuluri, Shivani Patel, Kesavan Melarcode Ramanan, Nikila Ravichandran, Indira Jayakumar, Lakshman Vaidhyanathan, Revathi Raj |
| Journal | Pediatric transplantation (Pediatr Transplant) Vol. 24 Issue 1 Pg. e13625 (02 2020) ISSN: 1399-3046 [Electronic] Denmark |
| PMID | 31821668 (Publication Type: Case Reports) |
| Copyright | © 2019 Wiley Periodicals, Inc. |
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