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- Title
Treosulfan‐based reduced toxicity hematopoietic stem cell transplantation in X‐linked agammaglobulinemia: A cost‐effective alternative to long‐term immunoglobulin replacement in developing countries.
- Authors
Vellaichamy Swaminathan, Venkateswaran; Uppuluri, Ramya; Patel, Shivani; Melarcode Ramanan, Kesavan; Ravichandran, Nikila; Jayakumar, Indira; Vaidhyanathan, Lakshman; Raj, Revathi
- 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.
- Subjects
HEMATOPOIETIC stem cell transplantation; DEVELOPING countries; AGAMMAGLOBULINEMIA; PROTEIN-tyrosine kinases
- Publication
Pediatric Transplantation, 2020, Vol 24, Issue 1, pN.PAG
- ISSN
1397-3142
- Publication type
Article
- DOI
10.1111/petr.13625