Incidence and risk factors of graft failure in allogeneic hematopoietic stem cell transplantation for mucopolysaccharidosis in a nationwide pediatric cohort. A study on behalf of the Francophone Society of Bone Marrow Transplantation and Cellular Therapy

Danhardt, Laura;Wiedemann, Arnaud;Michel, Gerard;Dalle, Jean‐Hugues;Pochon, Cécile;et.al.
(2024) eJHaem — Vol. 5, n° 6, p. 1295-1300 (2024)

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Authors
  • Danhardt, Laura
    Author
  • Wiedemann, Arnaud
    Author
  • Michel, Gerard
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  • Dalle, Jean‐Hugues
    Author
  • Brichard, BénédicteUCLouvain
    Author
  • Pochon, Cécile
    Author
  • et. al.
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Abstract
Mucopolysaccharidosis (MPS) requires urgent treatment to prevent neurological damage. While gene therapy holds promise for effectively treating these diseases with minimal toxicity, access remains limited for most patients. Consequently, advancing allogeneic hematopoietic stem cell transplantation (HSCT) for young children is crucial. Since the 2010s, cord blood (CB) transplants with reduced-toxicity conditioning (RTC) have become the standard of care.
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Danhardt, L., Wiedemann, A., Michel, G., Dalle, J.-H., Rialland, F., Renard, C., Jubert, C., Maertens, J., Sirvent, A., Buchbinder, N., Devalck, C., Brichard, B., Pochon, C., & et al. (2024). Incidence and risk factors of graft failure in allogeneic hematopoietic stem cell transplantation for mucopolysaccharidosis in a nationwide pediatric cohort. A study on behalf of the Francophone Society of Bone Marrow Transplantation and Cellular Therapy. eJHaem, 5(6), 1295-1300. https://doi.org/10.1002/jha2.1056 (Original work published 2024)