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

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

Abstract Context 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...

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Main Authors: Laura Danhardt, Arnaud Wiedemann, Gerard Michel, Jean‐Hugues Dalle, Fanny Rialland, Cécile Renard, Charlotte Jubert, Johan Maertens, Anne Sirvent, Nimrod Buchbinder, Christine Devalck, Bénédicte Brichard, Catherine Paillard, Stephanie Nguyen, Angelo Paci, David Combarel, Martin Castelle, Simona Pagliuca, Cecile Pochon
Format: Article
Language:English
Published: Wiley 2024-12-01
Series:eJHaem
Subjects:
cord blood
graft failure
GVHD
HSCT
mucopolysaccharidosis
outcomes
Online Access:https://doi.org/10.1002/jha2.1056
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