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.

Fiche publication

Date publication

décembre 2024

Journal

EJHaem

Auteurs

Membres identifiés du Cancéropôle Est :
Dr POCHON Cécile , Dr PAGLIUCA Simona

Tous les auteurs :
Danhardt L, Wiedemann A, Michel G, Dalle JH, Rialland F, Renard C, Jubert C, Maertens J, Sirvent A, Buchbinder N, Devalck C, Brichard B, Paillard C, Nguyen S, Paci A, Combarel D, Castelle M, Pagliuca S, Pochon C

Lien Pubmed

https://www.ncbi.nlm.nih.gov/pubmed/39691249

Résumé

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.