Outcomes of patients undergoing allogeneic haematopoietic stem cell transplantation for congenital amegakaryocytic thrombocytopenia; a study on behalf of the PDWP of the EBMT.

Journal

Bone marrow transplantation

ISSN: 1476-5365

Titre abrégé: Bone Marrow Transplant

Pays: England

ID NLM: 8702459

Informations de publication

Date de publication:
18 Sep 2024

Historique:

received: 17 03 2024

accepted: 05 09 2024

revised: 27 07 2024

medline: 18 9 2024

pubmed: 18 9 2024

entrez: 17 9 2024

Statut: aheadofprint

Résumé

Congenital amegakaryocytic thrombocytopenia is a rare, inherited bone marrow failure syndrome. Allogeneic haematopoietic stem cell transplantation (allo-HSCT) is currently the only curative treatment. In this retrospective study, we analysed 66 patients with allo-HSCT, reported in the European Society for Blood and Marrow Transplantation (EBMT) registry. Bone marrow (BM) was the most widely used stem cell source (n = 40; 61%) followed by peripheral blood (PB) (n = 18; 27%), and unrelated umbilical cord blood (UCB) (n = 8; 12%). Most frequently was a HLA-matched graft from related (n = 26; 39%) and unrelated (n = 15; 23%) donors after a myeloablative busulfan-based conditioning regimen. GvHD prophylaxis was mostly cyclosporine and methotrexate (53%). The 6-year cumulative incidence of graft-failure and second transplant were 25% and 17%, respectively. The 6-year disease-free survival (DFS) and overall survival (OS) were 66.9% and 85.6%, respectively. The 6-year transplant-related mortality (TRM) was 8.0%. In conclusion, most patients with CAMT benefit from allo-HSCT, but with many graft failures.

Identifiants

DOI: 10.1038/s41409-024-02416-x PMID: 39289521

pubmed: 39289521

doi: 10.1038/s41409-024-02416-x

pii: 10.1038/s41409-024-02416-x

doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

Informations de copyright

Références

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