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

Authors :
Aldebert, Clémence
Fahd, Mony
Galimard, Jacques-Emmanuel
Ghemlas, Ibrahim A.
Zecca, Marco
Silva, Juliana
Mohseny, Alexander
Kupesiz, Alphan
Hamladji, Rose-Marie
Miranda, Nuno
Güngör, Tayfun
Wynn, Robert F.
Merli, Pietro
Sundin, Mikael
Faraci, Maura
Diaz-de-Heredia, Cristina
Burkhardt, Birgit
Bordon, Victoria
Angoso, Marie
Bader, Peter
Ifversen, Marianne
Herrera Arroyo, Concepcion
Maximova, Natalia
Riesco, Susana
Stein, Jerry
Dalissier, Arnaud
Locatelli, Franco
Kalwak, Krzysztof
Dalle, Jean-Hugues
Corbacioglu, Selim
Source :
Bone Marrow Transplantation; 20240101, Issue: Preprints p1-9, 9p
Publication Year :
2024

Abstract

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.

Details

Language :
English
ISSN :
02683369 and 14765365
Issue :
Preprints
Database :
Supplemental Index
Journal :
Bone Marrow Transplantation
Publication Type :
Periodical
Accession number :
ejs67425003
Full Text :
https://doi.org/10.1038/s41409-024-02416-x