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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
- 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