Aldebert, Clemence and Fahd, Mony and Galimard, Jacques-Emmanuel and Ghemlas, Ibrahim A. and Zecca, Marco and Silva, Juliana and Mohseny, Alexander and Kupesiz, Alphan and Hamladji, Rose-Marie and Miranda, Nuno and Gungor, Tayfun and Wynn, Robert F. and Merli, Pietro and Sundin, Mikael and Faraci, Maura and Diaz-de-Heredia, Cristina and Burkhardt, Birgit and Bordon, Victoria and Angoso, Marie and Bader, Peter and Ifversen, Marianne and Herrera Arroyo, Concepcion and Maximova, Natalia and Riesco, Susana and Stein, Jerry and Dalissier, Arnaud and Locatelli, Franco and Kalwak, Krzysztof and Dalle, Jean-Hugues and Corbacioglu, Selim (2024) Outcomes of patients undergoing allogeneic haematopoietic stem cell transplantation for congenital amegakaryocytic thrombocytopenia; a study on behalf of the PDWP of the EBMT. BONE MARROW TRANSPLANTATION, 59 (12). pp. 1717-1725. ISSN 0268-3369, 1476-5365
Full text not available from this repository. (Request a copy)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.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | C-MPL MUTATIONS; DIAGNOSIS; |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation |
| Depositing User: | Dr. Gernot Deinzer |
| Date Deposited: | 06 Aug 2025 06:39 |
| Last Modified: | 06 Aug 2025 06:39 |
| URI: | https://pred.uni-regensburg.de/id/eprint/65678 |
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