Wachowiak, Jacek and Galimard, Jacques-Emmanuel and Dalissier, Arnaud and Rihani, Rawad and Alsaedi, Hawazen and Wynn, Robert F. and Dalle, Jean-Hugues and Peffault de Latour, Regis and Sedlacek, Petr and Balduzzi, Adriana and Schroeder, Thomas and Bodova, Ivana and Gonzalez Vicent, Marta and Gruhn, Bernd and Hamladji, Rose-Marie and Krivan, Gergely and Patrick, Katharine and Sobkowiak-Sobierajska, Agnieszka and Stepensky, Polina and Unal, Ali and Amrolia, Persis and Perez Martinez, Antonio and Rialland, Fanny and Aljurf, Mahmoud and Isgro, Antonella and Toren, Amos and Bierings, Marc and Corbacioglu, Selim and Kalwak, Krzysztof (2024) Outcomes of allogeneic haematopoietic cell transplantation for myelofibrosis in children and adolescents: the retrospective study of the EBMT Paediatric Diseases WP. BONE MARROW TRANSPLANTATION, 59 (8). pp. 1057-1069. ISSN 0268-3369, 1476-5365
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This retrospective study evaluated 35 children (median age 5.2 years; range 0.4-18) with myelofibrosis (MF), including 33 with primary myelofibrosis and 2 with secondary myelofibrosis transplanted from matched sibling donor (MSD) (n = 17) or non-MSD (n = 18) between 2000 and 2022. Conditioning was usually chemotherapy-based (n = 33) and myeloablative (n = 32). Fifteen patients received bone marrow (BM), 14 haematopoietic cells (HC) from peripheral blood (PB), and 6 from cord blood (CB). Day +100 acute GvHD II-IV incidence was significantly lower after MSD-haematopoietic cell transplantation (MSD-HCT) than after non-MSD-HCT [18.8% (4.3-41.1) vs 58.8% (31-78.6); p = 0.01]. Six-year non-relapse mortality (NRM) was 18% (7.1-32.8), relapse incidence was 15.9% (5.6-30.9), progression-free survival (PFS) was 66.1% (47-79.7), GvHD-free relapse-free survival was 50% (30.6-66.7), and overall survival (OS) was 71.1% (51.4-84). Six-year PFS and OS were significantly higher after BM transplantation compared to HCT from other sources [85.1% (52.3-96.1) vs 50.8% (26.3-71), p = 0.03, and 90.9% (50.8-98.7) vs 54% (28.1-74.2), p = 0.01, respectively], whereas NRM was significantly lower [0% vs 32% (12.3-53.9); p = 0.02]. This first multicentre study on outcomes of allogeneic HCT in children with myelofibrosis proves feasibility and curative effect of transplantation in these children, suggests that bone marrow transplantation is associated with better outcomes, and indicates the need for further studies.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | ESSENTIAL THROMBOCYTHEMIA; POLYCYTHEMIA-VERA; SPLENECTOMY; AGE; |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation |
| Depositing User: | Dr. Gernot Deinzer |
| Date Deposited: | 07 Aug 2025 04:35 |
| Last Modified: | 07 Aug 2025 04:35 |
| URI: | https://pred.uni-regensburg.de/id/eprint/65686 |
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