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Outcomes of allogeneic haematopoietic cell transplantation for myelofibrosis in children and adolescents: the retrospective study of the EBMT Paediatric Diseases WP
J. Wachowiak, JE. Galimard, A. Dalissier, R. Rihani, H. AlSaedi, RF. Wynn, JH. Dalle, R. Peffault de Latour, P. Sedlacek, A. Balduzzi, T. Schroeder, I. Bodova, M. Gonzalez Vicent, B. Gruhn, RM. Hamladji, G. Krivan, K. Patrick, A....
Language English Country England, Great Britain
Document type Journal Article, Multicenter Study
NLK
Free Medical Journals
from 1997 to 1 year ago
Freely Accessible Science Journals
from 1997 to 1 year ago
ProQuest Central
from 2000-01-01 to 1 year ago
Open Access Digital Library
from 1997-01-01
Health & Medicine (ProQuest)
from 2000-01-01 to 1 year ago
- MeSH
- Allografts MeSH
- Child MeSH
- Transplantation, Homologous methods MeSH
- Infant MeSH
- Humans MeSH
- Survival Rate MeSH
- Adolescent MeSH
- Child, Preschool MeSH
- Disease-Free Survival MeSH
- Primary Myelofibrosis * therapy mortality MeSH
- Transplantation Conditioning methods MeSH
- Retrospective Studies MeSH
- Hematopoietic Stem Cell Transplantation * methods MeSH
- Treatment Outcome MeSH
- Check Tag
- Child MeSH
- Infant MeSH
- Humans MeSH
- Adolescent MeSH
- Male MeSH
- Child, Preschool MeSH
- Female MeSH
- Publication type
- Journal Article MeSH
- Multicenter Study MeSH
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.
BMT and Cancer Immunotherapy Department Hadassah Hebrew University Medical Centre Jerusalem Israel
Centre Pierre et Marie Curie Service Hématologie Greffe de Moëlle Alger Algeria
Department of Blood and Marrow Transplant Royal Manchester Children's Hospital Manchester UK
Department of Bone Marrow Transplantation University of Essen Essen Germany
Department of Haematology Saint Antoine Hospital INSERM UMR 938 Sorbonne University Paris France
Department of Medicine and Surgery Milano Bicocca University Milano Italy
Department of Pediatric Hematology and Oncology University Hospital Motol Prague Czechia
Department of Pediatrics Jena University Hospital Jena Germany
EBMT Paris Study Office Paris France
Fondazione IME Policlinico Tor Vergata Rome Rome Italy
Great Ormond Street Hospital for Children NHS Foundation Trust London UK
Hematopoietic Stem Cell Transplant Unit Fondazione IRCCS San Gerardo dei Tintori Monza Italy
Hotel Dieu CHU Nantes Dept D'Hematologie Nantes France
King Faisal Specialist Hospital and Research Centre Riyadh Saudi Arabia
Robert Debré Hospital and Université de Paris Paris France
Sheffield Children's Hospital Western Bank Sheffield United Kingdom
Unidad de Trasplante Hematopoyético Hospital Niño Jesús Madrid Spain
References provided by Crossref.org
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- $a 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.
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