Haematopoietic cell transplantation for 106 infants and preschoolers with acquired and inherited bone marrow failures.

Autor: Loth G; Faculdades Pequeno Príncipe, Curitiba, Paraná, Brazil.; Bone Marrow Transplantation Unit, Hospital de Clínicas, Universidade Federal do Paraná, Curitiba, Paraná, Brazil.; Bone Marrow Transplantation Unit, Hospital Pequeno Príncipe, Curitiba, Paraná, Brazil., Dumke CCK; Bone Marrow Transplantation Unit, Hospital Pequeno Príncipe, Curitiba, Paraná, Brazil., Muratori RR; Bone Marrow Transplantation Unit, Hospital Nossa Senhora das Graças, Curitiba, Paraná, Brazil., Pelegrina PD; Faculdades Pequeno Príncipe, Curitiba, Paraná, Brazil.; Bone Marrow Transplantation Unit, Hospital Pequeno Príncipe, Curitiba, Paraná, Brazil., Peixoto CMA; Bone Marrow Transplantation Unit, Hospital Pequeno Príncipe, Curitiba, Paraná, Brazil., Bach JLM; Faculdades Pequeno Príncipe, Curitiba, Paraná, Brazil.; Bone Marrow Transplantation Unit, Hospital Pequeno Príncipe, Curitiba, Paraná, Brazil., Nichele S; Bone Marrow Transplantation Unit, Hospital de Clínicas, Universidade Federal do Paraná, Curitiba, Paraná, Brazil.; Bone Marrow Transplantation Unit, Hospital Nossa Senhora das Graças, Curitiba, Paraná, Brazil., Trennepohl JP; Bone Marrow Transplantation Unit, Hospital de Clínicas, Universidade Federal do Paraná, Curitiba, Paraná, Brazil.; Bone Marrow Transplantation Unit, Hospital Nossa Senhora das Graças, Curitiba, Paraná, Brazil., Koliski A; Bone Marrow Transplantation Unit, Hospital de Clínicas, Universidade Federal do Paraná, Curitiba, Paraná, Brazil., Mousquer RTG; Bone Marrow Transplantation Unit, Hospital de Clínicas, Universidade Federal do Paraná, Curitiba, Paraná, Brazil., Rodrigues AM; Faculdades Pequeno Príncipe, Curitiba, Paraná, Brazil.; Bone Marrow Transplantation Unit, Hospital de Clínicas, Universidade Federal do Paraná, Curitiba, Paraná, Brazil.; Bone Marrow Transplantation Unit, Hospital Pequeno Príncipe, Curitiba, Paraná, Brazil., Marchesini R; Bone Marrow Transplantation Unit, Hospital de Clínicas, Universidade Federal do Paraná, Curitiba, Paraná, Brazil., Feitosa MK; Bone Marrow Transplantation Unit, Hospital Pequeno Príncipe, Curitiba, Paraná, Brazil.; Histocompatibility/Immunogenetics Sector, Hospital de Clínicas, Universidade Federal do Paraná, Curitiba, Paraná, Brazil., Pilonetto DV; Histocompatibility/Immunogenetics Sector, Hospital de Clínicas, Universidade Federal do Paraná, Curitiba, Paraná, Brazil., Gouvea L; Bone Marrow Transplantation Unit, Hospital de Clínicas, Universidade Federal do Paraná, Curitiba, Paraná, Brazil.; Bone Marrow Transplantation Unit, Hospital Nossa Senhora das Graças, Curitiba, Paraná, Brazil., Benini FML; Bone Marrow Transplantation Unit, Hospital Pequeno Príncipe, Curitiba, Paraná, Brazil., Pirolli ES; Bone Marrow Transplantation Unit, Hospital Pequeno Príncipe, Curitiba, Paraná, Brazil., Tutumi RAF; Faculdades Pequeno Príncipe, Curitiba, Paraná, Brazil.; Bone Marrow Transplantation Unit, Hospital Pequeno Príncipe, Curitiba, Paraná, Brazil., Lima ACM; Histocompatibility/Immunogenetics Sector, Hospital de Clínicas, Universidade Federal do Paraná, Curitiba, Paraná, Brazil., Pasquini R; Bone Marrow Transplantation Unit, Hospital de Clínicas, Universidade Federal do Paraná, Curitiba, Paraná, Brazil.; Bone Marrow Transplantation Unit, Hospital Nossa Senhora das Graças, Curitiba, Paraná, Brazil., Bonfim C; Faculdades Pequeno Príncipe, Curitiba, Paraná, Brazil.; Bone Marrow Transplantation Unit, Hospital de Clínicas, Universidade Federal do Paraná, Curitiba, Paraná, Brazil.; Bone Marrow Transplantation Unit, Hospital Pequeno Príncipe, Curitiba, Paraná, Brazil.; Bone Marrow Transplantation Unit, Hospital Nossa Senhora das Graças, Curitiba, Paraná, Brazil.
Jazyk: angličtina
Zdroj: British journal of haematology [Br J Haematol] 2024 Oct 27. Date of Electronic Publication: 2024 Oct 27.
DOI: 10.1111/bjh.19831
Abstrakt: Aplastic anaemia in infants and young children presents unique challenges due to high prevalence of inherited bone marrow failure syndromes (IBMFS) in this age group. The objective of this study is assessing clinical characteristics and outcomes of haematopoietic cell transplantation in children ≤5 years with bone marrow failure syndromes. We analysied 106 patients (66% males), median age 4.6 years, including 40 with Fanconi anaemia (FA), 32 with Acquired Severe Aplastic anaemia (aSAA), 15 with Diamond-Blackfan Anaemia, 11 with Amegakaryocytic Purpura and 8 with other IBMFS. Molecular testing was limited (39%), with 25.4% confirmed genetically. Retrospective longitudinal study across three paediatric transplantation centres (1982-2020). Overall survival (OS) was 76.4% over a median 10-year follow-up. OS rates were similar between aSAA and IBMFS (FA 77.5%, other IBMFS 76.5%). Transplant-related mortality (TRM) was lower in aSAA (9.4%) compared with IBMFS (16.2%). Recent years showed improved outcomes, with TRM declining post-2010. Choice of stem cell source impacted OS, favouring bone marrow over umbilical cord, but showing encouraging results with haploidentical. Late complications were common, including endocrine-metabolic issues and delayed neuropsychomotor development. Diagnosing and managing bone marrow failures in young children pose significant challenges. Despite advancements in transplant practices, ongoing vigilance and comprehensive care are necessary to improve long-term survival rates.
(© 2024 British Society for Haematology and John Wiley & Sons Ltd.)
Databáze: MEDLINE
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