Hematopoietic cell transplantation in chronic granulomatous disease: a study of 712 children and adults

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art_CHIESA_Hematopoietic_cell_transplantation_in_chronic_granulomatous_disease_a_2020.PDF (829.98 KB)
Citações na Scopus
82
Tipo de produção
article
Data de publicação
2020
DOI
Scopus
Web of Science
PubMed
Título da Revista
ISSN da Revista
Título do Volume
Editora
AMER SOC HEMATOLOGY
Autores
CHIESA, Robert
WANG, Junfeng
BLOK, Henric-Jan
HAZELAAR, Sheree
NEVEN, Benedicte
MOSHOUS, Despina
SCHULZ, Ansgar
HOENIG, Manfred
HAUCK, Fabian
SERAIHY, Amal Al
Citação
BLOOD, v.136, n.10, p.1201-1211, 2020
Projetos de Pesquisa
Unidades Organizacionais
Fascículo
Resumo
Chronic granulomatous disease (CGD) is a primary immunodeficiency resulting in life-threatening infections and inflammatory complications. Allogeneic hematopoietic cell transplantation (allo-HCT) can cure the disease, but the indication to transplant remains controversial. We performed a retrospective multicenter study of 712 patients with CGD who underwent allo-HCT transplantation from March 1993 through December 2018. We studied 635 children (aged <18 years) and 77 adults. Median follow-up was 45 months. Median age at transplantation was 7 years (range, 0.1-48.6). Kaplan-Meier estimates of overall survival (OS) and event-free survival (EFS) at 3 years were 85.7% and 75.8%, respectively. In multivariate analysis, older age was associated with reduced survival and increased chronic graft-versus-host disease. Nevertheless, OS and EFS at 3 years for patients >= 18 years were 76% and 69%, respectively. Use of 1-antigen-mismatched donors was associated with reduced OS and EFS . No significant difference was found in OS, but a significantly reduced EFS was noted in the small group of patients who received a transplant from a donor with a >1 antigen mismatch. Choice of conditioning regimen did not influence OS or EFS. In summary, we report an excellent outcome after allo-HCT in CGD, with low incidence of graft failure and mortality in all ages. Older patients and recipients of 1-antigen-mismatched grafts had a less favorable outcome. Transplantation should be strongly considered at a younger age and particularly in the presence of a well-matched donor.
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URI
https://observatorio.fm.usp.br/handle/OPI/38014
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Coleções
Artigos e Materiais de Revistas Científicas - HC/ICr
Artigos e Materiais de Revistas Científicas - LIM/36