Hematopoietic Stem Cell Transplantation in Children with Inborn Errors of Immunity: a Multi-center Experience in Colombia.
Child, Preschool
Colombia
Combined Modality Therapy
Diagnosis, Differential
Female
Genetic Association Studies
Genetic Diseases, Inborn
/ diagnosis
Genetic Predisposition to Disease
Hematopoietic Stem Cell Transplantation
/ adverse effects
Humans
Infant
Lymphocyte Depletion
Male
Phenotype
Primary Immunodeficiency Diseases
/ diagnosis
Tissue Donors
Treatment Outcome
Hematopoietic stem cell transplantation
child
haploidentical transplantations
pediatrics
primary immunodeficiency diseases
transplant recipients
Journal
Journal of clinical immunology
ISSN: 1573-2592
Titre abrégé: J Clin Immunol
Pays: Netherlands
ID NLM: 8102137
Informations de publication
Date de publication:
11 2020
11 2020
Historique:
received:
02
07
2020
accepted:
25
08
2020
pubmed:
4
9
2020
medline:
13
10
2021
entrez:
4
9
2020
Statut:
ppublish
Résumé
To characterize the pediatric population with inborn errors of immunity (IEI) that was treated with hematopoietic stem cell transplantation (HSCT) in three reference centers in Colombia. What have been the characteristics and outcomes of hematopoietic stem cell transplantation in pediatric patients with inborn errors of immunity in three reference care centers in Colombia between 2007 and 2018? We conducted an observational, retrospective cohort study in children with a diagnosis of IEI who underwent HSCT between 2007 and 2018. Forty-seven patients were identified, and 5 were re-transplanted. Sixty-eight percent were male. The median age at diagnosis was 0.6 years, and for HSCT was 1.4 years. The most common diseases were chronic granulomatous disease (38%) followed by severe combined immune deficiencies (19%) and hemophagocytic lymphohistiocytosis (15%). Cord blood donors were the most used source of HSCT (44%). T cell-replete grafts from haploidentical donors using post-transplantation cyclophosphamide represent 37% of the cohort. All patients received conditioning, 62% with a non-myeloablative regimen. Calcineurin inhibitors were the main graft-versus-host disease prophylaxis (63.8%). Acute graft-versus-host disease developed in 35% of the total patients. The most frequent post-transplant infections were viral and fungal infections. The 1-year overall survival rates for the patients who received HSCT from identical, haploidentical, and cord sources were 80%, 72%, and 63%, respectively. The 5-year overall survival was 63%. HSCT is a curative treatment option for some IEI and can be performed with any donor type. Early and timely treatment in referral centers can improve survival.
Identifiants
pubmed: 32880086
doi: 10.1007/s10875-020-00856-w
pii: 10.1007/s10875-020-00856-w
doi:
Types de publication
Journal Article
Multicenter Study
Langues
eng
Sous-ensembles de citation
IM
Pagination
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