Hematopoietic Stem Cell Transplantation in Children with Inborn Errors of Immunity: a Multi-center Experience in Colombi
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ORIGINAL ARTICLE
Hematopoietic Stem Cell Transplantation in Children with Inborn Errors of Immunity: a Multi-center Experience in Colombia Manuela Olaya 1,2 & Alexis Franco 2,3 & Mauricio Chaparro 4 & Marcela Estupiñan 4 & David Aristizabal 4 & Natalia BuilesRestrepo 5 & José L Franco 6 & Andrés F Zea-Vera 7 & Mayra Estacio 8 & Eliana Manzi 2,8 & Estefania Beltran 8 & Paola Perez 2,9 & Jaime Patiño 2,9 & Harry Pachajoa 2,10 & Diego Medina-Valencia 2,3 Received: 2 July 2020 / Accepted: 25 August 2020 # Springer Science+Business Media, LLC, part of Springer Nature 2020
Abstract Purpose 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? Methods We conducted an observational, retrospective cohort study in children with a diagnosis of IEI who underwent HSCT between 2007 and 2018. Results 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-versushost disease prophylaxis (63.8%). Acute graft-versus-host disease developed in 35% of the total patients. The most frequent posttransplant 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%. Conclusions 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. Keywords Hematopoietic stem cell transplantation . transplant recipients . primary immunodeficiency diseases . pediatrics . haploidentical transplantations . child * Diego Medina-Valencia [email protected]
Andrés F Zea-Vera [email protected]
Manuela Olaya [email protected]
Mayra Estacio [email protected]
Alexis Franco [email protected]
Eliana Manzi [email protected]
Mauricio Chaparro [email protected]
Estefania Beltran [email protected]
Marcela Estupiñan [email protected] David Aristizabal [email protected] Natalia Builes-Restrepo [email protected] José L Franco [email protected]
Paola Perez [email protected] Jaime Patiño jaim
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