Manuela Olaya1,2, Alexis Franco2,3, Mauricio Chaparro4, Marcela Estupiñan4, David Aristizabal4, Natalia Builes-Restrepo5, José L Franco6, Andrés F Zea-Vera7, Mayra Estacio8, Eliana Manzi2,8, Estefania Beltran8, Paola Perez2,9, Jaime Patiño2,9, Harry Pachajoa2,10, Diego Medina-Valencia11,12. 1. Fundación Valle del Lili, Departamento Materno-infantil, Unidad de alergología e Inmunología, Cra 98 No. 18-49, Cali, 760032, Colombia. 2. Facultad de Ciencias de la Salud, Universidad Icesi, Cali, Colombia. 3. Fundación Valle del Lili, Departamento Materno-infantil, Unidad de trasplante de médula ósea, Cra 98 No. 18-49, Cali, 760032, Colombia. 4. Fundación HOMI-Hospital de la Misericordia, Unidad de Trasplante, Av Caracas #1-65, Bogotá, 111071, Colombia. 5. Hospital Pablo Tobón Uribe, Unidad de Trasplante de médula ósea, Cll 78b #69-240, Medellín, 11001, Colombia. 6. Grupo de Inmunodeficiencias Primarias, Universidad de Antioquia, Medellín, 50010, Colombia. 7. Departamento de Microbiología, Facultad de salud, Universidad del Valle, Calle 4B No. 36-00, Cali, 760036, Colombia. 8. Fundación Valle del Lili, Centro de Investigaciones Clínicas (CIC), Cra 98 No. 18-49, Cali, 760032, Colombia. 9. Fundación Valle del Lili, Departamento Materno-infantil, Unidad de Infectología pediátrica, Cra 98 No. 18-49, Cali, 760032, Colombia. 10. Fundación Valle del Lili, Departamento Materno-infantil, Servicio de Genética Clínica, Cra 98 No. 18-49, Cali, 760032, Colombia. 11. Facultad de Ciencias de la Salud, Universidad Icesi, Cali, Colombia. diego.medina@fvl.org.co. 12. Fundación Valle del Lili, Departamento Materno-infantil, Unidad de trasplante de médula ósea, Cra 98 No. 18-49, Cali, 760032, Colombia. diego.medina@fvl.org.co.
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-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%. 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.
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-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%. 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.
Authors: Teresa Del Rosal; Cristian Quintana-Ortega; Angela Deyá-Martinez; Pere Soler-Palacín; Walter Alfredo Goycochea-Valdivia; Nerea Salmón; Antonio Pérez-Martínez; Laia Alsina; Andrea Martín-Nalda; Laura Alonso; Olaf Neth; Luz Yadira Bravo-Gallego; Luis Ignacio Gonzalez-Granado; Ana Mendez-Echevarria Journal: Eur J Pediatr Date: 2022-09-14 Impact factor: 3.860