Diego Medina-Valencia1,2, Daniela Cleves3,4, Estefania Beltran4, Natalia Builes5, Alexis A Franco6,7, Andrés Felipe Escobar-González5, Manuela Olaya7,8. 1. Maternal and Child Department, Pediatric Stem Cell Transplant Service, Fundación Valle del Lili, Carrera 98 #18-49, Cali, 760032, Colombia. diego.medina@fvl.org.co. 2. School of Medicine, Universidad Icesi, Cali, Colombia. diego.medina@fvl.org.co. 3. Maternal and Child Department, Fundación Valle del Lili, Cra 98 No. 18 - 49, Cali, 760032, Colombia. 4. Centro de Investigaciones Clínicas, Fundación Valle del Lili, Cra 98 No. 18 - 49, Cali, 760032, Colombia. 5. Pediatric Stem Cell Transplant Unit, Hospital Pablo Tobón Uribe, Cll 78b #69-240, Medellín, 11001, Colombia. 6. Maternal and Child Department, Pediatric Stem Cell Transplant Service, Fundación Valle del Lili, Carrera 98 #18-49, Cali, 760032, Colombia. 7. School of Medicine, Universidad Icesi, Cali, Colombia. 8. Maternal and Child Department, Allergology and Immunology Unit, Fundación Valle del Lili, Cra 98 No. 18 - 49, Cali, 760032, Colombia.
Abstract
PURPOSE: Primary hemophagocytic lymphohistiocytosis is a severe and uncommon disease affecting pediatric patients. Genetic abnormalities have been related to altered apoptosis and exaggerated inflammatory reactions. Chemoimmunotherapy and stem cell transplantation are treatment options, but transplant is the only curative treatment. Here we aim to describe the treatment with hematopoietic stem cell transplantation with a novel strategy and the outcomes. METHODS: An observational, descriptive, case series study was performed in pediatric patients of two high complexity medical centers in Colombia. Data was collected retrospectively between 2015 and 2020. RESULTS: We describe five pediatric cases with a diagnosis of primary hemophagocytic lymphohistiocytosis. All were treated with replete-cell haploidentical hematopoietic stem transplantation, reduced-intensity conditioning, and post-transplant cyclophosphamide, in two high-complexity centers in Colombia. All patients are alive, and one is receiving management for chronic graft-versus-host disease. CONCLUSION: To the best of our knowledge, there are few reports in the literature with this strategy, promising a possible alternative when there are no other donor options.
PURPOSE: Primary hemophagocytic lymphohistiocytosis is a severe and uncommon disease affecting pediatric patients. Genetic abnormalities have been related to altered apoptosis and exaggerated inflammatory reactions. Chemoimmunotherapy and stem cell transplantation are treatment options, but transplant is the only curative treatment. Here we aim to describe the treatment with hematopoietic stem cell transplantation with a novel strategy and the outcomes. METHODS: An observational, descriptive, case series study was performed in pediatric patients of two high complexity medical centers in Colombia. Data was collected retrospectively between 2015 and 2020. RESULTS: We describe five pediatric cases with a diagnosis of primary hemophagocytic lymphohistiocytosis. All were treated with replete-cell haploidentical hematopoietic stem transplantation, reduced-intensity conditioning, and post-transplant cyclophosphamide, in two high-complexity centers in Colombia. All patients are alive, and one is receiving management for chronic graft-versus-host disease. CONCLUSION: To the best of our knowledge, there are few reports in the literature with this strategy, promising a possible alternative when there are no other donor options.