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oai:recercat.cat:11351/111102025-10-24T10:37:19Zcom_2072_378070com_2072_378040col_2072_378092

Hematopoietic stem cell transplantation in children with chronic granulomatous disease: the Spanish experience Alonso Garcia, Laura BUENO, DAVID Fernández Navarro, Jose Maria Regueiro, Alexandra Blanquer Blanquer, Miguel Benitez Carabante, Maria Isabel Uria Oficialdegui, Maria Luz Diaz de Heredia, Cristina Institut Català de la Salut [Alonso García L, Benitez Carabante MI, Uria Oficialdegui ML, Diaz de Heredia Rubio C] Servei d’Hematologia i Oncologia Pediàtriques, Vall d’Hebron Hospital Universitari, Barcelona, Spain. [Bueno Sánchez D] Servicio de Hemato-Oncología Pediátrica, Hospital Universitario La Paz, Madrid, Spain. [Fernández Navarro JM] Sección de Oncología y Hematología Infantil Hospital Universitari i Politècnic La Fe, Valencia, Spain. [Regueiro Garcia A] Departamento de Hematología y Oncología Pediátricas Hospital Clínico Universitario de Santiago, Santiago de Compostela, Spain. [Blanquer Blanquer M] Unidad de Trasplante Hematopoyético y Terapia Celular, Hospital Clínico Universitario Virgen de la Arrixaca, Instituto Murciano de Investigación Biosanitaria (IMIB), Murcia, Spain Vall d'Hebron Barcelona Hospital Campus Empelt contra l'hoste, Malaltia de l' Malalties congènites en els infants Cèl·lules mare hematopoètiques - Trasplantació ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Therapeutics::Biological Therapy::Cell- and Tissue-Based Therapy::Cell Transplantation::Stem Cell Transplantation::Hematopoietic Stem Cell Transplantation DISEASES::Hemic and Lymphatic Diseases::Hematologic Diseases::Leukocyte Disorders::Phagocyte Bactericidal Dysfunction::Granulomatous Disease, Chronic DISEASES::Immune System Diseases::Graft vs Host Disease TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::terapéutica::terapia biológica::tratamientos basados en células y tejidos::trasplante de células::trasplante de células madre::trasplante de células madre hematopoyéticas ENFERMEDADES::enfermedades hematológicas y linfáticas::enfermedades hematológicas::trastornos leucocitarios::disfunción bactericida del fagocito::enfermedad granulomatosa crónica ENFERMEDADES::enfermedades del sistema inmune::enfermedad injerto contra huésped Children; Chronic granulomatous disease; Graft failure Nens; Malaltia granulomatosa crònica; Fracàs de l'empelt Niños; Enfermedad granulomatosa crónica; Fracaso del injerto Introduction: Hematopoietic stem cell transplantation (HCT) can cure chronic granulomatous disease (CGD). However, transplant-associated morbidity or mortality may occur, and it is still controversial which patients benefit from this procedure. The aim of this retrospective study was to evaluate the outcome of pediatric patients who received HCT in one of the Spanish pediatric transplant units. Results: Thirty children with a median age of 6.9 years (range 0.6–12.7) were evaluated: 8 patients received a transplant from a sibling donor (MSD), 21 received a transplant from an unrelated donor (UD), and 1 received a haploidentical transplant. The majority of the patients received reduced-intensity conditioning regimens based on either busulfan plus fludarabine or treosulfan. Relevant post-HCT complications were as follows: i) graft failure (GF), with a global incidence of 28.26% (CI: 15.15–48.88), 11.1% in patients with MSD (1.64–56.70) and 37.08% in unrelated donors (19.33–63.17); and ii) chronic graft-versus-host disease (GVHD), with an incidence of 20.5% (8.9–43.2), 11.1% in patients with MSD (1.64–56.70) and 26.7% in unrelated donors (10.42–58.44). Post-HCT infections were usually manageable, but two episodes of pulmonary aspergillosis were diagnosed in the context of graft rejection. The 2-year OS was 77.3% (55.92–89.23). There were no statistically significant differences among donor types. Discussion: HCT in patients with CGD is a complex procedure with significant morbidity and mortality, especially in patients who receive grafts from unrelated donors. These factors need to be considered in the decision-making process and when discussing conditioning and GVHD prophylaxis. 2024-02-23T13:44:57Z 2024-02-23T13:44:57Z 2024-02-01 info:eu-repo/semantics/article info:eu-repo/semantics/publishedVersion Alonso García L, Bueno Sánchez D, Fernández Navarro JM, Regueiro Garcia A, Blanquer Blanquer M, Benitez Carabante MI, et al. Hematopoietic stem cell transplantation in children with chronic granulomatous disease: the Spanish experience. Front Immunol. 2024 Feb 1;15:1307932. 1664-3224 https://hdl.handle.net/11351/11110 10.3389/fimmu.2024.1307932 38370416 http://hdl.handle.net/11351/11110 eng Frontiers in Immunology;15 https://doi.org/10.3389/fimmu.2024.1307932 Attribution 4.0 International http://creativecommons.org/licenses/by/4.0/ info:eu-repo/semantics/openAccess application/pdf Frontiers Media Scientia