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oai:recercat.cat:11351/75372025-10-24T10:25:38Zcom_2072_378070com_2072_378040col_2072_378092

Use of Eculizumab in Pediatric Patients With Transplant Associated Thrombotic Microangiopathy Gomez Ganda, Laura Fernandez Polo, Aurora Muñoz López, Marina Renedo Miro, Berta Díaz de Heredia Rubio, Maria Cristina Benitez Carabante, Maria Isabel Ariceta Iraola, Gema Institut Català de la Salut [Gomez-Ganda L, Fernandez-Polo A, Renedo-Miro B] Servei de Farmàcia, Vall d'Hebron Hospital Universitari, Barcelona, Spain. [Benitez-Carabante MI, Diaz De Heredia C] Servei d’Oncologia i Hematologia Pediàtriques, Unitat de Trasplantament de Progenitors Hematopoètics (TPH), Vall d'Hebron Hospital Universitari, Barcelona, Spain. [Muñoz-Lopez M, Ariceta G] Servei de Nefrologia Pediàtrica, Vall d'Hebron Hospital Universitari, Barcelona, Spain Vall d'Hebron Barcelona Hospital Campus Pediatria Cèl·lules mare hematopoètiques - Trasplantació - Complicacions Trombocitopènia - Tractament DISEASES::Hemic and Lymphatic Diseases::Hematologic Diseases::Blood Platelet Disorders::Thrombocytopenia::Thrombotic Microangiopathies ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Therapeutics::Biological Therapy::Cell- and Tissue-Based Therapy::Cell Transplantation::Stem Cell Transplantation::Hematopoietic Stem Cell Transplantation Other subheadings::Other subheadings::Other subheadings::/adverse effects DISCIPLINES AND OCCUPATIONS::Health Occupations::Medicine::Pediatrics ENFERMEDADES::enfermedades hematológicas y linfáticas::enfermedades hematológicas::trastornos de las plaquetas sanguíneas::trombocitopenia::microangiopatías trombóticas 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 Otros calificadores::Otros calificadores::Otros calificadores::/efectos adversos DISCIPLINAS Y OCUPACIONES::profesiones sanitarias::medicina::pediatría Complement inhibitor; Eculizumab; Hematopoietic stem cell transplant (HSCT) Inhibidor del complemento; Eculizumab; Trasplante de células madre hematopoyéticas (TCMH) Inhibidor del complement; Eculizumab; Trasplantament de cèl·lules mare hematopoètiques (HSCT) Background: Transplant-associated thrombotic microangiopathy (TA-TMA) is a serious complication of hematopoietic stem cell transplantation (HSCT) associated with high morbidity and mortality. High-risk TA-TMA (hrTA-TMA) is characterized by multifactorial endothelial damage caused by environmental stressors, dysregulation of the complement system, and genetic predisposition. Complement inhibitors have significantly decreased mortality and are the current treatment of choice. In this article, we describe our experience with the use of eculizumab in pediatric patients diagnosed with hrT-TMA after HSCT. Method: Retrospective study of pediatric patients with hrTA-TMA treated with eculizumab between January 2016 and December 2020. Results: Four pediatric patients aged 1, 12, 14, and 17 years at the time of HSCT were diagnosed with hrTA-TMA and treated with eculizumab during the study. At diagnosis, they all had renal impairment with proteinuria, and hypertension under treatment with at least two antihypertensive drugs. The patient who presented multisystemic involvement died instead of treatment. The three patients with exclusive renal involvement achieved TA-TMA resolution after treatment with eculizumab for 65, 52, and 40.6 weeks and were able to stop treatment. The two patients with follow-up data one year after eculizumab withdrawal sustained a favorable response. Eculizumab was well tolerated, and with adequate vaccination and antibiotic prophylaxis, did not increase the risk of infection. Conclusions: Eculizumab appears to be both safe and effective for the treatment of hrTA-TMA in patients with renal impairment. Early diagnosis and initiation of treatment may improve response. Eculizumab withdrawal can be contemplated in patients who achieve laboratory and clinical resolution of TA-TMA. 2022-05-16T13:05:19Z 2022-05-16T13:05:19Z 2021-11 info:eu-repo/semantics/article info:eu-repo/semantics/publishedVersion Gomez-Ganda L, Benitez-Carabante MI, Fernandez-Polo A, Muñoz-Lopez M, Renedo-Miro B, Ariceta G, et al. Use of Eculizumab in Pediatric Patients With Transplant Associated Thrombotic Microangiopathy. Front Pediatr. 2021 Nov;9:761726. 2296-2360 https://hdl.handle.net/11351/7537 10.3389/fped.2021.761726 34858907 000733890100001 http://hdl.handle.net/11351/7537 eng Frontiers in Pediatrics;16 https://doi.org/10.3389/fped.2021.761726 Attribution 4.0 International http://creativecommons.org/licenses/by/4.0/ info:eu-repo/semantics/openAccess application/pdf Frontiers Media Scientia