Use of Eculizumab in Pediatric Patients With Transplant Associated Thrombotic Microangiopathy

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 st...

Descripción completa

Guardado en:

Detalles Bibliográficos
Autores principales:	Laura Gomez-Ganda, Maria Isabel Benitez-Carabante, Aurora Fernandez-Polo, Marina Muñoz-Lopez, Berta Renedo-Miro, Gema Ariceta, Cristina Diaz De Heredia
Formato:	article
Lenguaje:	EN
Publicado:	Frontiers Media S.A. 2021
Materias:	thrombotic microangiopathy (TA-TMA) hrTA-TMA eculizumab complement system hematopoietic stem cell transplant (HSCT) CH50 Pediatrics RJ1-570
Acceso en línea:	https://doaj.org/article/6f0a3f045b8d45d78d16fcb7bd192de4
Etiquetas:	Agregar Etiqueta Sin Etiquetas, Sea el primero en etiquetar este registro!

id	oai:doaj.org-article:6f0a3f045b8d45d78d16fcb7bd192de4
record_format	dspace
spelling	oai:doaj.org-article:6f0a3f045b8d45d78d16fcb7bd192de42021-11-11T09:28:01ZUse of Eculizumab in Pediatric Patients With Transplant Associated Thrombotic Microangiopathy2296-236010.3389/fped.2021.761726https://doaj.org/article/6f0a3f045b8d45d78d16fcb7bd192de42021-11-01T00:00:00Zhttps://www.frontiersin.org/articles/10.3389/fped.2021.761726/fullhttps://doaj.org/toc/2296-2360Background: 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.Laura Gomez-GandaMaria Isabel Benitez-CarabanteAurora Fernandez-PoloMarina Muñoz-LopezBerta Renedo-MiroGema AricetaCristina Diaz De HerediaFrontiers Media S.A.articlethrombotic microangiopathy (TA-TMA)hrTA-TMAeculizumabcomplement systemhematopoietic stem cell transplant (HSCT)CH50PediatricsRJ1-570ENFrontiers in Pediatrics, Vol 9 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	thrombotic microangiopathy (TA-TMA) hrTA-TMA eculizumab complement system hematopoietic stem cell transplant (HSCT) CH50 Pediatrics RJ1-570
spellingShingle	thrombotic microangiopathy (TA-TMA) hrTA-TMA eculizumab complement system hematopoietic stem cell transplant (HSCT) CH50 Pediatrics RJ1-570 Laura Gomez-Ganda Maria Isabel Benitez-Carabante Aurora Fernandez-Polo Marina Muñoz-Lopez Berta Renedo-Miro Gema Ariceta Cristina Diaz De Heredia Use of Eculizumab in Pediatric Patients With Transplant Associated Thrombotic Microangiopathy
description	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.
format	article
author	Laura Gomez-Ganda Maria Isabel Benitez-Carabante Aurora Fernandez-Polo Marina Muñoz-Lopez Berta Renedo-Miro Gema Ariceta Cristina Diaz De Heredia
author_facet	Laura Gomez-Ganda Maria Isabel Benitez-Carabante Aurora Fernandez-Polo Marina Muñoz-Lopez Berta Renedo-Miro Gema Ariceta Cristina Diaz De Heredia
author_sort	Laura Gomez-Ganda
title	Use of Eculizumab in Pediatric Patients With Transplant Associated Thrombotic Microangiopathy
title_short	Use of Eculizumab in Pediatric Patients With Transplant Associated Thrombotic Microangiopathy
title_full	Use of Eculizumab in Pediatric Patients With Transplant Associated Thrombotic Microangiopathy
title_fullStr	Use of Eculizumab in Pediatric Patients With Transplant Associated Thrombotic Microangiopathy
title_full_unstemmed	Use of Eculizumab in Pediatric Patients With Transplant Associated Thrombotic Microangiopathy
title_sort	use of eculizumab in pediatric patients with transplant associated thrombotic microangiopathy
publisher	Frontiers Media S.A.
publishDate	2021
url	https://doaj.org/article/6f0a3f045b8d45d78d16fcb7bd192de4
work_keys_str_mv	AT lauragomezganda useofeculizumabinpediatricpatientswithtransplantassociatedthromboticmicroangiopathy AT mariaisabelbenitezcarabante useofeculizumabinpediatricpatientswithtransplantassociatedthromboticmicroangiopathy AT aurorafernandezpolo useofeculizumabinpediatricpatientswithtransplantassociatedthromboticmicroangiopathy AT marinamunozlopez useofeculizumabinpediatricpatientswithtransplantassociatedthromboticmicroangiopathy AT bertarenedomiro useofeculizumabinpediatricpatientswithtransplantassociatedthromboticmicroangiopathy AT gemaariceta useofeculizumabinpediatricpatientswithtransplantassociatedthromboticmicroangiopathy AT cristinadiazdeheredia useofeculizumabinpediatricpatientswithtransplantassociatedthromboticmicroangiopathy
_version_	1718439225301401600

Use of Eculizumab in Pediatric Patients With Transplant Associated Thrombotic Microangiopathy

Ejemplares similares