Use of eculizumab in children with allogeneic haematopoietic stem cell transplantation associated thrombotic microangiopathy - a multicentre retrospective PDWP and IEWP EBMT study

Svec, Peter ; Elfeky, Reem ; Galimard, Jacques-Emmanuel ; Higham, Christine S. ; Dalissier, Arnaud ; Quigg, Troy C. ; Bueno Sanchez, David ; Han Lum, Su ; Faraci, Maura ; Cole, Theresa ; Pichler, Herbert ; Benítez-Carabante, Maria Isabel ; Horakova, Julia ; Gonzalez -Vicent, Marta ; Yanir, Asaf ; Fagioli, Franca ; Wölfl, Matthias ; von der Weid, Nicolas ; Protheroe, Rachel ; Krivan, Gergely ; Speckmann, Carsten ; James, Beki ; Avcin, Simona Lucija ; Bertrand, Yves ; Verna, Marta ; Riha, Petr ; Patrick, Katharine ; Cesaro, Simone ; Kalwak, Krzysztof ; Bierings, Marc ; Büchner, Jochen ; Mellgren, Karin ; Prohászka, Zoltán ; Neven, Bénédicte ; Lankester, Arjan ; Corbacioglu, Selim

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Details

Item type:	Article
Journal or Publication Title:	Bone Marrow Transplantation
Publisher:	SPRINGERNATURE
Place of Publication:	LONDON
Volume:	58
Number of Issue or Book Chapter:	2
Page Range:	pp. 129-141
Date:	2023
Institutions:	Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation
Identification Number:	Value Type 10.1038/s41409-022-01852-x DOI
Keywords:	RISK-FACTORS; COMPLEMENT; THERAPY; PATHOPHYSIOLOGY; CRITERIA; SUSCEPTIBILITY; INHIBITOR; DIAGNOSIS; ADULTS
Dewey Decimal Classification:	600 Technology > 610 Medical sciences Medicine
Status:	Published
Refereed:	Yes, this version has been refereed
Created at the University of Regensburg:	Yes
Item ID:	75390

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Abstract

Terminal complement blockade by humanised monoclonal antibody eculizumab has been used to treat transplantation-associated thrombotic microangiopathy (TA-TMA) in recent years. This retrospective international study conducted by the Paediatric Diseases (PDWP) and Inborn Error Working Party (IEWP) of the European Society for Blood and Marrow Transplantation (EBMT) describes outcome and response of ...

Abstract

Terminal complement blockade by humanised monoclonal antibody eculizumab has been used to treat transplantation-associated thrombotic microangiopathy (TA-TMA) in recent years. This retrospective international study conducted by the Paediatric Diseases (PDWP) and Inborn Error Working Party (IEWP) of the European Society for Blood and Marrow Transplantation (EBMT) describes outcome and response of 82 paediatric patients from 29 centres who developed TA-TMA and were treated with eculizumab between January 2014 and May 2019. The median time from hematopoietic stem cell transplantation (HSCT) to TA-TMA manifestation was 92 days (range: 7-606) and from TA-TMA diagnosis to the start of eculizumab treatment 6 days (range: 0-135). Most patients received eculizumab weekly (72%, n = 55) with a standard weight (kg)-based dose (78%, n = 64). Six months from beginning of eculizumab therapy, the cumulative incidence of TA-TMA resolution was 36.6% (95% CI: 26.2-47) and the overall survival (OS) was 47.1% (95% CI: 35.9-57.5). All 43 patients with unresolved TA-TMA died. The cause of death was HSCT-related in 41 patients. This study also documents poor outcome of patients without aGvHD and their frequent concomitant viral infections. Considering recent publications, intensified eculizumab dosing and complement monitoring could potentially improve upon outcomes observed in this study.

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