Svec, Peter and Elfeky, Reem and Galimard, Jacques-Emmanuel and Higham, Christine S. and Dalissier, Arnaud and Quigg, Troy C. and Sanchez, David Bueno and Lum, Su Han and Faraci, Maura and Cole, Theresa and Pichler, Herbert and Benitez-Carabante, Maria Isabel and Horakova, Julia and Vicent, Marta Gonzalez and Yanir, Asaf and Fagioli, Franca and Wölfl, Matthias and von der Weid, Nicolas and Protheroe, Rachel and Krivan, Gergely and Speckmann, Carsten and James, Beki and Avcin, Simona Lucija and Bertrand, Yves and Verna, Marta and Riha, Petr and Patrick, Katharine and Cesaro, Simone and Kalwak, Krzysztof and Bierings, Marc and Buchner, Jochen and Mellgren, Karin and Prohaszka, Zoltan and Neven, Benedicte and Lankester, Arjan and Corbacioglu, Selim (2023) Use of eculizumab in children with allogeneic haematopoietic stem cell transplantation associated thrombotic microangiopathy-a multicentre retrospective PDWP and IEWP EBMT study. BONE MARROW TRANSPLANTATION, 58. pp. 129-141. ISSN 0268-3369, 1476-5365
Full text not available from this repository. (Request a copy)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.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | RISK-FACTORS; COMPLEMENT; THERAPY; PATHOPHYSIOLOGY; CRITERIA; SUSCEPTIBILITY; INHIBITOR; DIAGNOSIS; ADULTS |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation |
| Depositing User: | Petra Gürster |
| Date Deposited: | 01 Sep 2023 04:37 |
| Last Modified: | 01 Sep 2023 04:37 |
| URI: | https://pred.uni-regensburg.de/id/eprint/58780 |
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