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Use of eculizumab in children with allogeneic haematopoietic stem cell transplantation associated thrombotic microangiopathy - a multicentre retrospective PDWP and IEWP EBMT study
P. Svec, R. Elfeky, JE. Galimard, CS. Higham, A. Dalissier, TC. Quigg, D. Bueno Sanchez, S. Han Lum, M. Faraci, T. Cole, H. Pichler, MI. Benítez-Carabante, J. Horakova, M. Gonzalez-Vicent, A. Yanir, F. Fagioli, M. Wölfl, N. von der Weid, R....
Jazyk angličtina Země Anglie, Velká Británie
Typ dokumentu multicentrická studie, časopisecké články
NLK
Free Medical Journals
od 1997 do Před 1 rokem
Freely Accessible Science Journals
od 1997 do Před 1 rokem
ProQuest Central
od 2000-01-01 do Před 1 rokem
Open Access Digital Library
od 1997-01-01
Health & Medicine (ProQuest)
od 2000-01-01 do Před 1 rokem
- MeSH
- dítě MeSH
- humanizované monoklonální protilátky MeSH
- lidé MeSH
- retrospektivní studie MeSH
- transplantace hematopoetických kmenových buněk * škodlivé účinky MeSH
- trombotické mikroangiopatie * farmakoterapie etiologie diagnóza MeSH
- Check Tag
- dítě MeSH
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
- multicentrická studie MeSH
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.
Bristol Royal Hospital for Children Bristol United Kingdom
Central Hospital of Southern Pest Budapest Hungary
EBMT Paris study office Paris France
Fondazione MBBM Milano Bicocca University Monza Italy
Great North Children's Hospital Newcastle Upon Tyne United Kingdom
Hospital Infantil La Paz Madrid Spain
Hospital Infantil Universitario 'Niño Jesus' Madrid Spain
Hospital Universitari Vall d'Hebron Barcelona Spain
Institut d'Hematologie et d'Oncologie Pediatrique Lyon France
IRCSS Institute G Gaslini Genova Italy
Leeds Children's Hospital Leeds United Kingdom
National Institute of Children's Diseases and Comenius Univeristy Bratislava Slovakia
Oslo University Hospital Oslo Norway
Paediatric Onco Haematology Regina Margherita Children's Hospital University of Torino Turin Italy
Pediatric Hematology Oncology Azienda Ospedaliera Universitaria Integrata Verona Verona Italy
Sahlgrenska Univ Hospital Goeteborg Sweden
Saint Antoine Hospital Sorbonne University Paris France
Semmelweis University Budapest Hungary
Sheffield Children's Hospital NHS Foundation Trust Sheffield United Kingdom
Texas Transplant Institute Methodist Children's Hospital San Antonio TX USA
The Royal Children's Hospital Melbourne Melbourne VIC Australia
UCSF Benioff Children's Hospital San Francisco CA USA
University Children's Hospital Basel Basel Switzerland
University children's hospital Ljubljana Ljubljana Slovenia
University Children's Hospital Würzburg Germany
University Hospital Motol Charles University Prague Czech Republic
Citace poskytuje Crossref.org
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