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Use of eculizumab to treat thrombotic microangiopathy associated with haematopoietic stem cell transplantation - a retrospective study on behalf of the Paediatric Diseases and Inborn Errors Working Parties of the European Society of Blood and Marrow Transplantation

Paediatric Diseases Working Party (PDWP)
Study number:
Type of transplant:
Short title:
Study status:
Statistical analysis/ Writing process
Deadline for data collection:
Study design:
Primary objective:
Primary objective is to determine the effectivity of eculizumab treatment in HSCT-TMA in children.The goal is to collect the unpublished real-life experience of eculizumab use that may help the current practice and shape the future research.
Key inclusion criteria:
- Age 0-18yrs
- Allogeneic haematopoietic stem cell transplantation recipient
- HSCT-TMA diagnosed according to any of the 6 criteria listed (defined in the questionnaire, attached))
- Treatment with eculizumab
- Informed consent given for EBMT registry data collection
Principal investigator:
Peter Svec
Principal investigator email:
EBMT Study coordinator:
Arnaud Dalissier