Alexion HSCT-TMA

• Research type

  Research Study

• Full title

  A Secondary Real-world Data Study of Pediatric and Adult Participants with Thrombotic Microangiopathy (TMA) after Hematopoietic Stem Cell Transplant (HSCT)

• IRAS ID

  349890

• Contact name

  Sarah Lawson

• Contact email

  sarah.lawson11@nhs.net

• Sponsor organisation

  Alexion Pharma GmbH

• Duration of Study in the UK

  1 years, 6 months, 0 days

• Research summary

  The goal of this study is to study overall survival results in patients diagnosed
  with hematopoietic stem cell transplant (HSCT) thrombotic microangiopathy
  (TMA) who did not get treatment with drugs called specific complement
  inhibitors.
  This is a global, observational, retrospective real-world data collection study of
  patients diagnosed with TMA within 52 weeks after HSCT. Patients will not get
  any treatment as part of this study.
  Real-world data are those pertaining to routine healthcare received by patients
  without being in any study.
  Data from 227 eligible patients (around 123 adult patients and around 104 child
  patients) will be collected from the medical records of about 50 study centres in
  about 11 countries and/or from the TriNetX data repository. TriNetX data
  repository option will not be applicable for the United Kingdom.
  Patients who were treated with drugs called complement inhibitors, including, but
  not limited to, eculizumab (SOLIRIS®) and ravulizumab (ULTOMIRIS®), will be
  excluded.
  The study will include child and adult patients who were diagnosed with TMA
  within 52 weeks after an HSCT treatment and who did not get complement
  inhibitor drugs

• REC name

  West of Scotland REC 3

• REC reference

  24/WS/0177

• Date of REC Opinion

  4 Mar 2025

• REC opinion

  Further Information Favourable Opinion