Long term outcome among SCID patients post HSCT

• Research type

  Research Study

• Full title

  Long term outcome among severe combined immunodeficiency (SCID) patients post haematopoietic stem cell transplantation (HSCT)

• IRAS ID

  319990

• Contact name

  Maaike Kusters

• Contact email

  maaike.kusters@gosh.nhs.uk

• Sponsor organisation

  Leiden University Medical Center

• Duration of Study in the UK

  1 years, 2 months, 1 days

• Research summary

  Severe combined immunodeficiency (SCID) comprises a group of rare diseases defined by severe T lymphocyte deficiency caused by genetic defect. Without curative treatment with urgent haematopoietic stem cell transplantation (HSCT), infants with SCID face a fatal outcome within the first year of life.
  
  Previous studies have focused on long-term clinical outcomes (mortality and morbidity) of SCID patients after HSCT, but large-scale data on patient reported quality of life (QoL) are lacking. There is an unmet need for a study that systematically looks at quality of life for SCID patients post-HSCT.

  This research project will be the first to systematically look at long-term outcomes and quality of life for SCID patient post-HSCT in a multicentre collaboration across Europe.
  Ultimately, this long-term quality of life data next to long-term clinical outcomes (morbidity/mortality) will allow a change in practice to further improve the long term outcome.

• REC name

  London - Westminster Research Ethics Committee

• REC reference

  24/PR/0063

• Date of REC Opinion

  14 Feb 2024

• REC opinion

  Favourable Opinion