Contribution of microbiome to inhibitor development in haemophilia A

  • Research type

    Research Study

  • Full title

    Does the composition of the microbiome correlate with the development of FVIII-neutralising antibodies in haemophila A patients?

  • IRAS ID

    271423

  • Contact name

    Carmen Coxon

  • Contact email

    carmen.coxon@nibsc.org

  • Sponsor organisation

    National Institute Biological Standards and Control

  • Duration of Study in the UK

    2 years, 11 months, 28 days

  • Research summary

    Development of FVIII-neutralising antibodies ('inhibitors') in haemophilia A (HA) patients is a serious complication of treatment. The microbiome influences many aspects of pathophysiology, including the immune system. Recent work (communicated at the ISTH 2019 meeting) revealed that loss of the microbiome from haemophila A mice (a experimental model for the human disease) resulted in an increase in 'inhibitor titre' (strength of the response). We are interested to know if this also affects inhibitor development in the human disease. To investigate this we will collect stool samples from severe HA patients at/before first exposure to FVIII and after 50 doses (by which point inhibitors have usually developed). We will compare the microbiome of patients that develop inhibitors to those that do not to determine if there is a correlation between the composition of the patient's microbiome and inhibitor development.

  • REC name

    London - Central Research Ethics Committee

  • REC reference

    20/LO/0406

  • Date of REC Opinion

    23 Mar 2020

  • REC opinion

    Favourable Opinion