Growing up with MCADD

  • Research type

    Research Study

  • Full title

    Understanding the experience and challenges associated with growing up with Medium chain acyl-CoA dehdrogenase deficiency (MCADD)

  • IRAS ID

    251612

  • Contact name

    Hilary Piercy

  • Contact email

    h.piercy@shu.ac.uk

  • Sponsor organisation

    Sheffield Hallam University

  • Duration of Study in the UK

    0 years, 9 months, days

  • Research summary

    MCADD (Medium chain acyl-CoA dehydrogenase deficiency) is a rare genetic condition where a person has problems breaking down fat to use an energy source. It is a lifelong condition that's present from birth and has been included in the newborn blood spot screening programme for several years. This has made it possible to detect and manage the condition from the first few days of life.
    When someone with MCADD is well, managing the condition involves eating regularly and avoiding prolonged periods of fasting. When unwell, their body's energy demands increase leading to the build-up of harmful substances in the body. Without rapid treatment to prevent the build-up of these harmful substances, liver damage, brain damage, and death can occur.
    We know from previous research about the anxieties and the challenges that parents of a child with MCADD face. We need to understand what it means to grow up with MCADD from the perspective of the young person so that care can be tailored to their needs to help them stay well.
    The aim of this project is to develop a detailed insight into young people's experience of having MCADD and how they manage their condition.

    We will recruit participants from a metabolic unit which cares for families affected by MCADD from birth to 16 years of age. A purposive sampling approach will be used to achieve a rich and comprehensive dataset. We aim to recruit 16 participants aged 10-16 years of age.

    Data will be collected through individual semi-structured interviews using a topic guide to ensure coverage of all areas of enquiry. Interviews will be digitally recorded and fully transcribed.

    We will secure written informed consent from a parent/guardian and informed written assent from the young person prior to data collection.

    Data will be analysed used a thematic analysis approach.

  • REC name

    London - Chelsea Research Ethics Committee

  • REC reference

    19/LO/0552

  • Date of REC Opinion

    20 Mar 2019

  • REC opinion

    Favourable Opinion