Caregiver Burden in Cystic Fibrosis

  • Research type

    Research Study

  • Full title

    Caregiver Burden Study in Cystic Fibrosis in the United States, Germany, United Kingdom, and Ireland



  • Contact name

    Ellison Suthoff

  • Contact email

  • Sponsor organisation

    Vertex Pharmaceuticals

  • Duration of Study in the UK

    1 years, 0 months, 0 days

  • Research summary

    Cystic fibrosis (CF) is an autosomal recessive condition, characterized by thickened mucus, that primarily affects the lungs, gastrointestinal tract, reproductive organs, and sweat glands. CF imposes a considerable humanistic and financial burden on patients and their caregivers, and this effect may be amplified during pulmonary exacerbations. The current understanding of overall burden of CF on caregivers, especially during an event of pulmonary exacerbation, is limited. This study aims to evaluate work productivity, quality of life, general health status, social impact, direct and indirect economic/financial impacts associated with CF, and particularly the impact of CF pulmonary exacerbations requiring hospitalization, among caregivers.

    This is a prospective observational cohort study of caregivers of pediatric CF patients. Recruitment will be conducted during a hospitalization due to pulmonary exacerbation. The study will include caregivers of patients who complete their acute IV antibiotic treatment entirely in the hospital setting as well as caregivers of patients who initiate treatment during a hospitalization and complete acute IV antibiotic treatment in the home setting.

    Data collection will consist of completion of a survey via paper at two time points: first (T1) during the acute IV antibiotic treatment (hospital or home setting), and second (T2) at least 4 weeks later when the pediatric CF patient has returned to a caregiver-reported “well” health status for at least 4 weeks. The T2 survey may be completed in the home or clinic setting. Each survey is expected to take 40-50 minutes to complete. The surveys consist of a combination of validated instruments and home-grown items. Prior to enrollment, the site coordinator will complete a screening form to ensure eligibility for the study, and will provide the pediatric CF patient’s FEV1 value and CF mutation type. Informed consent will be obtained prior to enrollment. Only de-identified data will be collected in this study.

  • REC name

    West Midlands - Coventry & Warwickshire Research Ethics Committee

  • REC reference


  • Date of REC Opinion

    18 Aug 2015

  • REC opinion

    Further Information Favourable Opinion