The RISE study protocol: Resilience lmpacted by positive Stressful Events for people with cystic fibrosis

Introduction For people with cystic fibrosis (pwCF), gaining access to elexacaftor/tezacaftor/ivacaftor (ETI) therapy, a new modulator drug combination, is perceived as a positive life event. ETI leads to a strong improvement of disease symptoms. However, some pwCF experience a deterioration in mental well-being after starting ETI therapy. The primary objective of this study is to investigate if and in which direction mental well-being of pwCF, defined as quality of life (QoL), changes after starting ETI therapy. Our secondary objectives include, among others, investigation of underlying biological and psychosocial factors associated with a change in QoL of pwCF after starting ETI therapy.

Methods and analysis The Resilience lmpacted by positive Stressful Events (RISE) study is a single arm, observational, prospective longitudinal cohort. It has a timeframe of 60 weeks: 12 weeks before, 12 weeks after, 24 weeks after, and 48 weeks after start ETI therapy. The primary outcome is the mental well-being, measured on each of these four time points. Patients are eligible when aged 12 years or older, qualifying for ETI therapy based on their CF-mutation, and being a patient in the University Medical Center Utrecht. Data will be analysed using a covariance pattern model with a general variance covariance matrix.

Ethics The RISE study was classified by the Institutional Review Board as exempt from the Medical Research Involving Human Subjects Act. Informed consent was obtained by both the child (12 to 16 years) and their caregivers or only provided by the participants themselves when aged 16 years and older.