Background and Importance

The new transmembrane conductance regulator (CFTR) modulator drugs (ivacaftor/tezacaftor/elexacaftor) are bringing about a major change in the treatment and quality of life of cystic fibrosis (CF) patients. There is a need to collect information on patient perception of benefit and safety.

Aim and Objectives

To assess the satisfaction and adverse effect (AE) profile reported by patients on ivacaftor/tezacaftor/elexacaftor treatment.

Material and Methods

Observational, prospective, single-centre study from March to June 2022. CF patients with at least one p.Phe508del mutation in treatment with CFTR modulators. Variables: sociodemographic (age and sex) and biochemical (GOT-ASAT, GPT-ALAT, bilirubin and CPK) collected from patients9 medical records. Treatment Satisfaction Questionnaire for Medication (TSQM 1.4) with 14 items in four scales assessing efficacy, side effects, convenience and overall satisfaction from the patient9s own perspective, the higher the score, the higher the satisfaction. Adverse Effects Questionnaire (ad hoc), Patient Informed Consent.

Results

Out of 58 patients on treatment, 43 answered the questionnaires, 17 (40%) female, median age 30 (26-37). For TSQM the median score for each item was 21 (20-21) over 21; 19 (16-20) over 21; 21 (18-21) over 21 and 17 (16-17) over 17 respectively. Regarding AEs: 39.47% reported increased appetite, 31.58% rash, 23.68% headache, 13.16% runny nose, increased blood pressure, diarrhoea and itchy skin, 10.53% abdominal pain or discomfort, common cold and itchy or stinging eyes and ≤ 2% flatulence, memory loss, yellowing of skin and eyes, muscle pain, fluid retention, insomnia, acne, nausea and vomiting. One patient presented with severe rash requiring discontinuation of treatment. Three months after starting treatment, only three patients had GPT-ASAT >3 LSN and only one patient >5 LSN, one patient had increased CPK >5 LSN and no patient had bilirubin >2 LSN.

Conclusion and Relevance

Although a high percentage of patients have experienced AEs, CFTR modulators are widely accepted drugs with a favourable AE profile. The most frequent AEs reported by patients were increased appetite, rash and headache. The AEs described by patients are described in the data sheet. More real-life studies are needed to confirm our study and to provide further evidence.

References and/or Acknowledgements

Conflict of Interest

No conflict of interest.