Ivacaftor (Kalydeco) for the treatment of cystic fibrosis in adults who have an R117H mutation
Ivacaftor (trade name: Kalydeco) has been approved in Germany since November 2015 for the treatment of cystic fibrosis in adults who have an R117H mutation. In 2019, the Institute for Quality and Efficiency (IQWiG, Germany) looked into whether ivacaftor has any advantages or disadvantages compared to the standard treatment for cystic fibrosis in this group of people. The standard treatment in this situation is best supportive care (BSC). It should be tailored to the patient's individual needs, relieve symptoms such as pain, and improve quality of life. BSC includes, for instance, inhalation treatments with saline (salt) solution, mucus-thinning medications, painkillers and physical therapy. The manufacturer provided one study involving a total of 50 people with usable data. One half of the participants were given one ivacaftor tablet (150 mg) every 12 hours, and the other half were given a placebo (fake medication). Everyone in the study also received BSC, but without all the usual treatments because inhalations with saline (salt) solution were not allowed as an additional treatment. Also, the study didn’t show what sort of treatment adjustments were made in BSC. These were the results in this group of patients after about half a year:
What are the advantages of ivacaftor?
Difficulty breathing: The results suggest that ivacaftor has an advantage here: Breathing improved in the ivacaftor group compared to the placebo group.
Emotional state: The study suggests that ivacaftor has an advantage compared to the placebo in this aspect of health-related quality of life, specifically in people who have a bacterial (Pseudomonas aeruginosa) respiratory infection. No difference was found in people who didn’t have this type of infection.
Vitality, social limitations and subjective impression of overall health: The study suggests that there was an improvement in these aspects of health-related quality of life in women. There was no difference here in men.
What are the disadvantages of ivacaftor?
Sore throat: The study suggests that ivacaftor has a disadvantage here: Nearly 17 out of 100 people who used ivacaftor had a sore throat, but none of the people who were given a placebo had this side effect.
Life expectancy: None of the participants passed away during the study.
Acute cystic fibrosis episodes affecting the airways: No difference was found here. All of the participants in the study had an average of 1 to 2 acute episodes per year.
Physical wellbeing, body image, eating disorders, stress resulting from the treatment and role functioning (the ability to carry out typical everyday activities): There was no difference in any of these aspects of health-related quality of life.
There were also no differences in terms of:
- Hospital stays due to cystic fibrosis episodes affecting the airways
- Gastrointestinal (stomach and bowel) problems
- Weight problems
- Serious side effects
- Treatment stopped due to side effects
Institute for Quality and Efficiency in Health Care (IQWiG, Germany). Ivacaftor (cystic fibrosis, 18 years and older, with R117H mutation) – Benefit assessment according to §35a Social Code Book V. Dossier assessment; Commission A19-68. November 28, 2019. (IQWiG reports; Volume 837).
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