- Mean absolute change in % predicted FEV1 was approx. 3% and mean relative change in % predicted FEV1 was approx. 5% (on the basis of pooled data). That means if a patient’s baseline FEV1 was 60%, on average they would have seen an increase to 63%. While this is less dramatic than the effect seen in G551Ds on Ivacaftor monotherapy, it was statistically significant and, for many people with CF, each % point maintained is important;
- Statistically significant increase in Body Mass Index (BMI)
- Delayed time to first pulmonary exacerbation;
- 30-39% reduction in pulmonary exacerbations (depending on the dose);
- 39-61% reduction in exacerbations requiring hospitalisation;
- 45-56% reduction in exacerbations requiring intravenous antibiotics;
- Some people on the trial had baseline FEV1 below 40%. While the eligibility criteria was 40%, some dipped below this threshold in the period between screening and first drug. These patients seem to have responded just as well as those with higher baseline FEV1;
- Approx. 90% of people on the trial decided to rollover into the open label extension including those who had been taking placebo during the trial period. This says something about how the drug made patients feel in themselves; and
- The Quality of Life questionnaire (which is validated by regulators) failed to show any improvement in quality of life. This seems at odds with the data above and, to me, suggests the instrument is not tuned into the things that really matter to patients. Perhaps there is a need to develop a new way of measuring quality of life. It would be a long process to get a new instrument validated but it could pay dividends and it something that could be done in partnership with patients and families.
Obviously exacerbations are disruptive to life so there is a clear and immediate quality of life impact for patients and families.
Personally, I wish I had known when I was younger that exacerbations tend to cause irreversible lung damage and knock you down a step. When I was a teenager and relatively healthy, I hated doing all my treatments particularly nebulisers and airway clearance. I sometimes used to think, “I’m fine and busy with my life. I won’t bother doing all my treatments. I'll wait until I get sick and then go to the hospital to get fixed". I didn’t think I was losing anything but I really was.