PROMISE: Working with the CF community to understand emerging clinical and research needs for those treated with highly effective CFTR modulator therapy

The Gut-Lung Axis in Cystic Fibrosis

“…Interestingly, in the pancreatic sufficient group, antibiotics had a greater impact on the intestinal microbiome than modulator therapy, a result consistent with that of a lung microbiota study on the effects of ivacaftor ( 159 ). A large upcoming study, PROMISE, will examine many effects of modulator therapies, including gut microbiota alterations ( 161 ). The PROMISE study is particularly important, as it will be the first to examine the effects of the most recently approved modulator combination therapy, elexacaftor/tezacaftor/ivacaftor (trikafta).…”

Assessment of a novel genetic counselling intervention to inform assisted reproductive technology treatments and other family-building options in adults with cystic fibrosis

“…The introduction of highly effective modular therapy may have a drastic effect on physical health and life expectancy in this population, which will undoubtedly change patients’ perceptions about parenthood and family building. However, given the novelty of these therapies, more studies are needed to assess the fertility impacts of modulator therapy, along with mental health and quality-of-life impacts ( Balfour-Lynn and King, 2020 , Nichols et al, 2021 ). As more is learned about these novel therapies and how they physically impact fertility, qualitative studies regarding changing perceptions about family building and parenthood are necessary.…”

“…Patient-reported outcomes that aim to assess health-related quality of life have been shown to improve with CFTR modulators ( Balfour-Lynn and King, 2020 ). The direct impact on fertility has not been assessed to date, given the novelty of the drug ( Nichols et al, 2021 ). Although it is too soon to collect evidence about life expectancy directly, it is possible that CF may not be a life-shortening disease in the future, which will fundamentally change patients’ perceptions about parenthood and family-building options ( Balfour-Lynn and King, 2020 ).…”

Cystic Fibrosis: A Disease in Transformation, yet More Work to Be Done!

“…The outcomes being evaluated are changes in ppFEV 1 , sweat chloride concentration (SCC), body mass index (BMI) and patient-reported outcomes (respiratory symptoms, CFQ-R). The PROMISE study has multiple sub-studies examining other disease manifestations including airway microbiology, gastrointestinal symptoms and glucose metabolism which will be reported at a later date (5).…”