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Lumacaftor/ivacaftor (LUM/IVA) improves outcomes in cystic fibrosis (CF) patients homozygous for Phe508del with ppFEV140%. There is limited safety or efficacy data in patients with ppFEV140%. We determined whether LUM/IVA in patients with ppFEV140 would reduce the rate of pulmonary exacerbations.This was a case control study performed on patients12 years, homozygous for Phe508del CFTR mutation and with ppFEV140%. Control subjects were matched for age, sex and ppFEV1, and had mutations ineligible for LUM/IVA. We assessed the rate of pulmonary exacerbations requiring intravenous antibiotics, the mean rate of change in ppFEV1 over 12 months and all adverse events.Data was collected from 7 Australian CF centres on 105 patients; 72 on LUM/IVA and 33 controls. LUM/IVA demonstrated a large reduction in exacerbations with an incident rate ratio of 0.455 (95%CI; 0.306 - 0.676), p 0.001 after adjusting for the number of exacerbations in the previous 12 months. LUM/IVA prolonged the time to first exacerbation and reduced the rate of decline in ppFEV1 over 12 months. Adverse events were common; chest tightness or dyspnoea was experienced by 55% and resulted in cessation of treatment in 32%.Treatment with LUM/IVA resulted in a substantially lower rate of pulmonary exacerbations, prolonged time to first exacerbation and slowed the rate of decline of ppFEV1 in participants with severe lung disease. Adverse reactions to LUM/IVA however were unacceptably frequent, and resulted in a very high discontinuation rate.

Objectives: Cystic fibrosis-related diabetes (CFRD) impacts approximately 40 to 50% of adults with cystic fibrosis (CF). Effective diabetes education has been shown to improve long-term self-management and quality of life. This survey explores the educational needs of patients with or developing CFRD and sets out to identify the optimal mode of delivery for diabetes education. Methods: Adult pre- and post-transplant patients with CFRD and early glucose metabolism abnormalities from the Royal Prince Alfred Hospital CF Clinic were invited to partake in an online survey via email or text message in December 2020. The study protocol was approved by the Sydney Local Health District Ethics Committee (X20-0434). Results: 46 participants completed the survey with a response rate of 45% (female 54%;CFRD 78%;mean duration since CFRD diagnosis: 13 years;post-transplant 15%). Main sources of diabetes education for participants were the CF Clinic (65%);Google search (30%) and their general practitioner (13%). 72% of participants with CFRD felt they had enough information to manage their diabetes but favoured having a diabetes education resource package. The majority of participants preferred to receive CFRD information via an email package (61%), while others favoured perusing an educational website (37%) or phone app (24%), when requested (44%) or every 6 months (31%). The most common categories of diabetes-related information requested by participants with CFRD were updated diabetic technology (75%), CF diet and food diaries (58%), exercise (56%), management of hyper-(58%) or hypoglycemia (47%) and diabetes and alcohol consumption (42%). Conclusion: Patients with CFRD and early glucose metabolism abnormalities have ongoing educational needs and may require improved resources, preferably via virtual platforms, to supplement in-person care. Future research on the clinical impact of increasing virtual education will be necessary during this COVID-19 pandemic and beyond.