A prospective randomised controlled mixed-methods pilot study of home monitoring in adults with cystic fibrosis.

BACKGROUND: Home monitoring (HM) is able to detect more pulmonary exacerbations (PEx) than routine care (RC) in individuals with cystic fibrosis (CF), but there is currently no evidence for benefits in health outcomes. Patient experiences of using HM and a health economics assessment have not been rigorously assessed to date. This study aimed to assess the effects of HM on hospital admissions, quality of life, antibiotic requirements, exacerbation frequency, lung function, nutritional outcomes, anxiety, depression, costs and health outcomes, as well as the qualitative effects on the patient experience. METHODS: This randomised controlled mixed-methods pilot study recruited CF adults cared for in one large regional CF centre. Participants were randomly allocated 1:1 to the intervention cohort [twice-weekly HM of symptoms measured by the Cystic Fibrosis Respiratory Symptom Diary - Chronic Respiratory Infection Symptom Score (CFRSD-CRISS) and forced expiratory volume in one second (FEV1)] or a control cohort (routine clinical care) for the 12-month study period. Measurements were recorded at study visits at baseline, 3, 6, 9 and 12 months. Spirometry, body weight, comorbidities, medications, hospital inpatient days, courses of antibiotics (oral and intravenous) and PEx (defined by the modified Fuchs criteria) were recorded at each study visit. Health status, capability and cost-effectiveness were measured at each study visit by the Hospital Anxiety and Depression Scale (HADS), the ICEpop CAPability measure for Adults (ICECAP-A), EuroQol 5 dimensions (EQ-5D-5L) questionnaire and an adapted resource use questionnaire. The patient experience of HM was assessed by semi-structured qualitative interviews at baseline and 12 months. RESULTS: Eighty-eight participants were recruited, with 44 (50%) randomised to receive HM and 44 (50%) randomised to receive RC. Patient hospital inpatient bed days per annum and overall health-related quality of life were similar between the groups. Protocol-defined PEx requiring intravenous and oral antibiotics were detected more frequently in the HM group, with no other differences between the groups in the secondary outcomes. The total mean National Health Service (NHS) costs were approximately £1500 more per patient for the RC arm than the HM group. The qualitative analysis demonstrated that the patient experience of HM was generally positive and overall the intervention was well accepted. CONCLUSION: The findings of this trial confirm that HM is effective in detecting PEx in adults with CF. There were no significant differences in hospital inpatient bed days and overall health-related quality of life between the groups. Despite the cost of the HM equipment and the salary of the research fellow to respond to the results, health economics analysis suggests the intervention was less expensive than RC. HM was generally well accepted, with most participants reporting that it resulted in them feeling more empowered and reassured. TRIAL REGISTRATION: The study protocol was registered with Clinicaltrials.gov (NCT02994706) on 16 July 2014 and published in a peer reviewed journal.Data from this trial has been presented in abstract form at the ECFS Conference in Lyon in September 2020.

Outcomes of pregnancy in women with cystic fibrosis (CF) taking CFTR modulators - an international survey.

BACKGROUND: As their long-term prognosis improves, women with CF are increasingly choosing to have children, but the safety of CFTR modulators in pregnancy and breastfeeding is currently unknown. METHODS: A survey was sent to lead clinicians of adult CF centres in Europe, the United Kingdom (UK), United States of America (USA), Australia and Israel requesting anonymised data on pregnancy outcomes in women using CFTR modulators before and during pregnancy and lactation. RESULTS: We identified 64 pregnancies in 61 women taking IVA (n = 31), LUM/IVA (n = 26) or TEZ/IVA (n = 7), resulting in 60 live births. In 44 pregnancies, CFTR modulators were either continued throughout pregnancy or temporarily stopped and then restarted. Two maternal complications were deemed related to CFTR modulator therapy; cessation of modulator therapy resulted in clinical decline in 9 women prompting resumption of therapy during pregnancy. No modulator-related complications were reported in infants exposed in utero and/or during breastfeeding. CONCLUSIONS: CFTR modulators were reported to be generally well tolerated in pregnancy and breastfeeding, with only 2 maternal complications that were deemed related to CFTR modulator therapy. Women stopping CFTR modulators in pregnancy may experience a decline in clinical status and in the cases identified in this survey, restarting therapy led to a clinical improvement. Current experience remains limited and longer-term prospective follow-up is required to exclude delayed adverse effects.

Characterising burden of treatment in cystic fibrosis to identify priority areas for clinical trials.

In a recent James Lind Alliance Priority Setting Partnership in cystic fibrosis (CF) the top priority clinical research question was: "What are effective ways of simplifying the treatment burden of people with CF?" We aimed to summarise the lived experience of treatment burden and suggest research themes aimed at reducing it. An online questionnaire was co-produced and responses subjected to quantitative and thematic analysis. 941 survey responses were received (641 from lay community). People with CF reported a median of 10 (interquartile range: 6-15) current treatments. Seven main themes relating to simplifying treatment burden were identified. Treatment burden is high, extending beyond time taken to perform routine daily treatments, with impact varying according to person-specific factors. Approaches to communication, support, evaluation of current treatments, service set-up, and treatment logistics (obtaining/administration) contribute to burden, offering scope for evaluation in clinical trials or service improvement.