Background:Lumacaftor/ivacaftor combination therapy is efficacious and generally safe for patients with cystic fibrosis (CF) homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation. However, long-term survival benefits of lumacaftor/ivacaftor (LUM/IVA) cannot yet be quantified. Simulation models can provide predictions about long-term health outcomes. In this study, we aimed to project long-term health outcomes of LUM/IVA plus standard care (SC) in patients with CF homozygous for F508del-CFTR.Methods:This modeling study was an individual patient simulation in US patients aged ⩾6 years with CF, homozygous for F508del-CFTR. The primary outcome was projected survival among (a) a cohort of patients who ever initiated LUM/IVA, accounting for treatment discontinuations, and (b) a cohort of patients who remain on continuous LUM/IVA. Patient characteristics and model parameters were derived from clinical trials: VX14-809-109, VX13-809-011B, TRAFFIC/TRANSPORT, and PROGRESS; published literature; and the US CF Foundation Patient Registry.Results:Lumacaftor/ivacaftor + SC is expected to increase median survival by 6.1 years versus SC alone, accounting for treatment discontinuations. The incremental median predicted survival versus SC assuming initiation of LUM/IVA at ages 6, 12, 18, and 25 years was 17.7, 12.6, 8.0, and 3.8 years, respectively. Assuming lifetime treatment with LUM/IVA, incremental median survival was predicted to be 7.8 years longer in the LUM/IVA + SC cohort. Initiating LUM/IVA at ages 6, 12, 18, and 25 years and assuming lifetime treatment resulted in incremental median predicted survival of 23.4, 18.2, 11.0, and 4.8 years, respectively.Conclusions:Lumacaftor/ivacaftor is projected to increase survival for patients with CF. Initiation at an early age and treatment persistence result in further increments in projected survival.

Ivacaftor therapy was associated with significant reductions in hospitalizations along with high rates of adherence to treatment over 12 months.

Background Trigeminal neuralgia (TN) causes severe episodic, unilateral facial pain and is initially treated with antiepileptic medications. For patients not responding or intolerant to medications, surgery is an option. Methods In order to expand understanding of the pain-related burden of illness associated with TN, a cross-sectional survey was conducted of patients at a specialist center that utilizes a multidisciplinary care pathway. Participants provided information regarding their pain experience and treatment history, and completed several patient-reported outcome (PRO) measures. Results Of 129 respondents, 69/128 (54%; 1 missing) reported no pain in the past 4 weeks. However, 84 (65%) respondents were on medications, including 49 (38%) on monotherapy and 35 (27%) on polytherapy. A proportion of patients had discontinued at least one medication in the past, mostly due to lack of efficacy (n = 62, 48%) and side effects (n = 51, 40%). A total of 52 (40%) patients had undergone surgery, of whom 30 had microvascular decompression (MVD). Although surgery, especially MVD, provided satisfactory pain control in many patients, 29% of post-surgical patients reported complications, 19% had pain worsen or stay the same, 48% were still taking pain medications for TN, and 33% reported new and different facial pain. Conclusions In most PRO measures, respondents with current pain interference had poorer scores than those without pain interference. In the Patient Global Impression of Change, 79% expressed improvement since beginning of treatment at this clinic. These results indicate that while the multidisciplinary approach can substantially alleviate the impact of TN, there remains an unmet medical need for additional treatment options.

of pre-index/index variables and receipt of PCE±14 days post-discharge on 6-month follow-up healthcare costs. Subjects receiving PCE-C were matched on age, gender, and time of admission to those not receiving PCE-C. Results: A total of 228 COPD admissions were identified (matched 1:1), all-cause costs in the 6-month period following discharge were similar between those who received PCE-C vs those that did not ($13,494 vs $10,856, p= 0.43). However, COPD-related follow-up costs were higher for those who received PCE-C ($2,431 vs $1,470, p= 0.01). Higher COPD-related costs were higher for older patients (RR= 1.05, p< 0.01) and those with greater pre-index COPD-related costs (RR= 1.004, p< 0.01). Similarly, factors associated with greater all-causefollow-up costs were age (RR= 1.03, p= 0.04), higher pre-index all-cause costs (RR= 1.01, p< 0.01), as well as longer length of stay (LOS) for baseline admission (RR= 1.04, p= 0.03). ConClusions: Greater follow-up costs may be related to the severity of COPD for those patients receiving a corticosteroid following a COPD-related admission. Higher follow-up healthcare costs were correlated with increased age, greater baseline healthcare costs, and a longer LOS of index admission. Sponsorship: GSK (HO-14-15081).

Objectives: The Assessment of Burden of COPD (ABC) tool evaluates and visualizes the health status of patients with chronic obstructive pulmonary disease (COPD). This tool may be used during consultations to monitor the burden of COPD and to adjust treatment. The ABC tool has items in 5 dimensions: symptoms, limitations, mental status, exacerbations, and fatigue. The aim of our study was to determine the burden of each of 15 elements in the ABC questionnaire. MethOds: A discrete choice experiment (DCE) was conducted using telephone-assisted personal interviews with 279 COPD patients. They were presented 13 sets of two patients, and decided which patient was in the worst health state. In order to make the choice task feasible despite the large number of 15 attributes, we generated an efficient partial profile design, that kept attributes in some dimensions constant and all at the same level (fold in), while varying the attributes in other dimensions (fold out). This reduced the burden on respondents by having them assess some attributes combined in categories, instead of as separate attributes. Multinomial logit was used to analyze the data. Results: Patients were considered to be in worst health if they had high levels of fatigue, exacerbations, anxiety, breathlessness at rest and limitations in moderate physical activities. These factors had three to five times as much impact on the burden of disease as lower levels of these attributes and other limitations. Coefficients were very small and/or statistically insignificant for breathlessness during physical activity, limitations on strenuous activity, coughing, small numbers of exacerbations, and most mental attributes. cOnclusiOns: It is possible to administer cognitively complicated DCE questionnaires using a fold-infold-out design. COPD-patients seem to accept being unable to perform strenuous activities. Gains in well-being can primarily be achieved by focusing on patients' ability to lead a relatively normal everyday life.

Purpose Due to the increasing incidence of dementia in Ireland, there is a need to prepare occupational therapy students for future careers in dementia-focused health care. The purpose of this study are to measure the knowledge and attitudes of Irish undergraduate occupational therapy students towards older people with dementia and examine related variables including year of study, personal and placement dementia experiences and future career choice. Design/methodology/approach Data was gathered using a cross-sectional online survey, incorporating the Alzheimer’s Disease Knowledge Scale and the Dementia Attitudes Scale, which was analysed using descriptive and inferential statistics. Findings Seventy-five responses were gathered and analysed, indicating more advanced occupational therapy students demonstrate higher levels of dementia knowledge, but dementia attitudes remain similar across different year groups. Students with clinical dementia experiences displayed comparable levels of dementia knowledge and attitudes to those without. However, students with familial dementia experiences displayed significantly more positive attitudes. The likelihood of selecting a future career with older adults with dementia significantly related to students’ positive dementia attitudes but not dementia knowledge. Originality/value To the best of the authors’ knowledge, this is the first Irish study that focuses on undergraduate occupational therapy students’ dementia knowledge and attitudes. The results could be used to inform and develop Irish undergraduate occupational therapy programmes.

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