Nicole L. Goh

Neurofibromatosis type-1 is a rare autosomal dominant disease, due to the loss of the NF1 tumour suppressor gene. Here we present a case of a 28-year-old man with neurofibromatosis type-1 lung disease and pleuroparenchymal fibroelastosis leading to recurrent pneumothoraxes requiring intervention.

Antifibrotic drugs, available for the best part of the last decade in many parts of the world, has improved outcomes in patients with idiopathic pulmonary fibrosis and progressive pulmonary fibrosis. However, it is unclear whether patients suffering from these devastating conditions have timely and adequate access to antifibrotic therapy in the Asia Pacific region (APAC). In this mixed-methods narrative review of 12 APAC countries, integration of questionnaire-based insights of 31 regional clinical experts in interstitial lung disease (ILD) with publicly available pharmaco-economic information has been used to understand how country-specific challenges impact on antifibrotic accessibility. Overall, a broad range of approaches are utilised to provide antifibrotic treatment including centrally or state-determined drug budgets, pharmaceutical industry-subsidised initiatives, charitable support and self-paying (out-of-pocket) options. Impediments to antifibrotic access commonly arise from prohibitive drug pricing in relation to income, absence of universal coverage for pharmaceutical costs, lack of formal pharmaco-economic analysis or restrictions on the use of generic preparations. Unequal access to antifibrotic drugs is a vital unmet therapeutic need in the APAC region, one that is likely to be exacerbated by a rising fibrotic interstitial lung disease burden.

Background: Advanced lung diseases are prevalent in women, yet are underrecognized and under-treated due to differing epidemiology and pathophysiology. Objective: To investigate any gender differences in access to palliative care and end-of-life management for patients with advanced lung diseases. Methods: A post-hoc analysis was conducted using three datasets that included information regarding the provision of palliative care to patients with advanced lung diseases - chronic obstructive pulmonary disease (COPD), fibrotic interstitial lung diseases (f-ILD) or non-small cell lung cancer (NSCLC) in tertiary and regional hospitals in Victoria, Australia, from 2004 to 2019. Results: 343 patients with advanced COPD, 67 with f-ILD and 1022 with NSCLC were included. Compared to men, women with COPD (n = 126, 36.7%) were less likely to have smoked (P = 0.024), had significantly worse lung function (P < 0.001), and were more likely to receive non-invasive ventilation at end of life (P = 0.021). Women with fibrotic ILDs (n = 30, 44.8%) had significantly worse lung function (P < 0.001) and were more likely to experience exacerbations during their last two years of life (P < 0.001). Women with NSCLC (n = 457, 44.7%) were significantly younger (P< 0.001), less likely to have smoked (P < 0.001) or had asbestos exposure (P < 0.001). There were no significant differences between men and women with advanced lung diseases regarding referral to palliative care services (P = 0.369), hospital place of death (P = 0.915), or end-of-life management. Conclusions: Despite differences in lung function, exacerbations and targeted therapies, men and women with advanced lung diseases received equal access to symptom palliation and palliative care services towards the end of life.

Objective: Idiopathic pulmonary fibrosis (IPF) is a chronic progressive lung disease with a poor prognosis. Antifibrotics slow the decline of pulmonary function after 12-months, but limited studies have examined the role of circulatory biomarkers in antifibrotic treated IPF patients. Methods: Serum from 98 IPF participants, from the Australian Idiopathic Pulmonary Fibrosis Registry were collected at four time-points over 1 year post-antifibrotic treatment and analysed as two separate cohorts. Patients were stratified as progressive, if they experienced ≥ 10% decline in FVC or ≥ 15% decline in DLCO or were deceased within 1 year of treatment initiation: or otherwise as stable. Ten molecules of interest were measured by ELISAs in patient serum. Results: Baseline MMP7 levels were higher in progressive than stable patients in Cohort 1 (p = 0.02) and Cohort 2 (p = 0.0002). Baseline MMP7 levels also best differentiated progressive from stable patients (Cohort 1, AUC = 0.74, p = 0.02; Cohort 2, AUC = 0.81, p = 0.0003). Regression analysis of the combined cohort showed that elevated MMP7 levels predicted 12-month progression (OR = 1.530, p = 0.010) and increased risk of overall mortality (HR = 1.268, p = 0.002). LASSO regression identified a multi-biomarker panel (MMP7, ICAM-1, CHI3L1, CA125) that differentiated progression more accurately than MMP7 alone. Furthermore, GAP combined with MMP7, ICAM-1, CCL18 and SP-D was more predictive of 3-year mortality than GAP alone. Conclusions: MMP7 along with a multi-biomarker and GAP panel can predict IPF progression and mortality, with the potential for optimising management.

Objective: Digital technologies offer opportunities for remote monitoring, increased patient engagement and incorporation of patient-reported outcome measures (PROMs) into interstitial lung disease (ILD) care and research. This study evaluated the usability and patient experience of the RE-BUILD (Registry for Better Understanding of ILD) application, an ILD-specific smartphone app. Methods: Patients with ILD aged ≥18 years were recruited from three tertiary ILD centres to use the RE-BUILD app for 6 months. The mHealth App Usability Questionnaire (MAUQ) was evaluated at 1, 3 and 6 months and patients received monthly prompts to enter clinical and PROM data. Qualitative interviews regarding patient experience were performed in a subset of 10. Results: Fifty patients, with mean age 66.9 ± 10.3 years, 25 (50%) female were included. Participants used the app for a median of 48 (IQR 21-178.3) sessions, equivalent to 8 sessions (IQR 3.5-29.71) per month. Median number of days that the app was accessed was 37 (IQR 14-96.8), with 13 (26%) patients using the app >30 times per month. The most accessed app feature was physical activity, followed by 'air quality'. Participants agreed or strongly agreed that the app was easy to use (76.0%) easy to learn to use (79.8%) and well-organized with accessible information (74.8%). The median overall MAUQ score for usability was 5.69 (IQR 5.03-6.19). There was also a high rate of engagement with app functionalities. Conclusions: RE-BUILD is a usable and intuitive platform for self-monitoring and data collection in ILD. Patients report a high degree of satisfaction and have provided valuable feedback for its further development.