Huang, Suiyuan

Objective Mycophenolate mofetil (MMF) can stabilize or improve lung function in systemic sclerosis–related interstitial lung disease (SSc‐ILD). We hypothesized that combining MMF with pirfenidone (PFD) would produce a significantly more rapid and/or greater improvement in lung function. Methods A randomized (1:1), double‐blind, placebo (PLA)‐controlled phase 2 trial was conducted in SSc‐ILD in which patients received PFD or PLA (801 mg three times daily) along with MMF (1,500 mg twice daily) for 18 months. The primary outcome was change in percent predicted forced vital capacity (FVC‐%). Linear mixed‐effects models assessed treatment differences in a modified intention‐to‐treat population. Results Only fifty‐one of 150 intended subjects (34%) were randomized (MMF+PLA, n = 24; MMF+PFD, n = 27). The FVC‐% improved from baseline to 18 months by 2.24 ± 1.35 (least‐squares mean [LSM] ± SEM) in the MMF+PLA group and 2.09 ± 1.28 in the MMF+PFD group (LSM treatment difference −0.14; P = 0.93). Median time to achieve a ≥3% improvement in FVC‐% was numerically faster in the MMF+PFD versus MMF+PLA group (12.3 vs 17.8 months, respectively), but the difference was not significant ( P = 0.33). For secondary outcomes, only the change over 18 months in the Patient‐Reported Outcomes Measurement Information System 29‐item physical function score, favoring MMF+PFD, reached statistical significance ( P = 0.04). Although other related patient‐reported outcomes (PROs) numerically favored the MMF+PFD group, as did quantitative high‐resolution computed tomography measures of ILD, the differences between groups did not reach statistical significance. Early withdrawals from study medication and adverse events of special interest were numerically greater with MMF+PFD (n = 8 vs 2 and n = 20 vs 7, respectively). Conclusion In this underpowered study, there was no statistically significant treatment difference in overall change in FVC‐% between groups. MMF+PFD was not as well tolerated as MMF+PLA.

Background and Objectives: Interstitial lung disease (ILD) is a common manifestation of connective tissue disease (CTD). (a) Pulmonary rehabilitation (PR) can be utilised to improve dyspnoea and quality of life in patients with chronic lung conditions, including both obstructive and restrictive lung diseases. (b) This study aims to determine if PR can be a useful therapy to improve functional capacity and self-reported measures of dyspnoea in patients with CTD-ILD. Methods: Patients with a diagnosis of CTD-ILD at a tertiary centre were prospectively enrolled to receive PR in this study. The primary outcome was a change in the 6-minute walk test (6MWT) between baseline and 8 weeks after PR. The secondary outcome measures included pulmonary function tests (PFTs) and Patient-Reported Outcomes Measurement Information System (PROMIS→) Dyspnoea, St George’s Respiratory Questionnaire (SGRQ), and Leicester Cough Questionnaire (LCQ), which were collected before and at 8 weeks after PR. P < .05 was considered statistically significant. Results: Twenty-one patients were included in the data analysis. The baseline characteristics are shown in Table 1 . Following PR, there was a statistically significant improvement in the 6MWT ( Figure 1 ). Improvement in scores was observed in LCQ and the symptom domain of the SGRQ. Trends towards improvement were noted in PROMIS → Dyspnoea scores. Conclusion: In CTD-ILD patients, an 8-week course of PR led to improvements in functional capacity (6MWT) and certain quality of life domains (LCQ and symptom domain of SGRQ). Future studies are required to determine the longer-term benefit of PR in CTD-ILD patients and if these improvements are sustained.

Abstract Objectives To establish a framework by which experts define disease subsets in systemic sclerosis associated interstitial lung disease (SSc-ILD). Methods A conceptual framework for subclinical, clinical and progressive ILD was provided to 83 experts, asking them to use the framework and classify actual SSc-ILD patients. Each patient profile was designed to be classified by at least four experts in terms of severity and risk of progression at baseline; progression was based on 1-year follow-up data. A consensus was reached if ≥75% of experts agreed. Experts provided information on which items were important in determining classification. Results Forty-four experts (53%) completed the survey. Consensus was achieved on the dimensions of severity (75%, 60 of 80 profiles), risk of progression (71%, 57 of 80 profiles) and progressive ILD (60%, 24 of 40 profiles). For profiles achieving consensus, most were classified as clinical ILD (92%), low risk (54%) and stable (71%). Severity and disease progression overlapped in terms of framework items that were most influential in classifying patients (forced vital capacity, extent of lung involvement on high resolution chest CT [HRCT]); risk of progression was influenced primarily by disease duration. Conclusions Using our proposed conceptual framework, international experts were able to achieve a consensus on classifying SSc-ILD patients along the dimensions of disease severity, risk of progression and progression over time. Experts rely on similar items when classifying disease severity and progression: a combination of spirometry and gas exchange and quantitative HRCT.