Patients in the LOTUSS study who were treated for systemic sclerosis-associated idiopathic pulmonary fibrosis (SSc-IPF) using Esbriet (pirfenidone) showed signs of drug tolerability, according to a presentation at the European League Against Rheumatism (EULAR) 2015 congress held in Rome. Esbriet, which was approved by the Food and Drug Administration in 2014 to treat idiopathic pulmonary fibrosis, may be effective in treating SSc-IPF.
Since pirfenidone has been used as a novel antifibrotic agent in patients with IPF, the study investigators were interested in the safety and tolerability of pirfenidone in patients with SSc-IPF, as interstitial lung disease is common and serious in patients with SSc. The clinical trial lasted 16 weeks, and all patients were aware of their treatment in the open-label trial design. Only patients who had undergone high resolution computed tomography to diagnose their IPF were eligible to participate. The study contained 63 patients, and the majority were female.
A dose titration period was used at the beginning of the trial. The target dose was 2403 mg/day, and participants received increasing doses until this target was reached. Throughout treatment, patients were monitored for modified Rodnan skin score (mRSS), forced vital capacity (FVC) percent predicted, and diffusing capacity of the lung for carbon monoxide (DLCO) percent predicted. However, by design, the study did not have enough participants to draw any conclusions regarding the efficacy of treatment.
At the end of the trial, treatment emergent adverse events (TEAEs) were experienced by nearly all (98.2%) of patients. The majority of these events were moderate TEAEs, with only 19% of TEAEs classified as severe. Most commonly (more than 10% of patients), nausea, headache, fatigue, and vomiting were experienced as TEAEs. These TEAEs were tolerated by the majority of patients, as only 9.5% of patients discontinued the study as a result of drug hypersensitivity, worsening fibromyalgia pain, pulmonary hypertension, photosensitivity reaction, or rash. These results were presented at EULAR 2015 in the presentation, “Safety and Tolerability of Pirfenidone in Patients with Systemic Sclerosis-Associated Interstitial Lung Disease – The LOTUSS Study.”
“In the 16-week, open label trial of pirfenidone in SSc-ILD, pirfenidone was generally well-tolerated in SSc-ILD patients, despite pre-existing co-morbidities, including underlying gastrointestinal disease, and concomitant use of mycophenolate mofetil,” stated the researchers. “The observed adverse events were expected and consistent with those previously seen with pirfenidone treatment in IPF trials.” Therefore, the researchers believe pirfenidone should be pursued as a treatment for SSc-IPF.