Abstract Rationale Refractory chronic cough (RCC) is a disease identified in a subset of people who experience chronic cough (defined in adults as a cough lasting 8 weeks), despite adequate treatment for known cough-related etiologies. Unexplained chronic cough (UCC) describes a chronic cough for which an underlying condition has not been determined, despite adequate investigation with diagnostic tests. Both conditions can substantially impact patients’ quality of life, yet currently, no approved medications for RCC or UCC exist in the United States (US). This retrospective chart review aimed to describe real-world characteristics of patients with RCC/UCC, currently used treatments and their effectiveness. Methods Physicians were recruited by a panel, and included pulmonologists, allergists/immunologists, otolaryngologists, gastroenterologists, and general practitioners. Each physician extracted detailed medical chart data for ≤5 adult patients diagnosed with RCC/UCC between January 1, 2019 and September 30, 2024. Patient characteristics, treatment patterns and effectiveness during the follow-up period (RCC/UCC diagnosis to last visit) were described, including reasons for discontinuation. Results Data from 621 patient charts were extracted by 254 physicians. Patients were mostly female (58.5%), White (73.3%), with a mean (standard deviation SD) age of 55.3 (15.6) years. In the 12 months prior to diagnosis, RCC/UCC-related symptoms or complications were reported in 49.4% of patients, including insomnia (25.1%), incontinence (12.7%), sleep apnea (10.8%), sleep disturbance (9.2%) and laryngeal dysfunction (8.9%). Pharmacologic (n = 2,404) and non-pharmacologic (n = 80) treatment regimens were analyzed. Treatment patterns varied widely; patients used a mean (SD) of 4.0 (4.1)/median 3.0 (range: 1.0-5.0) treatments during the follow-up period, over a median duration of 16.6 (range: 12.7-26.1) months (Figure). Physicians reported that 81.6% (n = 1,961) of pharmacologic treatments resulted in no response. Only 10.3% (n = 247) of treatment regimens yielded a response, including 9.0% (n = 216) that resulted in symptom improvement and 1.3% (n = 31) that resulted in symptom resolution. Most pharmacologic treatment regimens (61.2%) were discontinued after a median of 2.0 (range: 1.0-4.0) months. Lack of efficacy was cited as the primary reason for discontinuation (74.3%). Conclusions This study highlights the current challenges with diagnosis and management of RCC/UCC patients. The lack of US-approved medications means that patients remain reliant on trial-and-error approaches with off-label treatments, many of which do not provide meaningful relief. Our results reflect the inadequacy of current options, evidenced by the high treatment discontinuation rates and limited symptom resolution. Targeted, effective, and well-tolerated medications are needed for the management of RCC/UCC. This abstract is funded by: GSK (study number 306551)
Ghobrial et al. (Fri,) studied this question.