Patient perception of disease burden in diffuse cutaneous systemic sclerosis.

Systemic sclerosis is a rare multi-organ autoimmune rheumatic disease, resulting in progressive fibrosis of the skin/internal organs. This study aimed to understand the impact of diffuse cutaneous systemic sclerosis symptoms and disease burden from the patient's perspective. This was a mixed methodology, market research study involving ethnography, structured interviews, video diaries, and patient tasks. Patients had been diagnosed with diffuse cutaneous systemic sclerosis for ⩾ 6 months and were recruited via healthcare professionals or patient associations (France, Italy, the United Kingdom, and the United States). Patients filmed short (~15 min) daily video diaries about their lives over 7 days and participated in ethnographic sessions, patient tasks, and structured video interviews. In Germany and Spain, patients participated in 60-min telephone interviews. Twenty-three patients (mean age: 54 years; 83% women; minimum disease duration: 6 months) participated in the study. Time to diagnosis was prolonged, as patients overlooked their symptoms and some healthcare professionals attributed symptoms to other causes. Patients rarely received additional information or support services at diagnosis. Importantly, although patients were aware of the seriousness of organ involvement, they reported that skin changes, pain, and fatigue impaired their ability to perform routine tasks. Patients had a high prescription treatment burden (mean: 10 tablets/day; up to >25 tablets/day) with additional non-prescription medication taken for other comorbidities. Treatment discontinuation was common due to side effects. Patients experienced diffuse cutaneous systemic sclerosis as a loss of independence and self-esteem. Moreover, patients tended to have small support networks, and emotional support services were not offered as standard care. Patients with diffuse cutaneous systemic sclerosis had high treatment and disease burdens, with skin changes, pain, and fatigue profoundly affecting their lives. There is an unmet need for patient information at the time of diagnosis and emotional support services throughout the patient's journey with diffuse cutaneous systemic sclerosis. Based on the results of this study, we provide recommendations for improving diffuse cutaneous systemic sclerosis care.

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© The Author(s) 2019.

Déclaration de conflit d'intérêts

Declaration of conflicting interests: The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: D.K. consults for Bayer, Bristol-Myers Squibb, Boehringer Ingelheim, Cytori, CSL Behring, EMD Merck-Serono, Genentech/Roche, GlaxoSmithKline, Genkyotex, Sanofi-Aventis, UCB, Actelion, and Gilead; has stock options in Eicos Sciences; and has received research grants from National Institutes of Health/National Institute of Arthritis and Musculoskeletal and Skin Diseases, National Institutes of Health/National Institute of Allergy and Infectious Diseases, Bayer, Bristol-Myers Squibb, and Pfizer. Y.A. consults for Actelion, Bayer, Roche/Genentech, Inventiva, Medac, Pfizer, Sanofi, Servier, and UCB; and has received research grants from Bristol-Myers Squibb, Roche/Genentech, Inventiva, Pfizer, Sanofi, and Servier. C.P.D. consults for Bayer, Roche, GlaxoSmithKline, Actelion, Inventiva, CSL Behring, Takeda, Merck-Serono, MedImmune, and Biogen; and has received research grants from Actelion, GlaxoSmithKline, Novartis, and CSL Behring. M.M.-C. consults for Actelion, GlaxoSmithKline, Pfizer, and Bristol-Myers Squibb. J.P. consults for and/or has research grants with Actelion, Bayer, BMS Merck, Pfizer, and Roche. B.H. is an employee of Bayer AG. S.D. is an employee of Blueprint Partnership. J.d.O.P. has been an employee of Bayer US LLC. O.D. has been a consultant for Genentech/Roche, 4D Science GmbH, Actelion, Active Biotech, Bayer, Biogen Idec, Boehringer Ingelheim, Bristol-Myers Squibb, EpiPharm, Ergonex, espeRare Foundation, GlaxoSmithKline, Inventiva, Lilly, Mitsubishi Tanabe, Medac, MedImmune, Pfizer, Pharmacyclics, Serodapharm, and Sinoxa; and has received research funding from Actelion, Bayer, Boehringer Ingelheim, Ergonex, Pfizer, and Sanofi. O.D. has a patent for mir-29 for the treatment of systemic sclerosis.