Patient perception of disease burden in diffuse cutaneous systemic sclerosis

Abstract

Purpose: 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. Methods: 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. Results: 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. Conclusion: 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.

Development and validation of a patient-reported outcome measure for systemic sclerosis: the EULAR Systemic Sclerosis Impact of Disease (ScleroID) questionnaire

“…We have developed and validated the ScleroID questionnaire as a global measurement tool to assess the disease burden in SSc patients. The questionnaire is simple and easy to apply, has high internal consistency and shows good correlation symptoms or fatigue were also associated with a high self-reported disability and high disease burden in other reports from the literature [5,34].…”

Room for improvement in non-pharmacological systemic sclerosis care? — a cross-sectional online survey of 650 patients

“…Well-coordinated and integrated care is considered as one of the eight important indicators of quality and safety, from the patient perspective [30]. It is not easy to offer SSc patients appropriate and wellcoordinated care due to the complexity of the disease, the variability of the disease course, and the limited evidencesupported pharmacological and non-pharmacological treatment options [10,12,32]. However, poor communication and coordination, can create additional barriers to care access.…”

“…In recent years, an increased understanding of the disease and targeted research activities have led to an improved classification and a growing number of pharmacological treatment options for specific complications. Much effort has been made to identify the patients' perspective on their disease, quality of life and potential therapeutic targets [10][11][12]. Owing to the direct impact of the disease on daily functioning and psychosocial well-being of patients, nonpharmacological care is a key element of SSc care.…”

Consensus‐based recommendations on communication and education regarding primary care physical therapy for patients with systemic sclerosis

“…Studies have, therefore, recommended to increase awareness of non‐pharmacological support, evidence‐based guidelines for non‐pharmacological care, and education for health professionals to optimise care (Smith et al., 2018; Spierings et al., 2019). Probably due to the rarity, complexity and heterogeneity of SSc, and the limited evidence of pharmacological and non‐pharmacological treatment options (Becetti et al., 2019; Khanna et al., 2020; Nakayama et al., 2016), this is not an easy task and even more precision is needed due to the heterogeneous nature of the disease. Indeed, patients present with a variety of reasons to PT and essential contents of PT in SSc vary greatly between patients (Liem et al., 2021a).…”

“…Probably due to the rarity, complexity and heterogeneity of SSc, and the limited evidence of pharmacological and non-pharmacological treatment options (Becetti et al, 2019;Khanna et al, 2020;Nakayama et al, 2016), this is not an easy task and even more precision is needed due to the heterogeneous nature of the disease.…”