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Systemic sclerosis (scleroderma) is a rare, chronic, connective tissue disease with fibrosis of the skin and many organs, vascular damage, and production of autoantibodies leading to many heterogeneous signs and symptoms.
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Systemic sclerosis is one of the most severe connective tissue diseases with disability; altered appearance; organ damage of skin, gastrointestinal tract, lungs, pulmonary arteries, kidneys, and other organs; digital ulcers; and amputation, with significant emotional and social
Patient-Reported Outcome Measures in Systemic Sclerosis (Scleroderma)
Section snippets
Key points
Key elements of patient-reported outcome measurements in Systemic sclerosis
Performing a physical examination to assess skin disease or obtaining blood work to measure hemoglobin, renal function, and pulmonary function are well established and relatively easy ways to measure some aspects of SSc. However, it is not as simple to capture the patient experience. PROMs are tools that attempt to address this issue. The development and validation of these patient-centered instruments requires a rigorous process.
SSc is heterogeneous and can involve nearly every organ system.
Summary
SSc is a chronic, debilitating multifaceted disease. PROs in SSc are used in both clinical practice and research to measure the patient’s experience. The HAQ-DI has the patient rate function and is validated in many rheumatic diseases. More specific measures of HRQoL in SSc include the SHAQ, SBI, and UKFS. These tools offer the patient the opportunity to express their experience with disease, beyond the objective measures that can be collected by a physician. There are also many
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Cited by (12)
Characterization of adult patients with systemic sclerosis in a reference center from northwestern Colombia: a descriptive survey
2020, Revista Colombiana de ReumatologiaSystemic sclerosis: Advances towards stratified medicine
2020, Best Practice and Research: Clinical RheumatologyCitation Excerpt :In addition to providing the patient's perspective, PROMs tend to be easily administered electronically and provide an opportunity to engage patients and improve retention rates in trials [88]. PROMs for HRQoL, Raynaud's phenomenon, skin thickening, GI symptoms and dyspnoea have been partially validated in SSc patients (Table 3) [89,90]; however, the use of these tools varies among clinical trials making the results difficult to analyse across multiple studies. PROMs for other domains, such as SSc-PAH and cardiac disease, are yet to be developed and validated.
Factors associated with early postoperative survey completion in orthopaedic surgery patients
2020, Journal of Clinical Orthopaedics and TraumaCitation Excerpt :Better postoperative satisfaction has been seen in patients with higher preoperative knee function scores,13 which may corroborate our findings of a higher mean IKDC score in the group that completed their 2-week surveys. Although the validity of some patient-reported outcome measurements has been questioned,3 many tools, specifically PROMIS, have been shown to be highly reliable14 and are being studied in many other aspects of medicine as well.15–19 Our study is not without limitations.
Update on the epidemiology, risk factors, and disease outcomes of systemic sclerosis
2018, Best Practice and Research: Clinical RheumatologyCitation Excerpt :It includes 34 items and seven multi-item scales (reflux, distension/bloating, diarrhea, fecal soilage, constipation, emotional well-being, and social functioning) and a total GIT score to assess health-related quality of life (HRQoL) and severity of GIT symptoms [43,94,96]. The UCLA SCTC GIT 2.0 takes few minutes to complete, and it is reliable and valid [43,94,96]. The skin self-assessment is a partially validated measure of skin thickening [94].
The association between hand disease severity and fatigue in individuals with systemic sclerosis: a scoping review
2022, Disability and RehabilitationValidity and reliability of the Mouth Handicap of Systemic Sclerosis questionnaire in a UK population
2020, Journal of Oral Pathology and Medicine
Disclosure Statement: J.E. Pope consults and has performed research trials by Actelion, BMS, Celgene, Genentech, Pfizer, Roche. R.E. Pellar and T.M. Tingey have nothing to disclose.