Patient-Reported Outcome Measures in Systemic Sclerosis (Scleroderma)

https://doi.org/10.1016/j.rdc.2016.01.003Get rights and content

Section snippets

Key points

  • 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.

  • 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

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

First page preview

First page preview
Click to open first page preview

References (96)

  • S.R. Johnson et al.

    Quality of life and functional status in systemic sclerosis compared to other rheumatic diseases

    J Rheumatol

    (2006)
  • M.D. Mayes et al.

    Prevalence, incidence, survival, and disease characteristics of systemic sclerosis in a large US population

    Arthritis Rheum

    (2003)
  • E.C. Leroy et al.

    Scleroderma (systemic sclerosis): classification, subsets and pathogenesis

    J Rheumatol

    (1988)
  • J. Seibold

    Connective tissue diseases characterized by fibrosis

  • F. Wigley et al.

    Clinical features

  • D.L. Paquette et al.

    Cutaneous concerns of scleroderma patients

    J Dermatol

    (2003)
  • A.E. Smyth et al.

    A cross-sectional comparison of three self-reported functional indices in scleroderma

    Rheumatology (Oxford)

    (2003)
  • J.M. Ouimet et al.

    Work disability in scleroderma is greater than in rheumatoid arthritis and is predicted by high HAQ scores

    Open Rheumatol J

    (2008)
  • M. Hudson et al.

    Quality of life in patients with systemic sclerosis compared to the general population and patients with other chronic conditions

    J Rheumatol

    (2009)
  • S. Sekhon et al.

    The minimally important difference (MID) for patient centered outcomes including Health Assessment Questionnaire (HAQ), Fatigue, Pain, Sleep, Global VAS and SF-36 in Scleroderma (SSc)

    J Rheumatol

    (2010)
  • O. Schieir et al.

    Prevalence, severity, and clinical correlates of pain in patients with systemic sclerosis

    Arthritis Care Res (Hoboken)

    (2010)
  • G. El-Baalbaki et al.

    Association of pruritus with quality of life and disability in systemic sclerosis

    Arthritis Care Res (Hoboken)

    (2010)
  • M. Bassel et al.

    Frequency and impact of symptoms experienced by patients with systemic sclerosis: results from a Canadian National Survey

    Rheumatology (Oxford)

    (2010)
  • B.D. Thombs et al.

    Prevalence and clinical correlates of symptoms of depression in patients with systemic sclerosis

    Arthritis Rheum

    (2008)
  • L.R. Jewett et al.

    Sociodemographic and disease correlates of body image distress among patients with systemic sclerosis

    PLoS One

    (2012)
  • R. Knafo et al.

    (Not) talking about sex: a systematic comparison of sexual impairment in women with systemic sclerosis and other chronic disease samples

    Rheumatology (Oxford)

    (2009)
  • M. Hudson et al.

    Work disability in systemic sclerosis

    J Rheumatol

    (2009)
  • V.L. Malcarne et al.

    Psychosocial aspects of systemic sclerosis

    Curr Opin Rheumatol

    (2013)
  • B.D. Thombs et al.

    Psychological health and well-being in systemic sclerosis: State of the science and consensus research agenda

    Arthritis Care Res (Hoboken)

    (2010)
  • J. Pope

    Measures of systemic sclerosis (Scleroderma)

    Arthritis Care Res

    (2011)
  • M.E. Suarez-Almazor et al.

    Disease and symptom burden in systemic sclerosis: a patient perspective

    J Rheumatol

    (2007)
  • J.F. Fries et al.

    Measurement of patient outcome in arthritis

    Arthritis Rheum

    (1980)
  • S.R. Johnson et al.

    The health assessment questionnaire disability index and scleroderma health assessment questionnaire in scleroderma trials: an evaluation of their measurement properties

    Arthritis Rheum

    (2005)
  • P.A. Merkel et al.

    Measuring disease activity and functional status in patients with scleroderma and Raynaud’s phenomenon

    Arthritis Rheum

    (2002)
  • P.J. Clements et al.

    The disability index of the health assessment questionnaire is a predictor and correlate of outcome in the high-dose versus low-dose penicillamine in systemic sclerosis trial

    Arthritis Rheum

    (2001)
  • V.D. Steen et al.

    The value of the Health Assessment Questionnaire and special patient-generated scales to demonstrate change in systemic sclerosis patients over time

    Arthritis Rheum

    (1997)
  • D. Khanna et al.

    Does incorporation of aids and devices make a difference in the score of the health assessment questionnaire-disability index? Analysis from a scleroderma clinical trial

    J Rheumatol

    (2008)
  • M. Hudson et al.

    Discordance between patient and physician assessments of disease severity in systemic sclerosis

    J Rheumatol

    (2010)
  • P.J. Clements et al.

    Skin thickness score as a predictor and correlate of outcome in systemic sclerosis: high-dose versus low-dose penicillamine trial

    Arthritis Rheum

    (2000)
  • N. Sultan et al.

    The Health Assessment Questionnaire (HAQ) is strongly predictive of good outcome in early diffuse scleroderma: results from an analysis of two randomized controlled trials in early diffuse scleroderma

    Rheumatology (Oxford)

    (2004)
  • D. Khanna et al.

    Responsiveness of the SF-36 and the Health Assessment Questionnaire disability index in a systemic sclerosis clinical trial

    J Rheumatol

    (2005)
  • The EuroQol Group

    EuroQol-a new facility for the measurement of health-related quality of life

    Health Policy

    (1990)
  • R. Rabin et al.

    EQ-5D: a measure of health status from the EuroQol Group

    Ann Med

    (2001)
  • R. Gualtierotti et al.

    Health related quality of life assessment in patients with systemic sclerosis

    Reumatismo

    (2010)
  • H. Muller et al.

    Determinants of disability, quality of life and depression in dermatological patients with systemic scleroderma

    Br J Dermatol

    (2011)
  • G. Strickland et al.

    Predictors of health-related quality of life and fatigue in systemic sclerosis: evaluation of the EuroQol-5D and FACIT-F assessment tools

    Clin Rheumatol

    (2012)
  • L. Kwakkenbos et al.

    A comparison of the measurement properties and estimation of minimal important differences of the EQ-5D and SF-6D utility measures in patients with systemic sclerosis

    Clin Exp Rheumatol

    (2013)
  • Cited by (12)

    • Systemic sclerosis: Advances towards stratified medicine

      2020, Best Practice and Research: Clinical Rheumatology
      Citation 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 Trauma
      Citation 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 Rheumatology
      Citation 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].

    View all citing articles on Scopus

    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.

    View full text