Functional disability and its predictors in systemic sclerosis: a study from the DeSScipher project within the EUSTAR group

Jaeger, Veronika K.; Distler, Oliver; Maurer, Britta; Czirják, László; Lóránd, Veronika; Valentini, Gabriele; Vettori, Serena; Galdo, Francesco Del; Abignano, Giuseppina; Denton, Christopher P.; Nihtyanova, Svetlana I.; Allanore, Yannick; Avouac, Jérôme; Riemekasten, G.; Siegert, Elise; Huscher, Dörte; Matucci‐Cerinic, Marco; Guiducci, Serena; Frerix, Marc; Tarner, Ingo H.; Toth, B Garay; Fankhauser, Beat; Umbricht, Jörg; Zakharova, Anastasia; Mihai, Carina; Cozzi, Franco; Yavuz, Şule; Hunzelmann, Nicolas; Rednic, Simona; Vacca, Alessandra; Schmeiser, Tim; Riccieri, Valeria; Lefebvre, Paloma García de la Peña; Gabrielli, Armando; Krummel-Lorenz, Brigitte; Martinović, Duška; Ancuta, C.; Smith, Vanessa; Müller‐Ladner, Ulf; Walker, Ulrich A.

doi:10.1093/rheumatology/kex182

Cited by 65 publications

(69 citation statements)

References 28 publications

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“…It has been shown that multiple factors contribute to the potentially severe disease-related manifestations and the hindrance of patients' functional capacity (37,38). Together with an extended fibrosis of the skin, joint contractures, sarcopenia, gastrointestinal and cardiopulmonary involvement, the presence of Raynaud's phenomenon and DUs have an important impact on daily life activities and work-related endeavors (8,38).…”

Section: Discussionmentioning

confidence: 99%

“…The patient-centered approach to disability in scleroderma leads to the conclusion that SSc-related dyspnea often constitutes one of the main factors involved in the decrement of life quality as well as disability (47)(48)(49). According to recent data from the DeSScipher project within the EUSTAR (European Scleroderma Trials and Research) group, dyspnea and the presence of DUs emerge as major determinants of functional impairment in SSc patients (8). Other studies have reported similar findings regarding the importance of dyspnea in SSc-related quality of life (50,51).…”

Section: Discussionmentioning

confidence: 99%

“…Furthermore, studies proved that considerable loss in work ability occurs as early as 3 years from the onset of SSc (7). Interestingly, the patient-centered approach to the burden of symptoms often provides different insights compared to the physicians' view on the severity of the disease (8,9).…”

Section: Introductionmentioning

confidence: 99%

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A patient‑centered approach to the burden of symptoms in patients with scleroderma treated with Bosentan: A prospective single‑center observational study

et al. 2019

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Systemic sclerosis (SSc) is a rare and complex autoimmune disease associated with poor vital and functional outcomes. The functional hindrance in patients derives from various disease-specific manifestations, including Raynaud's phenomenon and digital ulcers (DUs). Bosentan is an endothelin receptor antagonist capable of preventing the appearance of new DUs in patients with scleroderma. The present study aimed to evaluate the effects of Bosentan on the severity of Raynaud's phenomenon, DU-related symptoms and functional impairment during the first year of treatment. A prospective study that included adult patients with SSc admitted to the Rheumatology Department between January 2016 and January 2017 that were candidates for Bosentan therapy, was performed. All patients were asked to evaluate the burden of symptoms secondary to Raynaud's and DUs using a visual analogue scale (VAS), whereas functional hindrance was assessed via Health Assessment Questionnaire Disability Index (HAQ-DI). The outcomes were assessed at baseline and every 3 months during 1 year of therapy. Among the 41 patients included initially, 2 participants discontinued the treatment after 1 month due to adverse events (elevation of liver enzymes). The study cohort exhibited a significant improvement in HAQ-DI, VAS-R and VAS-DU scores in response to Bosentan therapy over the 1-year follow-up period. Higher scores at baseline predicted a weaker treatment-related improvement, with the risk of a poor outcome being increased by 220% for VAS-R, 116% for VAS-DU, whereas no increase was observed for HAQ-DI. The post-treatment improvement in VAS-DU levels was associated with a better outcome for HAQ-DI (R=0.44; P=0.005). This association was not identified for VAS-R (R=0.24; P=0.137). Throughout the follow-up period, patients with dyspnea presented with significantly higher HAQ-DI scores compared with non-dyspneic patients. Bosentan therapy may indirectly influence functionality and quality of life in patients with scleroderma by reducing the burden of Raynaud's and DU-related symptoms. Nonetheless, patients with SSc with a decreased symptom burden at baseline exhibited improved outcomes.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

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A patient‑centered approach to the burden of symptoms in patients with scleroderma treated with Bosentan: A prospective single‑center observational study

et al. 2019

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“…Such changes influence symptoms such as dyspnea, which has been found to be a major factor contributing to their disability. [33] Another key feature of the PAH-CTD is the advancing age (PAH-CTD: 55 ± 13 vs IPAH: 46 ± 15 yr) and reduced exercise capacity (V O2: PAH-CTD: 0.82 ± 0.25 and 0.87 ± 0.30 vs IPAH: 1.10 ± 0.41 l.min -1 ) during the six minute walk test. [34,35]…”

Section: Central Factors (Cardiovascular and Pulmonary)mentioning

confidence: 99%

Exercise-based evaluations and interventions for pulmonary hypertension with connective tissue disorders

Babu

Morris

Arena

et al. 2018

Expert Review of Respiratory Medicine

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Exercise intolerance is a common and often significant limitation in pulmonary arterial hypertension (PAH). This intolerance greatly affects the quality of life and function of the individual with PAH, irrespective of its etiology. In PAH associated with connective tissue disorders (PAH-CTD), exercise intolerance is further amplified by the presence of coexisting musculoskeletal manifestations of CTD. The evaluation of exercise capacity and prescription for exercise training therefore becomes a challenge to the clinician. Areas covered: This review highlights factors contributing to exercise intolerance in PAH-CTD, evaluation methods of exercise capacity and an overview on exercise training and a roadmap for future research. Expert commentary: Exercise intolerance is a complex interplay of cardiovascular, pulmonary, and musculoskeletal systems. Data from cardiopulmonary exercise tests have shown predictive abilities for both diagnosis and prognosis. In its absence, the 6-min walk test can be used to provide similar information thereby making the role of exercise testing an invaluable evaluation method in PAH-CTD. Exercise training data in PAH-CTD are still sparse, though, data from PAH studies suggest potential benefit. However, more research is required in this area of testing and training for greater understanding on exercise hemodynamic, phenotypes, and training benefits.

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“…A survey of 50 patients with RA-ILD at a US center found that the severity of fatigue and dyspnea were the strongest predictors of physical health impairment, while the severity of cough, fatigue, and dyspnea were the strongest predictors of mental health impairment [28]. Dyspnea has been reported by patients with SSc as one of the main factors driving their functional disability [29].…”

Section: Introductionmentioning

confidence: 99%

Progressive fibrosing interstitial lung disease associated with systemic autoimmune diseases

Fischer

Distler

2019

Clin Rheumatol

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Interstitial lung disease (ILD) is a common manifestation of systemic autoimmune diseases and a leading cause of death in these patients. A proportion of patients with autoimmune ILDs develop a progressive fibrosing form of ILD, characterized by increasing fibrosis on high-resolution computed tomography, worsening of lung function, and early mortality. Autoimmune disease-related ILDs have a variable clinical course and not all patients will require treatment, but all patients should be monitored for signs of progression. Apart from systemic sclerosis-associated ILD, the limited evidence to support the efficacy of immunosuppression as a treatment for ILDs is based mainly on small retrospective series and expert opinion. Non-clinical data suggest that there are commonalities in the mechanisms that drive progressive fibrosis in ILDs with an immunological trigger as in other forms of progressive fibrosing ILD. This suggests that nintedanib and pirfenidone, drugs known to slow disease progression in patients with idiopathic pulmonary fibrosis, may also slow the progression of ILD associated with systemic autoimmune diseases. In the SENSCIS® trial, nintedanib reduced the rate of ILD progression in patients with systemic sclerosis-associated ILD. The results of other large clinical trials will provide further insights into the role of anti-fibrotic therapies in the treatment of autoimmune disease-related ILDs.

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Functional disability and its predictors in systemic sclerosis: a study from the DeSScipher project within the EUSTAR group

Abstract: SSc patients perceive dyspnoea, pain, digital ulcers, muscle weakness and gastrointestinal symptoms as the main factors driving their level of disability, unlike physicians who emphasize objective measures of disability.

Cited by 65 publications

References 28 publications

A patient‑centered approach to the burden of symptoms in patients with scleroderma treated with Bosentan: A prospective single‑center observational study

A patient‑centered approach to the burden of symptoms in patients with scleroderma treated with Bosentan: A prospective single‑center observational study

Exercise-based evaluations and interventions for pulmonary hypertension with connective tissue disorders

Progressive fibrosing interstitial lung disease associated with systemic autoimmune diseases

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