Recent advances steer the future of systemic sclerosis toward precision medicine

Lepri, Gemma; Hughes, Michael; Bruni, Cosimo; Cerinic, Marco Matucci; Randone, Silvia Bellando

doi:10.1007/s10067-019-04834-5

Cited by 17 publications

(8 citation statements)

References 34 publications

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“…Our future research agenda includes studies to better capture prevalence and prognostic impact including early SSc stages when SSc-pHI may be asymptomatic and subclinical, 40 as well as optimizing and tailoring use of cardiac assessments in the early diagnosis and follow-up of SSc-pHI. 41…”

Section: Discussionmentioning

confidence: 99%

Primary systemic sclerosis heart involvement: A systematic literature review and preliminary data-driven, consensus-based WSF/HFA definition

Buch¹

2021

Journal of Scleroderma and Related Disorders

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Introduction: Primary heart involvement in systemic sclerosis may cause morpho-functional and electrical cardiac abnormalities and is a common cause of death. The absence of a clear definition of primary heart involvement in systemic sclerosis limits our understanding and ability to focus on clinical research. We aimed to create an expert consensus definition for primary heart involvement in systemic sclerosis. Methods: A systematic literature review of cardiac involvement and manifestations in systemic sclerosis was conducted to inform an international and multi-disciplinary task force. In addition, the nominal group technique was used to derive a definition that was then subject to voting. A total of 16 clinical cases were evaluated to test face validity, feasibility, reliability and criterion validity of the newly created definition. Results: In total, 171 publications met eligibility criteria. Using the nominal group technique, experts added their opinion, provided statements to consider and ranked them to create the consensus definition, which received 100% agreement on face validity. A median 60(5–300) seconds was taken for the feasibility on a single case. Inter-rater agreement was moderate (mKappa (95% CI) = 0.56 (0.46–1.00) for the first round and 0.55 (0.44–1.00) for the second round) and intra-rater agreement was good (mKappa (95% CI) = 0.77 (0.47–1.00)). Criterion validity showed a 78 (73–84)% correctness versus gold standard. Conclusion: A preliminary primary heart involvement in systemic sclerosis consensus-based definition was created and partially validated, for use in future clinical research.

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Section: Discussionmentioning

confidence: 99%

Primary systemic sclerosis heart involvement: A systematic literature review and preliminary data-driven, consensus-based WSF/HFA definition

Buch¹

2021

Journal of Scleroderma and Related Disorders

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“…Although consensus and recommendations are nowadays available, these do not fully cover the different clinical scenarios, in particular regarding time to initiation and a possibly more effective treatment protocol. In this context, SSc experts still relay on their clinical experience and take into account the different abovementioned factors to guide their decision in a patient-tailored, customized treatment regimen [89], possibly informed also by molecular biomarkers [90].…”

Section: Discussionmentioning

confidence: 99%

The Treatment of Lung Involvement in Systemic Sclerosis

et al. 2021

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Systemic sclerosis (SSc) patients are often affected by interstitial lung disease (ILD) and, although there have been recent treatment advances, it remains the leading cause of death among SSc, with a 10-year mortality up to 40%. African Americans and subjects with diffuse cutaneous SSc or anti-topoisomerase 1 antibodies are most commonly affected. Currently, early ILD diagnosis can be made, and it is pivotal to improve the prognosis. The diagnostic mainstay test for SSc-ILD is high-resolution computed tomography for the morphology and pulmonary function tests for the functional aspects. Treatment planning and intensity are guided by the disease severity and risk of progression. Traditionally, therapy has depended on combinations of immunosuppressants, particularly cyclophosphamide and mycophenolate mofetil, which can be supplemented by targeted biological and antifibrotic therapies. Benefits have been observed in trials on hematopoietic autologous stem cell transplantation for patients with progressive SSc, whilst lung transplantation is reserved for refractory SSc-ILD cases. Herein, recent advances in SSc-ILD treatment will be explored.

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“…Furthermore, SSc is a very complex and heterogeneous disease, and the traditional classification of the patients into the limited or diffuse form based on the severity of skin involvement is an oversimplification. Lately, multiple studies have utilized intrinsic gene expression analyses to classify SSc patients into four categories: the inflammatory, fibroproliferative, limited, and normal-like subsets [ 206 ]. It is currently not certain if these categories are reflective of stable disease states that differ between patients.…”

Section: Conclusion and Future Perspectivesmentioning

confidence: 99%

Therapeutic Options for Systemic Sclerosis: Current and Future Perspectives in Tackling Immune-Mediated Fibrosis

et al. 2022

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Systemic sclerosis (SSc) is a severe auto-immune, rheumatic disease, characterized by excessive fibrosis of the skin and visceral organs. SSc is accompanied by high morbidity and mortality rates, and unfortunately, few disease-modifying therapies are currently available. Inflammation, vasculopathy, and fibrosis are the key hallmarks of SSc pathology. In this narrative review, we examine the relationship between inflammation and fibrosis and provide an overview of the efficacy of current and novel treatment options in diminishing SSc-related fibrosis based on selected clinical trials. To do this, we first discuss inflammatory pathways of both the innate and acquired immune systems that are associated with SSc pathophysiology. Secondly, we review evidence supporting the use of first-line therapies in SSc patients. In addition, T cell-, B cell-, and cytokine-specific treatments that have been utilized in SSc are explored. Finally, the potential effectiveness of tyrosine kinase inhibitors and other novel therapeutic approaches in reducing fibrosis is highlighted.

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Recent advances steer the future of systemic sclerosis toward precision medicine

Cited by 17 publications

References 34 publications

Primary systemic sclerosis heart involvement: A systematic literature review and preliminary data-driven, consensus-based WSF/HFA definition

Primary systemic sclerosis heart involvement: A systematic literature review and preliminary data-driven, consensus-based WSF/HFA definition

The Treatment of Lung Involvement in Systemic Sclerosis

Therapeutic Options for Systemic Sclerosis: Current and Future Perspectives in Tackling Immune-Mediated Fibrosis

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