A comprehensive framework for navigating patient care in systemic sclerosis: A global response to the need for improving the practice of diagnostic and preventive strategies in SSc

Saketkoo, Lesley Ann; Frech, Tracy; Varjú, Cecília; Domsic, Robyn T.; Farrell, Jessica; Gordon, Jessica; Mihai, Carina; Sandorfi, Nora; Shapiro, Lee; Poole, Janet L.; Volkmann, Elizabeth R.; Lammi, Monika; McAnally, Kendra J.; Alexanderson, Helene; Pettersson, Henrik; Hant, Faye N.; Kuwana, Masataka; Shah, Ami A.; Smith, Vanessa; Hsu, Vivien; Kowal‐Bielecka, Otylia; Assassi, Shervin; Cutolo, Maurizio; Kayser, Cristiane; Shanmugam, Victoria K.; Vonk, Madelon C.; Fligelstone, Kim; Baldwin, Nancy; Connolly, Kerri; Ronnow, Anneliese; Toth, B Garay; Suave, Maureen; Farrington, Sue; Bernstein, Elana J.; Crofford, Leslie J.; Czirják, László; Jensen, Kelly; Hinchclif, Monique; Hudson, Marie; Lammi, Matthew R.; Mansour, Jennifer; Morgan, Nadia D.; Mendoza, Fabian A.; Nikpour, Mandana; Pauling, John D; Riemekasten, Gabriela; Russell, Anne-Marie; Scholand, Mary Beth; Seigart, Elise; Rodríguez‐Reyna, Tatiana Sofía; Hummers, Laura K.; Walker, Ulrich A.; Steen, Virginia D.

doi:10.1016/j.berh.2021.101707

Cited by 28 publications

(21 citation statements)

References 96 publications

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“…Cardiopulmonary rehabilitation is feasible, safe, and effective regardless of severity of ILD [17 ▪ ,28 ▪ ,32 ▪ ,33–35,61,62,63 ▪ ]; and is the safest way for people who are deconditioned and limited by dyspnea to initiate and learn how to exercise and safely intensify exercise over time. Cardiopulmonary rehabilitation also provides key education on symptom reduction and self-management that improves capacity, exercise tolerance, mobility breathing, fatigue, and cough [32 ▪ ,33–35,64,65].…”

Section: Physical Activity and Exercise Across The Lifespanmentioning

confidence: 99%

Ageing with Interstitial lung disease: preserving health and well being

Saketkoo

Ndili

Patterson

et al. 2022

Current Opinion in Pulmonary Medicine

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Purpose of reviewAgeing, the accrual of molecular and cellular damage over a lifetime confers progressive physiologic dysfunction of bodily systems, leaving the body in a heightened state of vulnerability to biophysical and psychosocial stressors. The inflection point is frailty which easily leads to disability and death. Interstitial lung disease (ILD) creates biophysical and psychosocial stresses difficult for even optimally fit patients to cope with. With evolving ILD treatment pathways, people with ILD are living longer. Recent findingsILD and ageing are bi-directionally influential: ILD, its treatments, complications, and collateral systemic extra-pulmonary damage (hypoxic and oxidative stress) wear on the ageing person and ageing impacts a person's tolerance of ILD. ILD extent may proportionally accelerate age-related vulnerabilities. ILD related to inflammatory systemic diseases, e.g. connective tissue diseases or sarcoidosis, exert an even more complex biophysical impact on the body.

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Section: Physical Activity and Exercise Across The Lifespanmentioning

confidence: 99%

Ageing with Interstitial lung disease: preserving health and well being

Saketkoo

Ndili

Patterson

et al. 2022

Current Opinion in Pulmonary Medicine

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“…(28). Exercise and physical training are areas that are gaining momentum as systemic nonpharmacological treatment in inflammatory diseases and should be leveraged more frequently in sarcoidosis (65)(66)(67)(68)(69). Physical training/exercise is arguably both a systemic treatment that can modulate inflammation and a nonpharmacological therapy that cultivates physical capacity through amplification of neuromuscular and vascular networks and other bio-mechanical pathways that reduce symptom burden regardless of sarcoidosis disease activity status (67,(69)(70)(71).…”

Section: Non-pharmacological Therapies and Palliation Of Symptomsmentioning

confidence: 99%

Sarcoidosis: Updates on therapeutic drug trials and novel treatment approaches

et al. 2022

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Sarcoidosis is a systemic granulomatous inflammatory disease of unknown etiology. It affects the lungs in over 90% of patients yet extra-pulmonary and multi-organ involvement is common. Spontaneous remission of disease occurs commonly, nonetheless, over 50% of patients will require treatment and up to 30% of patients will develop a chronic progressive non-remitting disease with marked pulmonary fibrosis leading to significant morbidity and death. Guidelines outlining an immunosuppressive treatment approach to sarcoidosis were recently published, however, the strength of evidence behind many of the guideline recommended drugs is weak. None of the drugs currently used for the treatment of sarcoidosis have been rigorously studied and prescription of these drugs is often based on off-label” indications informed by experience with other diseases. Indeed, only two medications [prednisone and repository corticotropin (RCI) injection] currently used in the treatment of sarcoidosis are approved by the United States Food and Drug Administration. This situation results in significant reimbursement challenges especially for the more advanced (and often more effective) drugs that are favored for severe and refractory forms of disease causing an over-reliance on corticosteroids known to be associated with significant dose and duration dependent toxicities. This past decade has seen a renewed interest in developing new drugs and exploring novel therapeutic pathways for the treatment of sarcoidosis. Several of these trials are active randomized controlled trials (RCTs) designed to recruit relatively large numbers of patients with a goal to determine the safety, efficacy, and tolerability of these new molecules and therapeutic approaches. While it is an exciting time, it is also necessary to exercise caution. Resources including research dollars and most importantly, patient populations available for trials are limited and thus necessitate that several of the challenges facing drug trials and drug development in sarcoidosis are addressed. This will ensure that currently available resources are judiciously utilized. Our paper reviews the ongoing and anticipated drug trials in sarcoidosis and addresses the challenges facing these and future trials. We also review several recently completed trials and draw lessons that should be applied in future.

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“…Systemic sclerosis (SSc) is a chronic heterogeneous systemic autoimmune disease characterized by microvascular dysfunction, activation of the immune system, and cutaneous and visceral fibrosis. SSc has the highest morbidity and mortality rates among immune-mediated rheumatic diseases ( 1 ). However, the clinical manifestations and the clinical course of the disease are highly variable.…”

Section: Introductionmentioning

confidence: 99%

A cell-based assay for detection of anti-fibrillarin autoantibodies with performance equivalent to immunoprecipitation

Keppeke

Satoh²,

Kayser³

et al. 2022

Front. Immunol.

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Anti-fibrillarin autoantibodies are useful for the diagnosis and prognosis of systemic sclerosis (SSc). Anti-fibrillarin produces a clumpy nucleolar pattern in indirect immunofluorescence assay on HEp-2 cells (HEp-2 IFA). Here we develop and validate a reliable cell-based anti-fibrillarin assay (Fibrillarin/CBA) for use in clinical diagnostic laboratories. A TransMembrane Signal was fused to the human fibrillarin gene (TMS-fibrillarin). HEp-2 cells overexpressing transgenic TMS-fibrillarin at the cytoplasmic membrane were used as IFA substrate in the Fibrillarin/CBA. Sixty-two serum samples with nucleolar pattern in the HEp-2 IFA (41 clumpy; 21 homogeneous/punctate) were tested for anti-fibrillarin using Fibrillarin/CBA, immunoprecipitation (IP), line-blot and ELISA. In addition, samples from 106 SSc-patients were evaluated with Fibrillarin/CBA and the results were correlated with disease phenotypes. Thirty-eight of 41 samples with the clumpy nucleolar pattern (92.7%) were positive in the Fibrillarin/CBA, while all 21 samples with other nucleolar patterns were negative. Fibrillarin/CBA results agreed 100% with IP results. Among the 38 Fibrillarin/CBA-positive samples, only 15 (39.5%) and 11 (29%) were positive for anti-fibrillarin in line-blot and ELISA, respectively. Higher frequency of diffuse cutaneous SSc (dcSSc) phenotype (72.7% vs 36.8%; p=0.022), cardiac involvement (36.4% vs 6.5%; p=0.001) and scleroderma renal crisis (18.2% vs 3.3% p = 0.028) was observed in SSc patients with positive compared to negative Fibrillarin/CBA result. Performance of Fibrillarin/CBA in the detection of anti-fibrillarin autoantibodies was comparable to the gold standard IP. Positive Fibrillarin/CBA results correlated with disease phenotypes known to be associated with anti-fibrillarin autoantibodies, underscoring the clinical validation of this novel assay.

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A comprehensive framework for navigating patient care in systemic sclerosis: A global response to the need for improving the practice of diagnostic and preventive strategies in SSc

Cited by 28 publications

References 96 publications

Ageing with Interstitial lung disease: preserving health and well being

Ageing with Interstitial lung disease: preserving health and well being

Sarcoidosis: Updates on therapeutic drug trials and novel treatment approaches

A cell-based assay for detection of anti-fibrillarin autoantibodies with performance equivalent to immunoprecipitation

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