Patient reported outcome for physical function in idiopathic inflammatory myopathy.

OBJECTIVES: There is an unmet need to develop patient-reported outcomes (PRO) measures for Idiopathic Inflammatory Myopathies (IIM). To investigate the feasibility, compliance, and psychometric properties of NIH's Patient-Reported Outcomes Measurement Information System (PROMIS) physical function-20 (PF-20) in a large U.S. IIM population. METHODS: "Myositis Patient Centered Tele-Research" (My PACER) is a multicentre prospective observational study of IIM patients, competitively recruited through traditional in-person clinic visits (Center-Based Cohort [CBC]), and remotely using smartphone and web-based technology (Tele-Research Cohort [TRC]). The CBC was further randomly divided (1:1 ratio) into a traditional local sub-cohort, and a remote sub-cohort. Data collected included PRO and other patient self-assessments monthly for 6 months. Clinician-reported outcomes were obtained at baseline and 6 months. RESULTS: 120 IIM patients were enrolled (82 TRC/38 CBC, mean age 55 ± 13.4, 75% females, 81% Caucasians), with similar demographics and mean PROMIS PF-20 score between cohorts. The PROMIS PF-20 score was not associated with age, sex or race. The compliance and completion rates were similar between TRC and CBC as well as sub-cohorts. PROMIS PF-20 showed strong test-retest reliability at 1 month. PROMIS PF-20 was significantly associated with all core set measures except extra-muscular global and CK, as well as with most of symptoms, function and physical activity measures. PROMIS PF-20 illustrated concordant change with myositis response criteria and patient assessment, with a large effect size. CONCLUSIONS: PROMIS PF-20 demonstrates favorable psychometric properties including reliability, validity and responsiveness in a large cohort of myositis patients, with similar adherence in local or remotely enrolled patients.

Disease characteristics and medications use in idiopathic inflammatory myopathy: a multi-center prospective observational study of decentralized remote vs. traditional clinic enrollment.

OBJECTIVES: Idiopathic Inflammatory Myopathies (IIM) are rare and characterized by heterogeneous manifestations and clinical trajectories. Utilizing tele-research methods has the potential to improve participant recruitment and advance the understanding of the disease. We aimed to evaluate disease characteristics in IIM patients throughout the U.S. and compare these parameters between patients recruited remotely through mobile application or website vs those recruited locally in myositis clinics. METHODS: "Myositis Patient Centered Tele-Research" (My PACER) is a multicentre prospective observational study of U.S. IIM subjects, competitively recruited through traditional in-person clinic visits (Center-Based Cohort [CBC]), and remotely using mobile application or website and social media (Tele-Research Cohort [TRC]). Data collection comprised baseline demographic and clinical variables, encompassing symptoms, organ involvement, diagnostic tests results and medication use. RESULTS: The study included 120 IIM patients, 82 in the TRC and 38 in the CBC. The average age was 55 ± 13.4, 75% females and 81% Caucasians. Both cohorts exhibited similar demographic characteristics. Overall, 41% dermatomyositis, 27% polymyositis, 23% anti-synthetase syndrome, and 9% necrotizing myositis patients were enrolled, with comparable subtypes prevalence among cohorts (p= 0.85). The groups demonstrated similarities in multiple clinical factors, including muscle enzymes, diagnostic delay, employment status, various patient and physician-reported outcomes, functional tests, and the frequency of abnormal findings in chest CT, pulmonary function tests, and electromyography. TRC patients received biologics and csDMARDs more frequently (p< 0.001 and p= 0.013, respectively). CONCLUSION: Tele-research recruitment yielded a patient cohort resembling traditionally recruited patients demographically and clinically, indicating its effectiveness for robust and diverse patient recruitment in clinical studies.

Heterogeneity in nomenclature and abbreviation usage for anti-synthetase syndrome: a scoping review.

Anti-synthetase syndrome constitutes a dynamically evolving subset of Idiopathic Inflammatory Myopathy, however, the nomenclature and abbreviations for this syndrome are plagued by heterogeneity, leading to lack of consistency in literature. The objective of this study is to evaluate existing diversity in disease names and abbreviations, with a future goal to develop consensus on the nomenclature. A scoping review format was used for analysis. A comprehensive PUBMED search was conducted from January 1, 1984 (the initial description of anti-synthetase autoantibodies) to November 30, 2023, encompassing all pertinent articles published within this timeframe. Search terms included, ((antisynthetase syndrome) OR (anti synthetase syndrome)) OR (anti-synthetase syndrome)). The articles were screened for presence of terminology and abbreviations used. The search yielded 936 items with the specified terms. After excluding 303 irrelevant articles and 58 non-English publications, the remaining n = 575 articles underwent detailed review of the abstract and full article. Out of n = 575, 54.7% (n = 314) used 'antisynthetase syndrome' and 43.4% (n = 249) preferred 'anti-synthetase syndrome' with few novel names also. Among these, 394 articles used abbreviations while 181 did not. Most utilized term was ASS; in 64.7% (n = 255), followed AS in 11.9% (n = 47), ASSD in 9.9% (n = 39) and ASyS in 7.6% (n = 30). A discordance in nomenclature is evident, with about half using antisynthetase syndrome and other half using anti-synthetase syndrome. Moreover, significant heterogeneity exists in abbreviation use aswell. There is a pressing need to bridge this disparity and establish a uniform identifier for the disease with an objective to develop greater coherence in future research, educational initiatives, and interdisciplinary collaboration.

Clinical trials and novel therapeutics in dermatomyositis.

INTRODUCTION: Currently, there are no proven drugs that are FDA approved for the treatment of dermatomyositis (DM), even though multiple clinical trials are ongoing to evaluate safety and efficacy of novel therapeutics in DM. The purpose of this review is to highlight the biological plausibility, existing clinical evidence as well as completed and ongoing clinical trials for various drugs in pipeline for development for use in dermatomyositis. AREAS COVERED: The drugs with the strongest evidence have been included in this review with a focus on the mechanism of their action pertaining to the disease process, clinical studies including completed and ongoing trials. With better understanding of the underlying pathophysiologic process, there are new molecular targets that have been identified that can be targeted by these novel drugs, predominantly biologic drugs. EXPERT OPINION: There are various drugs being evaluated in phase II/III clinical trials that hold promise in DM. At the forefront of these are immunoglobulin, Lenabasum, and Abatacept for which phase III clinical trials are ongoing. In addition, promising clinical studies are ongoing or reported for KZR-616, anti-B cell therapy, anti-interferon drugs, and Repository Corticotrophin Injection (RCI).

Internet-based enrollment of a myositis patient cohort-a national experience.

INTRODUCTION: Recruitment for idiopathic inflammatory myopathies (IIM) research is a challenge due to the rarity of the disease and the scarcity of specialized myositis centers. Online recruitment may be a feasible alternative to reach rare disease patients. We evaluated various online recruitment methods in a large longitudinal IIM cohort. METHODS: The "Myositis Patient Centered Tele-Research" (My Pacer) is a prospective 6-month observational study of IIM, recruited online and through traditional clinic visits. We utilized diverse recruitment methods, such as physician referrals, social media, websites, direct emails, and partnerships with patient-support organizations. Participants self-enrolled and completed pre-screening, e-consenting, and release of medical information via the study-specific app or website. We compared the effectiveness of various recruitment and enrollment methods and the characteristics of the population recruited. RESULTS: A total of 841 participants completed the pre-screening; 408 completed e-consent and registration. From those, 353 (86.5%) were remotely recruited. Email (201; 49.26%) and social media (77; 18.87%) were important recruitment tools. Patient-support organizations were responsible for disseminating the study to 312 (75.46%) participants. The study app was used by 232 (65.72%) individuals for enrollment, with app users being slightly younger than website users (p = 0.001). Participants were mostly female 317 (77.76%), mean age of 54.84 years, White 328 (80.42%), Black 49 (12%), Asian 13 (3.26%), and non-Hispanic 378 (92.65%). Our study reached all U.S. regions and 45 (90%) U.S. states. CONCLUSIONS: Social media and partnerships with patient-support organizations lead to a high rate of recruitment, with a wide reach, and a reasonably diverse population.

A Narrative Review of Acthar Gel for the Treatment of Myositis.

Idiopathic inflammatory myopathies (IIMs) are autoimmune disorders characterized by symmetric proximal muscle weakness and chronic inflammation, with an increased risk of morbidity and mortality. The current standard of care includes traditional immunosuppressive pharmacotherapies; however, some patients cannot tolerate or do not adequately respond to these therapies, highlighting the need for alternative treatments for refractory disease. Acthar® Gel (repository corticotropin injection) is a naturally sourced mixture of adrenocorticotropic hormone analogs and other pituitary peptides that has been approved by the US Food and Drug Administration since 1952 for use in patients with two subgroups of IIMs, dermatomyositis (DM) and polymyositis (PM). However, it has not been routinely used in the treatment of IIMs. While Acthar may induce steroidogenesis, it also has a steroid-independent mechanism of action by exerting immunomodulatory effects through the activation of melanocortin receptors on immune cells, such as macrophages, B cells, and T cells. Recent clinical trials, retrospective analyses, and case reports add to the growing evidence suggesting that Acthar may be effective in patients with DM and PM. Here we review the current evidence supporting the safety and efficacy of Acthar for the treatment of refractory DM and PM.

Adalimumab-induced Anti-neutrophilic Cytoplasmic Antibody Vasculitis: A Rare Complication of an Increasingly Common Treatment.

Tumor necrosis factor (TNF) inhibitors are used for treatment of different autoimmune diseases. Interestingly they are also being noted to cause autoimmune side effects such as vasculitis, systemic lupus erythematosus, interstitial lung disease, etc. We describe one such case of granuloma annulare being treated with Adalimumab, who then developed pulmonary-renal syndrome form anti-neutrophilic cytoplasmic antibody (ANCA)-induced vasculitis. This was treated with steroids and immunosuppression, as well as discontinuation of the TNF inhibitor. However, he remains dependant on dialysis and immunosuppression. In this article, we review the existing literature on clinical presentation and course of TNF inhibitors-induced ANCA vasculitis. We also discuss the underlying mechanisms thought to be responsible for this.