In September 2018, the U.S. Food & Drug Administration (FDA) granted fast-track status to FCX‑013, a gene therapy product developed to treat moderate to severe localized scleroderma (morphea). Previously, the treatment received an orphan drug designation for localized scleroderma, as well as a rare pediatric disease designation. Phase 1 and 2 studies will assess safety…
Although the true cause of systemic sclerosis (SSc), or scleroderma, remains unknown, researchers have made progress in detecting the autoimmune disease’s early presence. Beyond the physiological signs of Raynaud’s phenomenon, a capillaroscopy can detect alterations in microcirculation and lab tests can confirm the presence of telltale autoantibodies, such as anti-topoisomerase 1, anti-centromere and anti-RNA polymerase…
Historically, the early approach for treating interstitial lung disease (ILD) due to systemic sclerosis (SSc) involved immunosuppressant therapy, primarily with cytotoxic agents.1 Glucocorticoids in combination with another immunosuppressant agent, such as oral azathioprine or cyclophosphamide, were often used to treat patients with severe, progressive SSc-ILD.2 However, direct evidence to support this therapeutic approach was lacking…
SNOWMASS VILLAGE, COLO.—Data from three recent trials in systemic sclerosis (scleroderma) provide information on a number of important issues related to screening and treatment. First presented at the 2018 ACR/ARHP Annual Meeting, the phase 2/3 trials assessed the safety and efficacy of targeted agents to treat patients with systemic sclerosis.1-3 In a follow-up presentation at…
A new study from Johns Hopkins University School of Medicine, published in the September 2018 issue of Arthritis Care & Research, identifies risk factors and clinical features associated with severe gastrointestinal (GI) dysmotility in patients with systemic sclerosis.1 The findings suggest a distinct pathological process may be at work in this patient group, says lead…
What happens when systemic sclerosis (SSc) overlaps with other systemic autoimmune rheumatic diseases? Patients with either diffuse cutaneous or limited cutaneous SSc sometimes develop systemic lupus erythematosus (SLE) as well. A new, large cohort study published in the Journal of Rheumatology reveals details on the epidemiology, clinical signs and survival data of SSc-SLE overlap syndrome.1…
Eosinophilic fasciitis generally presents with the acute onset of edema followed by progressive skin induration in the setting of hypergammaglobulinemia, an elevated erythrocyte sedimentation rate (ESR) and peripheral eosinophilia in 63–93% of patients.1,2 Skin involvement is typically limited to 20.1% of total body surface area and most commonly involves the extremities symmetrically.1,3 The condition was…
CHICAGO—Can non-drug interventions improve the lives of patients with scleroderma? Janet L. Poole, PhD, OTR/L, professor and director of the Occupational Therapy Graduate Program of the School of Medicine, University of New Mexico, Albuquerque, addressed this question when she presented the ARHP Distinguished Lecture at the 2018 ACR/ARHP Annual Meeting. She discussed the challenges faced…
Recent studies point to a relationship between scleroderma and autoantibodies (e.g., anti-RNA polymerase III or anti-RNPC3), and an increased cancer risk within a short interval of scleroderma onset. Mechanistic studies provide further evidence that cancer may trigger scleroderma in patients with these autoantibodies. However, many questions remain unanswered. A study in the Annals of the…
“You have systemic sclerosis,” says the physician. Other somber words patients may hear the day they learn their diagnosis include, “rare, chronic, no treatment.” But a ray of hope could soon enter those exam rooms thanks to research conducted by a team from the UK. Rizgar A. Mageed, PhD, FRCP, FRCPath, is professor of experimental…