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…
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…
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…
A yearlong pilot study that evaluated the safety and efficacy of belimumab in a small group of patients with early diffuse systemic sclerosis found no significant difference in the number of adverse events between those treated with the drug and those who received a placebo. Currently, no drugs are approved specifically for the treatment of…
A 58-year-old African American woman with a past medical history of hypertension (HTN), hyperlipidemia, severe gastroesophageal reflux disease (GERD) and limited cutaneous systemic sclerosis (lcSSc) presented to the emergency department with shortness of breath (SOB) and progressive bilateral lower extremity swelling for three weeks. She denied any chest pain, but endorsed generalized fatigue and dyspnea…
A recently published study gives new insight into an innovative approach to treating patients with severe scleroderma and internal organ involvement. During the six-year study, researchers demonstrated the durability of the beneficial effects of stem cell transplantation in this patient population. Their data also suggest the treatment has the potential to renormalize the autoreactive immune system…
The year was 1991. It was my first Tuesday as a rheumatology fellow at the University of Pittsburgh’s Presbyterian Hospital. Navigating a maze of buildings and hallways, I delivered myself to the entrance to the scleroderma clinic. Running late and not knowing whether there was a separate entrance for staff, I clicked open the door….
SAN DIEGO—Fibrosis affects all organ systems, but isn’t always systemic sclerosis. Experts on less common forms discussed patient presentations, diagnosis and treatment at the 2017 ACR/ARHP Annual Meeting in San Diego on Nov. 6. Retroperitoneal Fibrosis Formerly called Ormond’s disease, retroperitoneal fibrosis (RPF) is usually an IgG4-related disease, but has some unique characteristics, said John…