News for Patients


Systematic Autoantigen Analysis Identifies a Distinct Subtype of Scleroderma with Coincident Cancer

Author: George J. Xu, Ami A. Shahf, Mamie Z. Li, Qikai Xu, Antony Rosen, Livia Casciola-Rosen, and Stephen J. Elledge
Date Published: November-2016
Source: PNAS

In this study, we created a barcoded whole-genome ORF mRNA display library and combined it with phage-immunoprecipitation sequencing to look for autoantibodies in sera from patients with scleroderma who also had coincident cancer without a known autoantibody biomarker. Using these two technologies, we found that 25% of these patients had autoantibodies to RNA Binding Region Containing 3 (RNPC3) and multiple other components of the minor spliceosome. There was evidence of intra- and intermolecular epitope spreading within RNPC3 and the complex. These combined technologies are highly effective for rapidly discovering autoantibodies in patient subgroups, which will be useful tools for patient stratification and discovery of pathogenic pathways.

 

Parenting and Scleroderma: How Does That Work?

Author: Kim Tocker
Date Published: November-2016
Source: Scleroderma News

“If you didn’t have scleroderma then you’d be a normal Mum, aye?”

Our youngest was making a comment about how he loves going for bike rides with his Dad, and wished I could come, too. In the same conversation, he talked about missing me at the latest school sports afternoon, and described how lots of the other kids’ mums had participated in the parents’ running races.

Immediately I had visions of myself in slow motion. His mum crawling over the finish line about half an hour after the race actually finished, with a few of the parents who felt sorry for me obligingly clapping from the sidelines. I also could visualize my prednisone fat quivering in the sun as I lay there gasping for breath. No, it all would be awfully unmanageable, and also extremely traumatic, for those having to witness that sight.

 

Rituximab Effective Long-Term for Systemic Sclerosis

Author: Pauline Anderson
Date Published: November-2016
Source: MedPage Today

New research provides additional evidence that rituximab, a B-cell depletion therapy, improves lung fibrosis and reduces skin thickening in patients with systemic sclerosis (SSc).

Over 7 years, pulmonary function was stabilized or improved in SSc patients with interstitial lung disease receiving rituximab, and the drug also helped resolve skin thickening in these patients. As well, the study showed that cessation of rituximab therapy was associated with a decline in pulmonary function, and that the drug had an acceptable safety profile.

 

New Study Suggests Way to Slow Skin Fibrosis in Scleroderma

Author: HSS
Date Published: November-2016
Source: Press Release

The prognosis for patients diagnosed with scleroderma - an autoimmune disease characterized by fibrosis of the skin - is not typically a rosy one. With limited treatment options available, those suffering from the disorder can face disabling hardening and tightening of their skin. Scleroderma can also affect the blood vessels, lungs and other internal organs.

 

New Study Suggests Way to Slow Skin Fibrosis in Scleroderma

Author: HSS
Date Published: November-2016
Source: Press Release

The prognosis for patients diagnosed with scleroderma - an autoimmune disease characterized by fibrosis of the skin - is not typically a rosy one. With limited treatment options available, those suffering from the disorder can face disabling hardening and tightening of their skin. Scleroderma can also affect the blood vessels, lungs and other internal organs.

 

Targeted Therapy for Scleroderma Fibrosis

Author: Sara R. Schoenfeld, MD, & Flavia V. Castelino, MD
Date Published: October-2016
Source: The Rheumatologist

Scleroderma, or systemic sclerosis (SSc), is an autoimmune disease characterized by vasculopathy and fibrosis. Although relatively rare, with a prevalence in North America of approximately 300 per 1 million people, SSc is associated with significant morbidity and high rates of mortality.1 Patients with scleroderma have four times greater mortality than age- and sex-matched controls, with the majority of deaths related to interstitial lung disease (ILD) and pulmonary hypertension (PH).2

 

PAH Medications, Tracleer and Opsumit, Seen to Block Fibrosis in Systemic Sclerosis in Early Study

Author: Joana Fernandes, PHD
Date Published: October-2016
Source: Scleroderma News

Two approved treatments for pulmonary arterial hypertension — Tracleer (bosentan) and Opsumit (macitentan) — can block a molecular pathway that promotes fibrosis in systemic sclerosis (SSc), and may be a potential therapy for these patients as well, according to an early study. The study, “Bosentan And Macitentan Prevent The Endothelial-To-Mesenchymal Transition (Endomt) In Systemic Sclerosis: In Vitro Study,” was published in the journal Arthritis Research & Therapy.

 

Many Systemic Sclerosis Patients with Raynaud’s Syndrome Soon Develop Other Conditions

Author: Joana Fernandes, PHD
Date Published: October-2016
Source: Scleroderma News

Patients with systemic sclerosis and Raynaud’s syndrome have a high risk of developing other organ complications within two years after the onset of Raynaud’s, according to a study published in the journal PLoS One. The authors reported that these complications mainly occur in the skin, gastrointestinal tract, lungs, heart, kidneys and prostate. The study, “Incidences and Risk Factors of Organ Manifestations in the Early Course of Systemic Sclerosis: A Longitudinal EUSTAR Study,” was conducted by Veronika Jaeger and her colleagues from the University Hospital Basel in Switzerland.

 

Adempas PAH Benefits Extend to SSc

Author: Nancy Walsh
Date Published: July-2016
Source: MedPage Today

Treatment with riociguat (Adempas) increased exercise capacity among patients whose pulmonary arterial hypertension was associated with connective tissue disease (PAH-CTD) in a post-hoc analysis of a phase III clinical trial, researchers reported.

Among PAH-CTD patients randomized to receive riociguat in dosages up to 2.5 mg three times per day, the mean 6-minute walking distance increased by 18 meters at week 12 compared with a decrease of 8 m (about 26 ft.) for those given placebo, for a mean treatment difference of +28 m (95% CI -4 to 61), according to Christopher P. Denton, MD, of University College London, and colleagues.

 

Mycophenolate mofetil versus oral cyclophosphamide in scleroderma-related interstitial lung disease (SLS II): a randomised controlled, double-blind, parallel group trial

Author: D.P. Tashkin, MD, M.D. Roth, MD, P.J. Clements, MD, D.E. Furst, MD, D. Khanna, MD, E.C. Kleerup, MD, J. Goldin, MD, E. Arriola, PharmD, E.R. Volkmann, MD, et al
Date Published: July-2016
Source: The Lancet

12 months of oral cyclophosphamide has been shown to alter the progression of scleroderma-related interstitial lung disease when compared with placebo. However, toxicity was a concern and without continued treatment the efficacy disappeared by 24 months. We hypothesised that a 2 year course of mycophenolate mofetil would be safer, better tolerated, and produce longer lasting improvements than cyclophosphamide.

 

Hand Impairment in Systemic Sclerosis: Various Manifestations and Currently Available Treatment

Author: Amber Young, Rajaie Namas, Carole Dodge, Dinesh Khanna
Date Published: July-2016
Source: Springer Link

Systemic sclerosis (SSc) is an autoimmune disease initially recognized by hand involvement due to characteristic Raynaud’s phenomenon (RP), puffy hands, skin thickening, and contractures resembling claw deformities. SSc contributes to hand impairment through inflammatory arthritis, joint contractures, tendon friction rubs (TFRs), RP, digital ulcers (DU), puffy hands, skin sclerosis, acro-osteolysis, and calcinosis. These manifestations, which often coexist, can contribute to difficulty with occupational activities and activities of daily living (ADL), which can result in impaired quality of life. However, despite this knowledge, most diagnostic and treatment principles in SSc are focused on visceral manifestations due to known associations with morbidity and mortality. Treatment of inflammatory arthritis is symptom based and involves corticosteroids ≤10 mg daily, methotrexate, tumor necrosis factor inhibitors, tocilizumab, and abatacept. Small joint contractures are managed by principles of occupational hand therapy and rarely surgical procedures.

 

Advanced Echo Detection of Heart Disease in Scleroderma

Author: Johns Hopkins Rheumatology
Date Published: July-2016
Source: Johns Hopkins Medicine

A new imaging technique reveals evidence of heart dysfunction in Scleroderma patients with undiagnosed heart disease. In a team effort between the Johns Hopkins Divisions of Rheumatology, Cardiology, and Pulmonary/Critical Care Medicine, led by Monica Mukherjee, M.D. and Ami A. Shah, M.D., researchers coupled traditional echocardiography (echo) with a new technique called “speckle-tracking” to reveal the presence of right heart dysfunction that was not detected by traditional methods. This new method may identify patients with Scleroderma who are at high risk for developing disability and even death due to severe heart disease.

 

Taking it personally, cutting-edge biotech snags $45 million

Author: Ron Leuty
Date Published: July-2016
Source: San Francisco Business Times

This year-old company is taking on fibrotic diseases — and it's a personal battle.

 

Digital ulcers in systemic sclerosis

Author: Christopher P. Denton
Date Published: July-2016
Source: The Lancet

Systemic sclerosis (scleroderma) is one of the most recalcitrant of the immune-mediated rheumatic diseases because of its clinical impact and the high mortality associated with the internal organ complications that it causes.1 Lung fibrosis has emerged as a major cause of scleroderma-related death, and with better treatments for pulmonary arterial hypertension, the other main lethal lung manifestation of scleroderma, this looks set to continue.

 

Systemic sclerosis: The need for structured care

Author: Kathleen Morrisroe, Tracy Frech, Janine Schniering, Britta Maurer, Mandana Nikpour
Date Published: July-2016
Source: Clinical Rheumatology

Autoimmune connective tissue diseases (CTDs) have a propensity to affect multiple organ systems as well as physical function, quality of life, and survival. Their clinical heterogeneity, multisystem involvement, and low worldwide prevalence present challenges for researchers to establish a study design to help better understand the course and outcomes of CTDs.

Systemic sclerosis (SSc) is a notable example of a CTD, wherein longitudinal cohort studies (LCS) have enabled us to elucidate disease manifestations, disease course, and risk and prognostic factors for clinically important outcomes, by embedding research in clinical practice. Nevertheless, further efforts are needed to better understand SSc especially with regard to recognizing organ involvement early, developing new therapies, optimizing the use of existing therapies, and defining treatment targets.

 

Raynaud’s Symptoms in SSc Seen to Ease with Iloprost and Bosentan Treatment

Author: Magdalena Kegel
Date Published: July-2016
Source: Scleroderma News

Iloprost (brand name, Ventavis) in combination with bosentan (Tracleer) can improve blood flow in microscopic vessels called capillaries in the hands and feet of patients with systemic sclerosis, where poor capillary function often gives rise to Raynaud’s phenomenon.

The study, “Effect of treatment with iloprost with or without bosentan on nailfold videocapillaroscopic alterations in patients with systemic sclerosis,“ recently published in the journal Modern Rheumatology, inspected capillaries at the nail fold of the finger using video microscopy to evaluate if the two drugs improve the workings of these blood vessels, potentially easing Raynaud’s symptoms.

 

Actelion initiates Phase III study to evaluate macitentan (Opsumit) in children with PAH

Author: Magdalena Kegel
Date Published: July-2016
Source: Scleroderma News

Actelion Ltd (ATLN.VX) announced today that it will be initiating a Phase III study to evaluate the effect of macitentan on delaying disease progression in children with PAH using a pediatric formulation of macitentan (Opsumit).

TOMORROW (pediaTric use Of Macitentan tO delay disease pRogRessiOn in PAH Worldwide) is a multicenter, controlled, randomized, open-label event-driven study to assess the efficacy, safety and pharmacokinetics of macitentan versus standard of care in children with PAH.

 

 

Clinical characteristics and predictors of gangrene in patients with systemic sclerosis and digital ulcers in the Digital Ulcer Outcome Registry: a prospective, observational cohort

Author: Daniel Rosenberg, Barbara Schwierin and Marco Matucci-Cerinic Yannick Allanore, Christopher P Denton, Thomas Krieg, Peter Cornelisse
Date Published: June-2016
Source: Annals of the Rheumatic Diseases

Digital vasculopathy in systemic sclerosis (SSc) consists of a spectrum of Raynaud’s phenomenon (RP), digital ulcers (DUs), critical digital ischaemia and escalation to gangrene. The complications of severe digital vasculopathy often require hospital-based management with intravenous therapies and surgery.1–3 Although gangrene is not infrequent in the clinic, data on the prevalence and implications of gangrene in patients with SSc are scarce.

 

Actemra may reduce scleroderma skin thickening

Author: Ara Freeman
Date Published: June-2016
Source: Dermatology News Digital Network

The interleukin-6 inhibitor tocilizumab (Actemra) substantially reduced skin thickening in patients with systemic sclerosis in the “proof-of concept” faSScinate trial, the results of which have now been published in the Lancet. The mean change in the primary endpoint of a modified Rodnan skin score at 24 weeks was –3.92 in the tocilizumab group and –1.22 in the placebo group, with an overall difference of –2.70 (95% confidence interval, –5.85-0.45; P = .0915). The effect was sustained at 48 weeks’ follow-up (–6.33 vs. –2.77; 95% CI, –7.2-0.12; P = .0579).

 

An Open-label, Phase II Study of the Safety and Tolerability of Pirfenidone in Patients with Scleroderma-associated Interstitial Lung Disease: the LOTUSS Trial

Author: Dinesh Khanna, Carlo Albera, Aryeh Fischer, Nader Khalidi, Ganesh Raghu, Lorinda Chung, Dan Chen, Elena Schiopu, Margit Tagliaferri, James R. Seiboldand Eduard Gorina
Date Published: June-2016
Source: The Journal of Rheumatology

Systemic sclerosis-associated interstitial lung disease (SSc-ILD) shares a number of clinical features and pathogenic mechanisms with idiopathic pulmonary fibrosis (IPF). This study was designed to evaluate the tolerability of the IPF treatment pirfenidone in SSc-ILD. The known gastrointestinal, skin, and liver adverse events (AE) of pirfenidone are of importance given the involvement of these organs in SSc.