News for Patients


Lung Ailment in Systemic Sclerosis More Aggressive in Men -- But survival the same as in women

Author: Wayne Kuznar
Date Published: February-2016
Source: MedPage Today

Although pulmonary arterial hypertension (PAH) is more common in women with systemic sclerosis (SSc), men had a shorter interlude after an SSc diagnosis to the occurrence of PAH, were younger at the time of PAH diagnosis, and had a shorter PAH disease duration, Canadian researchers found.

 

The crowded crossroad to angiogenesis in systemic sclerosis: where is the key to the problem?

Author: Mirko Manetti, Serena Guiducci and Marco Matucci-Cerinic
Date Published: January-2016
Source: Arthritis Research & Therapy

Dysregulated expression of several proangiogenic and antiangiogenic factors has been implicated in the dysfunction of angiogenesis in systemic sclerosis (SSc). Into this complex scenario comes the study recently published in Arthritis Research & Therapy by Hirigoyen et al. [1], who report new data implicating an inhibitory splice variant of vascular endothelial growth factor (VEGF)-A, namely VEGF165b, in the inhibition of angiogenesis by platelets in SSc. These findings are part of an intriguing chain of data as several studies have shown that VEGF-A, one of the most potent promoters of angiogenesis, is markedly increased in SSc skin and circulation despite clear evidence of an insufficient angiogenic response [2, 3].

 

Treprostinil iontophoresis improves digital blood flow during local cooling in systemic sclerosis

Author: Florence Gaillard-Bigot, Matthieu Roustit, Sophie Blaise, et al
Date Published: January-2016
Source: Microcirculation

Severe Raynaud's syndrome and digital ulcers are the most prevalent manifestations of systemic sclerosis (SSc) peripheral microvascular disease. We tested whether treprostinil iontophoresis on the finger pad of patients with SSc would improve digital blood flow during hand cooling.

 

Genetic factors and systemic sclerosis

Author: Giuseppe Murdacaa, Miriam Contatorea, Rossella Gullib, Paola Mandichb, Francesco Puppoa
Date Published: January-2016
Source: Autoimmunity Reviews

Systemic sclerosis (SSc) is a rare connective tissue disease of unknown etiology characterized by chronic inflammation and fibrosis of the skin, vascular abnormalities, and variable involvement of organs including kidneys, gastrointestinal tract, heart, and lungs. SSc shows a complex etiology in which both environmental and genetic factors seem to influence the onset and outcome of the disease. We provide an extensive overview of the genetic factors and epigenetic modifications and what their knowledge has revealed in terms of etiopathogenesis of SSc.

 

My Hands and Feet Are Always Cold — Should I Worry?

Author: Heart & Vascular Team
Date Published: February-2016
Source: Cleveland Clinic

It’s a familiar scene. You turn up the thermostat; your partner turns it down. Some people struggle with feeling cold all the time — especially when it comes to their hands and feet. If you feel like your extremities are often cold, you may wonder when you should see a doctor. Here’s what you need to know. When blood vessels constrict “Cold hands and feet are a common complaint,” says vascular specialist Natalie Evans, MD. “But generally, when this happens in young healthy people, it isn’t anything to worry about.”

 

Scleroderma related interstitial lung disease can be treated with new promising treatment

Author:
Date Published: January-2016
Source: Bel Marra Health

Scleroderma related interstitial lung disease can be treated with new promising treatment. Interstitial lung disease is common in systemic sclerosis and is the leading cause of death. If discovered early on, though, prognosis is positive for patients. Interstitial lung disease is a term to describe a large group of disorders which refers to the scarring of lung tissue and supporting of the air sacs. This scarring can contribute to lung stiffness which affects a person’s ability to breathe which limits oxygen from entering the bloodstream.

 

Colonic Microbial Signatures in Systemic Sclerosis How do they affect clinical manifestations?

Author:
Date Published: January-2016
Source: Rheumatology News

Compared with healthy controls, patients with systemic sclerosis (SSc) harbor a distinct colonic microbial signature that may perpetuate immunological aberrations and contribute to clinical manifestations of SSc. This according to a study of 17 patients with SSc and 17 age- and sex-matched healthy controls. Researchers found:

 

Anti-IFI16 Antibodies in Scleroderma - Studying associations with digital gangrene

Author:
Date Published: January-2016
Source: Rheumatology News

Anti-interferon inducible protein 16 (Anti-IFI16) antibodies are associated with digital gangrene in scleroderma. This according to a study of sera from 94 patients with scleroderma and 47 healthy controls and a separate case-control study of 39 patients with scleroderma and digital gangrene and 39 patients with scleroderma and Raynaud’s alone. Researchers found:

 

Telling Jewels From Junk in DNA

Author: Carl Zimmer
Date Published: January-2016
Source: New York Times

When you learned about genes in high school, chances are it went something like this: Our DNA holds about 20,000 protein-coding genes. To make a protein, a cell makes a copy of the corresponding gene, in the form of a single-stranded molecule called RNA. The cell uses the RNA molecule as a template to make the protein. And then the protein floats off to do its job. That’s certainly true. But there’s more to the story.

 

Experimental Compound Shows Promise in Reversing Skin Disease Associated with Systemic Sclerosis

Author: Collee Labbe
Date Published: January-2016
Source: NIAMS Spotlight on Research

A new drug appears to alter the expression of certain genes associated with systemic sclerosis by blocking a key protein, and also leads to clinical improvements in the skin, according to a study funded in part by the NIH’s National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS). The study was published in the Journal of Clinical Investigation. Systemic sclerosis, a severe form of scleroderma, is a chronic connective tissue disease that affects multiple organs and tissues in the body. It can lead to fibrosis, or hardening, of the skin and other organs such as the lungs, kidneys and heart. Currently, no effective treatments exist. Because the disease affects everyone differently, finding therapies has been challenging.

 

Idiopathic Pulmonary Fibrosis Drugs Makes Inroads in Systemic Sclerosis Market

Author:
Date Published: January-2016
Source: Clinical Trials Arena

Pharmaceutical giant, Boehringer Ingelheim (BI), is poised to begin a Phase III trial of its idiopathic pulmonary fibrosis (IPF) drug, Ofev (nintedanib) as a treatment for systemic sclerosis-related interstitial lung disease (SSc-ILD). Ofev initially gained approval by the Food and Drug Administration (FDA) in October 2014 and by the European Medicines Agency (EMA) in January 2015. Roche and Genentech's Esbriet, arguably BI's closest competitor in the field, is more widely known than Ofev. Despite being available in Europe since 2011, Esbriet gained FDA approval on the same day as Ofev.

 

SteadyMed’s Treyvent Awarded Orphan Drug Designation from FDA

Author: Dainela Semedo
Date Published: January-2016
Source: Pulmonary Hypertension News

SteadyMed, Ltd., a company that develops products to treat orphan and high-value conditions with unmet parenteral delivery needs, recently announced that its lead product candidate, Trevyent® has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of pulmonary arterial hypertension (PAH).

 

Anti-IFI16 antibodies in scleroderma are associated with digital gangrene

Author: Zsuzsanna H. McMahan, Ami A. Shah, Dhananjay Vaidya, Fredrick M. Wigley, Antony Rosen andLivia Casciola-Rosen
Date Published: December-2015
Source: Arthritis Research & Therapy

Objective: Our aim was to examine the association between anti-interferon inducible protein 16 (IFI16) antibodies and clinical features of scleroderma. Methods: Sera from a discovery sample of 94 scleroderma patients and 47 healthy controls were assayed for anti-IFI16 antibodies by ELISA, and associations were examined using regression analyses. As anti-IFI16 autoantibodies strongly associated with digital gangrene in the discovery sample, a subsequent 1:1 disease-duration matched case-control study was designed for further exploration. Cases were patients with scleroderma and digital gangrene, while controls had scleroderma and Raynaud's alone (n=39 pairs). Nonparametric unadjusted matched-pair analyses, univariate and multivariable conditional logistic regression were performed.

 

Predictors of lung function decline in scleroderma-related interstitial lung disease based on high-resolution computed tomography: implications for cohort enrichment in systemic sclerosis–associated interstitial lung disease trials

Author: Dinesh Khanna, Vivek Nagaraja, Chi-hong Tseng, Fereidoun Abtin, Robert Suh, et al
Date Published: December-2015
Source: Arthritis Research & Therapy

Background: The extent of lung involvement visualized by high-resolution computed tomography (HRCT) is a predictor of decline in forced vital capacity (FVC) in scleroderma–interstitial lung disease. Our objective was to evaluate the performance of three different HRCT-defined staging systems in the Scleroderma Lung Study I (SLS I) over a 1-year period. Methods: We assessed two visual semiquantitative scores: the maximum fibrosis score (MaxFib, the fibrosis score in the zone of maximal lung involvement) and visual assessment of total lung involvement (TLI) as proposed by Goh and Wells. In addition, we evaluated the computer-aided diagnosis and calculated the quantitative percentage with fibrosis (QLF) and TLI.

 

Recent developments in the classification, evaluation, pathophysiology, and management of scleroderma renal crisis

Author: Cybele Ghossein, John Varga, Andrew Z. Fenves
Date Published: December-2015
Source: Current Rheumatology Reports

Scleroderma renal crisis (SRC) is an uncommon complication of systemic sclerosis. Despite the advent of angiotensin-converting inhibitor therapy, SRC remains a life-threatening complication. Recent studies have contributed to a better understanding of SRC, but much remains unknown regarding its pathophysiology, risk factors, and optimal management. Genetic studies provide evidence that immune dysregulation might be a contributing factor, providing hope that further research in this direction might illuminate pathogenesis and provide novel predictors for this complication.

 

EADV: Fresolimumab shows early promise in scleroderma

Author: Bruce Jancin
Date Published: December-2015
Source: Rheumatology News

The recent success of fresolimumab in treating early diffuse cutaneous systemic sclerosis in a proof-of-concept study signals better days ahead in the treatment of scleroderma, Dr. Thomas Krieg predicted at the annual congress of the European Academy of Dermatology and Venereology. “There will be new and better treatments based upon our improved understanding of the pathophysiology of this complex multifaceted disease. New targeted therapies are likely,” according to Dr. Krieg, chairman of the department of dermatology and venereology and dean of the medical faculty at the University of Cologne (Germany).

 

FDA Approves New Orphan Drug to Treat Pulmonary Arterial Hypertension

Date Published: December-2015
Source: FDA

On December 21, the U.S. Food and Drug Administration approved Uptravi (selexipag) tablets to treat adults with pulmonary arterial hypertension (PAH), a chronic, progressive, and debilitating rare lung disease that can lead to death or the need for transplantation. “Uptravi offers an additional treatment option for patients with pulmonary arterial hypertension,” said Ellis Unger, M.D., director of the Office of Drug Evaluation I in the FDA’s Center for Drug Evaluation and Research. “The FDA supports continued efforts to provide new treatment options for rare diseases.”

 

New Biomarker Assay to Aid in Systemic Sclerosis Diagnosis

Author: Dainela Semedo
Date Published: December-2015
Source: Scleroderma News

Protagen AG, a leader in the development of novel molecular diagnostics for autoimmune diseases, recently launched the Multilisa BICD2, an ELISA (enzyme-linked immunosorbent assay) that together with clinical symptoms will aid on the diagnosis of systemic sclerosis (SSc). SSc is a complex, multi-organ autoimmune disease characterized by increased fibroblast activity that results in abnormal growth of connective tissue, causing vascular damage and fibrosis. Fibrosis can occur in the skin, the gastrointestinal (GI) tract and other internal organs. The clinical manifestations of SSc are extremely heterogeneous and dependent on the presence and degree of organ involvement. SSc is a rare disorder, with an annual incidence in the United States of about 20 cases in every 1 million adults.

 

New Phase III Study of Nintedanib in people with Systemic Sclerosis and Lung Fibrosis

Date Published: December-2015
Source: BI

Boehringer Ingelheim announced today that the first person has been enrolled in the SENSCIS™ (Safety and Efficacy of Nintedanib in Systemic SClerosIS) study. The global Phase III trial, which is now open for recruitment, is investigating the efficacy and safety of nintedanib in people with a rare disease called systemic sclerosis (SSc) who also developed interstitial lung disease (SSc-ILD) (NCT02597933).1 In total, 520 people are expected to be enrolled in clinical trial centres worldwide. Systemic sclerosis, also known as scleroderma, is a rare disease characterised by the thickening and scarring of connective tissue of multiple organs in the body.2 The scarring can also affect the lungs and when this occurs the condition is called SSc-ILD.2 The presence of ILD with SSc often indicates a poor prognosis and higher risk of death.

 

Digital Ulcers Predict Systemic Sclerosis Complications

Author: Nancy Walsh
Date Published: December-2015
Source: MedPage Today

Patients with systemic sclerosis (SSc) who have chronic digital ulcers experience high rates of severe complications and medication use, a retrospective European study found. Among a cohort of almost 1,500 SSc patients who either had a history of digital ulcers or ulcers present at the time of enrollment and who were followed for 2 years, gangrene and amputation were reported in 22% and 15.9% of those who had ulcers present at each follow-up clinic visit compared with 1.5% for both outcomes among patients who had no new ulcers at any study visit, according to Marco Matucci-Cerinic, MD, of the University of Florence, and colleagues.