News for Patients


DETECT, ASIG Algorithms Outperform ESC/ERS Guidelines for Pulmonary Arterial Hypertension in Systemic Sclerosis

Author: Daniela Semedo
Date Published: January-2015
Source: Scleroderma News

Systemic sclerosis (SSc) is a multisystem connective tissue disease characterized by vasculopathy and fibrosis. Pulmonary arterial hypertension (PAH) is one of the most severe organ complications and a leading cause of death in SSc. Estimations are that patients with SSc that have associated PAH have a 3-year survival of just 50%. Since early identification has been found to improve the outcome, researchers have been working on pinpointing the optimal care management for patients with SSc.

 

SSc Patients May Improve with Grafts of Their Own Fat

Author: Alisa Woods
Date Published: January-2015
Source: Scleroderma News

Italian scientists may have identified a way to improve systemic sclerosis (SSc) symptoms with fat grafts. A recent study titled “Autologous Fat Grafting in the Treatment of Fibrotic Perioral Changes in Patients with Systemic Sclerosis” published in the journal Cell Transplantation showed that the fat grafts improved mouth movement, improved blood vessel regrowth, and helped to restore the skin.

 

High Systolic Pulmonary Artery Pressure is a Red Flag in Systemic Sclerosis Patients

Author: Patricia Silva
Date Published: January-2015
Source: Lung Disease News

A recent study published in the journal Rheumatology revealed that a high systolic pulmonary artery pressure (sPAP) can elevate the mortality risk in individuals with systemic sclerosis (SSc). SSc is a rare, chronic connective tissue disease, characterized by increased fibroblast activity which results in abnormal growth of connective tissue and thickening of the skin. It is a disorder more common among women and it has two forms: limited cutaneous, which is restricted to the skin on the face, hands and feet, and diffuse cutaneous that has the risk of progressing to visceral organs such as the lungs.

 

Pain Management During Scleroderma Digital Ulcer Debridement Results In Improved Outcomes

Author: Charles Moore
Date Published: January-2015
Source: Scleroderma News

Controlling procedural pain in the debridement of scleroderma-associated digital ulcers led to better outcomes and treatment adherence among patients, according to recent research. Originally described at the American College of Rheumatology Annual Meeting 2013, the study monitored treatment of 51 digital ulcers in 32 systemic sclerosis (SSc) patients at a rheumatology clinic in Italy between January 2012 and December 2012 inclusive. Procedural pain was addressed in an ascending sequence beginning with local lidocaine and/or prilocaine cream (25 mg/g of either or 5% EMLA cream).

 

Pulmonary Arterial Hypertension in Systemic Sclerosis: Improved Screening Methods Identified

Author: Alissa Woods, PhD
Date Published: January-2015
Source: Scleroderma News

A collaborative group of researchers, based in Australia and China, have found that early screening for pulmonary arterial hypertension (PAH) in systemic sclerosis (SSc) using two recently developed methods resulted in identification of PAH in 100% of individuals screened. The study, titled A Comparison of the Predictive Accuracy of Three Screening Models for Pulmonary Arterial Hypertension in Systemic Sclerosis appeared January 18th, 2015 in Arthritis Research & Therapy.

 

NIH Grants for Cureveda's Systemic Sclerosis and COPD Studies

Author: Curevedas
Date Published: January-2015
Source: Advance Healthcare Network

The National Institutes of Health (NIH) recently granted two $450,000 Phase I Small Business Technology Transfer (STTR) grants to Cureveda LLC, who is currently developing new therapies for the treatment of inflammatory and fibrotic diseases, such as scleroderma. The company will invest the grants in its ongoing preclinical studies that are going to evaluate its lead investigational product VEDA-1209 to treat systemic sclerosis and chronic obstructive pulmonary disease (COPD).

 

Bristol-Myers Squibb and California Institute For Biomedical Research Collaborate On Anti-Fibrotic Therapy

Author: I. Santos
Date Published: January-2015
Source: Pulmonary Hypertension News

A new collaboration between Bristol-Myers Squibb and the California Institute for Biomedical Research (Calibr) was announced today. The new collaboration aims to develop new small molecule anti-fibrotic therapies and includes an agreement that enables Bristol-Myers Squibb to develop, produce and commercialize the preclinical compounds that can result from this same collaboration. Carl Decicco, Ph.D., from Bristol-Myers Squibb said in a press release: “Bristol-Myers Squibb’s collaboration with Calibr further strengthens our Specialty portfolio and advances the company’s fibrotic diseases pipeline with the addition of this promising program. Calibr’s innovative discovery program in fibrosis represents an opportunity to develop new treatment approaches for patients.”

 

Scleroderma-Related Raynaud’s Phenomenon Treated with Alprostadil

Author: Maureen Newman
Date Published: January-2015
Source: Scleroderma News

Important to the development of any treatment for scleroderma is a clinical study investigating the pharmacodynamics and pharmacokinetics of a potential drug candidate. NexMed (USA), Inc., a subsidiary of Apricus Biosciences, Inc., is sponsoring a currently enrolling clinical trial investigating these two aspects of alprostadil in systemic sclerosis (SSc) patients with a secondary diagnosis of Raynaud’s phenomenon, also known as scleroderma-related Raynaud’s phenomenon.

 

Clinical Trial for MEDI-551 in Scleroderma Completed

Author: Maureen Newman
Date Published: January-2015
Source: Scleroderma News

A Phase 1 clinical trial recently ended that involved “A Study of the Safety and Tolerability of MEDI-551 in Scleroderma.” MedImmune LLC, sponsor of the study, last updated the results of the trial in an abstract presented through Annals of the Rheumatic Diseases. According to “Safety and Tolerability of MEDI-551 in Subjects with Systemic Sclerosis (SSC): Results from A Phase 1 Randomized, Placebo-Controlled Escalating Single-Dose Study,” the pharmacodynamics and safety profile of MEDI-551 warrant further clinical development. Although patients taking MEDI-551 experienced adverse and serious events potentially due to treatment, and patients taking placebo experienced none, events were singular, and the only death was related to scleroderma worsening, not treatment.

 

ASSET (Abatacept Systemic SclErosis Trial)

Author:
Date Published: January-2015
Source: University of Michigan Scleroderma Program

ASSET (Abatacept Systemic SclErosis Trial) is an international, placebo-controlled, double blind, randomized trial of early diffuse cutaneous systemic sclerosis (SSc). This trial assesses abatacept (Orencia®, a recombinant fusion protein consisting of the extracellular domain of human CTLA4 is FDA approved biologic medication for rheumatoid arthritis and juvenile arthritis) in patients with early diffuse SSc with less than or equal to 36 months.

 

Is Rituximab Ready for Prime Time in SSc? Experts: new data support rituximab for reducing lung fibrosis in systemic sclerosis.

Author: Diana Swift
Date Published: January-2015
Source: MedPage Today

B-cell depletion via rituximab (Rituxan) infusion reduced the progression of skin thickening and lung fibrosis in systemic sclerosis (SSc), according to a review article, and clinicians need to consider rituximab therapy in these patients. Fiona McQueen, MD, of the University of Auckland, and Kamal Solanki, MD, of Waikato Hospital in Hamilton, both in New Zealand, looked at observational, case-control data from the European League Against Rheumatism Scleroderma Trials and Research (EUSTAR) group and summarized their findings in Rheumatology.

 

Genetic Markers Aid in Tracking CVD Risk in SSc - Two markers appear to be useful for identifying SSc patients at risk for pulmonary hypertension.

Author: Diana Swift
Date Published: January-2015
Source: MedPage Today

Patients with systemic sclerosis (SSc) who are free of traditional cardiovascular risk factors can still have increased levels of two biomarkers indicative of myocyte injury: high-sensitivity cardiac troponin (HS-cTnT) and N-terminal fragment of pro-brain natriuretic peptide (NT-proBNP). "Elevated levels of these markers may reflect subclinical myocardial damage and dysfunction caused by the disease [SSc] itself," wrote French investigators online in Arthritis Care and Research.

 

Surgical treatment of systemic sclerosis-is it justified to offer peripheral sympathectomy earlier in the disease process?

Author: Momeni A, Sorice SC, Valenzuela A, Fiorentino DF, Chung L, Chang J.
Date Published: January-2015
Source: Microsurgery

Systemic sclerosis (SSc) is a rare connective tissue disease associated with significant digital vasculopathy. Peripheral sympathectomy is frequently offered late in the disease process after severe digital ischemia has already occurred with patients being symptomatic for numerous years. The purpose of the present study was to analyze the results of peripheral sympathectomy in patients with a confirmed diagnosis of SSc.

 

ZEBRA Clinical Trial Testing Treatment for Scleroderma Renal Crisis

Author: Maureen Newman
Date Published: January-2015
Source: Scleroderma News

“Zibotentan Better Renal Scleroderma Outcome Study (ZEBRA)” is more than a standard clinical trial for scleroderma patients. Rather, the phase 2 clinical trial is a three-part clinical trial seeking to help patients with mild, moderate, or severe kidney disease due to scleroderma. University College, London is sponsoring the trial in collaboration with the Medical Research Council. The aim is to study Zibotentan in patients with mild or moderate kidney disease in ZEBRA 1, severe acute kidney disease (renal crisis) without dialysis in ZEBRA 2A, and renal crisis with dialysis in ZEBRA 2B.

 

Laboratory Testing for Diagnosis, Management of Patients with Rheumatic Disease

Author: Peter H. Schur, MD
Date Published: December-2014
Source: The Rheumatologist

Laboratory testing is an essential element in the diagnosis and management of patients with rheumatic disease. This article focuses on a diverse array of serological markers that can provide unique information on the status of the patient’s immune system that is important in clinical evaluation as well as scientific inquiry. These tests help in the diagnosis of a particular disease, and importantly, they may help monitor disease activity. Indeed, immunological testing represents one of the bedrocks of rheumatology and is a distinguishing feature of our specialty.

 

Actelion Submits New Drug for Pulmonary Arterial Hypertension to FDA

Author: Leonor Mateus Ferreira
Date Published: December-2014
Source: Lung Disease News

Actelion has submitted its new treatment for pulmonary arterial hypertension (PAH) to the U.S. Food and Drug Administration (FDA). The company is applying for a New Drug Application (NDA) for selexipag (Uptravi), after having also submitted the drug to the European Medicines Agency (EMA). Actelion is expecting to offer selexipag as a treatment for patients who suffer from PAH, which will make it the first selective oral IP prostacyclin receptor agonist for the disease. The drug, which was originally developed by the Kyoto, Japan-based research company Nippon Shinyaku, has been evaluated in a phase III pivotal study called GRIPHON, whose encouraging results supported Actelion’s recent submission efforts.

 

Carbon Dioxide Lasers Studied for SSc Therapy

Author: Maureen Newman
Date Published: December-2014
Source: Scleroderma News

“Fractional Carbon Dioxide Laser Versus UVA 1 in Treatment of Localized Scleroderma,” a clinical trial currently recruiting patients, is looking to add another possible remedy to the list for systemic sclerosis (SSc). In the trial, fractional carbon dioxide laser treatment will be compared against UVA1 phototherapy in terms of its ability to improve the clinical presentation of SSc symptoms.

 

Adiponectin May Prevent Skin Fibrosis in Systemic Sclerosis

Author: Alissa Woods, PhD
Date Published: December-2014
Source: Pulmonary Hypertension News

An international collaboration of researchers from the United States and China reported that adiponectin (APN), a protein that regulates glucose levels and fatty acid breakdown in the body, may be a naturally-occurring anti-fibrotic agent for systemic sclerosis (SSc). They presented their study at the 2014 American College of Rheumatology Meeting in November. Systemic sclerosis is an autoimmune and connective tissue disease. It is an uncommon, chronic disorder in which skin degeneration and scarring occurs, causing accumulation of collagen (fibrosis). Damage to the joints, internal organs and blood vessel abnormalities — particularly damage to small arteries — may also be present in SSc. Although anti-fibrotic agents have been studied for the treatment of skin fibrosis in SSc, so far no agent has been shown to be consistently beneficial.

 

Procedural pain management in the treatment of scleroderma digital ulcers

Author: Giuggioli D, et al
Date Published: December-2014
Source: MD Linx

The authors report the results of the experience on procedural pain management in scleroderma digital ulcers (DU). Therefore, the present study showed valuable control of procedural pain during DU debridement with sequential, combined analgesic treatment.

 

Connective tissue diseases: New criteria improve recognition of early systemic sclerosis

Author: Yoshihide Asano & Shinichi Sato
Date Published: November-2014
Source: Nature Reviews Rheumatology

In systemic sclerosis (SSc), new classification criteria highly sensitive to early and limited forms of the disease could facilitate not only the recognition of early SSc, but also the discovery of biomarkers of poor prognosis, with potential benefits for conducting clinical trials.