For Patients

If you have scleroderma, you are not alone. The scleroderma community is made up of tens of thousands of patients and their loved ones worldwide. The SRF is here to help.

The first time many people hear about scleroderma is when they, a family member or friend are diagnosed with the disease. Scleroderma is a complex and surprisingly widespread illness, affecting as many people as more commonly recognized diseases such as multiple sclerosis and muscular dystrophy.

In addition to funding the most promising research aimed at improved therapies and a cure, the Scleroderma Research Foundation provides information that may help scleroderma patients better understand their disorder and more effectively manage its symptoms.

To learn more about the various forms and subtypes of scleroderma, please click here. This section of the Scleroderma Research Foundation’s Web site provides information for patients to educate themselves, as well as their caretakers and loved ones, about this serious disease.

Please remember, information provided on this Website and others is intended as a guide. Specific medical advice can only be provided by your health care professional.

 
 

Research News

Transforming growth factor β—at the centre of systemic sclerosis

Author: Robert Lafyatiso
Date Published: August-2014
Source: Nature Reviews Rheumatology

Transforming growth factor β (TGF-β) has long been implicated in fibrotic diseases, including the multisystem fibrotic disease systemic sclerosis (SSc). Expression of TGF-β-regulated genes in fibrotic skin and lungs of patients with SSc correlates with disease activity, which points to this cytokine as the central mediator of pathogenesis. Patients with SSc often develop pulmonary arterial hypertension (PAH), a particularly lethal complication caused by vascular dysfunction.

The role of genetics and epigenetics in the pathogenesis of systemic sclerosis

Author: Jasper C. A. Broen, Timothy R. D. J. Radstake & Marzia Rossato
Date Published: August-2014
Source: Nature Reviews Rheumatology

Systemic sclerosis (SSc) is a complex autoimmune disease of unclear aetiology. A multitude of genetic studies, ranging from candidate-gene studies to genome-wide association studies, have identified a large number of genetic susceptibility factors for SSc and its clinical phenotypes, but the contribution of these factors to disease susceptibility is only modest. However, in an endeavour to explore how the environment might affect genetic susceptibility, epigenetic research into SSc is rapidly expanding.

Lipoic acid plays a role in scleroderma: insights obtained from scleroderma dermal fibroblasts

Author: P. Tsou, B. Balogh, A. Pinney, G. Zakhem, et al
Date Published: August-2014
Source: Arthritis Research & Therapy

Systemic sclerosis (SSc) is a connective tissue disease characterized by fibrosis of the skin and organs. Increase in oxidative stress and platelet-derived growth factor receptor (PDGFR) activation promote collagen I (Col I) production, leading to fibrosis in SSc. Lipoic acid (LA) and its active metabolite dihydrolipoic acid (DHLA) are naturally occurring thiols that act as cofactors and antioxidants, and are produced by lipoic acid synthetase (LIAS). The goal of this study was to examine whether LA and LIAS was deficient in SSc patients and determine the effect of DHLA on the phenotype of SSc dermal fibroblasts.

Sclerosis: Autologous Cell Transfers May Help Patients

Author: Jennifer Garcia
Date Published: August-2014
Source: Medscape Medical News

Injections of autologous stromal vascular fraction (SVF) may improve hand function and decrease pain among patients with systemic sclerosis (SSc), according to a new study published online August 11 in the Annals of the Rheumatic Diseases. The phase 1, open-label study enrolled 12 female patients with SSc and a Cochin Hand Function Scale score higher than 20/90. Patients receiving vasodilator or immunosuppressive therapy in the 3 months before or 6 months after enrollment were excluded from the study. Patients were administered subcutaneous injections of autologous SVF into each finger of both hands and were evaluated over the course of a 6-month period.

Twenty-two points to consider for clinical trials in systemic sclerosis, based on EULAR standards

Author: D. Khanna, D. Furst, Y. Allanore, S. Bae, V. Bodukam, P. Clements, M. Cutolo, et al
Date Published: August-2014
Source: Oxford Rheumatology

SSc is clinically and aetiopathogenically heterogeneous. Consensus standards for more uniform trial design and selection of outcome measures are needed. The objective of this study was to develop evidence-based points to consider (PTCs) for future clinical trials in SSc.

News for Patients

4 keys to appealing a rejected insurance claim

Author: Tom Murhpy
Date Published: August-2014
Source: Associated Press

Keep calm and take notes. Stay true to this principle and you can improve your odds of successfully fighting a health insurer's claim rejection. Experts who help with the appeals process say patients have a 50 percent chance or better of prevailing. They say a winning argument may require heavy doses of research and persistence, but the end result is a decision that can stave off thousands of dollars in medical bills.

Sclerosis: Autologous Cell Transfers May Help Patients

Author: Jennifer Garcia
Date Published: August-2014
Source: Medscape Medical News

Injections of autologous stromal vascular fraction (SVF) may improve hand function and decrease pain among patients with systemic sclerosis (SSc), according to a new study published online August 11 in the Annals of the Rheumatic Diseases. The phase 1, open-label study enrolled 12 female patients with SSc and a Cochin Hand Function Scale score higher than 20/90. Patients receiving vasodilator or immunosuppressive therapy in the 3 months before or 6 months after enrollment were excluded from the study. Patients were administered subcutaneous injections of autologous SVF into each finger of both hands and were evaluated over the course of a 6-month period.

Pulmonary Arterial Hypertension Treatment Guidelines: New Answers and Even More Questions

Author: Anna R. Hemnes, MD
Date Published: August-2014
Source: Chest

I vividly remember my first patient with pulmonary arterial hypertension (PAH) during my internship in 1999. We admitted a young woman with pulmonary hypertension, clearly miserable from right-sided heart failure. Although she had been followed in our pulmonary hypertension clinic, there was little to offer her until she clearly required the only medication that was known to be efficacious in patients with PAH at that time: IV epoprostenol.1 Within a few days of starting therapy, she was a new person: walking through the hall, heart failure resolved, and ready to go home to her family.

Pharmacologic Therapy for Pulmonary Arterial Hypertension in Adults: CHEST Guideline and Expert Panel Report

Author: D. Taichman, MD, PhD, FCCP; J. Ornelas, MS; L., MD; J. Klinger, MD, FCCP; et al
Date Published: August-2014
Source: Chest

OBJECTIVE: Choices of pharmacologic therapies for pulmonary arterial hypertension (PAH) are ideally guided by high-level evidence. The objective of this guideline is to provide clinicians advice regarding pharmacologic therapy for adult patients with PAH as informed by available evidence. METHODS: This guideline was based on systematic reviews of English language evidence published between 1990 and November 2013, identified using the MEDLINE and Cochrane Library databases. The strength of available evidence was graded using the Grades of Recommendations, Assessment, Development, and Evaluation methodology. Guideline recommendations, or consensus statements when available evidence was insufficient to support recommendations, were developed using a modified Delphi technique to achieve consensus.

Low Blood Pressure Increases Risk of Death in Patients with Systemic Sclerosis-Associated Pulmonary Arterial Hypertension

Author: Lara C. Pullen, PhD
Date Published: July-2014
Source: The Rheumatologist

Patients with pulmonary arterial hypertension (PAH) associated with systemic sclerosis (SSc-APAH) have a higher mortality rate than non-SSc-APAH patients with idiopathic disease as well as those with other connective tissue diseases (CTD-APAH). In a recent study, researchers pinpoint specific predictors that put patients with SSc-APAH at higher risk of death.

Ways to Give

There are many ways that you can support the work of the Scleroderma Research Foundation. We are grateful for your commitment to helping the SRF fund research that will result in improved therapies and, ultimately, a cure.

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