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

Impaired BMPRII Signalling in a TGFβ Dependent Mouse Model of Pulmonary Hypertension and in Systemic Sclerosis.

Author: A. Gilbane, E. Derrett-Smith, S. Trinder, R. Good, A. Pearce , C. Denton , and A. Holmes
Date Published: January-2015
Source: American Journal of Respiratory and Critical Care Medicine

Rationale: Up to 10 percent of systemic sclerosis (SSc) patients develop pulmonary arterial hypertension (PAH). This risk persists throughout the disease and is time-dependent, suggesting that SSc is a susceptibility factor. Outcome for SSc-PAH is poor compared with heritable (hPAH) or idiopathic (iPAH) forms, despite clinical and pathological similarities. Whereas susceptibility in hPAH and iPAH is strongly associated with gene mutations leading to reduced expression of bone morphogenetic protein type II receptor (BMPRII), these mutations have not been observed in SSc-PAH.

Clinical Trial for MEDI-551 in Scleroderma Completed

Author: Mahoney JM, Taroni J, Martyanov V, Wood TA, Greene CS, Pioli PA, Hinchcliff ME, Whitfield ML
Date Published: January-2015
Source: PLoS One

Systemic sclerosis (SSc) is a rare systemic autoimmune disease characterized by skin and organ fibrosis. The pathogenesis of SSc and its progression are poorly understood. The SSc intrinsic gene expression subsets (inflammatory, fibroproliferative, normal-like, and limited) are observed in multiple clinical cohorts of patients with SSc. Analysis of longitudinal skin biopsies suggests that a patient's subset assignment is stable over 6-12 months. Genetically, SSc is multi-factorial with many genetic risk loci for SSc generally and for specific clinical manifestations.

Serum adhesion molecule levels as prognostic markers in patients with early systemic sclerosis: a multicentre, prospective, observational study.

Author: Hasegawa M, Asano Y, Endo H, Fujimoto M, Goto D, Ihn H
Date Published: January-2015
Source: PLoS One

To assess the utility of circulating adhesion molecule levels as a prognostic indicator of disease progression in systemic sclerosis (SSc) patients with early onset disease. METHODS: Ninety-two Japanese patients with early onset SSc presenting with diffuse skin sclerosis and/or interstitial lung disease were registered in a multicentre, observational study. Concentrations of intercellular adhesion molecule (ICAM) -1, E-selectin, L-selectin, and P-selectin in serum samples from all patients were measured by enzyme-linked immunosorbent asssay (ELISA). In 39 patients, adhesion molecule levels were measured each year for four years. The ability of baseline adhesion molecule levels to predict subsequent progression and severity in clinical and laboratory features were evaluated statistically.

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.

Systemic sclerosis in 2014: Advances in cohort enrichment shape future of trial design

Author: Yannick Allanore & Oliver Distler
Date Published: January-2015
Source: Nature Reviews Rheumatology

The treatment and study of systemic sclerosis (SSc) is entering a new era with the reporting and preparation of several randomized controlled trials according to an improved understanding of SSc pathogenesis. Advances in trial designs reported in 2014 should now be built upon with further improvements to patient selection to enable targeting of therapies to specific subgroups of patients with SSc.

News for Patients

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.

ASSET (Abatacept Systemic SclErosis Trial)

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.

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).

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.

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.

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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