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

Digital ulcers in SSc treated with oral treprostinil: a randomized, double-blind, placebo-controlled study with open-label follow-up

Author: J. Seibold, F. Wigley, E. Schiopu, C. Denton, et al
Date Published: February-2017
Source: JSRD

Methods: The literature was reviewed. In addition, a survey was conducted of 315 rheumatologists/internists with a self-declared interest in SSc, to determine their preferred use of echocardiograms.

Background: Prostacyclins are routinely used to treat vascular features of systemic sclerosis (SSc, scleroderma) but require parenteral infusion or inhalation. This study evaluated the safety and efficacy of oral treprostinil in digital ulcers secondary to SSc. Methods: This was a randomized (1:1) placebo-controlled, multicenter study in adults with SSc and at least one active digital ulcer at entry. Oral treprostinil was administered twice daily and titrated to maximum tolerated dose with clinical assessments at Weeks 5, 10, 15 and 20. The primary endpoint was change in net digital ulcer burden. Secondary outcomes included ulcer healing and prevention, measures of hand function, quality of life, Raynaud phenomenon and global assessments. Simplified data were gathered during open-label follow up.

Pros and cons of echocardiography in the screening, diagnosis and follow-up of patients with systemic sclerosis pulmonary arterial hypertension – a rheumatologist’s perspective

Author: Murray Baron
Date Published: February-2017
Source: JSRD

Purpose: Our objective was to review the evidence regarding echocardiography in patients with systemic sclerosis (SSc) and possible pulmonary hypertension (PH).

Methods: The literature was reviewed. In addition, a survey was conducted of 315 rheumatologists/internists with a self-declared interest in SSc, to determine their preferred use of echocardiograms.

Results: The most relevant literature findings come from two studies, the DETECT study and one from the Australian Scleroderma Interest Group. In both these studies, it appears that the use of non-echocardiographic variables such as pulmonary function tests (PFTs) and values of serum N-terminal pro-brain natriuretic peptide (NT-proBNP), are adequate on their own in suggesting which patients are at high risk of PH. Echocardiograms added very little information and in fact may confuse the picture by appearing to be normal when in fact underlying PH is present.

 

Treatment outcome in early diffuse cutaneous systemic sclerosis: the European Scleroderma Observational Study (ESOS)

Author: A. Herrick, X. Pan, S. Peytrignet, M. Lunt, R. Hesselstrand, L. Mouthon, A. Silman, E. Brown, L. Czirják, J. Distler, O. Distler, et al
Date Published: February-2017
Source: Annals of the Rheumatic Diseases

Objectives The rarity of early diffuse cutaneous systemic sclerosis (dcSSc) makes randomised controlled trials very difficult. We aimed to use an observational approach to compare effectiveness of currently used treatment approaches.

Methods This was a prospective, observational cohort study of early dcSSc (within three years of onset of skin thickening). Clinicians selected one of four protocols for each patient: methotrexate, mycophenolate mofetil (MMF), cyclophosphamide or ‘no immunosuppressant’. Patients were assessed three-monthly for up to 24 months. The primary outcome was the change in modified Rodnan skin score (mRSS). Confounding by indication at baseline was accounted for using inverse probability of treatment (IPT) weights. As a secondary outcome, an IPT-weighted Cox model was used to test for differences in survival.

Changes in plasma CXCL4 levels are associated with improvements in lung function in patients receiving immunosuppressive therapy for systemic sclerosis-related interstitial lung disease

Author: E. Volkmann, D. Tashkin, M. Roth, P. Clements, D. Khanna, D. Furst, M. Mayes, J. Charles, C. Tseng, R. Elashoff and S. Assassi
Date Published: January-2017
Source: Arthritis Research & Therapy

Increased circulatory levels of the chemokine CXCL4 have been associated with the presence of interstitial lung disease (ILD) in an observational study of patients with systemic sclerosis (SSc). The purpose of the present study was to evaluate the relationship between baseline CXCL4 level and extent of ILD in the context of a randomized controlled trial and to determine whether changes in CXCL4 levels in response to immunosuppression are associated with future progression of SSc-ILD.

Systematic autoantigen analysis identifies a distinct subtype of scleroderma with coincident cancer

Author: Robert Linda
Date Published: January-2017
Source: Johns Hopkins Rheumatology

A study by Livia Casciola-Rosen, Ph.D. and Aim Shah, M.D. from the Johns Hopkins Division of Rheumatology in collaboration with Steve J. Elledge, Ph.D. and collegues at MIT and Harvard, used cutting-edge technologies to identify a new subgroup of antibodies present in people without classical scleroderma-associated antibodies who develop cancer and scleroderma within a short period of time.

News for Patients

Pneumocystis jiroveci pneumonia is a rare but serious complication for patients with rheumatic diseases

Author: Erika Darrah
Date Published: March-2017
Source: Johns Hopkins Medicine

A research team from the Johns Hopkins Division of Rheumatology lead by Christopher Mecoli, M.D., M.H.S, studied medical records of patients with rheumatic diseases who were admitted to the Johns Hopkins Hospital for pneumocystis jiroveci pneumonia (PJP) infection over a 20-year period (1996-2015). PJP is an uncommon but severe lung infection that can occur in patients with rheumatic disease, in particular in those who are taking medications that suppress the immune system.

Treatment outcome in early diffuse cutaneous systemic sclerosis: the European Scleroderma Observational Study (ESOS)

Author: A. Herrick1, X. Pan, S. Peytrignet, M. Lunt, R. Hesselstrand, et al
Date Published: February-2017
Source: Annals of the Rheumatic Diseases

Objectives The rarity of early diffuse cutaneous systemic sclerosis (dcSSc) makes randomised controlled trials very difficult. We aimed to use an observational approach to compare effectiveness of currently used treatment approaches. Methods This was a prospective, observational cohort study of early dcSSc (within three years of onset of skin thickening). Clinicians selected one of four protocols for each patient: methotrexate, mycophenolate mofetil (MMF), cyclophosphamide or ‘no immunosuppressant’. Patients were assessed three-monthly for up to 24 months. The primary outcome was the change in modified Rodnan skin score (mRSS). Confounding by indication at baseline was accounted for using inverse probability of treatment (IPT) weights. As a secondary outcome, an IPT-weighted Cox model was used to test for differences in survival.

If the Shoe Fits … It’s a Major Feat!

Author: Lisa Goodman-Helfand
Date Published: March-2017
Source: Scleroderma News

Buying a new pair of shoes used to be one of my favorite things to do. The unsightly red spots, telangiectasia, which are generously sprinkled all over my body, limit the clothing I choose to wear. Anything backless, strapless, sleeveless, scoop-necked, or low-cut is immediately ruled out. Being that I am not by nature a conservative person, or Amish, having scleroderma has sort of sucked the joy out of shopping for clothes. But with shoes, the sky was the limit.

Though scleroderma impacted nearly every aspect of my appearance since my diagnosis at age 10, it had thankfully left my feet alone. High heels, platform shoes, open-toed sandals, flip-flops … no shoe was out of bounds for me. Aside from adhering to a reasonable budget, nothing stood between me and whatever shoes my heart desired.

Combo Tx Aids in Scleroderma PAH

Author: Nancy Walsh
Date Published: January-2017
Source: MedPage Today

Initial combination therapy with ambrisentan (Letairis) and tadalafil (Cialis) was more effective than monotherapy with either agent alone among patients with pulmonary arterial hypertension associated with connective tissue disease (CTD-PAH), a post-hoc subgroup analysis of the AMBITION trial found. Among patients with CTD-PAH randomized to receive both agents, the risk of having a first clinical event such as hospitalization for worsening PAH was 57% lower than in the two pooled monotherapy groups (HR 0.43, 95% CI 0.24-0.77), according to John Gerry Coghlan, MD, of the Royal Free Hospital in London, and colleagues.

Changes in plasma CXCL4 levels are associated with improvements in lung function in patients receiving immunosuppressive therapy for systemic sclerosis-related interstitial lung disease

Author: E. Volkmann, D. Tashkin, M. Roth, P. Clements, D. Khanna, D. Furst, M. Mayes, J. Charles, C. Tseng, R. Elashoff and S. Assassi
Date Published: January-2017
Source: Arthritis Research & Therapy

Increased circulatory levels of the chemokine CXCL4 have been associated with the presence of interstitial lung disease (ILD) in an observational study of patients with systemic sclerosis (SSc). The purpose of the present study was to evaluate the relationship between baseline CXCL4 level and extent of ILD in the context of a randomized controlled trial and to determine whether changes in CXCL4 levels in response to immunosuppression are associated with future progression of SSc-ILD.

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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