What is scleroderma (types and symptoms)

What is Scleroderma?

Judy Williamson,
Scleroderma Patient

The word “scleroderma” is Greek for hard skin, the most visible characteristic of the disease. In fact, scleroderma is much more than this; chronic, complex and debilitating, it often affects the internal organs with life-threatening consequences. Depending on the subtype of illness, scleroderma can damage the lungs, kidneys and gastrointestinal tract with grave results. Peripheral vasculature damage due to scleroderma can result in loss of digits or limbs. In some cases, the joints and muscles are affected, resulting in a loss of mobility.

Scleroderma falls into several different disease categories: It is a vascular disease because it can constrict and injure tiny blood vessels. It is a connective tissue disease because it can cause abnormal changes to the skin, tendons and bones. Like many other rheumatic disorders, scleroderma is believed to be an autoimmune disease because the disease can apparently trigger the body to make antibodies against itself.

The symptoms and severity of scleroderma vary from one person to another and the course of the disease is often unpredictable. The number of women affected with scleroderma is disproportionately high with some estimates suggesting as many as four out of every five patients being female. The disease most often strikes between the ages of 20 and 50; however, children and those above age 50 across all ethnic groups are also affected.

Today, there is no way to prevent scleroderma and there is no cure. Treatments are available for some, but not all of the most serious complications of the disease. Current treatments include medications that modulate the immune system, chemotherapy drugs, vasodilators and ACE-inhibitors. Presently, most treatments act to slow the progression of the disease and limit damage rather than truly arresting the disease. In addition, some of the drugs currently in use can have serious side effects. There is much work that remains to be done.

Despite the number of people affected by scleroderma and the devastating effect the disease can have, scleroderma research remains critically underfunded by the National Institutes of Health. Contributions made to the Scleroderma Research Foundation support promising exploratory projects as well as innovative research studies that may provide the basis for longer term investment by federal research funding programs. Until new therapies are made possible by advances in medical research, people living with scleroderma continue to have hope, knowing that scientists are working every day on their behalf.

For a more detailed description of scleroderma, click here.

The SRF strives to keep you informed of headlines, reports and announcements affecting the scleroderma community. Click on the links to visit our library where you will find articles intended to keep you up to date on current news for patients, research and SRF related news.

You can help fund promising research aimed at helping people living with scleroderma. Please donate online today.

 
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Research News

SRF-funded Harvard Researchers Explore Cellular Self-destruction as Scleroderma Treatment

Author: Magdalena Kegel
Date Published: January-2018
Source: Scleroderma News

Organ fibrosis in scleroderma patients might be reversed with treatments that allow fibrotic cells to self-destruct, Harvard scientists suggest.

Their study, funded by the SRF, “Targeted apoptosis of myofibroblasts with the BH3 mimetic ABT-263 reverses established fibrosis,” appeared in the journal Science Translational Medicine. It demonstrated that the survival–self-destruction balance of fibrosis-producing myofibroblast cells can be tipped by using a compound now being explored as a cancer treatment.

Getting Started on Methotrexate: What You Should Know

Author: Johns Hopkins
Date Published: January-2018
Source: Johns Hopkins Rheumatology

Methotrexate is a medication commonly used to treat rheumatoid arthritis and many other autoimmune and inflammatory conditions. Nurse Victoria Ruffing talks about what you should know when getting started on Methotrexate, as well as the side effects of Methotrexate.

Pathogenesis of systemic sclerosis: recent insights of molecular and cellular mechanisms and therapeutic opportunities

Author: John Varga, Maria Trojanowska, Masataka Kuwana
Date Published: July-2017
Source: Journal of Scleroderma and Related Disorders

Systemic sclerosis (SSc) is a complex disease characterized by early microvascular abnormalities, immune dysregulation and chronic inflammation, and subsequent fibrosis of the skin and internal organs. Excessive fibrosis, distinguishing hallmark of SSc, is the end result of a complex series of interlinked vascular injury and immune activation, and represents a maladaptive repair process. Activated vascular, epithelial, and immune cells generate pro-fibrotic cytokines, chemokines, growth factors, lipid mediators, autoantibodies, and reactive oxygen species. These paracrine and autocrine cues in turn induce activation, differentiation, and survival of mesenchymal cells, ensuing tissue fibrosis through increased collagen synthesis, matrix deposition, tissue rigidity and remodeling, and vascular rarefaction.

The mighty fibroblast and its utility in scleroderma research

Author: Sara M. Garrett, DeAnna Baker Frost, Carol Feghali-Bostwick
Date Published: May-2017
Source: Journal of Scleroderma and Related Disorders

Fibroblasts are the effector cells of fibrosis characteristic of systemic sclerosis (SSc, scleroderma) and other fibrosing conditions. The excess production of extracellular matrix (ECM) proteins is the hallmark of fibrosis in different organs, such as skin and lung. Experiments designed to assess the pro-fibrotic capacity of factors, their signaling pathways, and potential inhibitors of their effects that are conducted in fibroblasts have paved the way for planning clinical trials in SSc. As such, fibroblasts have proven to be valuable tools in the search for effective anti-fibrotic therapies for fibrosis. Herein we highlight the characteristics of fibroblasts, their role in the etiology of fibrosis, utility in experimental assays, and contribution to drug development and clinical trials in SSc.This article is available for you to download for free for 1 week until 2nd August 2017.

Systemic Sclerosis Linked With Altered Gut Microbiome

Author: Gregory M. Weiss, MD
Date Published: July-2017
Source: Rheumatology Network

The numbers of Bacteroidetes bacteria in the GI tracts of 2 separate cohorts of patients with systemic sclerosis were significantly reduced when compared with healthy controls. American patients with systemic sclerosis had more extensive alterations in their intestinal microbiota than those in a Norwegian cohort. An abundance of Prevotella species was associated with moderate-to-severe GI symptoms in patients with systemic sclerosis. Clostridium species abundance was associated with low GI symptom severity, and Lactobacillus with none-to-mild constipation.

News for Patients

SRF-funded Harvard Researchers Explore Cellular Self-destruction as Scleroderma Treatment

Author: Magdalena Kegel
Date Published: January-2018
Source: Scleroderma News

Organ fibrosis in scleroderma patients might be reversed with treatments that allow fibrotic cells to self-destruct, Harvard scientists suggest.

Their study, funded by the SRF, “Targeted apoptosis of myofibroblasts with the BH3 mimetic ABT-263 reverses established fibrosis,” appeared in the journal Science Translational Medicine. It demonstrated that the survival–self-destruction balance of fibrosis-producing myofibroblast cells can be tipped by using a compound now being explored as a cancer treatment.

Getting Started on Methotrexate: What You Should Know

Author: Johns Hopkins
Date Published: January-2018
Source: Johns Hopkins Rheumatology

Methotrexate is a medication commonly used to treat rheumatoid arthritis and many other autoimmune and inflammatory conditions. Nurse Victoria Ruffing talks about what you should know when getting started on Methotrexate, as well as the side effects of Methotrexate.

Raynaud’s Phenomenon : What You Should Know

Author: Johns Hopkins
Date Published: January-2018
Source: Johns Hopkins Rheumatology

Raynaud’s Phenomenon is a condition that results in decreased blood flow to your fingers and toes. There are two types of Raynaud’s: primary and secondary. SRF-funded investigator, Dr. Zsuzsanna McMahan from the Johns Hopkins Scleroderma Center dives into what this condition is, things to look out for, how to manage it, and when you should speak with a doctor.

Uptravi Slows Progression of Pulmonary Hypertension Stemming from Connective Tissue Disease, Study Finds

Author: Iqra Mumal
Date Published: August-2017
Source: Pulmonary Hypertension News

Actelion’s Uptravi (selexipag) delays the progression of pulmonary arterial hypertension that stems from connective tissue disease, a follow-up analysis of Phase 3 clinical trial findings indicates. People with PAH-CTD also tolerate the therapy well, according to the study. The PAH part of PAH-CTD refers to high blood pressure in the lungs’ arteries.

Prucalopride Can Help Improve Gastrointestinal Symptoms in SSc Patients, Study Shows

Author: Alice Melao
Date Published: August-2017
Source: Scleroderma News

Prucalopride, marketed under the name Resolor in Europe and Resotran in Canada, can safely manage mild to severe gastrointestinal (GI) symptoms associated with systemic sclerosis (SSc), including bowel movement and transit, reflux disease, and bloating, finds a study published in the journal Arthritis Research and Therapy. Doctors have proposed using medicines to improve prokinetics, or motility of the GI tract, in treating SSc patients. But the lack of proper clinical trials demonstrating the therapeutic potential of such drugs in the SSc population discouraged their use in clinics.

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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