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Scleroderma study: Hope for a longer life for patients with rare autoimmune disorder

January 12, 2018

An unusual autoimmune disease that causes skin and lung damage can be treated effectively by stem cell transplant, a new study in the New England Journal of Medicine has found. The approach could represent the first new treatment to improve survival in patients with severe scleroderma in more than four decades.

The experimental procedure uses chemotherapy and radiation to destroy the body's malfunctioning immune system, then replaces it via a stem-cell transplant. The stem cells are taken from the patient's own blood and given back to the patient after the chemotherapy.

In a multisite clinical trial, the approach proved more successful than the existing treatment, significantly improving survival and reported quality of life. "This is a major advance in the treatment of severe scleroderma," said Karen Ballen, MD, a co-investigator on the study and the director of stem cell transplantation at the University of Virginia Cancer Center.

Scleroderma Study

The study compared the most effective existing treatment, the drug cyclophosphamide, with the new approach. Thirty-four trial participants received cyclophosphamide, while 33 received the stem cell transplant treatment. After 72 months, 86 percent of those who received the stem-cell transplant remained alive, compared with only 51 percent of those who received infusions of cyclophosphamide.

In the New England Journal of Medicine paper detailing their findings, the researchers conclude: "At four and a half years of follow up, participants who received a transplant experienced significantly better outcomes overall than those who received cyclophosphamide. In addition, 44 percent of participants who received cyclophosphamide had begun taking anti-rheumatic drugs for progression of their scleroderma, compared to only 9 percent of those who received a transplant."

Both treatment options carried risks of infections and low blood-cell counts, the researchers reported. The overall infection rates were similar.

All participants had severe scleroderma that affected their lungs or kidneys. The researchers noted that their study had limitations that suggest the findings may not apply to all patients with scleroderma.
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Scleroderma Findings Published

The researchers have published their findings in the New England Journal of Medicine. The study was led by Keith Sullivan, MD, of Duke University.

The research was supported by the National Institutes of Health's National Institute of Allergy and Infectious Diseases, grants N01-AI05419, HHSN272201100025C, N01-AI25481, HHSN272200900057C and 1UMZAI117870.

To learn more about scleroderma, visit https://uvahealth.com/services/dermatology/skin-conditions/scleroderma. To keep up with the latest medical research news from UVA, subscribe to the Making of Medicine blog at http://makingofmedicine.virginia.edu.

University of Virginia Health System

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Scleroderma: The Proven Therapy that Can Save Your Life
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Scleroderma, which affects as many as 400,000 Americans, starts off like skin cancer but is far more deadly. This new edition is updated with new information about the best therapy for this disease, including the results of the landmark first, and a new, second clinical trial of the only therapy to report reversal and remission of this deadly disease. View Details


The First Year: Scleroderma: An Essential Guide for the Newly Diagnosed (The First Year Series)
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Scleroderma—a chronic autoimmune condition that causes hardening, thickening, or tightening of the skin and attacks the heart, lungs, kidneys, and gastrointestinal tract—is extraordinarily difficult to diagnose and can take a huge toll on the psychological well-being of the individual. From the first moment of her diagnosis, author Karen Gottesman took charge and educated herself on every aspect of her condition. Now, as a "patient-expert," she guides those newly diagnosed step by step through their first year with scleroderma. She provides crucial information about the nature of the... View Details


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SCLERODERMA: Symptoms, Causes, Diagnosis and Effective Treatments – Official Guide from the National Institute of Skin Diseases

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Breathing Again. . . A Joyous Miracle: My Journey with Scleroderma
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Focused on her busy career, rising through the ranks of management in high-tech, it was easy for Linda Edwards to dismiss individual symptoms as they appeared, especially when doctors told her they were not life-threatening. Even with her eventual diagnosis of scleroderma, her positive outlook, resilience, and faith kept her pushing through life. It wasn’t until she was facing death, frail, and her breathing compromised, that she realized it would take a miracle to save her. Join Linda on her journey of survival. In Breathing Again… A Joyous Miracle: My Journey with Scleroderma, she... View Details


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The Scleroderma Book: A Guide for Patients and Families by Maureen D. Mayes (1999-07-15)
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An autoimmune disease in the family with Lupus, Rheumatoid Arthritis, and Polymyositis, Scleroderma afflicts hundreds of thousands of people in the United States. And yet there are very few sources of information for men and women with this disease. Now, in The Scleroderma Book, one of the nation's leading authorities provides a comprehensive guide written specifically for patients and their families.
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