Scleroderma Treatment is a collection of diseases of the skin and connective tissues. The disease results in a hardening of the skin and tightening of connective tissues throughout the patient’s body. This callusing is caused by an overproduction and buildup of collagen in body tissue. Scleroderma can affect the patient’s internal organs, digestive tract, blood vessels, and skin.
The cause of this autoimmune disease is unknown, but symptoms are often brought on by exposure to airborne and chemical pollutants. It is a long-term disease which is also called progressive systemic sclerosis. The disease typically affects patients between 25 and 55 years of age and is more prevalent in women at a rate of 4 to 1 over men.
Children also can contract scleroderma which is typically a type of the disease called localized scleroderma.
Scleroderma manifests in different ways depending on the affected organs. There are two types of systemic scleroderma. The limited form also called limited systemic sclerosis primarily only affects the skin of the patient. The second form of the disease is the diffuse form, also known as diffuse systemic sclerosis. This form of the disease also affects the skin but additionally impacts internal organs including the lungs and kidneys of the patient.
Common Symptoms of Scleroderma
Skin
- Hardening and tightening of various areas of the skin affect almost every patient with scleroderma. Based on the type of scleroderma determines the size and area of the skin that is involved.
Fingers and Toes/Raynaud’s Disease
- An early symptom of scleroderma is intense reactions to emotional stress and cold temperatures. This response results in discoloration, pain, and numbness in the fingers and toes, which is named Raynaud’s disease.
Digestive System
- Difficulty absorbing nutrients
- Slow motility of digestive systemorbing nutrients
- Acid reflux
Common Symptoms associated with References on Scleroderma
Sullivan KM, Goldmuntz EA, Keyes-Elstein L, McSweeney PA, Pinckney A, Welch B, Mayes MD, Nash RA, Crofford LJ, Eggleston B, Castina S, Griffith LM, Goldstein JS, Wallace D, Craciunescu O, Khanna D, Folz RJ, Goldin J, St Clair EW, Seibold JR, Phillips K, Mineishi S, Simms RW, Ballen K, Wener MH, Georges GE, Heimfeld S, Hosing C, Forman S, Kafaja S, Silver RM, Griffing L, Storek J, LeClercq S, Brasington R, Csuka ME, Bredeson C, Keever-Taylor C, Domsic RT, Kahaleh MB, Medsger T, Furst DE; SCOT Study Investigators.
Myeloablative Autologous Stem-Cell Transplantation for Severe Scleroderma. N Engl J Med. 2018 Jan 4;378(1):35-47. doi: 10.1056/nejmoa1703327. PMID: 29298160; PMCID: PMC5846574. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5846574/
BACKGROUND: Despite current therapies, diffuse cutaneous systemic sclerosis (scleroderma) often has a devastating outcome. We compared myeloablative CD34+ selected autologous hematopoietic stem-cell transplantation with immunosuppression by means of 12 monthly infusions of cyclophosphamide in patients with scleroderma.
METHODS: We randomly assigned adults (18 to 69 years of age) with severe scleroderma to undergo myeloablative autologous stem-cell transplantation (36 participants) or to receive cyclophosphamide (39 participants). The primary end point was a global rank composite score comparing participants with each other on the basis of a hierarchy of disease features assessed at 54 months: death, event-free survival (survival without respiratory, renal, or cardiac failure), forced vital capacity, the score on the Disability Index of the Health Assessment Questionnaire, and the modified Rodnan skin score.
RESULTS: In the intention-to-treat population, global rank composite scores at 54 months showed the superiority of transplantation (67% of 1404 pairwise comparisons favored transplantation and 33% favored cyclophosphamide, P = 0.01). In the per-protocol population (participants who received a transplant or completed ≥9 doses of cyclophosphamide), the rate of event-free survival at 54 months was 79% in the transplantation group and 50% in the cyclophosphamide group (P = 0.02).
At 72 months, Kaplan–Meier estimates of event-free survival (74% vs. 47%) and overall survival (86% vs. 51%) also favored transplantation (P = 0.03 and 0.02, respectively). A total of 9% of the participants in the transplantation group had initiated disease-modifying antirheumatic drugs (DMARDs) by 54 months, as compared with 44% of those in the cyclophosphamide group
P = 0.001). Treatment- related mortality in the transplantation group was 3% at 54 months and 6% at 72 months, as compared with 0% in the cyclophosphamide group.
CONCLUSIONS: Myeloablative autologous hematopoietic stem-cell transplantation achieved long-term benefits in patients with scleroderma, including improved event-free and overall survival, at a cost of increased expected toxicity. Rates of treatment-related death and post-transplantation use of DMARDs were lower than those in previous reports of nonmyeloablative transplantation. (Funded by the National Institute of Allergy and Infectious Diseases and the National Institutes of Health; ClinicalTrials.gov number, NCT00114530.)
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