General Medicine

Lasting Benefits for Scleroderma With Stem-Cell Transplantation

Scleroderma is a rare autoimmune disease that leads to hardening of connective tissue; it is estimated that fewer than 500,000 people in the United States have scleroderma.17 The etiology of scleroderma remains unclear; however, many patients have a personal or family history of thyroid diseases and other autoimmune disorders. In one small study, 7 of 18 patients with systemic scleroderma had a familial history of thyroid disease and 8 of 18 had a thyreopathy.18 Because of the high prevalence of thyroid disorders in patients with systemic scleroderma, routine screening for thyroid disorders might be warranted, especially in patients with risk factors for thyroid disorders and/or atypical clinical follow-up.18 Despite being a heterogeneous disorder, almost all patients have skin involvement manifest as a loss of cutaneous elasticity followed by skin thickening.19 Hands and fingers are often the first areas affected. Calcinosis, pruritus from dry skin, and joint contracture are other common findings.19 Image credit: ISM / CID

Scleroderma is a rare autoimmune disease that leads to hardening of connective tissue; it is estimated that fewer than 500,000 people in the United States have scleroderma.17 The etiology of scleroderma remains unclear; however, many patients have a personal or family history of thyroid diseases and other autoimmune disorders. In one small study, 7 of 18 patients with systemic scleroderma had a familial history of thyroid disease and 8 of 18 had a thyreopathy.18 Because of the high prevalence of thyroid disorders in patients with systemic scleroderma, routine screening for thyroid disorders might be warranted, especially in patients with risk factors for thyroid disorders and/or atypical clinical follow-up.18

Despite being a heterogeneous disorder, almost all patients have skin involvement manifest as a loss of cutaneous elasticity followed by skin thickening.19 Hands and fingers are often the first areas affected. Calcinosis, pruritus from dry skin, and joint contracture are other common findings.19 Image credit: ISM / CID

Myeloablative autologous hematopoietic stem-cell transplantation linked to improved survival in severe scleroderma.

HealthDay News — For patients with scleroderma, myeloablative CD34+ selected autologous hematopoietic stem-cell transplantation is associated with lasting benefits, according to a study published in the New England Journal of Medicine.

Keith M. Sullivan, MD, from the Duke University Medical Center in Durham, NC, and colleagues randomized adults with severe scleroderma to undergo myeloablative autologous stem-cell transplantation or receive cyclophosphamide (36 and 39 participants, respectively).

The researchers found that at 54 months the global rank composite scores showed the superiority of transplantation (67 and 33% of 1404 pairwise comparisons favored transplantation and cyclophosphamide, respectively; P =.01) in the intention-to-treat population. The rate of event-free survival at 54 months was 79 and 50% in the transplantation and cyclophosphamide groups, respectively, in the per-protocol population (P =.02). Transplantation was also favored in Kaplan-Meier estimates of event-free survival (74 vs 47%; P =.03) and overall survival (86 vs 51%; P =.02) at 72 months. By 54 months, 9 and 44% of patients in the transplantation and cyclophosphamide groups, respectively, had initiated disease-modifying antirheumatic drugs (P =.001). Treatment-related mortality was 3 and 6% at 54 and 72 months, respectively, in the transplantation group versus 0% in the cyclophosphamide group.

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“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,” the authors wrote.

Disclosures: One author disclosed financial ties to Novartis.

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Reference

Sullivan KM, Goldmuntz EA, Keyes-elstein L, et al. Myeloablative autologous stem-cell transplantation for severe scleroderma. N Engl J Med. 2018;378(1):35-47.

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