Stem-Cell Transplantation Beneficial for Scleroderma

Myeloablative autologous hematopoietic stem-cell transplantation linked to improved survival


WEDNESDAY, Jan. 3, 2018 (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 Jan. 4 issue of the New England Journal of Medicine.

Keith M. Sullivan, M.D., from the Duke University Medical Center in Durham, N.C., 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 percent of 1,404 pairwise comparisons favored transplantation and cyclophosphamide, respectively; P = 0.01) in the intention-to-treat population. The rate of event-free survival at 54 months was 79 and 50 percent in the transplantation and cyclophosphamide groups, respectively, in the per-protocol population (P = 0.02). Transplantation was also favored in Kaplan-Meier estimates of event-free survival (74 versus 47 percent; P = 0.03) and overall survival (86 versus 51 percent; P = 0.02) at 72 months. By 54 months, 9 and 44 percent of patients in the transplantation and cyclophosphamide groups, respectively, had initiated disease-modifying antirheumatic drugs (P = 0.001). Treatment-related mortality was 3 and 6 percent at 54 and 72 months, respectively, in the transplantation group versus 0 percent in the cyclophosphamide group.

"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 write.

One author disclosed financial ties to Novartis.

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