Significant improvement in spleen volume for untreated adults with type 1 disease
TUESDAY, Feb. 17, 2015 (HealthDay News) — A novel oral substrate reduction therapy, eliglustat, can safely reverse clinical manifestations in untreated adults with Gaucher’s disease type 1, according to a study published in the Feb. 17 issue of the Journal of the American Medical Association.
Pramod K. Mistry, M.D., Ph.D., from the Yale University School of Medicine in New Haven, Conn., and colleagues examined whether eliglustat safely reverses clinical manifestations in untreated adults with Gaucher’s disease type 1. Forty patients were stratified by spleen volume and randomized in a 1:1 ratio to receive eliglustat or placebo for nine months.
The researchers observed a 27.77 percent decrease in the least-square mean spleen volume in the eliglustat group, compared with a 2.26 percent increase in the placebo group (absolute treatment difference, −30.03 percent; P < 0.001). The least-square mean absolute differences between groups favored eliglustat for secondary end points, with a 1.22-g/dL increase in hemoglobin level (P < 0.001), 6.64 percent reduction in liver volume (P = 0.007), and 41.06 percent increase in platelet count (P < 0.001). There were no serious adverse events; one patient withdrew from the eliglustat group (non-treatment related).
“The clinical significance of these findings is uncertain, and more definitive conclusions about clinical efficacy and utility will require comparison with the standard treatment of enzyme replacement therapy as well as longer-term follow-up,” the authors write.
Several authors disclosed financial ties to pharmaceutical companies, including Genzyme, which manufactures eliglustat and funded the trial.
Abstract
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