Amgen presents data from first Nplate study in children with chronic ITP

NEW ORLEANS A drug made by biotech company Amgen was effective at treating a serious bleeding disorder in children, according to results of an early-stage, placebo-controlled clinical study presented at the annual meeting of the American Society of Hematology in New Orleans Monday.

Amgen said the drug Nplate (romiplostim) was effective in treating thrombocytopenia resulting from immune thrombocytopenic purpura in children ages 1 to 18, with 88% of the 17 patients achieving a blood platelet count of 50,000 per microliter during two consecutive weeks of treatment. ITP is an autoimmune disorder that causes low platelet counts, which can lead to heavy bleeding events.

Safety results showed that adverse side effects were similar between patients who received Nplate and those who received placebo, with side effects being mild to moderate in severity.

“Currently, most drug treatment options for children with chronic ITP involve immunosuppression,” University of Texas Southwestern Medical Center pediatrics professor George Buchanan stated. “This is the first study of an agent that stimulates platelet production in the pediatric population.”