FDA Approves Medication to Treat Pediatric Patients With Immune Thrombocytopenia

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The Food and Drug Administration has approved Nplate for pediatric patients 1 year of age and older with immune thrombocytopenia who have had an insufficient response to medications, such as corticosteroids,or surgery to remove the spleen.

The Food and Drug Administration (FDA) has approved Nplate (romiplostim), an injectable protein medication, for the treatment of pediatric patients, who are 1 year old or older, with immune thrombocytopenia (ITP) for at least six months. These patients also have to have had an insufficient response to other medications, such as corticosteroids, or surgery to remove the spleen.

ITP, which can affect children and adults, is a condition in which platelets (part of the blood that helps control bleeding) are destroyed by the immune system. The low platelet count may cause patients to bruise and bleed easily.

The FDA based its approval on two double-blind, placebo-controlled trials in this patient population. The first study included 62 patients with relapsed/refractory disease, who had one or more previous ITP treatment, to either receive Nplate or placebo. Researchers determined that 52 percent of patients in the Nplate group achieved durable platelet response compared with 10 percent of the placebo group. Seventy-one percent versus 20 percent of patients in the both groups, respectively, had overall platelet response.

In the second trial, 22 patients randomly received Nplate or placebo. Eighty-eight percent of patients in the Nplate group reached the desired endpoints regarding platelet count and an increase in platelet count for 2 consecutive weeks. Neither outcome measured was reached by any patient treated with placebo.

More than 25 percent of patients experienced all-grade side effects, including bruising, upper respiratory tract infection and pain that affects the throat and neck.

Data were recently presented at the 2018 American Society of Hematology Annual Meeting for an integrated analysis of data from five clinical trials that showed that most pediatric patients with ITP had rapid and durable platelet responses when treated with romiplostim.

ITP affects at least 3,000 children under the age of 16 each year in the United States.