Romiplostim for the treatment of chronic immune (idiopathic) thrombocytopenic purpura

What Was Tested

This randomized controlled trial paper looks at Romiplostim.

On August 22, 2008, Romiplostim (Nplate for Injection) received approval from the US Food and Drug Administration (FDA) for the treatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenic purpura (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.

This report summarizes the FDA analyses of the clinical data supporting this approval.

Who Took Part

The age groups tagged for this paper are adults, middle-aged adults, older adults and people over 80, both men and women.

In the controlled studies, enrolled patients had completed at least one prior treatment for chronic ITP and had a platelet count < or = 30 x 10(9)/L.

One study enrolled patients who had undergone splenectomy; the other enrolled patients who had not undergone splenectomy.

How the Study Was Run

The design is described as randomized, double-blind and placebo-controlled.

Because people in this Romiplostim study were split into groups at random, differences between the groups are less likely to reflect who happened to join each one.

Blinding on both sides kept the participants and researchers unaware of who received Romiplostim.

The comparison arm used a placebo, so changes seen with Romiplostim could be set against what happens without it.

The FDA reviewed data from two double-blind, placebo-controlled clinical studies, an uncontrolled extension study, and supportive studies.

The primary endpoint in both controlled studies was durable platelet response.

What Was Measured

PubMed files the paper under topics including Carrier Proteins, Chronic Disease, Placebo Effect, Platelet Count, Prognosis, Purpura, Thrombocytopenic, Idiopathic and Receptors, Fc.

What This Means in Plain Language

The paper is a randomized, double-blind and placebo-controlled study of Romiplostim.

Its findings take up roughly 8 sentences of the abstract and are left out here on purpose; the source paper is the place to read them.

The publication is from 2009 (Oncology (Williston Park, N.Y.)), a journal based in United States.

More context: the Romiplostim compound page, all human trials.