New results indicate that the once-daily, non-peptide oral platelet growth factor eltrombopag (RevoladeTM/PromactaTM) boosts platelet counts and cuts bleeding in adults with chronic idiopathic thrombocytopenia purpura (ITP) who have failed conventional ITP treatments.
The results, from a phase III trial, were reported at the 12th Congress of the European Hematology Association (EHA).
“While ITP and its potentially severe clinical consequences have long been recognized, there has been a disproportionate lack of progress in treatment,” Adrian Newland, MD, an investigator in the ITP Clinical Trials Program and Professor of Hematology at Barts and the London NHS Trust in London, UK, said at a news conference. “This is unfortunate since thrombocytopenia predisposes patients to bleeding and can lead to postponements in surgery and medical treatments such as cancer chemotherapy as well as antiviral therapy in patients with hepatitis C.”
The new results were presented at the conference by Drew Provan, MD, Senior Lecturer in the Hematology Division at the Royal London Hospital in London, UK.
Overall, 114 patients were randomly assigned to six weeks of treatment with eltrombopag, 50 mg, or placebo. Dose escalation to 75 mg or matching placebo was permitted after three weeks in subjects whose platelet count remained below 30,000 /µL
At the start of the trial, patients had chronic ITP with platelet counts less than 30,000/µL and had failed or become unresponsive to conventional ITP treatments within the past three months which included corticosteroids, immunoglobulins, and/or splenectomy.
The treatment groups were similar with respect to baseline demographic and clinical characteristics.
Results showed that 59 percent of eltrombopag-treated patients and 16 percent of placebo-treated patients achieved the study’s primary endpoint of a platelet count greater than or equal to 50,000/ µL with up to six weeks of dosing, p less than 0.001.
Clinically significant bleeding, defined as World Health Organization (WHO) grades 2 to 4, occurred in 16 percent of the eltrombopag and 36 percent of the placebo group, p=0.029.
Dr. Provan said that the phase III results confirm the effectiveness of eltrombopag for the short-term treatment of patients with chronic ITP and added that long-term and repetitive treatment studies are presently underway in patients with chronic ITP.
Eltrombopag is an investigational compound that has not received regulatory approval in any market for any indication at this time.
The study was sponsored by GlaxoSmithKline.
Jill Steing
Jill Stein is a Paris-based freelance medical writer.
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