Eltrombopag Raises Platelet Blood Cell Count And Decreases Bleeding In Patients With Clotting Cell Shortage

Main Category: Blood / Hematology
Article Date: 20 Feb 2009 - 5:00 PDT

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A shortage of the blood cells which help clotting (platelets) can be corrected by used of the drug eltrombopag. This is the conclusion of an Article in this week's edition of The Lancet, written by Dr James B Bussel, Weill-Cornell Medical College of Cornell University, New York, USA, and colleagues.

Idiopathic thrombocytopenic purpura (ITP)is is the condition of having a low platelet count (thrombocytopenia ) of no known cause (idiopathic ), which can cause bruising (purpura) or more serious bleeding events. It is usually autoimmune. In this phase III randomised controlled trial, the authors studied 114 patients from 63 sites in 23 countries, all with ITP, and platelet counts of less than 30000 per µL of blood (normal range is 150,000-400,000), and one or more previous ITP treatment. They were randomised to receive either standard care plus once-daily eltrombopag (76 patients) or placebo (38). After three weeks, patients who still had platelet counts less than 50,000 per µL could increase study drug to 75mg. The primary endpoint was the proportion of patients achieving platelet counts of 50,000 per µL or more by day 43.

The researchers found that 59% of eltrombopag patients and 16% of placebo patients achieved the platelet count of 50,000 per µL. Statistical analysis revealed that eltrombopag patients were almost 10 times more likely to reach the target platelet count as placebo patients. Of the 34 eltrombopag patients who increased their dose of eltrombopag, 29% responded. Generally, platelet counts returned to the patients' baseline levels within two weeks after the end of treatment. Eltrombopag patients were around half as likely to have bleeds at any time during the study than placebo patients. Serious adverse events, including those leading to treatment discontinuation, were rare in both groups.

The authors conclude: "In this large, multicentre, randomised, placebo-controlled trial of eltrombopag treatment in chronic ITP, substantially more patients in the eltrombopag group achieved platelet counts of 50 000 per μL or greater than did those in the placebo group. Increases of platelet counts to 50 000 per μL or greater were seen within two weeks in more than half of patients given eltrombopag. In conjunction with the rise in platelet count, a prospective assessment showed a significant reduction in bleeding events both during and at the end of the study."

"Eltrombopag is an effective treatment for managment of thrombocytopenia in chronic ITP."

In an accompanying Comment, Professor Simon Panzer and Professor Ingrid Pabinger, Medical University Vienna, Austria, say: "The role of eltrombopag in long-term treatment, and in deferring splenectomy, will be determined by its price and especially by its safety in long-term observational studies."

The Lancet

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