January 01, 2013
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Eltrombopag maintained efficacy after cessation in immune thrombocytopenia

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Approximately one-third of patients with immune thrombocytopenia who were treated with a thrombopoietin-receptor agonist maintained adequate platelet counts 6 months after stopping treatment, according to study results.

Perspective from Keith McCrae, MD

Researchers conducted the study to evaluate patients with immune thrombocytopenia who had electively discontinued eltrombopag (Promacta, GlaxoSmithKline) without substituting additional therapy. They wanted to observe what impact, if any, this discontinuation would have on platelet counts.

Fifteen patients who had been taking the drug for a minimum of 4 months before the start of the study were included. A platelet count of ≥30,000/mcL and ≥20,000/mcL above initial baseline for 6 months off eltrombopag without intervening treatment (other than rescue) served as the primary endpoint. Being stable off therapy at 4 weeks after discontinuing eltrombopag served as the secondary endpoint.

Five of the patients enrolled in the study demonstrated response for 5 months or longer, while 10 patients were classified as non-responders. The fifth responder discontinued therapy 5 months ago and had platelet counts ≥195,000/mcL.

Two of the responders had undergone splenectomy, and all had three or more previous therapies.

Non-significant factors associated with response included age, duration of immune thrombocytopenia, duration of eltrombopag therapy, splenectomy status, number of prior immune thrombocytopenia treatments, bleeding history and platelet count at the time that eltrombopag was discontinued.

Responders demonstrated a lower absolute immature platelet fraction value when treatment was stopped (P=.022).

Reference:

Leven E. Abstract #1085.

Disclosure:

Leven reports no relevant financial disclosures.