Eltrombopag treatment of patients with secondary immune thrombocytopenia: retrospective EHR analysis

Abstract

Immune thrombocytopenia (ITP) may occur in isolation (primary) or in association with a predisposing condition (secondary ITP [sITP]). Eltrombopag is a well-studied treatment for primary ITP, but evidence is scarce for sITP. We evaluated real-world use of eltrombopag for sITP using electronic health records. Eligible patients had diagnoses of ITP and a qualifying predisposing condition, and eltrombopag treatment. We described patient characteristics, treatment patterns, platelet counts, and thrombotic and bleeding events. We identified 242 eligible patients; the most common predisposing conditions were hepatitis C and systemic lupus erythematosus. Average duration of eltrombopag treatment was 6.1 months. Most (81.4%) patients achieved a platelet count ≥ 30,000/µL at a mean of 0.70 months, 70.2% reached ≥ 50,000/µL at a mean of 0.95 months, and 47.1% achieved a complete response of > 100,000/µL at a mean of 1.43 months after eltrombopag initiation. At eltrombopag discontinuation, 105 patients (43%) experienced a treatment-free period for a mean 3.3 months. Bleeding events occurred with similar frequency before and during eltrombopag treatment whereas thrombotic events were less frequent during eltrombopag treatment. Our results suggest similar rates of platelet response with eltrombopag in patients with sITP as compared with primary ITP. In addition, a treatment-free period is possible for a substantial minority of patients.

Expert Report on Immune Thrombocytopenia: Current Diagnostics and Treatment – Recommendations from an Expert Group from Austria, Germany, and Switzerland

“…Secondary ITP frequently relapses after corticosteroid therapy but seems to respond well to TPO-RAs [99], although most of these agents are approved only for primary ITP. An uncomplicated and timely commitment for health insurance to cover the cost of off-label TPO-RAs would be desirable.…”

Expertenreport Immunthrombozytopenie – Aktuelle Diagnostik und Therapie

“…Wenn die Thrombozytopenie im Vordergrund steht, wird man sich nach dem üblichen Vorgehen wie bei der primären ITP richten. Während die sekundäre ITP nach Glukokortikoiden häufig rezidiviert scheint sie auf TPO-RAs gut anzusprechen [99]. Leider sind alle TPO-RAs arzneimittelrechtlich nur für die primäre, nicht für die sekundäre ITP zugelassen.…”