Safety and efficacy of eltrombopag plus pulsed dexamethasone as first‐line therapy for immune thrombocytopenia

Abstract

Summary Current first‐line treatments for immune thrombocytopenia (ITP) usually have transient effects and sustained platelet response off therapy remains low. We evaluated whether eltrombopag plus pulsed dexamethasone as first‐line therapy can increase the proportion of patients maintaining platelet counts >50 × 109/l for a prolonged period without further ITP therapy. Treatment consisted of eltrombopag 25–75 mg daily according to platelet response for 12 weeks plus dexamethasone, 40 mg daily for four consecutive days every four weeks for 1–3 courses. Primary endpoint was durable response off therapy defined as maintaining platelet counts >50 × 109/l for more than six months without further ITP therapy. Fifty ITP subjects were enrolled between November 2014 and March 2019. Out of 46 evaluable subjects, 26 (56·5%) had achieved the primary endpoint. The median platelet counts at six months off‐treatment follow‐up were 158 × 109/l. Only two out of 26 responders had relapsed at eight‐ and nine‐month follow‐up. The remaining 24 are still maintaining platelet counts >50 × 109/l, the longest over three years. All subjects tolerated treatment well and no Grade 3 or above adverse effects were reported. Eltrombopag plus pulsed dexamethasone as a first‐line therapy could result in durable response off therapy in a significant number of ITP subjects.

High‐dose dexamethasone plus recombinant human thrombopoietin vs high‐dose dexamethasone alone as frontline treatment for newly diagnosed adult primary immune thrombocytopenia: A prospective, multicenter, randomized trial

“…Initial response was achieved in 100% of patients at day 33, and SR was kept in 75% of patients at 6 months and 66.7% at 12 months. The promising results of this combination strategy as first‐line therapy for treatment‐naïve ITP were further validated in another single‐arm, pilot study, which reported a SR rate of 56.5% at 6 months 22 . However, the efficacy of corticosteroids plus TPO‐RAs as a frontline treatment for ITP has not yet been evaluated in a prospective, randomized trial.…”

“…The promising results of this combination strategy as first-line therapy for treatment-naïve ITP were further validated in another singlearm, pilot study, which reported a SR rate of 56.5% at 6 months. 22 However, the efficacy of corticosteroids plus TPO-RAs as a frontline treatment for ITP has not yet been evaluated in a prospective, randomized trial. Here, we reported the results of a prospective, multicenter, randomized, controlled, open-label, phase three clinical trial, in which we compared the efficacy and safety of HD-DXM plus rhTPO vs HD-DXM alone in newly diagnosed, treatment-naïve adult ITP patients.…”

Immune Thrombocytopenia (ITP): Current Limitations in Patient Management

“…As a combination for intensified first-line therapy, a single-arm study with dexamethasone followed by a 28-day course of eltrombopag in treatment-naïve subjects showed 83% of patients had an early response and 50% of patients had a complete response at six months [29]. Simultaneous combination of eltrombopag and high-dose dexamethasone therapy could generate a sustained, complete, long-term response in adult patients and be used as a viable first-line treatment [30]. TPO-RAs are not universally considered curative, although in one study, 30% of adult patients with ITP maintained platelet counts >50 × 10 9 /L after discontinuation of romiplostim [31].…”

Risk Factors and Psychological Analysis of Chronic Immune Thrombocytopenia in Children

“…Childhood immune thrombocytopenia is an acquired autoimmune disease characterized by a decrease in platelet count (<100×10 9 /L) and an increased risk of bleeding. 1,2 This thrombocytopenia may be due to reduced platelet production and immune-mediated destruction, but usually involves glycoprotein autoantibodies expressed on megakaryocytes. [3][4][5] ITP is the most common hemorrhagic disease in children, accounting for one-third of hemorrhagic diseases, 6 Its onset is usually acute and short-lived, with an incidence of approximately (2~5)/10 million.…”