Efficacy and safety of romiplostim in adult Iraqi patients with refractory immune thrombocytopenia

Abstract

BACKGROUND: Immune thrombocytopenia (ITP) is an immune-mediated acquired disease characterized by decrease of the platelet count. The majority of adult patients progress to chronic stage with some of them failed to respond or relapsed after the second-line therapy. Romiplostim is a thrombopoietin receptor agonist recently approved for patients with chronic ITP, it was recently licensed and introduced for use in Iraq. The aim of this study was to assess the efficacy and safety of romiplostim among patients with refractory ITP. PATIENTS AND METHODS: This prospective study was conducted between April 2017 and June 2018 in two hematology centers in Baghdad and Basra. A group of 56 adult patients with refractory ITP enrolled in this study. The patients were evaluated before the weekly scheduled romiplostim and followed for efficacy and safety of the treatment. RESULTS: the median age was 39 years with a female-to-male ratio of 4:1. The response rate (platelet ≥50 × 109/l) was observed in 75% of the enrolled patient. The time to initial response was 2.5 weeks, and the mean dose of romiplostim used to achieve a sustained response was 3.1 mcg/kg. The response rate was significantly higher among splenectomized (94.4% vs. 65.8%). There was no difference in response regarding the age and gender. The most frequent adverse effects were joint pain (35.7%), followed by headache (32.1%) and fatigue (21.4%), resolved spontaneously within 2 days. Six patients developed thrombocytosis without any thrombotic event. CONCLUSIONS: Romiplostim is an effective option among Iraqi patients with refractory ITP as a long-term treatment or as a bridge for another intervention, with a relatively safe toxicity profile.

Keywords

• efficacy
• immune thrombocytopenia
• refractory
• romiplostim
• safety