Summary

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• Nplate (romiplostim) is indicated for the treatment of thrombocytopenia in adult patients with immune thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. It is also indicated for the treatment of thrombocytopenia in pediatric patients 1 year of age and older with ITP for at least 6 months who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.
• This summary is based on the review of eight systematic review(s)/meta-analysis(es). ^[1-8]
• Romiplostim significantly improved durable platelet response (RR=6.34, P=0.003) and overall response (RR=3.62, P=0.002) in pediatric ITP compared to placebo, with consistent platelet response observed in both pediatric and adult ITP populations.
• In adults with ITP, romiplostim was among the top-ranking treatments for platelet response and effectively reduced severe (grade ≥3) bleeding events, while no significant difference was observed in the necessity for rescue treatments in pediatric studies.
• Comparison among treatments indicated no clear superiority between romiplostim and eltrombopag in platelet response, while avatrombopag showed a higher overall platelet response rate compared to both romiplostim and eltrombopag in adults.
• There were no significant differences in the overall incidence of adverse events (AEs) between romiplostim and placebo in both pediatric and adult populations with ITP.
• The incidence of serious adverse events (SAEs) and thrombotic events in romiplostim users was not significantly different from control groups, with an odds ratio of 0.92 (95% CI: 0.14-6.13, P=0.93) for thrombotic events.
• Significant improvements in platelet response and reduction in bleeding events were observed in pediatric and adult ITP patients treated with romiplostim, while patients with myelodysplastic syndromes (MDS) experienced reduced bleeding events with mixed results in overall response rates.