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Safety and efficacy of non-first-line drugs in the treatment of immune thrombocytopenia: a systematic review and network meta-analysis

Xin Zhou, Mengran Li, Xiaohui Sui, Ai Li, Xiao-Yan Li, Ning-ning Shan

Expert Review of Hematology, 2025

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Abstract

ABSTRACT Background This systematic review and network meta-analysis(NMA) comprehensively evaluated the efficacy and safety of non-first-line drugs in the treatment of adult patients with immune thrombocytopenia (ITP). Researchdesign and methods PubMed, Web of Science, Embase, and the Cochrane Library were systematically searched. Randomized controlled trials (RCTs) investigating Count data were extracted in the form of event occurrences/non-occurrences. NMA was carried out via R. Results 29 RCTs were encompassed. In contrast to placebo, the avatrombopag 20 mg group demonstrated the highest PR (RR = 12.23, 95% CrI: 5.48–33.72). The combination of eltrombopag and danazol exhibited the lowest incidence of bleeding events (RR = 0.31, 95% CrI: 0.16–0.57), while the avatrombopag 5 mg group had the lowest incidence of SAEs (RR = 0.44, 95% CrI: 0.22–0.84). The comprehensive evaluation suggested that romiplostim, initiated at a dose of 1 μg/kg within a dose-adjustment regimen, may confer one of the most favorable benefit – risk profiles, with a relatively high PR (SUCRA = 75.1%) and a low incidence of bleeding events (SUCRA = 54.8%). Conclusions When initiating therapy with romiplostim at a dose of 1 μg/kg and subsequently titrating the dosage according to the patient’s platelet response, romiplostim may represent one of the most effective therapeutic options. Registration The study protocol for this systematic review was registered in the International Prospective Registry of…

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