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

Abstract

Background: Thissystematic review and network meta-analysis(NMA) comprehensively evaluated theefficacy and safety of non-first-line drugs in the treatment of adult patientswith 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 Systematic Reviews (PROSPERO) database (www.crd.york.ac.uk/prospero/), and it was allocated the PROSPERO identification number.

Keywords: ITP; Network meta-analysis; Non-first-line drugs; Thrombopoietin receptor agonists; cost-effectiveness.