Results from a systematic review and network meta-analysis of clinical outcomes in patients with persistent immune thrombocytopenia (ITP) suggest second-line treatment with the thrombopoietin receptor stimulators eltrombopag and romiplostim provides high efficacy and safety, especially when compared with treatment with rituximab.

In this analysis, published in the British Journal of Haematology, researchers assessed clinical outcomes in patients with persistent ITP, who require second-line therapy. They included 12 randomized controlled trials with a total of 1313 adults with persistent ITP; the included trials reported information on platelet response, platelet count, and any bleeding or serious adverse events.

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Eltrombopag and romiplostim had the best platelet response outcomes compared with placebo, though platelet response outcomes of eltrombopag did not yield a statistically significant advantage (risk ratio [RR], 1.10; 95% CI: 0.46-2.67).

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Both eltrombopag and romiplostim were better than rituximab (RR for eltrombopag, 4.56; 95% CI: 1.89-10.96; RR for romiplostim, 4.13; 95% CI: 1.56-10.94) and recombinant human thrombopoietin (rhTPO) plus rituximab (RR for eltrombopag, 4.18; 95% CI: 1.21-14.49; RR for romiplostim, 3.79; 95% CI: 1.02-14.09).

Romiplostim ranked the highest for improving platelet count, followed by eltrombopag, rhTPO plus rituximab, and rituximab. Rituximab had the lowest risk for bleeding, followed by eltrombopag and romiplostim.

The highest risk for serious adverse events occurred with rhTPO plus rituximab, followed by rituximab, eltrombopag, and romiplostim.

Results from clustered ranking indicated romiplostim had the best balance between short-term efficacy and serious adverse events. Eltrombopag was second best in this clustered ranking.

“Considering clinical efficacy and adverse events simultaneously using clustered ranking indicated that the treatment with the best balance between high short-term efficacy with regard to platelet response, platelet count, low risk of bleeding, and adverse events was romiplostim, followed by eltrombopag. Rituximab had the lowest clinical efficacy and highest risk for serious adverse events,” wrote the authors.

Though these results indicate good efficacy and safety of romiplostim and eltrombopag and suggest rituximab may not be beneficial, the authors noted that randomized clinical trials with long-term clinical outcomes are needed. Additionally, impact analysis and assessment of cost effectiveness would improve clinical understanding of second-line therapy for persistent adult ITP.


1. Puavilai T, Thadanipon K, Rattanasiri S, et al. Treatment efficacy for adult persistent immune thrombocytopenia: a systematic review and network meta-analysis. Br J Haematol. doi:10.1111/bjh.16161