Treatment efficacy for adult persistent immune thrombocytopenia: a systematic review and network meta‐analysis

Abstract

Persistent immune thrombocytopenia (ITP) patients require second-line treatments, for which information on clinical outcomes are lacking. A systematic review and network meta-analysis (NMA) were conducted. Only randomised controlled trials (RCT) of second-line drugs in adult persistent ITP patients with platelet response, platelet count, any bleeding or serious adverse events (SAE) outcome were eligible. Twelve RCTs (n = 1313) were included in NMA. For platelet response outcome, eltrombopag and romiplostin were the best relative to placebo; the former had a non-significant advantage [risk ratio (RR) = 1Á10 (95% confidence interval: 0Á46, 2Á67)]. Both treatments were superior to rituximab and recombinant human thrombopoietin (rhTPO)+rituximab, with corresponding RRs of 4Á56 (1Á89, 10Á96) and 4Á18 (1Á21, 14Á49) for eltrombopag; 4Á13 (1Á56, 10Á94) and 3Á79 (1Á02, 14Á09) for romiplostim. For platelet count, romiplostim ranked highest, followed by eltrombopag, rhTPO+rituximab, and rituximab. For bleeding, rituximab had lowest risk, followed by eltrombopag and romiplostim. For SAEs, rhTPO+rituximab had highest risk, followed by rituximab, eltrombopag and romiplostim. From clustered ranking, romiplostim had the best balance between short-term efficacy and SAEs, followed by eltrombopag. In conclusion, romiplostim and eltrombopag may yield high efficacy and safety. Rituximab may not be beneficial due to lower efficacy and higher complications compared with the thrombopoietin receptor agonists. RCTs with long-term clinical outcomes are required.

Mediterranean Diet Maintained Platelet Count within a Healthy Range and Decreased Thrombocytopenia-Related Mortality Risk: A Randomized Controlled Trial

“…Beyond the maintenance of platelet count within normal ranges, MedDiet interventions appeared to lower the risk of developing thrombocytopenia in older adults at high cardiovascular risk, and attenuated all-cause mortality risk among individuals presenting the condition. Deficient levels of platelets and their consequences are traditionally addressed by pharmacological treatments [ 34 ]. However, to the best of our knowledge, this is the first intervention study to date to describe the protective effect of a healthy diet on the development of low platelet count and their associated mortality.…”

Second‐line treatments and outcomes for immune thrombocytopenia: A retrospective study with electronic health records

“…TPO‐RAs have been demonstrated to be effective and safe, although no direct comparisons between eltrombopag and romiplostim or between TPO‐RAs and other second‐line treatment options have been performed to date. Systematic reviews and network meta‐analyses have suggested similar efficacy of eltrombopag and romiplostim and superior efficacy of both agents compared with rituximab 12,13 . In the current study, key outcomes were compared among patients receiving second‐line treatment with eltrombopag, romiplostim, rituximab, or splenectomy.…”

“…The proportion of patients who experienced BREs in the 12month follow-up period differed significantly across treatment cohorts, ranging from 27.5% for eltrombopag to 35.6% for romiplostim (P = .01) ( Table 3) and superior efficacy of both agents compared with rituximab. 12,13 In the current study, key outcomes were compared among patients receiving second-line treatment with eltrombopag, romiplostim, rituximab, or splenectomy. In contrast to a clinical trial, a real-world sample provides a more diverse population of patients treated in a routine practice setting.…”

Primary Immune Thrombocytopenia: Novel Insights into Pathophysiology and Disease Management

“…On the other hand, progressive multifocal leukoencephalopathy seem to be rare [ 137 ]. Taken together, due to the lower efficacy and higher complications compared with TPO-RAs [ 138 ], rituximab should be avoided as first line therapy and used only if there is high evidence for remission [ 4 ].…”