Romiplostim in adults with newly diagnosed or persistent immune thrombocytopenia

Abstract

Introduction: Three distinct phases are recognized in immune thrombocytopenia (ITP): newly diagnosed (≤3 months after diagnosis), persistent (>3-12 months after diagnosis), and chronic (>12 months). Several international guidelines/expert recommendations have been released in the past 2 years regarding the treatment of newly diagnosed/persistent ITP. Areas covered: Across the guidelines/expert recommendations, thrombopoietin receptor agonists (TPO-RAs), including romiplostim (the focus of this review), are recommended in newly diagnosed or persistent ITP for patients who fail to respond to corticosteroids or intravenous immunoglobulin (or where these are contraindicated). To identify data relating to romiplostim in adults with newly diagnosed or persistent ITP, we conducted a search of PubMed (with no time limit applied) and abstracts from 2019 EHA/ASH meetings using the term 'romiplostim.' Expert opinion: The findings from nine clinical trials, six real-world studies and ten case reports provide insight into the early use of romiplostim, which could help to reduce exposure to the adverse effects associated with prolonged corticosteroid use, as well as reduce the risk of severe bleeding. Additionally, given the durable responses observed in patients with newly diagnosed/persistent ITP, as well as the potential for treatment-free responses following discontinuation, romiplostim might help to avoid the need for subsequent treatment.

Effectiveness and safety of romiplostim among patients with newly diagnosed, persistent and chronic ITP in routine clinical practice in central and Eastern Europe: an analysis of the PLATON study

“…A number of observational studies and case reports demonstrate that romiplostim has already been used in the newly diagnosed and persistent settings in clinical practice (although off-label at the time) [21]. This realworld evidence from unselected daily practice supports finding from clinical trials that romiplostim is effective and well tolerated in patients <1 year from ITP diagnosis (see review by Lozano et al [21]).…”

“…A number of observational studies and case reports demonstrate that romiplostim has already been used in the newly diagnosed and persistent settings in clinical practice (although off-label at the time) [21]. This realworld evidence from unselected daily practice supports finding from clinical trials that romiplostim is effective and well tolerated in patients <1 year from ITP diagnosis (see review by Lozano et al [21]). For example, Snell Taylor et al [18,23] recently reported that 32% of 64 newly diagnosed, 53% of 50 persistent, and 49% of 226 chronic ITP patients achieved a durable platelet response (same definition as used within our study) and all non-romiplostim ITP medications were discontinued in 74% of newly diagnosed and over half of persistent (69%) and chronic (51%) ITP patients.…”

“…In recent guidelines [1,[12][13][14], TPO-RAs are generally recommended as a second-line treatment in the persistent phase of ITP (after three months). A key potential benefit of using romiplostim earlier in the treatment paradigm (rather than in the chronic setting) is to help avoid unnecessary adverse events associated with prolonged use of steroids, thereby potentially improving quality of life [21]. Indeed, guidelines recommend keeping the initial treatment period with corticosteroid-associated side effects to no longer than 8 weeks [1,[12][13][14].…”

“…In clinical studies, romiplostim treatment led to sustained platelet responses in adults with newly diagnosed or persistent ITP [17]. While evidence from real-world studies and case reports indicates that romiplostim is already being used in the newly diagnosed and persistent settings [18][19][20][21], efficacy and safety data are based on studies with relatively small numbers of patients. The aim of the present research was to analyse previously published data from the PLATON (Prospective Analysis of Management of ITP Patients) study [19] according to duration of primary ITP to extend the knowledge base on the effectiveness and safety of romiplostim in adults with newly diagnosed and persistent ITP.…”

Novel therapies for immune thrombocytopenia

“…Of the four current TPO receptor agonists, three have been approved by multiple regulatory agencies for treating ITP (romiplostim, eltrombopag, avatrombopag) and the fourth (lusutrombopag) only for patients with chronic liver disease undergoing procedures. 6,131,132 A fourth non-peptide TPO receptor agonist, hetrombopag olamine, is currently under development in China for several indications and was approved in China for second-line treatment of ITP on 16 June 2021. 133 Its structure and function are similar to those of eltrombopag (Fig 4).…”