Primary immune thrombocytopenia (ITP) treated with romiplostim in routine clinical practice: retrospective study from the United Kingdom ITP Registry

Doobaree, Indraraj Umesh; Newland, Adrian C.; McDonald, Vickie; Nandigam, Raghava; Mensah, Lesley; Leroy, Sandrine; Seesaghur, Anouchka; Patel, Hitan; Wetten, Sally

doi:10.1111/ejh.13221

Cited by 17 publications

(13 citation statements)

References 36 publications

(66 reference statements)

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“… 79 Data from observational, real-world studies, which often describe effects on concomitant medications, such as corticosteroid dose reduction or discontinuation, have also shown that romiplostim often improved platelet counts and reduced bleeding events and hospitalizations in patients. 81 , 82 The efficacy and safety results were similar to those observed in clinical trials. 81 …”

Section: Clinical Evidence and Rationale For Use Of Romiplostim In Chronic Itpsupporting

confidence: 78%

“…The patient had consecutive platelet counts ≥340 × 10 9 / L. 79 Data from observational, real-world studies, which often describe effects on concomitant medications, such as corticosteroid dose reduction or discontinuation, have also shown that romiplostim often improved platelet counts and reduced bleeding events and hospitalizations in patients. 81,82 The efficacy and safety results were similar to those observed in clinical trials. 81 Patients have also maintained sustained platelet counts after discontinuing romiplostim; 73,[83][84][85][86] whether this is related to romiplostim or reflects spontaneous improvement of ITP is not known.…”

Section: Clinical Evidence and Rationale For Use Of Romiplostim In Chronic Itpsupporting

confidence: 76%

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A Review of Romiplostim Mechanism of Action and Clinical Applicability

Bussel¹,

Soff²,

Balduzzi³

et al. 2021

DDDT

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Thrombocytopenia results from a variety of conditions, including radiation, chemotherapy, autoimmune disease, bone marrow disorders, pathologic conditions associated with surgical procedures, hematopoietic stem cell transplant (HSCT), and hematologic disorders associated with severe aplastic anemia. Immune thrombocytopenia (ITP) is caused by immune reactions that accelerate destruction and reduce production of platelets. Thrombopoietin (TPO) is a critical component of platelet production pathways, and TPO receptor agonists (TPO-RAs) are important for the management of ITP by increasing platelet production and reducing the need for other treatments. Romiplostim is a TPO-RA approved for use in patients with ITP in the United States, European Union, Australia, and several countries in Africa and Asia, as well as for use in patients with refractory aplastic anemia in Japan and Korea. Romiplostim binds to and activates the TPO receptor on megakaryocyte precursors, thus promoting cell proliferation and viability, resulting in increased platelet production. Through this mechanism, romiplostim reduces the need for other treatments and decreases bleeding events in patients with thrombocytopenia. In addition to its efficacy in ITP, studies have shown that romiplostim is effective in improving platelet counts in various settings, thereby highlighting the versatility of romiplostim. The efficacy of romiplostim in such disorders is currently under investigation. Here, we review the structure, mechanism, pharmacokinetics, and pharmacodynamics of romiplostim. We also summarize the clinical evidence supporting its use in ITP and other disorders that involve thrombocytopenia, including chemotherapy-induced thrombocytopenia, aplastic anemia, acute radiation syndrome, perisurgical thrombocytopenia, post-HSCT thrombocytopenia, and liver disease.

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Section: Clinical Evidence and Rationale For Use Of Romiplostim In Chronic Itpsupporting

confidence: 78%

Section: Clinical Evidence and Rationale For Use Of Romiplostim In Chronic Itpsupporting

confidence: 76%

A Review of Romiplostim Mechanism of Action and Clinical Applicability

Bussel¹,

Soff²,

Balduzzi³

et al. 2021

DDDT

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show abstract

“…initiated within the first year in 34% of patients despite the 'chronic' indication [62,73]. Similar results were seen in a UK study, where 27% initiated romiplostim before 1 year of diagnosis (12% within 3 months and 6% within 3-6 months) [63]. Likewise, in a Spanish observational study of patients receiving TPO-RAs, 16% were newly diagnosed and 17% had persistent disease [64].…”

Section: Real-world Evaluation Of Romiplostim In Adults With Newly DIsupporting

confidence: 56%

Romiplostim in adults with newly diagnosed or persistent immune thrombocytopenia

Lozano

Godeau

Grainger

et al. 2020

Expert Review of Hematology

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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.

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“…Treating mice with the TPO analog romiplostim triggered a proper response and did significantly increase platelet counts in these animals, which could be due to the already increased number of MK and MKP that may give the Gfi1b -DN mice a starting advantage. The effect of romiplostim, which is a drug already approved for some patients suffering from chronic idiopathic thrombocytopenia purpura, but was also reported in off-label uses such as treatment of persistent thrombocytopenia associated with stem cell transplantation or congenital amegakaryocytic thrombocytopenia, 48-50 confirmed that heterozygous Gfi1b -DN mutant MK are still functional. This finding is important because it suggests that patients suffering from GFI1B -RT could potentially respond to a treatment with thrombopoietic agonists such as romiplostim.…”

Section: Discussionmentioning

confidence: 74%