High dose romiplostim as a rescue therapy for adults with severe bleeding and refractory immune thrombocytopenia

Roumier, Mathilde; Burel, S. Le; Audia, Sylvain; Chauchet, Adrien; Gousseff, Marie; Hamidou, M.; Liferman, François; Moulis, Guillaume; Galicier, Lionel; Ebbo, M.; London, Jonathan; Poutrel, Solène; Terriou, Louis; Zarrouk, Virginie; Michel, Marc; Godeau, Bertrand; Mahévas, Matthieu

doi:10.1002/ajh.26040

Cited by 11 publications

(8 citation statements)

References 5 publications

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“…The IWG recommends the concomitant use of IVIg, intravenous corticosteroids, and platelet transfusion with general supportive care. TPO-RAs can also be administered concurrently at relatively more aggressive dosages to increase the response rate and reduce early relapse [ 11 ], especially for patients with refractory ITP in an emergency bleeding situation [ 120 ]. Vinca alkaloids may also be considered, but the risk of peripheral neuropathy should be noted.…”

Section: Emergency Treatmentmentioning

confidence: 99%

How we treat primary immune thrombocytopenia in adults

Liu

Hou

2023

J Hematol Oncol

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Primary immune thrombocytopenia (ITP) is an immune-mediated bleeding disorder characterized by decreased platelet counts and an increased risk of bleeding. Multiple humoral and cellular immune abnormalities result in accelerated platelet destruction and suppressed platelet production in ITP. The diagnosis remains a clinical exclusion of other causes of thrombocytopenia. Treatment is not required except for patients with active bleeding, severe thrombocytopenia, or cases in need of invasive procedures. Corticosteroids, intravenous immunoglobulin, and anti-RhD immunoglobulin are the classical initial treatments for newly diagnosed ITP in adults, but these agents generally cannot induce a long-term response in most patients. Subsequent treatments for patients who fail the initial therapy include thrombopoietic agents, rituximab, fostamatinib, splenectomy, and several older immunosuppressive agents. Other potential therapeutic agents, such as inhibitors of Bruton’s tyrosine kinase and neonatal Fc receptor, are currently under clinical evaluation. An optimized treatment strategy should aim at elevating the platelet counts to a safety level with minimal toxicity and improving patient health-related quality of life, and always needs to be tailored to the patients and disease phases. In this review, we address the concepts of adult ITP diagnosis and management and provide a comprehensive overview of current therapeutic strategies under general and specific situations.

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Section: Emergency Treatmentmentioning

confidence: 99%

How we treat primary immune thrombocytopenia in adults

Liu

Hou

2023

J Hematol Oncol

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“…Platelet transfusion is only used in ITP where there is life-threatening bleeding or there is a high risk of bleeding. In emergency situations we may use romiplostim 10 µg/kg/week as a therapeutic alternative to the use of IVIg [ 11 ].…”

Section: First-line Treatment For Itpmentioning

confidence: 99%

Proposal for a New Protocol for the Management of Immune Thrombocytopenia (ITP)

González-López

2022

Adv Ther

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For many decades immune thrombocytopenia (ITP) was managed using therapies which had not undergone randomised clinical trials and included corticosteroids, immune suppression or splenectomy. These older therapies are associated with an increase in morbidity and mortality. These empirical therapies have variable efficacy and well-described side effects for many patients with minimal benefit to the patient. Over the past 10 years there has been a shift away from immune suppression and non-evidence-based therapies towards using treatments with reduced or no immune suppression with an increasing reliance on the recently developed and approved thrombopoietin receptor agonists. The recent COVID-19 pandemic has made it more urgent that we develop non-immune suppressive strategies for ITP. In this commentary we describe our proposal for a contemporary approach to the management of ITP in adults that is based on our hospital practices and published guidelines.

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“…The Updated International Consensus Report recommends the use of TPO-RA in the case of a life-threatening bleeding if initial treatments with corticosteroids, IVIG and thrombocyte transfusion fails to increase the platelet count [ 11 ]. Roumier et al used high dose romiplostim (10 μg/kg body weight) together with vinca alkaloids in 30 patients with severe bleeding and compared the results with a historical patient group treated with vinca alkaloids only [ 101 ]. Both groups constituted of patients who failed to achieve a response after initial therapy with IVIG, corticosteroids and/or platelet transfusion [ 101 ].…”

Section: Treatment Of Itpmentioning

confidence: 99%

“…Roumier et al used high dose romiplostim (10 μg/kg body weight) together with vinca alkaloids in 30 patients with severe bleeding and compared the results with a historical patient group treated with vinca alkaloids only [ 101 ]. Both groups constituted of patients who failed to achieve a response after initial therapy with IVIG, corticosteroids and/or platelet transfusion [ 101 ]. At day 7, complete response (60% vs. 29%, p < 0.05), and at day 14, both partial response (80% vs. 43%, p < 0.05) and complete response (70 vs. 17%, p < 0.0001) were significantly higher in the romiplostim plus vinca alkaloid group compared to the vinca alkaloid group alone [ 101 ].…”

Section: Treatment Of Itpmentioning

confidence: 99%

“…Both groups constituted of patients who failed to achieve a response after initial therapy with IVIG, corticosteroids and/or platelet transfusion [ 101 ]. At day 7, complete response (60% vs. 29%, p < 0.05), and at day 14, both partial response (80% vs. 43%, p < 0.05) and complete response (70 vs. 17%, p < 0.0001) were significantly higher in the romiplostim plus vinca alkaloid group compared to the vinca alkaloid group alone [ 101 ]. Although this study shows the effective use of high dose romiplostim in life-threatening bleeding in ITP patients, two patients (6.6%) treated with high dose romiplostim developed major thromboembolic events.…”

Section: Treatment Of Itpmentioning

confidence: 99%

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Primary Immune Thrombocytopenia: Novel Insights into Pathophysiology and Disease Management

Singh¹,

Uzun²,

Bakchoul³

2021

JCM

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Immune thrombocytopenia (ITP) is an autoimmune disorder defined by a significantly reduced number of platelets in blood circulation. Due to low levels of platelets, ITP is associated with frequent bruising and bleeding. Current evidence suggests that low platelet counts in ITP are the result of multiple factors, including impaired thrombopoiesis and variations in immune response leading to platelet destruction during pathological conditions. Patient outcomes as well as clinic presentation of the disease have largely been shown to be case-specific, hinting towards ITP rather being a group of clinical conditions sharing common symptoms. The most frequent characteristics include dysfunction in primary haemostasis and loss of immune tolerance towards platelet as well as megakaryocyte antigens. This heterogeneity in patient population and characteristics make it challenging for the clinicians to choose appropriate therapeutic regimen. Therefore, it is vital to understand the pathomechanisms behind the disease and to consider various factors including patient age, platelet count levels, co-morbidities and patient preferences before initiating therapy. This review summarizes recent developments in the pathophysiology of ITP and provides a comprehensive overview of current therapeutic strategies as well as potential future drugs for the management of ITP.

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High dose romiplostim as a rescue therapy for adults with severe bleeding and refractory immune thrombocytopenia

Abstract: Life-threatening bleeding is a rare event in immune thrombocytopenia (ITP) and intracranial hemorrhage (ICH) occurs in less than 1% but is

Cited by 11 publications

References 5 publications

How we treat primary immune thrombocytopenia in adults

How we treat primary immune thrombocytopenia in adults

Proposal for a New Protocol for the Management of Immune Thrombocytopenia (ITP)

Primary Immune Thrombocytopenia: Novel Insights into Pathophysiology and Disease Management

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