Eltrombopag safety and efficacy for primary chronic immune thrombocytopenia in clinical practice

González-López, Tomás José; Álvarez‐Román, María Teresa; Pascual, Cristina; Sánchez‐González, Blanca; Fernández-Fuentes, Fernando; Jarque, Isidro; Pérez‐Rus, Gloria; Pérez-Crespo, Susana; Bernat, Silvia; Hernández‐Rivas, José Ángel; Andrade, Marcio; Cortés, Montserrat; Gómez-Núñez, Marta; Olivera, Pável; Martínez-Robles, Violeta; Fernández-Rodríguez, Ángeles; Fuertes-Palacio, Miguel Angel; Fernández-Miñano, Carmen; Cabo, Erik de; Fisac, Rosa; Aguilar, Carlos; Bárez, Abelardo; Peñarrubia, María Jesús; García-Frade, Luis Javier; González‐Porras, José Ramón

doi:10.1111/ejh.12725

Cited by 39 publications

(51 citation statements)

References 22 publications

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“…Eltrombopag is effective and well tolerated in primary ITP according to pivotal trials (Cheng et al , ; Saleh et al , ). In addition, our results from an extra‐clinical trial are consistent with the excellent results for eltrombopag obtained in ITP clinical trials (Gonzalez‐Porras et al , ; Gonzalez‐Lopez et al , ). However, the role of eltrombopag in secondary ITP has not been thoroughly studied.…”

Section: Discussionsupporting

confidence: 90%

Use of eltrombopag for secondary immune thrombocytopenia in clinical practice

et al. 2017

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Eltrombopag is a second-line treatment in primary immune thrombocytopenia (ITP). However, its role in secondary ITP is unknown. We evaluated the efficacy and safety of eltrombopag in secondary ITP in daily clinical practice. Eighty-seven secondary ITP patients (46 with ITP secondary to autoimmune syndromes, 23 with ITP secondary to a neoplastic disease subtype: lymphoproliferative disorders [LPDs] and 18 with ITP secondary to viral infections) who had been treated with eltrombopag were retrospectively evaluated. Forty-four patients (38%) had a platelet response, including 40 (35%) with complete responses. Median time to platelet response was 15 days (95% confidence interval, 7-28 days), and was longer in the LPD-ITP group. Platelet response rate was significantly lower in the LPD-ITP than in other groups. However, having achieved response, there were no significant differences between the durable response of the groups. Forty-three patients (49·4%) experienced adverse events (mainly grade 1-2), the commonest being hepatobiliary laboratory abnormalities. There were 10 deaths in this case series, all of which were related to pre-existing medical conditions. In routine clinical practice, eltrombopag is effective and well-tolerated in unselected patients with ITP secondary to both immune and infectious disorders. However, the response rate in LPD-ITP is low.

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Section: Discussionsupporting

confidence: 90%

Use of eltrombopag for secondary immune thrombocytopenia in clinical practice

et al. 2017

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“…The availability of the well tolerated, nonimmunosuppressive thrombopoietin receptor agonists (TPO-RAs), eltrombopag and romiplostim, as second line treatment options for ITP has started to shift the paradigm of ITP management and delay splenectomy [7, 10–13]. In the subset of patients who do not achieve adequate responses to TPO-RAs, one must consider the optimal management of these agents prior to splenectomy.…”

Section: Introductionmentioning

confidence: 99%

Balancing Therapy with Thrombopoietin Receptor Agonists and Splenectomy in Refractory Immune Thrombocytopenic Purpura: A Case of Postsplenectomy Thrombocytosis Requiring Plateletpheresis

Zimmerman

Norsworthy

2016

Case Reports in Hematology

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Immune thrombocytopenic purpura (ITP) causes thrombocytopenia through the autoimmune destruction of platelets. Corticosteroids remain the first line of therapy, and traditionally splenectomy has been the second. While the availability of thrombopoietin receptor agonists (TPO-RAs) has expanded treatment options, there is little data for the ideal management of these agents in preparation for splenectomy. Thrombocytosis has been reported after splenectomy in patients treated with TPO-RA preoperatively, with one prior case requiring plateletpheresis for symptomatic thrombocytosis. We present a case report and review of the literature pertaining to this complication and provide recommendations for preventing postsplenectomy thrombocytosis in ITP patients on TPO-RAs.

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“…71 In clinical practice, the response rate is somewhat lower (74% to 94%), reflecting perhaps the increased heterogeneity of a nonclinical trial population. [72][73][74] The literature also suggests that patients who are intolerant of 1 TPO-RA can successfully switch to the other. 75 Long-term follow-up data are now available for both TPO-RAs for adult patients.…”

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Clinical updates in adult immune thrombocytopenia

Lambert

Gernsheimer

2017

Blood

364

319

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Immune thrombocytopenia (ITP) occurs in 2 to 4/100 000 adults and results in variable bleeding symptoms and thrombocytopenia. In the last decade, changes in our understanding of the pathophysiology of the disorder have led to the publication of new guidelines for the diagnosis and management of ITP and standards for terminology. Current evidence supports alternatives to splenectomy for second-line management of patients with persistently low platelet counts and bleeding. Long-term follow-up data suggest both efficacy and safety, in particular, for the thrombopoietin receptor agonists and the occurrence of late remissions. Follow-up of patients who have undergone splenectomy for ITP reveals significant potential risks that should be discussed with patients and may influence clinician and patient choice of second-line therapy. Novel therapeutics are in development to address ongoing treatment gaps.

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Eltrombopag safety and efficacy for primary chronic immune thrombocytopenia in clinical practice

Abstract: Eltrombopag is highly effective and well tolerated in unselected patients with primary chronic ITP.

Cited by 39 publications

References 22 publications

Use of eltrombopag for secondary immune thrombocytopenia in clinical practice

Use of eltrombopag for secondary immune thrombocytopenia in clinical practice

Balancing Therapy with Thrombopoietin Receptor Agonists and Splenectomy in Refractory Immune Thrombocytopenic Purpura: A Case of Postsplenectomy Thrombocytosis Requiring Plateletpheresis

Clinical updates in adult immune thrombocytopenia

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