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Management of primary immune thrombocytopenia in a real-world setting in Japan: eltrombopag versus corticosteroids

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Abstract

This real-world study in Japan assessed the long-term safety of persistent use of eltrombopag compared to corticosteroids. Overall, 1887 patients with primary immune thrombocytopenia were included in the study cohort, based on hospital claims data. Eltrombopag was frequently used as a second- or third-line therapy (monotherapy: 13.1% and 25.7%; combination: 24.39% and 16.52%, respectively). The risk of bleeding was approximately 30% lower in the eltrombopag group (as monotherapy and in combination with other drugs including corticosteroids) than the corticosteroid group (hazard ratio, 0.66; 95% confidence interval, 0.45–0.96). Results from univariate and multivariate Cox models indicated that patients aged ≥ 60 years, male patients and patients who received the drugs for peptic ulcer or gastroesophageal reflux disease have a higher risk of cerebral haemorrhage or gastrointestinal bleeding. Surgeries were more common among patients on corticosteroids compared to patients on eltrombopag (39.1% vs 34.6%, P = 0.004), while splenectomies were very rare. There was no significant difference in the costs of scheduled, emergency, or any type of hospitalisations between the exposure groups. The risk of infections, cataracts, and thrombosis did not differ between the exposure groups.

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Acknowledgements

This study was funded by Novartis Pharma KK. Authors thank Atulya Nagarsenkar, PhD, of Novartis Healthcare Pvt Ltd for his medical editorial assistance. Financial support for medical editorial assistance was provided by Novartis Pharma KK.

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Authors and Affiliations

  1. Novartis Pharma KK, Tokyo, Japan

    Tze Fang Wong

  2. Creativ-Ceutical, Kraków, Poland

    Renata Majewska

  3. Department of Blood Transfusion, Osaka University Hospital, Suita, Osaka, Japan

    Yoshiaki Tomiyama

Authors
  1. Tze Fang Wong
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  2. Renata Majewska
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  3. Yoshiaki Tomiyama
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Contributions

TFW and YT designed the study. TFW and RM designed the statistical analysis plan. All the authors contributed in drafting the manuscript.

Corresponding author

Correspondence to Yoshiaki Tomiyama.

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Conflict of interest

Tze Fang Wong is the employee of Novartis KK, the manufacturer and retailer of eltrombopag. Renata Majewska is the employee of Creativ-Ceutical, which received funding from Novartis KK. Yoshiaki Tomiyama reports honoraria and speakers bureau fees from Novartis and Kyowa Kirin, and advisory role for Sysmex. Yoshiaki Tomiyama is an associate editor of International Journal of Hematology.

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Wong, T.F., Majewska, R. & Tomiyama, Y. Management of primary immune thrombocytopenia in a real-world setting in Japan: eltrombopag versus corticosteroids. Int J Hematol 114, 152–163 (2021). https://doi.org/10.1007/s12185-021-03149-z

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  • DOI: https://doi.org/10.1007/s12185-021-03149-z

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