Annals of Clinical Epidemiology
Online ISSN : 2434-4338
ORIGINAL ARTICLE
Trends of Treatments for Pediatric Patients with Immune Thrombocytopenia in Japan
Akifumi Endo , Nobuaki Michihata, Masato Yanagi, Masamune Higashigawa, Makiko Mori, Hidemi Toyoda, Akira Ishiguro, Hideo Yasunaga
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Supplementary material

2026 Volume 8 Issue 1 Pages 1-9

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Abstract

BACKGROUND

New drugs for pediatric immune thrombocytopenia have recently been developed; however, the practice patterns of the different treatments remain unclear in Japan.

METHODS

Using a national inpatient database, we analyzed recent trends in the treatment practice patterns of pediatric patients with immune thrombocytopenia.

RESULTS

From 2010 to 2021, the proportion of corticosteroid use did not change from 35.2 to 33.4%, whereas the proportion of intravenous immunoglobulin increased from 66.0 to 76.2% (P trend 0.003), and rituximab, romiplostim, and eltrombopag increased from 0.2 to 0.7%, 0.0 to 1.4%, and 0.3 to 7.5%, respectively, (P trend <0.001, 0.003, and <0.001, respectively). The proportion of no-treatment decreased from 20.2 to 6.5% (P trend <0.001). None of the patients had undergone splenectomy since 2016. Intravenous immunoglobulin and corticosteroids were mostly used at 1.0 g/kg and 2.0 mg/kg per day, respectively. We compared clinical practice patterns and outcomes between the only one-treatment and no-treatment groups. Intravenous immunoglobulin tended to be used in non-academic hospitals and younger patients; however, more risk of readmissions within 6 months occurred. Second-line treatment tended to be used in academic hospitals and older patients.

CONCLUSIONS

We observed an increasing trend in intravenous immunoglobulin use and second-line treatment, no change in corticosteroid use, and a decreasing trend in no-treatment approaches, using a national inpatient database. The hospital type might have affected the practice patterns. Clinicians might have chosen the optimal treatment for pediatric patients with immune thrombocytopenia considering several factors and patient backgrounds.

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© 2026 Society for Clinical Epidemiology

This article is licensed under a Creative Commons [Attribution-NonCommercial-NoDerivatives 4.0 International] license.
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