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

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.