High‐dose Cepharanthin for pediatric chronic immune thrombocytopenia in Japan

Background A nationwide, multicenter and observational study was retrospectively conducted to evaluate the clinical utility of Cepharanthin (CEP) for pediatric patients with chronic immune thrombocytopenia (ITP). Methods Clinical and laboratory data for 46 Japanese patients aged 12 months, were analyzed. Results Median daily CEP dose was 1 mg/kg (range, 0.12–2 mg/kg). Median platelet count prior to CEP was 20.5 × 109/L (IQR, 8.3–53.0 × 109/L), and then significantly increased to 58.5 × 109/L (IQR, 22.8–115.0 × 109/L) and 69.0 × 109/L (IQR, 23.0–134.0 × 109/L) at 12 and 24 months of treatment, respectively. No life‐threatening bleeds or moderate–severe adverse events were reported. Of 38 patients who received both corticosteroids (CS) and CEP, 17 patients (45%) were weaned from CS, and 15 patients (39%) attained the reduced dose of CS. The duration from the start of CEP to the stopping of CS was a median of 413 days (range, 49–1734 days) in patients who were weaned from CS. Conclusions CEP alone or combined with CS was useful for the management of pediatric chronic ITPs.