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 <16 years who were diagnosed as having chronic ITP in 14 hospitals during 2001-2011, and were treated with CEP for >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 x 10(9)/L (IQR, 8.3-53.0 x 10(9)/L), and then significantly increased to 58.5 x 10(9)/L (IQR, 22.8-115.0 x 10(9)/L) and 69.0 x 10(9)/L (IQR, 23.0-134.0 x 10(9)/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.