High-dose Cepharanthin for pediatric chronic immune thrombocytopenia in Japan

Taro Yamazaki, Atsushi Shibuya, Saori Ishii, Nobuyuki Miura, Akira Ohtake, Nozomu Sasaki, Ryuichiro Araki, Yatio Ota, Mitsuhiro Fujiwara, Yuji Miyajima, Kimiaki Uetake, Keigo Hamahata, Koji Kato, Kiyoshi Kawakami, Hidemi Toyoda, Naohiko Moriguchi, Masahiko Okada, Masanori Nishi, Yoshiyasu Ogata, Tomohito TakimotoShouichi Ohga, Shigeru Ohta, Shin Amemiya

Research output: Contribution to journalArticlepeer-review

10 Citations (Scopus)


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 × 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.

Original languageEnglish
Pages (from-to)303-308
Number of pages6
JournalPediatrics International
Issue number3
Publication statusPublished - Mar 1 2017

All Science Journal Classification (ASJC) codes

  • Pediatrics, Perinatology, and Child Health


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