Taro Yamazaki1, Atsushi Shibuya1, Saori Ishii1, Nobuyuki Miura1, Akira Ohtake1, Nozomu Sasaki1, Ryuichiro Araki2, Yatio Ota3, Mitsuhiro Fujiwara4, Yuji Miyajima5, Kimiaki Uetake6, Keigo Hamahata7, Koji Kato8, Kiyoshi Kawakami9, Hidemi Toyoda10, Naohiko Moriguchi11, Masahiko Okada12, Masanori Nishi13, Yoshiyasu Ogata13, Tomohito Takimoto14, Shouichi Ohga14,15, Shigeru Ohta16, Shin Amemiya1. 1. Department of Pediatrics, Saitama Medical University Hospital, Iruma, Saitama, Japan. 2. Community Health Science Center, Saitama Medical University, Iruma-gun, Saitama, Japan. 3. Toyohira Ota Children's Clinic, Sapporo, Hokkaido, Japan. 4. Department of Pediatrics, Kurashiki Central Hospital, Kurashiki, Okayama, Japan. 5. Department of Pediatrics, Anjo Kosei Hospital, Anjo, Aichi, Japan. 6. Department of Pediatrics, Obihiro Kosei Hospital, Obihiro, Hokkaido, Japan. 7. Department of Pediatrics, Japanese Red Cross Wakayama Medical Center, Wakayama City, Wakayama, Japan. 8. Department of Hematology and Oncology, Children's Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Aichi, Japan. 9. Department of Pediatrics, Kagoshima City Hospital, Kagoshima City, Kagoshima, Japan. 10. Department of Pediatrics, Mie University Hospital, Tsu, Mie, Japan. 11. Department of Pediatrics, Sakai Hospital, Kindai University, Sakai, Osaka, Japan. 12. Department of Pediatrics, Nagasaki University Hospital, Nagasaki City, Nagasaki, Japan. 13. Department of Pediatrics, Saga University Hospital, Saga City, Saga, Japan. 14. Department of Pediatrics, Kyushu University Hospital, Fukuoka City, Fukuoka, Japan. 15. Department of Pediatrics, Yamaguchi University Hospital, Ube, Yamaguchi, Japan. 16. Department of Clinical and Education of Doctor Center, Shiga University of Medical Science, Otsu, Shiga, Japan.
Abstract
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.
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.