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 × 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.
Department of Pediatrics, Saitama Medical University Hospital, Iruma, Saitama, Japan.
Taro
T
Yamazaki
Department of Pediatrics, Saitama Medical University Hospital, Iruma, Saitama, Japan.
Atsushi
A
Shibuya
Department of Pediatrics, Saitama Medical University Hospital, Iruma, Saitama, Japan.
Saori
S
Ishii
Department of Pediatrics, Saitama Medical University Hospital, Iruma, Saitama, Japan.
Nobuyuki
N
Miura
Department of Pediatrics, Saitama Medical University Hospital, Iruma, Saitama, Japan.
Akira
A
Ohtake
Department of Pediatrics, Saitama Medical University Hospital, Iruma, Saitama, Japan.
Nozomu
N
Sasaki
Community Health Science Center, Saitama Medical University, Iruma-gun, Saitama, Japan.
Ryuichiro
R
Araki
Toyohira Ota Children's Clinic, Sapporo, Hokkaido, Japan.
Yatio
Y
Ota
Department of Pediatrics, Kurashiki Central Hospital, Kurashiki, Okayama, Japan.
Mitsuhiro
M
Fujiwara
Department of Pediatrics, Anjo Kosei Hospital, Anjo, Aichi, Japan.
Yuji
Y
Miyajima
Department of Pediatrics, Obihiro Kosei Hospital, Obihiro, Hokkaido, Japan.
Kimiaki
K
Uetake
Department of Pediatrics, Japanese Red Cross Wakayama Medical Center, Wakayama City, Wakayama, Japan.
Keigo
K
Hamahata
Department of Hematology and Oncology, Children's Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Aichi, Japan.
Koji
K
Kato
Department of Pediatrics, Kagoshima City Hospital, Kagoshima City, Kagoshima, Japan.
Kiyoshi
K
Kawakami
Department of Pediatrics, Mie University Hospital, Tsu, Mie, Japan.
Hidemi
H
Toyoda
Department of Pediatrics, Sakai Hospital, Kindai University, Sakai, Osaka, Japan.
Naohiko
N
Moriguchi
Department of Pediatrics, Nagasaki University Hospital, Nagasaki City, Nagasaki, Japan.
Masahiko
M
Okada
Department of Pediatrics, Saga University Hospital, Saga City, Saga, Japan.
Masanori
M
Nishi
Department of Pediatrics, Saga University Hospital, Saga City, Saga, Japan.
Yoshiyasu
Y
Ogata
Department of Pediatrics, Kyushu University Hospital, Fukuoka City, Fukuoka, Japan.
Tomohito
T
Takimoto
Department of Pediatrics, Kyushu University Hospital, Fukuoka City, Fukuoka, Japan.|Department of Pediatrics, Yamaguchi University Hospital, Ube, Yamaguchi, Japan.
Shouichi
S
Ohga
Department of Clinical and Education of Doctor Center, Shiga University of Medical Science, Otsu, Shiga, Japan.
Shigeru
S
Ohta
Department of Pediatrics, Saitama Medical University Hospital, Iruma, Saitama, Japan.
Shin
S
Amemiya
D000293:Adolescent; D000894:Anti-Inflammatory Agents, Non-Steroidal / Q000627:therapeutic use*; D044182:Benzylisoquinolines / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D002908:Chronic Disease; D004334:Drug Administration Schedule; D005260:Female; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D007564:Japan; D008297:Male; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy*; D012189:Retrospective Studies; D016896:Treatment Outcome; D052117:Benzodioxoles
D000894:Anti-Inflammatory Agents, Non-Steroidal; D044182:Benzylisoquinolines; C006947:cepharanthine; D052117:Benzodioxoles
Cepharanthin; child; corticosteroid; idiopathic thrombocytopenic purpura; platelet count