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High-dose Cepharanthin for pediatric chronic immune thrombocytopenia in Japan
Taro Yamazaki1, Atsushi Shibuya1, Saori Ishii1
1Department of Pediatrics, Saitama Medical University Hospital, Iruma, Saitama, Japan.
Insights
Cepharanthin (CEP) effectively increased platelet counts in pediatric patients with chronic immune thrombocytopenia (ITP). This treatment, alone or with corticosteroids, proved useful and safe for managing chronic ITP in children.
Area of Science:
- Hematology
- Pediatric Medicine
- Pharmacology
Background:
- Chronic immune thrombocytopenia (ITP) affects pediatric patients, necessitating effective treatment options.
- Cepharanthin (CEP) is being evaluated for its clinical utility in managing pediatric chronic ITP.
Purpose of the Study:
- To evaluate the clinical utility of Cepharanthin (CEP) in pediatric patients with chronic immune thrombocytopenia (ITP).
- To assess the safety and efficacy of CEP in improving platelet counts and reducing corticosteroid dependence.
Main Methods:
- Retrospective, multicenter, observational study of 46 Japanese pediatric patients (<16 years) with chronic ITP.
- Analysis of clinical and laboratory data for patients treated with CEP for over 12 months.
- Evaluation of platelet counts, adverse events, and corticosteroid (CS) weaning rates.
Main Results:
- Median platelet counts significantly increased from 20.5 × 109 /L to 58.5 × 109 /L at 12 months and 69.0 × 109 /L at 24 months.
- No life-threatening bleeds or severe adverse events were reported.
- 45% of patients on both CS and CEP were weaned off CS, and 39% achieved a reduced CS dose.
Conclusions:
- Cepharanthin (CEP) is a useful therapeutic option for pediatric chronic ITP.
- CEP, alone or in combination with corticosteroids, demonstrates efficacy and safety in managing pediatric chronic ITP.
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.
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