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Cyclosporin A for persistent or chronic immune thrombocytopenia in children
Anthony P Y Liu1, Daniel K L Cheuk2, Ana H Y Lee2
1Department of Paediatrics and Adolescent Medicine, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Queen Mary Hospital, Hong Kong Special Administrative Region, China. apyliu@hku.hk.
Insights
Cyclosporin A (CSA) effectively treats chronic immune thrombocytopenia (ITP) in children, showing a response in over half of patients. This treatment may reduce the need for steroids or splenectomy in children with persistent ITP.
Area of Science:
- Pediatric Hematology
- Immunology
- Pharmacology
Background:
- Immune thrombocytopenia (ITP) affects children, with 20% developing a chronic form.
- Established treatment strategies for chronic ITP in pediatric patients are limited.
- Cyclosporin A (CSA) has shown promise in small studies for chronic ITP.
Purpose of the Study:
- To evaluate the efficacy and safety of Cyclosporin A (CSA) in children with persistent or chronic immune thrombocytopenia (ITP).
- To assess CSA's potential to reduce reliance on chronic steroid therapy or splenectomy.
Main Methods:
- Retrospective review of 30 pediatric patients (<18 years) with persistent/chronic ITP treated with CSA.
- Data collected on patient demographics, ITP diagnosis, CSA treatment duration, dosage, and response.
- Analysis of platelet counts and adverse events.
Main Results:
- Complete or partial response to CSA was observed in 17 patients (57%).
- Sustained response was achieved in 7 patients (23%).
- Observed side effects, primarily hirsutism, were generally tolerable and reversible.
Conclusions:
- Cyclosporin A (CSA) demonstrates efficacy in approximately half of pediatric patients with persistent or chronic immune thrombocytopenia (ITP).
- CSA is a safe second-line treatment option for managing chronic ITP in children.
- Further research may explore CSA's role in minimizing long-term treatments like steroids or surgery.
Abstract:
Twenty percent of children with immune thrombocytopenia (ITP) develop a chronic course where treatment strategy is less established. Cyclosporin A (CSA) has been shown to be effective in small series of children with chronic ITP and might reduce the need for chronic steroid therapy and/or splenectomy. We reviewed consecutive patients below 18 years old with persistent or chronic ITP treated with CSA in our unit between January 1998 and June 2015. Thirty patients (14 boys and 16 girls) were included. The median age at initial diagnosis of ITP was 5 years (range 0.5-16.2 years). CSA was started at a median of 13.9 months (range 3.4-124 months) after initial diagnosis and given for a median duration of 9.3 months (range 0.2-63.9 months). The median platelet count before commencement was 12 × 10(9)/L (range 4-199 × 10(9)/L). The median dose of CSA was 6 mg/kg/day (range 2.4-7.5 mg/kg/day). Complete response (CR) or response (R) was achieved in 17 patients (57 %), and 7 (23 %) had sustained response. Side effects (most commonly hirsutism) were tolerable and reversible. CSA appeared effective in about half of persistent or chronic ITP patients and safe as a second-line agent in managing these children.
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