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Dapsone: An Old but Effective Therapy in Pediatric Refractory Immune Thrombocytopenia
Sanjeev Khera1, Suman Kumar Pramanik2, Uday Yanamandra2
1Department of Pediatrics, Army Hospital Research and Referral, Delhi, 110010 India.
Insights
Dapsone effectively treats chronic immune thrombocytopenia (ITP) in children refractory to other therapies. This economical drug shows a 72% response rate with a good safety profile, offering a viable option for pediatric ITP management.
Area of Science:
- Pediatric Hematology
- Immunology
- Pharmacology
Background:
- Management of chronic or refractory immune thrombocytopenia (ITP) in children lacks definitive guidelines.
- Dapsone is an underutilized, cost-effective, and potentially efficacious treatment for chronic ITP.
Purpose of the Study:
- To evaluate the efficacy and safety of dapsone in managing chronic ITP in pediatric patients.
- To assess dapsone's response rates, time to response, and side effects in children with refractory ITP.
Main Methods:
- Retrospective analysis of children under 14 years with chronic ITP and significant bleeding, refractory to splenectomy, rituximab, or eltrombopag.
- Dapsone therapy was initiated at 1-2 mg/kg/day, excluding patients with dapsone intolerance or G6PD deficiency.
- Outcomes were assessed based on international working group definitions for response, time to response, and adverse events.
Main Results:
- Overall response to dapsone was observed in 72% of 29 analyzed children (7% complete response, 48% partial response).
- Responses were sustained for at least 3 months, with a median time to response of 2.9 months.
- Major side effects included methemoglobinemia (1 case) and skin ulceration (2 cases); 21% relapse rate was noted.
Conclusions:
- Dapsone is an economical and effective therapeutic option for children with chronic or refractory immune thrombocytopenia.
- The drug demonstrates a favorable safety profile, making it a valuable treatment choice in pediatric ITP management.
- Dapsone offers a viable alternative for patients unresponsive to established therapies like splenectomy, rituximab, or eltrombopag.
Abstract:
There are no definitive guidelines for management of chronic or refractory immune thrombocytopenia (ITP) in children. Dapsone is an inexpensive and efficacious, yet neglected, therapeutic option for treatment of chronic ITP. We evaluated the efficacy and safety of dapsone in the management of chronic ITP in children. Children with chronic ITP < 14 years with minimum grade 2 bleeds refractory to either splenectomy/rituximab/eltrombopag; who were offered dapsone therapy were retrospectively analyzed. Dapsone intolerance and G6PD deficiency were excluded. Dapsone was started at a dose of 1-2 mg/kg/day. Response to dapsone as per international working group definitions, time to response along with side-effects were noted. Forty-four children enrolled; 29 analyzed. Nineteen were refractory to rituximab, 8 to splenectomy and 6 to eltrombopag. Median age was 9.8 years (3-14) with 16/29 males. Median dapsone dose was 1.59 mg/kg/day (range 1-2.1). Overall response was seen in 21/29 (72%): Complete Response in 7/29 (24%), Partial Response in 14/29 (48%). All responses were sustained for minimum 3 months. Median duration to response was 2.9 months (2-6.6). Median follow up was 28 months (6-73) and relapse rate-21%. Major side effects noted: Methemoglobinemia-01, skin ulceration-02. In three cases dapsone could be tapered and stopped without relapse. Dapsone is an economical and efficacious agent with good safety profile in childhood chronic/refractory ITP.
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