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.

中文翻译：

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氨苯砜：一种古老但有效的小儿难治性免疫性血小板减少症疗法

对于儿童慢性或难治性免疫性血小板减少症 (ITP) 的管理没有明确的指南。氨苯砜是治疗慢性 ITP 的一种廉价且有效但被忽视的治疗选择。我们评估了氨苯砜治疗儿童慢性 ITP 的有效性和安全性。患有慢性 ITP < 14 岁且至少 2 级出血的儿童对脾切除术/利妥昔单抗/艾曲波帕均无效；对接受氨苯砜治疗的患者进行回顾性分析。排除氨苯砜不耐受和 G6PD 缺乏症。氨苯砜的起始剂量为 1–2 mg/kg/天。根据国际工作组的定义对氨苯砜的反应、反应时间以及副作用都进行了记录。四十四名儿童入学；29 分析。19 例对利妥昔单抗无效，8 例对脾切除术无效，6 例对艾曲波帕无效。中位年龄为 9.8 岁 (3-14)，男性为 16/29。氨苯砜的中位剂量为 1.59 mg/kg/天（范围 1-2.1）。在 21/29 (72%) 中看到总体响应：在 7/29 (24%) 中完全响应，在 14/29 (48%) 中部分响应。所有反应持续至少 3 个月。中位缓解持续时间为 2.9 个月 (2–6.6)。中位随访时间为 28 个月 (6-73)，复发率为 21%。注意到的主要副作用：高铁血红蛋白血症-01，皮肤溃疡-02。在三种情况下，氨苯砜可以逐渐减量并停止而不会复发。氨苯砜是一种经济有效的药物，在儿童慢性/难治性 ITP 中具有良好的安全性。所有反应持续至少 3 个月。中位缓解持续时间为 2.9 个月 (2–6.6)。中位随访时间为 28 个月 (6-73)，复发率为 21%。注意到的主要副作用：高铁血红蛋白血症-01，皮肤溃疡-02。在三种情况下，氨苯砜可以逐渐减量并停止而不会复发。氨苯砜是一种经济有效的药物，在儿童慢性/难治性 ITP 中具有良好的安全性。所有反应持续至少 3 个月。中位缓解持续时间为 2.9 个月 (2–6.6)。中位随访时间为 28 个月 (6-73)，复发率为 21%。注意到的主要副作用：高铁血红蛋白血症-01，皮肤溃疡-02。在三种情况下，氨苯砜可以逐渐减量并停止使用而不会复发。氨苯砜是一种经济有效的药物，在儿童慢性/难治性 ITP 中具有良好的安全性。