Khera Sanjeev, Pramanik Suman Kumar, Yanamandra Uday, Mishra Kundan, Kapoor Rajan, Das Satyaranjan
Department of Pediatrics, Army Hospital Research and Referral, Delhi, 110010 India.
Department of Hematology, Army Hospital Research and Referral, Delhi, India.
Indian J Hematol Blood Transfus. 2020 Oct;36(4):690-694. doi: 10.1007/s12288-020-01286-9. Epub 2020 May 7.
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.
目前尚无针对儿童慢性或难治性免疫性血小板减少症(ITP)的明确管理指南。氨苯砜是一种价格低廉且有效的治疗慢性ITP的方法,但却被忽视了。我们评估了氨苯砜治疗儿童慢性ITP的疗效和安全性。对年龄小于14岁、至少有2级出血且对脾切除术/利妥昔单抗/艾曲泊帕难治的慢性ITP儿童进行回顾性分析,这些儿童接受了氨苯砜治疗。排除氨苯砜不耐受和葡萄糖-6-磷酸脱氢酶(G6PD)缺乏的情况。氨苯砜起始剂量为1-2mg/kg/天。记录根据国际工作组定义的对氨苯砜的反应、反应时间以及副作用。44名儿童入组;29名进行分析。19名对利妥昔单抗难治,8名对脾切除术难治,6名对艾曲泊帕难治。中位年龄为9.8岁(3-14岁),29名中有16名男性。氨苯砜中位剂量为1.59mg/kg/天(范围1-2.1)。29名中有21名(72%)出现总体反应:完全缓解7名(24%),部分缓解14名(48%)。所有反应至少持续3个月。中位反应持续时间为2.9个月(2-6.6)。中位随访时间为28个月(6-73),复发率为21%。主要副作用包括:高铁血红蛋白血症1例,皮肤溃疡2例。3例中氨苯砜可逐渐减量并停药且无复发。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。