Childhood chronic immune thrombocytopenic purpura (cITP) is a rare disease. In severe cases, there is no evidence for the optimal therapeutic strategy. Our aim was to describe the real‐life management of non‐selected children with cITP at diagnosis. Since 2004, patients less than 18 years old with cITP have been enrolled in the national prospective cohort, OBS’CEREVANCE. From 1990 to 2014, in 29 centres, 392 children were diagnosed with cITP. With a median follow‐up of six years (2·0–25), 45% did not need second‐line therapy, and 55% (n  = 217) received one or more second lines, mainly splenectomy (n  = 108), hydroxychloroquine (n  = 61), rituximab (n  = 61) or azathioprine (n  = 40). The overall five‐year further second‐line treatment‐free survival was 56% [95% CI 49·5–64.1]. The use of splenectomy significantly decreased over time. Hydroxychloroquine was administered to children with positive antinuclear antibodies, more frequently older and girls, and reached 55% efficacy. None of the patients died. Ten years after the initial diagnosis, 55% of the 56 followed children had achieved complete remission. Children with cITP do not need second‐line treatments in 45% of cases. Basing the treatment decision on the pathophysiological pathways is challenging, as illustrated by ITP patients with positive antinuclear antibodies treated with hydroxychloroquine.

中文翻译：

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392例慢性免疫性血小板减少性紫癜儿童的二线治疗趋势和长期结果：过去25年的法国经验。

儿童慢性免疫性血小板减少性紫癜（cITP）是一种罕见的疾病。在严重的情况下，没有最佳治疗策略的证据。我们的目的是描述诊断时未选择cITP的儿童的现实生活管理。自2004年以来，年龄小于18岁的cITP患者已被纳入全国前瞻性队列OBS'CEREVANCE。从1990年到2014年，在29个中心中，有392名儿童被诊断出患有cITP。中位随访期为6年（2·0-25），其中45％的患者不需要二线治疗，而55％的患者（n  = 217）接受了一个或多个二线治疗，主要是脾切除术（n  = 108），羟氯喹（n  = 61），利妥昔单抗（n  = 61）或硫唑嘌呤（n = 40）。五年后无二线治疗的总生存期为56％[95％CI 49·5-64.1]。随着时间的推移，脾切除术的使用显着减少。羟氯喹被用于抗核抗体阳性的儿童，年龄较大的女孩和女童更为常见，并达到55％的疗效。没有患者死亡。最初诊断后十年，在56名跟随的儿童中，有55％的儿童已完全缓解。患有cITP的儿童在45％的病例中不需要二线治疗。以病理生理学途径为基础的治疗决策具有挑战性，正如ITP患者接受用羟氯喹治疗的抗核抗体阳性的患者所说明的那样。