Immune thrombocytopenia (ITP) is an autoimmune disease associated with isolated thrombocytopenia, which is caused by an imbalance between platelet production and platelet destruction. Petechial and mucous membrane hemorrhages are characteristic of ITP, but life-threatening bleeding rarely occurs. Depending on the bleeding symptoms, ITP can be treated with glucocorticoids (GC), intravenous immunoglobulins (IVIG), or in severe cases, platelet transfusions. Mild bleeding does not necessarily require therapy. Using the German Surveillance Unit for rare Pediatric Diseases (ESPED) we conducted a prospective survey on ITP patients in all German Children's Hospitals between September 2018 and August 2019. We collected data on ITP, including the clinical course, therapy implementation recommendations (according to the Association of German Scientific Medical Societies guidelines), outcome, and influence of treatment regimens depending on the treating physician´s experience with ITP patients. Of the 287 recorded cases of children with ITP, 268 questionnaires were sent to the authors. Two hundred seventeen of the questionnaires fulfilled the inclusion criteria. ITP affected boys and girls similarly, and the median age of manifestation was 3.5 years. The main reasons for hospitalization were thrombocytopenia, bleeding signs, hematomas, and/or petechiae. Bleeding scores were ≤ 3 in 96% of children, which corresponded to a low-to-moderately low risk of bleeding. No life-threatening bleeding was documented. The most common therapies were IVIG (n = 59), GC (n = 33), or a combination of these (n = 17). Blood products (i.e., red blood cells, platelet concentrate, and fresh frozen plasma) were given to 13 patients. Compared to the established guidelines, 67 patients were over-treated, and 2 patients were under-treated. Adherence to German ITP treatment guidelines is currently limited. To improve patient safety and medical care, better medical training and dissemination of the guidelines are required in line with targeted analyses of patients with serious bleeding events to identify potential risk constellations.

中文翻译：

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ESPED 儿童新诊断免疫性血小板减少症调查：我们给予多少治疗？

免疫性血小板减少症 (ITP) 是一种与孤立性血小板减少症相关的自身免疫性疾病，其由血小板生成和血小板破坏之间的不平衡引起。点状和粘膜出血是 ITP 的特征，但很少发生危及生命的出血。根据出血症状，ITP 可以用糖皮质激素 (GC)、静脉注射免疫球蛋白 (IVIG) 或在严重的情况下输注血小板来治疗。轻度出血不一定需要治疗。2018 年 9 月至 2019 年 8 月，我们使用德国罕见儿科疾病监测部门 (ESPED) 对所有德国儿童医院的 ITP 患者进行了前瞻性调查。我们收集了 ITP 的数据，包括临床病程、治疗实施建议（根据德国科学医学会协会指南）、结果和治疗方案的影响取决于治疗医师对 ITP 患者的经验。在记录的 287 例 ITP 儿童病例中，向作者发送了 268 份问卷。217 份问卷符合纳入标准。ITP 对男孩和女孩的影响相似，表现的中位年龄为 3.5 岁。住院的主要原因是血小板减少、出血迹象、血肿和/或瘀点。96% 的儿童出血评分≤ 3，这对应于低至中低的出血风险。没有记录到危及生命的出血。最常见的疗法是 IVIG (n = 59)、GC (n = 33) 或这些的组合 (n = 17)。血液制品（即红细胞、浓缩血小板和新鲜冷冻血浆）给予 13 名患者。与既定指南相比，67 名患者过度治疗，2 名患者治疗不足。目前对德国 ITP 治疗指南的遵守有限。为了提高患者安全和医疗保健，需要更好的医疗培训和指南的传播，以配合对严重出血事件患者的针对性分析，以确定潜在的风险群。