Haematology Unit, Department of Paediatric and Public Health Sciences, University of Turin, Turin, Italy.
Paediatric Haematology and Oncology Unit, "Policlinico-Vittorio Emanuele" Hospital, University of Catania, Catania, Italy.
Blood Transfus. 2020 Sep;18(5):396-405. doi: 10.2450/2020.0041-20. Epub 2020 Jul 22.
The aim of the present study was to assess management strategies for immune thrombocytopenia (ITP) among Italian paediatric haematologists, and to compare these with those of recent international guidelines. Predictors of early remission or disease chronicity were also evaluated.
During a period of 1 year, 205 children (age: 1 month-18 years) with newly diagnosed ITP were prospectively enrolled by 16 centres belonging to the Italian Association of Paediatric Haematology and Oncology (AIEOP). We collected the subjects demographic data, history, clinical symptoms, platelet count and treatment at presentation and at subsequent visits.
Of the 205 patients, 47 (23%) were initially managed with a wait-and-see approach. Compared to these patients, children administered platelet-enhancing therapies were significantly younger (median age: 4.75 vs 7.96 years; p<0.001) and had lower platelet counts. At the 3-month follow-up, 92/202 patients (46%) had persistent ITP. Recovery within 3 months was predicted by younger median age (5.3 vs 7.8 years; p<0.001), and recent viral infection (p<0.001) . At 1 year, 56 patients had chronic ITP, which was associated with older median age (7.54 vs 5.35 years; p<0.001), and a family history of autoimmunity (p<0.05; relative risk: 1.81; 95% confidence interval: 1.09-2.98). In total, 357 pharmacological treatments were recorded (216 intravenous immunoglobulins, 80 steroids). Response to intravenous immunoglobulins did not have an effect on remission rate at 12 months.
Pediatric hematologists in Italian Centre treat over three-quarters of patients with newly diagnosed ITP, despite recent international guidelines. Almost 80% of patients with mild clinical symptoms received pharmacological treatment at diagnosis, which was significantly associated with younger age. Chronicity at 12 months was not affected by different therapeutic approaches at diagnosis or response to therapy.
本研究旨在评估意大利儿科血液学家对免疫性血小板减少症 (ITP) 的治疗策略，并将其与近期国际指南进行比较。还评估了早期缓解或疾病慢性的预测因素。
在 1 年期间，由意大利儿科血液学和肿瘤学协会 (AIEOP) 的 16 个中心前瞻性招募了 205 名新诊断为 ITP 的儿童（年龄：1 个月至 18 岁）。我们收集了患者的人口统计学数据、病史、临床症状、血小板计数以及就诊时和后续就诊时的治疗情况。
在 205 名患者中，47 名（23%）最初采用观望治疗方法。与这些患者相比，接受血小板增强治疗的患儿年龄明显较小（中位数年龄：4.75 岁 vs 7.96 岁；p<0.001），血小板计数较低。在 3 个月的随访中，202 名患者中有 92 名（46%）持续性 ITP。3 个月内恢复的预测因素是年龄中位数较小（5.3 岁 vs 7.8 岁；p<0.001）和近期病毒感染（p<0.001）。在 1 年时，56 名患者患有慢性 ITP，这与年龄中位数较大（7.54 岁 vs 5.35 岁；p<0.001）和自身免疫性疾病家族史（p<0.05；相对风险：1.81；95%置信区间：1.09-2.98）相关。共记录了 357 种药物治疗（216 种静脉内免疫球蛋白，80 种类固醇）。静脉内免疫球蛋白的反应对 12 个月时的缓解率没有影响。
尽管有近期的国际指南，但意大利中心的儿科血液学家对超过四分之三的新诊断 ITP 患者进行了治疗。近 80%有轻度临床症状的患者在诊断时接受了药物治疗，这与年龄较小显著相关。12 个月时的慢性率不受诊断时不同治疗方法或对治疗的反应的影响。
