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儿童急性免疫性血小板减少症管理的建议:来自意大利儿科血液学与肿瘤学协会的共识会议。

Recommendations for the management of acute immune thrombocytopenia in children. A Consensus Conference from the Italian Association of Pediatric Hematology and Oncology.

发表日期:2023 Jul 27
作者: Giovanna Russo, Emilia Parodi, Piero Farruggia, Lucia D Notarangelo, Silverio Perrotta, Maddalena Casale, Simone Cesaro, Giovanni Del Borrello, Giovanni C Del Vecchio, Fiorina Giona, Chiara Gorio, Saverio Ladogana, Giuseppe Lassandro, Antonio Marzollo, Karolina Maslak, Maurizio Miano, Margherita Nardi, Giuseppe Palumbo, Francesca Rossi, Marco Spinelli, Alessandra Tolva, Paola Saracco, Ugo Ramenghi, Paola Giordano
来源: Experimental Hematology & Oncology

摘要:

免疫性血小板减少性紫癜(ITP)是一种通过免疫系统介导引起的以单纯性血小板减少为特征的获得性出血性疾病。在儿科患者中,每年发病率估计为1.9-6.4/10万人口。儿童IPT通常是一种自限性和良性疾病。由于缺乏关于该疾病管理的可靠随机试验,儿童ITP的临床管理常存在争议。治疗在临床实践中差异很大,血液学学会关于临床治疗的现行指南主要基于专家意见而非强有力的证据。意大利小儿血液学与肿瘤学协会(AIEOP)凝血障碍工作组制定了此文件,以收集关于新诊断ITP管理的共享专家意见,更新先前的指南并向儿科医生提供建议。每个声明都被给予了一个表达证据强度、适应性和参与者之间一致性的分数。给出了对临床期的定义和临床响应的明确定义。给出了关于出血症状分类、出血风险评估、诊断和预后因素的建议。对于治疗的具体建议包括一线治疗(静脉免疫球蛋白、类固醇)和二线治疗(联合治疗、血小板生成素受体激动剂、免疫抑制药物、利妥昔单抗)的适应症,以及出血紧急治疗和支持性治疗,包括紧急脾切除术。还讨论了最佳的随访时间表、ITP与疫苗之间的关系以及与健康相关的生活质量问题。该专家组对于如何治疗新诊断的儿童ITP达成了广泛共识,并对所有相关临床方面进行了全面审查。
Immune thrombocytopenia (ITP) is an acquired immune-mediated bleeding disorder characterized by isolated thrombocytopenia. Its estimated yearly incidence in the pediatric population is 1.9-6.4/100,000. ITP in children is usually a self-limiting and benign disorder. The clinical management of children with ITP often remains controversial, as robust randomized trials on the management of this disorder are lacking. Treatments vary widely in clinical practice and existing guidelines from hematology societies on clinical management offer indications based largely on expert opinion rather than strong evidence.The Coagulative Disorder Working Group of the Italian Association of Pediatric Hematology and Oncology (AIEOP) developed this document to collect shared expert opinions on the management of newly diagnosed ITP, updating previous guidelines and providing recommendations to pediatricians. Each statement has been given a score expressing the strength of evidence, appropriateness and agreement among participants.Clear-cut definitions of the clinical phases of the disease and clinical response are stated. Recommendations are given regarding the classification of bleeding symptoms, evaluation of bleeding risk, diagnosis, and prognostic factors. Specific recommendations for treatment include indications for first-line (intravenous immunoglobulins, steroids) and second-line (combined therapy, thrombopoietin receptor agonists, immunosuppressive drugs, rituximab) therapeutic agents, as well as hemorrhagic emergency and supportive treatment, including emergency splenectomy. The optimal follow-up schedule, the relation between ITP and vaccines and health-related quality-of-life issues are also discussed.The panel achieved broad consensus on issues related to how to treat children with newly diagnosed ITP, providing a comprehensive review of all relevant clinical aspects.