Knöfler Ralf, Streif Werner
Children's Hospital, Carl Gustav Carus Dresden University Hospital, Dresden, Germany.
Transfus Med Hemother. 2010;37(5):231-235. doi: 10.1159/000320762. Epub 2010 Sep 15.
Inherited disorders of platelet function are a rare and heterogeneous group of diseases usually characterised by a mild to moderate bleeding tendency. Typical bleeding symptoms are easy bruising, epistaxis, menorrhagia as well as mucocutaneous and perioperative bleeding. The performance of platelet function diagnostics in children is hampered by age-dependent restriction of blood sample size, poor venous access, and the lack of reproducible test reference ranges for children of different age groups. Platelet function testing is limited to specialised centres, because platelet function test procedures are complicated and time-consuming, which most likely results in a relevant number of undiagnosed and incorrectly classified children with clinically relevant platelet function defects. Evaluation of bleeding history and bleeding symptoms is essential for a rational step-by-step approach to diagnosis. Platelet function diagnostics should be preceded by the exclusion of thrombocytopenia, von Willebrand disease, and secondary haemostasis defects. Light transmission aggregometry is still considered the standard for the assessment of platelet function. Every effort should be made to classify the specific platelet function defect in the patient, because this is essential for accurate treatment and counselling.
遗传性血小板功能障碍是一类罕见且异质性的疾病,通常表现为轻度至中度出血倾向。典型的出血症状包括容易出现瘀斑、鼻出血、月经过多以及黏膜皮肤出血和围手术期出血。儿童血小板功能诊断受到血样采集量受年龄限制、静脉穿刺困难以及不同年龄组儿童缺乏可重复的检测参考范围等因素的阻碍。血小板功能检测仅限于专业中心,因为血小板功能检测程序复杂且耗时,这很可能导致相当数量具有临床相关血小板功能缺陷的儿童未被诊断或分类错误。评估出血史和出血症状对于合理的逐步诊断方法至关重要。在进行血小板功能诊断之前,应排除血小板减少症、血管性血友病和继发性止血缺陷。光透射聚集法仍被视为评估血小板功能的标准方法。应尽一切努力对患者的特定血小板功能缺陷进行分类,因为这对于准确的治疗和咨询至关重要。