PDF(Pubmed)
Abstract：
Atypical haemolytic uremic syndrome (aHUS) is a rare disorder characterised by complement-mediated thrombotic microangiopathy (TMA). Despite clinical guidelines, the diagnosis and treatment of aHUS in its early stages remains challenging. This study examined the annual trends in aHUS clinical practices in Japan and explored factors influencing early diagnosis and treatment. Using data from the 2011-2020 Diagnosis Procedure Combination database, 3096 cases with the HUS disease code were identified, of which 217 were confirmed as aHUS and treated with eculizumab or plasma exchange. Early initiation, defined as starting eculizumab or plasma exchange within 7 days of admission, was the focus of the study. Our study revealed no significant changes over time in the number of aHUS diagnoses, cases treated with eculizumab, or early initiation cases. Early initiation cases underwent haemodialysis earlier and had ADAMTS13 activity measured earlier, shorter hospital stays, and lower hospitalisation costs than late initiation cases. In conclusion, we found no increase in the number of newly diagnosed aHUS cases or early treatment initiation over time. Early recognition of TMA and differentiation of the causative disease are crucial for identifying potential aHUS cases, which may lead to better patient prognoses.
摘要：
非典型溶血性尿毒综合征（aHUS）是一种罕见的疾病，其特征是补体介导的血栓性微血管病（TMA）。尽管有临床指南，aHUS的早期诊断和治疗仍然具有挑战性.这项研究调查了日本aHUS临床实践的年度趋势，并探讨了影响早期诊断和治疗的因素。使用2011-2020年诊断程序组合数据库的数据，确定了3096例HUS疾病代码，其中217例被证实为aHUS,并接受依库珠单抗或血浆置换治疗.早期启动，定义为在入院后7天内开始依库珠单抗或血浆置换，是研究的重点。我们的研究表明，随着时间的推移，aHUS诊断的数量没有显著变化，用依库珠单抗治疗的病例，或早期启动病例。早期开始的病例较早进行了血液透析，并且较早地测量了ADAMTS13活性，缩短住院时间,住院费用低于延迟启动病例。总之,我们发现,随着时间的推移,新诊断的aHUS病例数或早期治疗开始数没有增加.早期识别TMA和区分致病疾病对于识别潜在的aHUS病例至关重要。这可能会导致更好的患者预后。
