Annual trends in atypical haemolytic uremic syndrome management in Japan and factors influencing early diagnosis and treatment: A retrospective study

Abstract

Abstract Atypical haemolytic uremic syndrome (aHUS) is a rare disorder characterised by complement-mediated thrombotic microangiopathy (TMA). Despite existing clinical guidelines, early diagnosis and treatment of aHUS remain 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, a total of 3,096 cases with the HUS disease code were identified. Among them, 217 were confirmed as aHUS cases and received treatment with eculizumab or plasma exchange. Early initiation, defined as starting eculizumab or Plasma exchange within seven days of admission, was the focus of the study. The research revealed that over time, there were no significant changes in the number of aHUS diagnoses, cases treated with eculizumab, or early initiation cases. Compared to late initiation cases, early initiation cases underwent haemodialysis and had ADAMTS13 activity measured earlier, had shorter hospital stays, and lower hospitalization costs. In conclusion, this study did not find an increase in the number of newly diagnosed aHUS cases or early treatment initiation over time. Timely recognition of TMA, especially through the prompt measurement of ADAMTS13 activity, is crucial for identifying potential aHUS cases and initiating treatment promptly.