부산대학교 도서관

Aims: To describe the baseline characteristics and treatment of Australian patients diagnosed with atypical haemolytic uraemic syndrome (aHUS) reported to the Global aHUS Registry. Methods: Descriptive analysis of the Australian cohort with aHUS (n = 106) was undertaken for demographics, disease characteristics and prior treatment with eculizumab; comparing with the global cohort (n = 1688) for certain pre‐specified disease characteristics. Results: In Australia, almost two‐thirds of patients diagnosed with aHUS were female and over 80% of patients were Caucasians, with similar proportions reported in the global cohort. Less than 6% of patients in the Australia and global cohorts were reported to have a history of autoimmune disease (4% vs 2%, respectively; P =.21) or cancer (5% vs 5%, respectively; P =.93), conditions that have been associated with secondary HUS. In the Australian cohort, 26% had received a kidney transplant and 68% of patients had received eculizumab. Kidneys were the most common organ involvement, followed by gastrointestinal tract (26%) and cardiovascular system (19%), with 35% of patients reported to have had at least two organs involved within 6 months prior to baseline visit or entry into the registry. Complement factor H was the most common pathogenic complement gene variant in the Australian patients. Conclusion: Data from the aHUS registry confirms and defines region‐specific disease characteristics among a selected group of Australian children and adults with aHUS reported to the registry. Ongoing and more inclusive data will provide further information about temporal trends and treatment outcomes, representing a unique opportunity for clinicians and researchers to further develop knowledge surrounding this rare disease. SUMMARY AT A GLANCE: The registry shows baseline characteristics of patients with atypical haemolytic uraemic syndrome (aHUS) in Australia in which almost two‐thirds were female and over 80% were Caucasians, similar to global numbers. Less than 6% of patients have a history of autoimmune disease or cancer; 26% had received a kidney transplant and 68% of patients had received eculizumab. Kidneys were the most common organ involvement, and complement factor H was the most common pathogenic complement gene variant. [ABSTRACT FROM AUTHOR]