Abstract
Atypical hemolytic uremic syndrome (aHUS) is a rare, life-threatening thrombotic microangiopathy (TMA) caused by complement dysregulation, often leading to end-stage renal disease and death. Recent real-world and post-marketing surveillance studies underscore the critical importance of initiating eculizumab, a terminal complement inhibitor, within seven days of disease onset, which is associated with sustained inhibition of TMA and marked improvement in renal outcomes and survival compared with delayed treatment. We report the clinical course of a 61-year-old previously healthy woman with aHUS who received early empiric eculizumab therapy. She initially presented with intractable vomiting and was hospitalized with a presumed diagnosis of acute pancreatitis; however, diagnostic imaging was unremarkable. On the next day of her hospitalization, laboratory findings consistent with TMA emerged, accompanied by rapid deterioration in renal function and platelet counts, prompting initiation of eculizumab on day 3. Treatment resulted in rapid inhibition of TMA, with substantial improvement in hematologic parameters and renal function reaching near-normal levels by hospital discharge (day 24), with continued improvement observed at follow-up (day 47). This case reinforces the importance of early eculizumab initiation in aHUS and aligns with real-world evidence demonstrating rapid clinical recovery and potential economic benefits through reduced hospitalization and healthcare costs.
Keywords
Atypical hemolytic uremic syndrome, Thrombotic microangiopathies, Eculizumab