Functional Assessment of Fatigue and Other Patient-Reported Outcomes in Patients Enrolled in the Global aHUS Registry

Research output: Contribution to journalArticle


Introduction: Atypical hemolytic uremic syndrome (aHUS) is a progressive and potentially life-threatening disease characterized by complement-mediated thrombotic microangiopathy. Patients with aHUS may experience fatigue, which can negatively impact their lives, but there is a knowledge gap regarding disease burden in these patients. Methods: In this longitudinal study, patients with aHUS from the Global aHUS Registry who completed patient-reported outcome assessments (Functional Assessment of Chronic Illness Therapy-Fatigue scale [FACIT-Fatigue], general health status, and work status) at ≥2 time points were assessed relative to treatment status: (i) never treated with eculizumab; (ii) on eculizumab at registry enrollment and continued therapy; and (iii) started eculizumab after registry enrollment. Results: Patients who started eculizumab after the baseline visit (n = 23) exhibited improvements in fatigue (nearly 75% achieved clinically meaningful improvement), improved general health status (55%), and 25% to 30% rate reduction in symptoms of fatigue, weakness, irritability, nausea/vomiting, and swelling at last follow-up. Among patients already on eculizumab at registry enrollment (n = 295) and those never treated (n = 233), these parameters changed minimally relative to the baseline. Emergency room visits and hospital admissions were similar between groups. The number of health care provider visits and work days missed were higher in patients who started eculizumab after registry enrollment. Conclusion: These real-world findings confirm the detrimental effects of aHUS on patients’ daily lives, including high levels of fatigue and impairments in general health status. The results suggest clinically meaningful improvement in fatigue, other patient-reported outcomes, and symptoms with eculizumab initiation after enrollment into the aHUS registry.


  • Larry A. Greenbaum
  • Christoph Licht
  • Vasileios Nikolaou
  • Imad Al-Dakkak
  • Janet Green
  • Christian Stefan Haas
  • Elena Román-Ortiz
  • Hae Il Cheong
  • Lisa Sartz
  • Rita Swinford
  • Ioannis Tomazos
  • Benjamin Miller
  • Spero Cataland
External organisations
  • Children's Healthcare of Atlanta
  • Hospital for Sick Children, Toronto
  • Philipp University of Marburg
  • Hospital Universitario Doctor Peset
  • Seoul National University Hospital
  • Skåne University Hospital
  • The Ohio State University Wexner Medical Center
  • Emory University
  • Parexel International
  • Alexion Pharmaceuticals
  • No affiliation available (private)
  • University of Texas
Research areas and keywords

Subject classification (UKÄ) – MANDATORY

  • Urology and Nephrology


  • aHUS, atypical hemolytic uremic syndrome, complement, FACIT-Fatigue, fatigue, patient-reported outcomes
Original languageEnglish
JournalKidney International Reports
Publication statusE-pub ahead of print - 2020
Publication categoryResearch