The Patient-Reported Outcomes Measurement Information System perspective of adults with long-standing atypical hemolytic uremic syndrome.

BACKGROUND

Atypical hemolytic uremic syndrome (aHUS) is a thrombotic microangiopathy driven by a dysregulation of the complement system, disproportionately affecting the kidneys. While terminal complement blockade has reduced the risk of death and progression to end stage renal disease, patients face significant chronic health challenges. The patient experience in the current treatment era has not been well-characterized.

OBJECTIVES

To evaluate patient-reported health outcomes (PRO) across global health domains, including physical and cognitive function and social isolation.

METHODS

We performed a cross-sectional analysis of adults with aHUS using validated National Institute of Health Patient-Reported Outcomes Measurement Information System instruments.

RESULTS

Compared with a normative reference population, our aHUS cohort (N = 50) had significantly lower mean ± SD T-scores for physical function (44.6 ± 8.9; P < .001) and cognitive function (41.4 ± 12.0; P < .001), and higher scores for anxiety (59.2 ± 9.7; P < .001), depression (55.8 ± 9.7; P < .001), fatigue (59.3 ± 10.7; P < .001), and sleep disturbance (53.6 ± 7.5, P < .001), suggesting overall worse functioning in several domains. Patients with self-reported anxiety or depression had higher scores for depression and anxiety and worse functioning across all measured outcomes.

CONCLUSION

In this first study of PROs in patients with long-standing aHUS in the era of complement-directed therapies, patients reported a chronically impaired global health status with reduced physical and cognitive function and higher levels of fatigue, anxiety, depression, and sleep disturbances compared with the general population. Our data underscore that aHUS is a multisystem disease that may be associated with persistent symptoms even during periods of hematologic and renal quiescence.