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

Greenbaum, Larry A. ; Licht, Christoph ; Nikolaou, Vasileios ; Al-Dakkak, Imad ; Green, Janet ; Haas, Christian Stefan ; Román-Ortiz, Elena ; Cheong, Hae Il ; Sartz, Lisa LU and Swinford, Rita , et al. (2020) In Kidney International Reports
Abstract

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... (More)

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.

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organization
publishing date
type
Contribution to journal
publication status
epub
subject
keywords
aHUS, atypical hemolytic uremic syndrome, complement, FACIT-Fatigue, fatigue, patient-reported outcomes
in
Kidney International Reports
publisher
Elsevier Inc.
external identifiers
  • scopus:85087768012
  • pmid:32775815
ISSN
2468-0249
DOI
10.1016/j.ekir.2020.05.003
language
English
LU publication?
yes
id
7ffe7333-9451-492e-9bd8-32d7c187edaa
date added to LUP
2020-07-24 08:57:20
date last changed
2020-11-16 03:40:51
@article{7ffe7333-9451-492e-9bd8-32d7c187edaa,
  abstract     = {<p>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.</p>},
  author       = {Greenbaum, Larry A. and Licht, Christoph and Nikolaou, Vasileios and Al-Dakkak, Imad and Green, Janet and Haas, Christian Stefan and Román-Ortiz, Elena and Cheong, Hae Il and Sartz, Lisa and Swinford, Rita and Tomazos, Ioannis and Miller, Benjamin and Cataland, Spero},
  issn         = {2468-0249},
  language     = {eng},
  publisher    = {Elsevier Inc.},
  series       = {Kidney International Reports},
  title        = {Functional Assessment of Fatigue and Other Patient-Reported Outcomes in Patients Enrolled in the Global aHUS Registry},
  url          = {http://dx.doi.org/10.1016/j.ekir.2020.05.003},
  doi          = {10.1016/j.ekir.2020.05.003},
  year         = {2020},
}