Evaluation of health-related quality of life in hemolytic uraemic syndrome patients treated with eculizumab: a systematic evaluation on basis of EMPRO

Mukherjee, Anwesha; Kandhare, Amit D.; Bodhankar, Subhash L.

doi:10.1080/0886022x.2018.1427110

Cited by 15 publications

(8 citation statements)

References 51 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…For example, eculizumab is approved only as an interval medication for secondary prevention of sudden exacerbations of NMOSD symptoms. Nonetheless, eculizumab is being used off‐label in patients with NMOSD suffering from severe symptom exacerbations, while PEX provides little to no benefit, as well as during acute episodes of hemolytic‐uremic syndrome and additional diseases linked to acute complement factor 5 (C5) activation 33 . Therefore, if further research is performed, our selected proteins could serve as significant biomarkers in personalized treatment plans to optimize therapeutic effectiveness and minimize the risks and costs associated with side effects.…”

Section: Discussionmentioning

confidence: 99%

Blood‐based inflammatory protein biomarker panel for the prediction of relapse and severity in patients with neuromyelitis optica spectrum disorder: A prospective cohort study

Wei,

Li,

Zhao

et al. 2024

CNS Neurosci Ther

View full text Add to dashboard Cite

BackgroundTo date, most existing models for predicting neuromyelitis optica spectrum disorder (NMOSD) are based primarily on clinical characteristics. Blood‐based NMOSD severity and prognostic predictive immune‐ and inflammation‐related biomarkers are needed. We aimed to investigate the associations between plasma inflammatory biomarkers and relapse and attack severity in NMOSD.MethodsThis two‐step, single‐center prospective cohort study included discovery and validation cohorts. We quantified 92 plasma inflammatory proteins by using Olink's proximity extension assay and identified differentially expressed proteins in the relapse group (relapse within 1 year of follow‐up) and severe attack group. To define a new molecular prognostic model, we calculated the risk score of each patient based on the key protein signatures and validated the results in the validation cohort.ResultsThe relapse prediction model, including FGF‐23, DNER, GDNF, and SLAMF1, predicted the 1‐year relapse risk. The severe attack prediction model, including PD‐L1 and MCP‐2, predicted the severe clinical attack risk. Both the relapse and severe attack prediction models demonstrated good discriminative ability and high accuracy in the validation cohort.ConclusionsOur discovered biomarker signature and prediction models may complement current clinical risk stratification approaches. These inflammatory biomarkers could contribute to the discovery of therapeutic interventions and prevent NMOSD progression.

show abstract

Section: Discussionmentioning

confidence: 99%

Blood‐based inflammatory protein biomarker panel for the prediction of relapse and severity in patients with neuromyelitis optica spectrum disorder: A prospective cohort study

Wei,

Li,

Zhao

et al. 2024

CNS Neurosci Ther

View full text Add to dashboard Cite

show abstract

“…This analysis of data from the Global aHUS Registry represents the first evaluation of fatigue and other patient-reported outcomes directly from patients enrolled in the registry, including general health status, health care resource utilization, work status, and symptoms, and provides important insights into the burden of aHUS. Previous descriptions of health-related quality of life in patients with aHUS have focused on the effect of eculizumab on health-related quality of life, 21 whereas this analysis provides insights into the overall burden and quality of life in patients with aHUS, regardless of treatment.…”

Section: Discussionmentioning

confidence: 99%

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

Greenbaum

Licht

Nikolaou

et al. 2020

Kidney International Reports

View full text Add to dashboard Cite

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 ¼ 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 ¼ 295) and those never treated (n ¼ 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.

show abstract

“…The FACIT-F scale was originally designed to assess the fatigue among cancer patients, showing good internal consistency reliability and discriminant and convergent validity� 49 The FACIT-F instrument has been evaluated in rheumatoid arthritis and psoriatic arthritis, primary Sjogren's syndrome, osteoarthritis, inflammatory bowel disease, chronic immune thrombocytopenia, Parkinson disease, and systemic lupus erythematosus, as well as many other long-term conditions (e.g., multiple sclerosis, cancer, neurologic disorders)� 48,50,51,[60][61][62][63][64] In a systematic evaluation of quality of life in patients with aHUS treated with eculizumab based on the Evaluating Measures of Patient-Reported Outcomes tool, the psychometric determinants properties of FACIT-F instrument were assessed and rated� This rating was done on the basis on 3 studies where this instrument had been used among patients with aHUS to determine their HRQoL� 10,65,66 Scores generated by the tool were considered reasonably acceptable when they exceeded ≥ 50 points, and the maximum theoretical points were 100. Based on this scale, the reliability, validity, and responsiveness of FACIT-F instrument achieved 25.00, 54.17, and 55.56, respectively, showing a low score for reliability and slightly above the overall threshold of acceptable validity and responsiveness� 52…”

Section: Facit-f Scalementioning

confidence: 92%

Ravulizumab (Ultomiris)

CADTH¹

2023

cjht

View full text Add to dashboard Cite

CADTH reimbursement reviews are comprehensive assessments of the clinical effectiveness and cost-effectiveness, as well as patient and clinician perspectives, of a drug or drug class. The assessments inform non-binding recommendations that help guide the reimbursement decisions of Canada's federal, provincial, and territorial governments, with the exception of Quebec. This review assesses ravulizumab (Ultomiris) 10 mg/mL solution for IV infusion. Indication: For the treatment of adults and pediatric patients with atypical hemolytic uremic syndrome to inhibit complement-mediated thrombotic microangiopathy.

show abstract

Evaluation of health-related quality of life in hemolytic uraemic syndrome patients treated with eculizumab: a systematic evaluation on basis of EMPRO

Cited by 15 publications

References 51 publications

Blood‐based inflammatory protein biomarker panel for the prediction of relapse and severity in patients with neuromyelitis optica spectrum disorder: A prospective cohort study

Blood‐based inflammatory protein biomarker panel for the prediction of relapse and severity in patients with neuromyelitis optica spectrum disorder: A prospective cohort study

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

Ravulizumab (Ultomiris)

Contact Info

Product

Resources

About