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

Greenbaum, Larry A.; Licht, Christoph; Nikolaou, Vasileios S.; Al-Dakkak, Imad; Green, Janet; Haas, Christian; Román-Ortiz, Elena; Cheong, Hae Il; Sartz, Lisa; Swinford, Rita D.; Tomazos, Ioannis; Miller, Benjamin L.; Cataland, Spero R.

doi:10.1016/j.ekir.2020.05.003

Cited by 15 publications

(14 citation statements)

References 25 publications

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“… Patient research Patients are leading or partnering with others (e.g. academic or industry partners) to conduct research and have recognized the need to share their findings by authoring publications in peer-reviewed publications [ 16 , 17 ]. Anecdotally, it seems that the opportunities for patients to author publications may have increased during the COVID-19 pandemic.…”

Section: Factors Driving the Increase In Patient Authorshipmentioning

confidence: 99%

Hidden in plain sight? Identifying patient-authored publications

Oliver¹,

Lobban²,

Dormer

et al. 2022

Res Involv Engagem

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Background Patient advocates are increasingly authoring peer-reviewed publications that could enhance patient care and understanding of the lived experience. Although patient authorship may be seen as an innovation in the peer-reviewed publication environment and some may not be aware of or accept patient authorship, we know patient-authored publications exist. However, identifying patient-authored publications is often challenging and time-consuming. Main body In this commentary, we propose a definition for a patient author and patient-authored publications. We outline factors driving the increase in patient authorship, including patient interest, recognition of the value of including the patient voice and major funders recognising the importance of involving patient advocates in research. Evidence and experience-based guidance on patient authorship is emerging, and we highlight practical guidance for patient advocates on authoring peer-reviewed publications. To gain a better understanding of patient authorship, an efficient method is needed to identify patient-authored publications. A dataset on patient-authored publications could be used for a range of quantitative and qualitative research studies. The affiliation search function in PubMed can provide an easy, and reproducible way to identify a dataset of patient-authored publications in the international peer-reviewed literature, but only if patient authors include a standard metatag, (e.g. Patient Author) as one of their listed affiliations, combined with other affiliations as appropriate. From 2020 to 2021, there was a nine-fold increase in patient-authored publications in PubMed identified using the Patient Author tag. We recognize that terminology can be contentious and some authors may prefer alternative metatags. Further efforts are required to gain consensus on a suitable, standard metatag or set of metatags to use to show the true extent of patient authorship. Conclusion Patient authorship is not only legitimate, but it also exemplifies the principles of diversity, equity and inclusion. Stakeholders in the publication community need to review their policies and procedures to identify and address barriers to patient authorship. Patient advocates, funders, researchers and publishers could all help to promote awareness and acceptance of patient authorship and the merits of using a standard metatag or set of metatags, so that patient-authored publications are no longer hidden in plain sight.

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Section: Factors Driving the Increase In Patient Authorshipmentioning

confidence: 99%

Hidden in plain sight? Identifying patient-authored publications

Oliver¹,

Lobban²,

Dormer

et al. 2022

Res Involv Engagem

View full text Add to dashboard Cite

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“…This could be attributed to this state causing symptoms that limit HRQoL and the possible impact on an individual's ability to continue with their daily activities. Some patients with aHUS can expect relatively frequent hospital admissions and lengthy stays [40,41]. The prospect of a year in which a patient can expect to end up in intensive care and on the general ward for a period was associated with a disutility of between −0.063 and −0.048.…”

Section: Discussionmentioning

confidence: 99%

Utility Values Associated with Atypical Hemolytic Uremic Syndrome-Related Attributes: A Discrete Choice Experiment in Five Countries

et al. 2021

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Background Atypical hemolytic uremic syndrome is a rare disease caused by complement dysregulation that can lead to progressive kidney damage or death if untreated. Owing to its rarity, the impact of atypical hemolytic uremic syndrome and available therapies (eculizumab and ravulizumab) on patients' health-related quality of life is difficult to describe, but such data are required for an economic evaluation. Objective The objective of this study was to estimate utility values for atypical hemolytic uremic syndrome-related attributes in five countries for an economic evaluation. Methods Using discrete choice experiment surveys, key atypical hemolytic uremic syndrome-related attributes (life expectancy, administration frequency, risk of meningitis, need for hospitalization, and risk of kidney impairment) were evaluated in adult general population samples from Australia, Canada, the Netherlands, Sweden, and the UK. Survey choice sets were constructed using a published orthogonal array. A mixed-effects logit model estimated preference strength for each attribute. Utilities were estimated using marginal substitution rates between overall survival and other attributes, weighted against average life expectancy. Results Across all countries (N = 2382), utility weights revealed a consistent pattern: participants were averse to the risk of kidney impairment (disutility/utility weight range: −0.185 to −0.158), risk of meningitis (−0.041 to −0.032), and the need for hospitalization (−0.063 to −0.048), but preferred 8-weekly vs 2-weekly infusions over 1 h (0.013-0.039). Conclusions Although all attributes played a role in determining treatment preferences, the largest drivers were life expectancy and risk of kidney impairment. Participants favored 8-weekly dosing (corresponding to ravulizumab administration frequency) vs 2-weekly dosing. The discrete choice experiment was designed such that estimated (dis)utility weights can be used in future cost-effectiveness models in atypical hemolytic uremic syndrome.

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“…In this edition, Greenbaum et al address the issue of patientreported outcomes (PROs) related to eculizumab therapy in the context of atypical hemolytic uremic syndrome (aHUS). 1 This longitudinal study was spawned by the Global aHUS Registry as an original and collaborative partnership between patients and clinicians committed to a better alignment of clinical research goals with patient needs. 2 Ever since its inception in 2012, this innovative worldwide program, bolstered by industry support, has mustered patient organizations and expert clinicians from 16 countries in a bid to identify and prioritize clinical issues pertaining to aHUS.…”

Section: See Clinical Research On Page 1161mentioning

confidence: 99%

“…address the issue of patient-reported outcomes (PROs) related to eculizumab therapy in the context of atypical hemolytic uremic syndrome (aHUS). 1 …”

mentioning

confidence: 99%