PROMIS® and Neuro-QoL<sup>TM</sup> measures are valid measures of health-related quality of life among patients with familial chylomicronemia syndrome

Fox, Rina S.; Peipert, John Devin; Vera-Llonch, Montserrat; Phillips, Glenn; Cella, David

doi:10.1080/14779072.2020.1748011

Cited by 10 publications

(14 citation statements)

References 35 publications

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“…The following symptoms, which were mentioned by patients in the prior qualitative study, were excluded from our list of the most import symptoms: blurred vision, poor appetite, difficulty concentrating, weight loss, indigestion, muscle weakness. The 12 impacts shown in Table 1 expand upon the findings by Davidson by, for example, detailing specific impacts related to mental and emotional well-being and adding the concept of sleep disruption [ 6 ].…”

Section: Resultsmentioning

confidence: 77%

“…Although items drawn from PROMIS have undergone cognitive debriefing to evaluate language, comprehensibility, ambiguity, and relevance [ 16 ], we conducted cognitive interviews of the draft item set with individuals with FCS to confirm item clarity, meaning, and relevance for FCS. Participants for the cognitive interviews were recruited from a sample of individuals with FCS who participated in the 2018 study, “PROMIS®-Based Survey of Health-Related Quality of Life in Familial Chylomicronemia Syndrome.”[ 6 ]. All participants reported that they had a diagnosis of FCS, were 18 years of age or older, and lived in the United States.…”

Section: Methodsmentioning

confidence: 99%

“…Fox et al [ 6 ] recently administered nine measures from The National Institutes of Health Patient-Reported Outcomes Measurement Information System® (PROMIS®) PROMIS and Quality of Life in Neurological Disorders™ (Neuro-QoL™) via an online survey to a sample of FCS patients and found that these measures captured worse global physical and mental health, anxiety, depression, physical function, fatigue, pain interference, cognitive function, and belly pain among individuals with FCS, relative to the general population. Moreover, FCS patients reported worse sleep disturbance, self-efficacy for managing social interactions and stigma relative to other chronic illness populations.…”

Section: Introductionmentioning

confidence: 99%

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Qualitative development of the PROMIS Profile v1.0-Familial Chylomicronemia Syndrome (FCS) 28

et al. 2022

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Purpose Familial chylomicronemia syndrome (FCS) is a rare genetic disorder characterized by high triglyceride levels, significant disease burden, and negative impacts on health-related quality of life. This project aimed to create a PROMIS-based patient-reported outcome measure that represents valid and important concerns for patients with FCS. Methods We reviewed the literature and data from a previous qualitative study of FCS to identify key FCS symptoms and impacts, which were mapped to PROMIS domains to create a pool of eligible items. Candidate items were reduced per expert feedback and patients with FCS completed cognitive interviews to confirm content validity and measure content. Results Literature and qualitative data review identified ten key symptoms and 12 key impacts of FCS, including abdominal pain, fatigue, difficulty thinking, and worry about pancreatitis attacks. We identified 96 items primarily from PROMIS, supplemented with items from the Quality of Life in Neurological Disorders™ (Neuro-QoL™) and the Functional Assessment of Chronic Illness Therapy (FACIT) measurement systems. This pool was reduced to 32 candidate items, which were assessed via cognitive interviews with eight participants with FCS. Cognitive interview results and additional expert feedback led to the removal of four items and finalization of the PROMIS Profile v1.0—familial chylomicronemia syndrome (FCS) 28. Conclusions The PROMIS Profile v1.0—familial chylomicronemia syndrome (FCS) 28 provides strong content validity for assessing quality of life among patients with FCS. The benefits of PROMIS, including norm-referenced mean values for each measure, will facilitate comparison of patients with FCS to other clinical populations.

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Section: Resultsmentioning

confidence: 77%

Section: Methodsmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Qualitative development of the PROMIS Profile v1.0-Familial Chylomicronemia Syndrome (FCS) 28

et al. 2022

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“…Impacts, too, reflect both the unpredictability and chronic course of the disease and the ways in which patients attempt to manage their FCS and its symptoms. In a recent study, patients with FCS reported both abdominal (“belly”) pain and pain interference considered clinically meaningful based on comparisons to general population norms [ 11 , 12 ]. Similar findings were also reported for cognition impacts [ 11 ].…”

Section: Discussionmentioning

confidence: 99%

“…In a recent study, patients with FCS reported both abdominal (“belly”) pain and pain interference considered clinically meaningful based on comparisons to general population norms [ 11 , 12 ]. Similar findings were also reported for cognition impacts [ 11 ]. In the current study, worry or anxiety was a common theme when patients described ongoing impacts of FCS.…”

Section: Discussionmentioning

confidence: 99%

Development of a novel PRO instrument for use in familial chylomicronemia syndrome

Davidson

Slota

Vera-Llonch

et al. 2021

J Patient Rep Outcomes

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Background Familial chylomicronemia syndrome (FCS), a rare genetic disorder characterized by high levels of circulating triglycerides, negatively impacts multiple organs, including the liver and pancreas. Objective The objective of this study was to develop and support the content validity of a novel patient-reported outcome (PRO) measure addressing FCS symptoms and impacts. To facilitate use in clinical trials of new treatments, evidence supporting the new measure needed to be consistent with regulatory guidance and requirements. Methods A pool of items addressing symptoms and impacts of FCS was initially developed based on data from a large burden-of-illness study with patients with FCS as well as a review of available literature and existing PRO measures. Two rounds of qualitative interviews were conducted with patients with FCS (N = 10) to refine the draft items and support the measure’s content validity. Each interview began with concept elicitation followed by cognitive debriefing of the draft FCS measure. Results Patient-reported symptoms and impacts of FCS were generally consistent with those identified by the burden-of-illness study; abdominal pain was particularly prevalent and salient for patients. Suggested changes to the draft item pool were generally minor. Comprehensibility and ease of completion for the final instrument were confirmed during the second set of interviews. Conclusion The content validity of the final 17-item FCS Symptoms and Impacts Scale is strongly supported by patient input gathered through both a large burden-of-illness study and qualitative research. To further support use in clinical trials, psychometric evaluation of the measure is underway.

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Prevalence and correlates of self‐reported cognitive difficulties in deployment‐injured U.S. military personnel

Jurick

McCabe

Watrous

et al. 2022

Journal of Traumatic Stress

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Cognitive difficulties typically resolve within days to weeks following mild traumatic brain injury (mTBI); however, a sizable proportion of individuals continue to report cognitive symptoms months to years later that are often associated with posttraumatic stress disorder (PTSD) and depression to a greater degree than a history of mTBI. The current study sought to evaluate the prevalence of self-reported cognitive difficulties as well as the relative contributions of demographic, injury-related, and mental health variables in a large study of U.S. military personnel injured during deployment since 2001. Slightly fewer than half (42.0%) of participants reported elevated cognitive difficulties compared with a normative population; however, this was driven primar-

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PROMIS® and Neuro-QoL^TM measures are valid measures of health-related quality of life among patients with familial chylomicronemia syndrome

Abstract: & David Cella (2020) PROMIS® and Neuro-QoL TM measures are valid measures of healthrelated quality of life among patients with familial chylomicronemia syndrome, Expert Review of

Cited by 10 publications

References 35 publications

Qualitative development of the PROMIS Profile v1.0-Familial Chylomicronemia Syndrome (FCS) 28

Qualitative development of the PROMIS Profile v1.0-Familial Chylomicronemia Syndrome (FCS) 28

Development of a novel PRO instrument for use in familial chylomicronemia syndrome

Prevalence and correlates of self‐reported cognitive difficulties in deployment‐injured U.S. military personnel

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PROMIS® and Neuro-QoLTM measures are valid measures of health-related quality of life among patients with familial chylomicronemia syndrome

Abstract: & David Cella (2020) PROMIS® and Neuro-QoL TM measures are valid measures of healthrelated quality of life among patients with familial chylomicronemia syndrome, Expert Review of

Cited by 10 publications

References 35 publications

Qualitative development of the PROMIS Profile v1.0-Familial Chylomicronemia Syndrome (FCS) 28

Qualitative development of the PROMIS Profile v1.0-Familial Chylomicronemia Syndrome (FCS) 28

Development of a novel PRO instrument for use in familial chylomicronemia syndrome

Prevalence and correlates of self‐reported cognitive difficulties in deployment‐injured U.S. military personnel

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PROMIS® and Neuro-QoL^TM measures are valid measures of health-related quality of life among patients with familial chylomicronemia syndrome