scholarly journals REiNS: Patient Report of Hearing in Neurofibromatosis Type 2: Recommendations for Clinical Trials

Neurology ◽  
2021 ◽  
pp. 10.1212/WNL.0000000000012424
Author(s):  
Heather L. Thompson ◽  
Ann Blanton ◽  
Barbara Franklin ◽  
Vanessa L. Merker ◽  
Kevin H. Franck ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Clinical Trials ◽  
Neurofibromatosis Type ◽  
The Self ◽  
Self Assessment ◽  
Hearing Function ◽  
Patient Reported ◽  
Related Quality

Objective:To systematically evaluate published patient-reported outcome measures for the assessment of hearing function and hearing-related quality of life, and subsequently recommend measures selected by the Response Evaluation in Neurofibromatosis and Schwannomatosis International Collaboration (REiNS) as endpoints for clinical trials in Neurofibromatosis Type 2.Methods:The REiNS patient-reported outcomes working group systematically evaluated published patient-reported outcome measures of (1) hearing function and (2) hearing-related quality of life for individuals with hearing loss of various etiologies using previously published REiNS rating procedures. Ten measures of hearing functioning and 11 measures of hearing-related quality of life were reviewed. Measures were numerically scored and compared primarily on their participant characteristics (including participant age range and availability of normative data), item content, psychometric properties, and feasibility for use in clinical trials.Results:The Self-Assessment of Communication and the Self-Assessment of Communication-Adolescent were identified as most useful for adult and pediatric populations with NF2, respectively, for the measurement of both hearing function and hearing-related quality of life. Measures were selected for their strengths in participant characteristics, item content, psychometric properties, and feasibility for use in clinical trials.Conclusions:Currently, REiNS recommends the Self-Assessment of Communication adult and adolescent forms for the assessment of patient-reported hearing function and hearing-related quality of life for clinical trials for neurofibromatosis type 2. Further work is needed to demonstrate the utility of these measures in evaluating pharmacological and/or behavioral interventions.

scholarly journals Toward Patient-Centered Innovation

2020 ◽  
Vol 15 (10) ◽  
pp. 1522-1530
Author(s):  
Jennifer E. Flythe ◽  
Tandrea S. Hilliard ◽  
Kourtney Ikeler ◽  
San Keller ◽  
Debbie S. Gipson ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Clinical Trials ◽  
Conceptual Framework ◽  
Drug Administration ◽  
Food And Drug Administration ◽  
Health Related Quality ◽  
Patient Reported ◽  
Related Quality ◽  
Health Related

Individuals with dialysis-dependent kidney failure experience considerable disease- and treatment-related decline in functional status and overall well-being. Despite these experiences, there have been few substantive technological advances in KRT in decades. As such, new federal initiatives seek to accelerate innovation. Historically, integration of patient perspectives into KRT product development has been limited. However, the US Food and Drug Administration recognizes the importance of incorporating patient perspectives into the total product life cycle (i.e., from product conception to postmarket surveillance) and encourages the consideration of patient-reported outcomes in regulatory-focused clinical trials when appropriate. Recognizing the significance of identifying patient-reported outcome measures (PROMs) that capture contemporary patient priorities, the Kidney Health Initiative, a public–private partnership between the American Society of Nephrology and US Food and Drug Administration, convened a workgroup to (1) develop a conceptual framework for a health-related quality of life PROM; (2) identify and map existing PROMs to the conceptual framework, prioritizing them on the basis of their supporting evidence for use in the regulatory environment; and (3) describe next steps for identifying PROMs for use in regulatory clinical trials of transformative KRT devices. This paper summarizes the proposed health-related quality-of-life PROM conceptual framework, maps and prioritizes PROMs, and identifies gaps and future needs to advance the development of rigorous, meaningful PROMS for use in clinical trials of transformative KRT devices.

scholarly journals Health-related Quality of Life of Individuals With Neurofibromatosis Type 2

2016 ◽  
Vol 37 (5) ◽  
pp. 574-579 ◽  
Author(s):  
Vanessa L. Merker ◽  
Amanda L. Bergner ◽  
Ana-Maria Vranceanu ◽  
Alona Muzikansky ◽  
William Slattery ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Neurofibromatosis Type ◽  
Neurofibromatosis Type 2 ◽  
Health Related Quality ◽  
Related Quality ◽  
Health Related

Lessons Learned in the Assessment of Health-Related Quality of Life: Selected Examples From the National Cancer Institute of Canada Clinical Trials Group

2007 ◽  
Vol 25 (32) ◽  
pp. 5078-5081 ◽  
Author(s):  
Michael Brundage ◽  
David Osoba ◽  
Andrea Bezjak ◽  
Dongsheng Tu ◽  
Michael Palmer ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Clinical Trials ◽  
National Cancer Institute ◽  
Lessons Learned ◽  
Quality Data ◽  
Health Related Quality ◽  
Patient Reported ◽  
Related Quality ◽  
Health Related

In this article, we provide a brief historical review of the development of patient-reported outcome measurement, analysis, and reporting in clinical trials of the National Cancer Institute of Canada Clinical Trials Group (NCIC CTG). In doing so, we examine selected lessons learned in furthering the quality of these data and their application to clinical practice. We conclude that sequential institution of key policies within the NCIC CTG and the development of a collective philosophy within the group has enabled the routine incorporation of health-related quality of life into clinical trial protocols according to robust scientific principles; that collection of quality data is possible in a variety of circumstances (although not universally so); that patient-reported data on subjective experiences is likely to be more reliable and valid than conventional toxicity information; and that simple analyses that report group trends as well as individual patient response rates are preferred.

scholarly journals Monitoring patients with juvenile idiopathic arthritis using health-related quality of life

2021 ◽  
Vol 19 (1) ◽  
Author(s):  
Martijn J. H. Doeleman ◽  
Sytze de Roock ◽  
Nathan Buijsse ◽  
Mark Klein ◽  
Gouke J. Bonsel ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Disease Activity ◽  
Roc Curves ◽  
Health Related Quality ◽  
Self Assessment ◽  
Diagnostic Characteristics ◽  
Patient Reported ◽  
Related Quality ◽  
Health Related

Abstract Background Pediatric patients with juvenile idiopathic arthritis (JIA) are at risk for a lower health-related quality of life compared to their healthy peers. Remote monitoring of health-related quality of life using electronic patient-reported outcomes could provide important information to treating physicians. The aim of this study was to investigate if self-assessment with the EuroQol five-dimensional ‘youth’ questionnaire with five levels (EQ-5D-Y-5 L) inside a mobile E-health application could identify JIA patients in need of possible treatment adjustments. Methods The EQ-5D-Y-5 L was completed via a mobile application (Reuma2Go) between October 2017 and January 2019. The clinical juvenile arthritis disease activity score with 71 joint count (cJADAS-71) was reported at every corresponding visit as reference for disease activity. Previously described cJADAS-71 thresholds were used to identify patients in possible need of treatment adjustments. Discriminatory power of the EQ-5D-Y-5 L was assessed by ROC-curves and diagnostic characteristics. Results Sixty-eight JIA patients completed the EQ-5D-Y-5 L questionnaire. Median cJADAS-71 indicated low disease activity overall in the studied population. ROC curves and diagnostic characteristics demonstrated that self-assessment with the EQ-5D-Y-5 L could distinguish between patients with inactive disease (or minimal disease activity) and moderate to high disease activity with good accuracy (87%), sensitivity (85%), specificity (89%) and negative predictive value (86%). Conclusions Results demonstrate that the EQ-5D-Y-5 L was able to identify JIA patients in need of possible treatment adjustments in our studied population. Remote monitoring of health-related quality of life and patient-reported outcomes via E-health applications could provide important additional information to determine the frequency of clinical visits, assess therapeutic efficacy and guide treat-to-target strategies in pediatric patients with JIA.

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