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Quality of Life Research

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26-05-2018

Engaging patients and caregivers in prioritizing symptoms impacting quality of life for Duchenne and Becker muscular dystrophy

Auteurs: Ilene L. Hollin, Holly Peay, Ryan Fischer, Ellen M. Janssen, John F. P. Bridges

Gepubliceerd in: Quality of Life Research | Uitgave 9/2018

Abstract

Purpose

Patient preference information (PPI) have an increasing role in regulatory decision-making, especially in benefit–risk assessment. PPI can also facilitate prioritization of symptoms to treat and inform meaningful selection of clinical trial endpoints. We engaged patients and caregivers to prioritize symptoms of Duchenne and Becker muscular dystrophy (DBMD) and explored preference heterogeneity.

Methods

Best–worst scaling (object case) was used to assess priorities across 11 symptoms of DBMD that impact quality of life and for which there is unmet need. Respondents selected the most and least important symptoms to treat among a subset of five. Relative importance scores were estimated for each symptom, and preference heterogeneity was identified using mixed logit and latent class analysis.

Results

Respondents included patients (n = 59) and caregivers (n = 96) affected by DBMD. Results indicated that respondents prioritized “weaker heart pumping” [score = 5.13; 95% CI (4.67, 5.59)] and pulmonary symptoms: “lung infections” [3.15; (2.80, 3.50)] and “weaker ability to cough” [2.65; (2.33, 2.97)] as the most important symptoms to treat and “poor attention span” as the least important symptom to treat [− 5.23; (− 5.93, − 4.54)]. Statistically significant preference heterogeneity existed (p value < 0.001). At least two classes existed with different priorities. Priorities of the majority latent class (80%) reflected the aggregate results, whereas the minority latent class (20%) did not distinguish among pulmonary and other symptoms.

Conclusions

Estimates of the relative importance for symptoms of Duchenne muscular dystrophy indicated that symptoms with direct links to morbidity and mortality were prioritized above other non-skeletal muscle symptoms. Findings suggested the existence of preference heterogeneity for symptoms, which may be related to symptom experience.

vorige artikel Trajectory of health-related quality of life and its determinants in patients who underwent lumbar spine surgery: a 1-year longitudinal study

volgende artikel Anxiety in the orthopedic patient: using PROMIS to assess mental health

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Titel: Engaging patients and caregivers in prioritizing symptoms impacting quality of life for Duchenne and Becker muscular dystrophy
Auteurs: Ilene L. Hollin
Holly Peay
Ryan Fischer
Ellen M. Janssen
John F. P. Bridges
Publicatiedatum: 26-05-2018
Uitgeverij: Springer International Publishing
Gepubliceerd in: Quality of Life Research / Uitgave 9/2018
Print ISSN: 0962-9343
Elektronisch ISSN: 1573-2649
DOI: https://doi.org/10.1007/s11136-018-1891-7

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Abstract

Purpose

Methods

Results

Conclusions

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