ArticleJournal of genetic counseling2016
Prioritizing Parental Worry Associated with Duchenne Muscular Dystrophy Using Best-Worst Scaling.
Article in Journal of genetic counseling, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
18 citing papers in PubMed, 1 synthesis or guideline pooled it, 34 citations in OpenAlex.
- Using Best-Worst Scaling to Investigate Preferences in Health Care.PharmacoEconomics · 2016Pooled it
- Development and Evaluation of Decision Partner: A Decision Aid for HIV Remission Clinical Trial Participation.AIDS and behavior · 2025Article
- Draft Guidance for Industry Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and Related Dystrophinopathies - Developing Potential Treatments for the Entire Spectrum of Disease.Journal of neuromuscular diseases · 2024Article
- GM1-gangliosidosis: The caregivers' assessments of symptom impact and most important symptoms to treat.American journal of medical genetics. Part A · 2023Article
- A Comparison of Caregiver and Patient Preferences for Treating Duchenne Muscular Dystrophy.The patient · 2022Article
- Diagnostic experiences of Duchenne families and their preferences for newborn screening: A mixed-methods study.American journal of medical genetics. Part C, Seminars in medical genetics · 2022Article
- A best-worst scaling experiment to prioritize concern about ethical issues in citizen science reveals heterogeneity on people-level v. data-level issues.Scientific reports · 2021Observational
- Comparing the Preferences of Patients and the General Public for Treatment Outcomes in Type 2 Diabetes Mellitus.The patient · 2021Article
- Challenges to informed consent for exome sequencing: A best-worst scaling experiment.Journal of genetic counseling · 2019Article
- Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best-worst scaling experiment in caregivers and adult patients.Orphanet journal of rare diseases · 2019Article
- Article
- Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy: A qualitative preference study of patients and parents.PloS one · 2019Article
- Exploring variation in parental worries about HPV vaccination: a latent-class analysis.Human vaccines & immunotherapeutics · 2019Article
- Parents' Views on the Best and Worst Reasons for Guideline-Consistent HPV Vaccination.Cancer epidemiology, biomarkers & prevention : a publication of the American Association for Cancer Research, cosponsored by the American Society of Preventive Oncology · 2018Article
- Psychosocial Needs and Facilitators of Mothers Caring for Children with Duchenne/Becker Muscular Dystrophy.Journal of genetic counseling · 2018Article
- Would You Use It With a Seal of Approval? Important Attributes of 2,4-Dinitrophenol (2,4-DNP) as a Hypothetical Pharmaceutical Product.Frontiers in psychiatry · 2018Article
- Developing and piloting an instrument to prioritize the worries of patients with acute myeloid leukemia.Patient preference and adherence · 2018Article
- Experimental measurement of preferences in health and healthcare using best-worst scaling: an overview.Health economics review · 2016Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Duchenne muscular dystrophy (DMD) is a progressive, fatal pediatric disorder with significant burden on parents. Assessing disease impact can inform clinical interventions. Best-worst scaling (BWS) was used to elicit parental priorities among 16 short-term, DMD-related worries identified through community engagement. Respondents viewed 16 subsets of worries, identified using a balanced, incomplete block design, and identified the most and least worrying items. Priorities were assessed using best-worst scores (spanning +1 to -1) representing the relative number of times items were endorsed as most and least worrying. Independent-sample t-tests compared prioritization of parents with ambulatory and non-ambulatory children. Participants (n = 119) most prioritized worries about weakness progression (BW score = 0.64) and getting the right care over time (BW = 0.25). Compared to parents of non-ambulatory children, parents of ambulatory children more highly prioritized missing treatments (BW = 0.31 vs. 0.13, p < 0.001) and being a good enough parent (BW = 0.06 vs. -0.08, p = 0.010), and less prioritized child feeling like a burden (BW = -0.24 vs. -0.07, p < 0.001). Regardless of child's disease stage, caregiver interventions should address the emotional impact of caring for a child with a progressive, fatal disease. We demonstrate an accessible, clinically-relevant approach to prioritize disease impact using BWS, which offers an alternative to the use of traditional rating/ranking scales.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.