Differences in Preferences for Neonatal Outcomes Among Health Care Professionals, Parents, and Adolescents

Saigal, Saroj; Stoskopf, Barbara; Feeny, David; Furlong, William; Burrows, Elizabeth; Rosenbaum, Peter; Hoult, Lorraine

doi:10.1001/jama.281.21.1991

Cited by 325 publications

(213 citation statements)

References 32 publications

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“…This change of internal standards or “response‐shift”41 is not represented in the HUI. In the majority of patients, the responses were generated by parents and health professionals, who tend to provide lower valuations for HRQL than patients,39, 42, 43 suggesting that the discrepancy between HRQL as perceived by patients and as perceived by society may even be greater. No agreement exists on how to do justice to both the objective function and the subjective judgment of HRQL.…”

Section: Discussionmentioning

confidence: 99%

Natural History of Vanishing White Matter

Hamilton

Lei

Vermeulen

et al. 2018

Annals of Neurology

173

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ObjectiveTo comprehensively describe the natural history of vanishing white matter (VWM), aiming at improving counseling of patients/families and providing natural history data for future therapeutic trials.MethodsWe performed a longitudinal multicenter study among 296 genetically confirmed VWM patients. Clinical information was obtained via disease‐specific clinical questionnaire, Health Utilities Index and Guy's Neurological Disability Scale assessments, and chart review.ResultsFirst disease signs occurred at a median age of 3 years (mode = 2 years, range = before birth to 54 years); 60% of patients were symptomatic before the age of 4 years. The nature of the first signs varied for different ages of onset. Overall, motor problems were the most common presenting sign, especially in children. Adolescent and adult onset patients were more likely to exhibit cognitive problems early after disease onset. One hundred two patients were deceased. Multivariate Cox regression analysis revealed a positive relation between age at onset and both preservation of ambulation and survival. Absence of stress‐provoked episodes and absence of seizures predicted more favorable outcome. In patients with onset before 4 years, earlier onset was associated with more severe disability and higher mortality. For onset from 4 years on, disease course was generally milder, with a wide variation in severity. There were no significant differences for sex or for the 5 eIF2B gene groups. The results confirm the presence of a genotype–phenotype correlation.InterpretationThe VWM disease spectrum consists of a continuum with extremely wide variability. Age at onset is a strong predictor for disease course. Ann Neurol 2018;84:274–288

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Section: Discussionmentioning

confidence: 99%

Natural History of Vanishing White Matter

Hamilton

Lei

Vermeulen

et al. 2018

Annals of Neurology

173

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“…The choice of whose values to use to generate the scoring algorithms for generic preference based instruments applicable for young people may have important policy implications because there is evidence to indicate that adults' preferences for identical health states may differ from adolescents' preferences (Ratcliffe et al, 2015b;Ratcliffe et al, 2012b;Norquist et al, 2008;Saigal et al, 1999) In an empirical comparison of adult versus adolescent specific scoring algorithms for the CHU9D and the AQoL-6D Adolescent, Ratcliffe et al (2012b) found notable differences. For the CHU9D instrument, employment of the adolescent algorithms resulted in lower mean health state values than the adult algorithm.…”

Section: Introductionmentioning

confidence: 99%

Valuing the Child Health Utility 9D: Using profile case best worst scaling methods to develop a new adolescent specific scoring algorithm

Ratcliffe

Huynh

Chen

et al. 2016

Social Science & Medicine

115

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In contrast to the recent proliferation of studies incorporating ordinal methods to generate health state values from adults, to date relatively few studies have utilized ordinal methods to generate health state values from adolescents. This paper reports upon a study to apply profile case best worst scaling methods to derive a new adolescent specific scoring algorithm for the Child Health Utility 9D (CHU9D), a generic preference based instrument that has been specifically designed for the estimation of quality adjusted life years for the economic evaluation of health care treatment and preventive programs targeted at young people. A survey was developed for administration in an online format in which consenting community based Australian adolescents aged 11 to 17 years (N=1982) indicated the best and worst features of a series of 10 health states derived from the CHU9D descriptive system. The data were analyzed using latent class conditional logit models to estimate values (part worth utilities) for each level of the nine attributes relating to the CHU9D. A marginal utility matrix was then estimated to generate an adolescent-specific scoring algorithm on the full health = 1 and dead = 0 scale required for the calculation of QALYs. It was evident that different decision processes were being used in the best and worst choices. Whilst respondents appeared readily able to choose 'best' attribute levels for the CHU9D health states, a large amount of random variability and indeed different decision rules were evident for the choice of 'worst' attribute levels, to the extent that the best and worst data should not be pooled from the statistical perspective. The optimal adolescent-specific scoring algorithm was therefore derived using data obtained from the best choices only. The study provides important insights into the use of profile case best worst scaling methods to generate health state values with adolescent populations.3

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“…15 In addition, there is a tendency to emphasize objective measures, deficits and impairments rather than more subjective humanistic values. 16 It is clear that those providing counseling must be well trained, aware of their own inherent biases and willing and able to impart a variety of viewpoints and provide ready access to a wide spectrum of information sources.…”

Section: Volume 34 No 4 -November 2007mentioning

confidence: 99%