Physician and patient-oriented outcomes in progressive neurological disease

Jc, Hobart; Ja, Freeman; Lamping, D

doi:10.1097/00019052-199612000-00008

Cited by 36 publications

(19 citation statements)

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“…However, patients' and clinicians' perceptions of the impact of disease and therapy may differ, and be equally valid [16].Accordingly, there is a growing emphasis on the importance of patient-reported outcome measures, particularly in chronic conditions such as neurological disorders [5,16,39].…”

Section: Introductionmentioning

confidence: 99%

Testing the SF-36 in Parkinson's disease

2008

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Rating scales are increasingly the primary outcome measures in clinical trials. However, clinically meaningful interpretation of such outcomes requires that the scales used satisfy basic requirements (scaling assumptions) within the data. These are rarely tested. The SF-36 is the most widely used patient-reported rating scale. Its scaling assumptions have been challenged in neurological disorders but remain untested in Parkinson's disease (PD). We therefore tested these by analyzing SF-36 data from 202 PD patients (54% men; mean age 70) to determine if it was legitimate to report scores for the eight SF-36 scales and its two summary measures of physical and mental health, and if those scores were reliable and valid. Results supported generation of the eight SF-36 scale scores and their reliabilities were generally good (> or = 0.74 in all but one instance). However, we found limitations that question the meaningfulness of four scales and other limitations that restrict the ability of four scales to detect change in clinical trials (floor/ceiling effects, 19.6-46.2 %). The two SF-36 summary measures were not found to be valid indicators of physical and mental health. This study demonstrates important limitations of the SF-36 and provides the first evidence-based guidelines for its use in PD. The limitations of the SF-36 demonstrated here may explain some unexpected findings in previous studies. However, the main implication is a general one for the clinical research community regarding requirements for reporting rating scale endpoints. Specifically, investigators should routinely provide scale evaluations based on data from within major clinical trials.

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Section: Introductionmentioning

confidence: 99%

Testing the SF-36 in Parkinson's disease

2008

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“…2,3 Children with SB, because of loss of motor function related to the primary lesion, are restricted in the performance of daily activities such as standing, ambulation and the voluntary control of bladder and bowel functions. Consequently, these neurological deficits may greatly impair the QoL of patients and objective information is scarce.…”

Section: Introductionmentioning

confidence: 99%

Assessment of health status in children with spina bifida

et al. 2005

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Study design: Prospective multidimensional study by means of: (1) clinical assessment, (2) parental-administered questionnaire for general health (CHQ-PF50), and (3) standardised disability measurements. Objectives: To assess the health-related quality of life (QoL) and disability in children with spina bifida (SB) and to correlate them with the clinical picture and our previous study on adolescents with SB. Setting: SB Centre at a University Hospital in Italy. Methods: A total of 29 consecutive children with SB (mean age 11.4, range 4-14 years) were evaluated through Child Health Questionnaire Parental Form (CHQ-PF50), the FIM instrument, and the Barthel index. Results: Disability was inversely related only (r ¼ 0.49; P ¼ 0.007) to the physical aspect of the QoL of children. Similarly, the disability was inversely related (r ¼ 0.37; P ¼ 0.005) to the emotional aspect of QoL of patient's parents. Unexpectedly, for the mental aspects of QoL of patients, major disability was not associated with higher psychological distress and severe role disability due to emotional problems. At clinical examination, findings especially for continence and number of catheterisations were usually related to deterioration of physical aspects of QoL (r ¼ À2.28; P ¼ 0.02) in children. Conclusion: The multiperspective assessment showed that there is a linear inverse correlation between disability and QoL in children with SB only for physical aspects. Conversely, there is linear inverse correlation between disability and QoL in patient's parents regarding only emotional aspects. Moreover, this study provided useful information for clinical practice underlining that continence problems are those that most affect QoL in children with SB and their parents.Spinal Cord (2005) 43, 230-235.

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“…2,3 Although outcomes measurement in clinical PD trials has been highly biased toward motor impairment, 4 there has been an increased demand for outcome measures that capture patients' perceptions of their illness in recent years. 5,6 For example, patient-reported outcomes have been advocated in the assessment of cell replacement and functional neurosurgical therapies, 7 and the potential and relevance of such outcomes as means of monitoring disease progression in neuroprotective PD trials have been highlighted recently. 8 The most widely used patient-reported outcomes have been health status questionnaires, typically tapping various physical, mental, and social aspects of health, thus broadly operationalizing the World Health Organization's definition of health.…”

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confidence: 99%

Health status measurement in Parkinson's disease: Validity of the PDQ‐39 and Nottingham Health Profile

et al. 2003

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We assessed the feasibility and psychometric properties of two commonly used health status questionnaires in Parkinson's disease (PD): the generic Nottingham Health Profile (NHP) and the disease-specific 39-item Parkinson's disease Questionnaire (PDQ-39), from a cross-sectional postal survey of PD patients (N = 81), using traditional and Rasch measurement methodologies. Overall response rate was 88%. Both questionnaires were found feasible, although the NHP performed less well. The PDQ-39 had fewer floor effects and was better able to separate respondents into distinct groups than the NHP, whereas the latter exhibited less ambiguous dimensionality and better targeting of respondents with non-extreme scores. Reliability and validity indices were similar, and potential differential item functioning by age and gender groups was found for both questionnaires. PDQ-39 response alternatives indicated ambiguity. With few exceptions, questionnaire scales were unable to meet recommended standards fully. While preliminary, this study illustrates the need for thorough evaluation of outcome measures and has implications beyond the questionnaires used here. Although promising, both questionnaires warrant further developmental work and stronger support of measurement validity before they could be considered fully suitable for valid use in PD, in particular in earlier stages of the disease.

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Physician and patient-oriented outcomes in progressive neurological disease

Cited by 36 publications

References 0 publications

Testing the SF-36 in Parkinson's disease

Testing the SF-36 in Parkinson's disease

Assessment of health status in children with spina bifida

Health status measurement in Parkinson's disease: Validity of the PDQ‐39 and Nottingham Health Profile

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