Assessing the costs of healthcare technologies in clinical trials.
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Biomedical subjects
Publications and source records attributed to R Fitzpatrick.
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BACKGROUND: Routine follow-up of breast cancer patients in specialist clinics is standard practice in most countries. Follow-up involves regularly scheduled breast cancer check-ups during the disease-free period. The aims of follow-up are to detect breast cancer recurrence and to provide psychosocial support to the patient; however, little is known about patients' views on breast cancer follow-up. AIM: To assess the effect on patient satisfaction of transferring primary responsibility for follow-up of women with breast cancer in remission from hospital outpatient clinics to general practice. METHOD: Randomized controlled trial with 18 months' follow-up in which women received routine follow-up either in hospital outpatient clinics or from their own general practitioner. Two hundred and ninety-six women with breast cancer in remission receiving regular follow-up care at two district general hospitals in England were included in the study. Patient satisfaction was measured by means of a self-administered questionnaire supplied three times during the 18-month study period. RESULTS: The general practice group selected responses indicating greater satisfaction than did the hospital group on virtually every question. Furthermore, in the general practice group there was a significant increase in satisfaction over baseline; a similar significant increase in satisfaction over baseline was not found in the hospital group. CONCLUSION: Patients with breast cancer were more satisfied with follow-up in general practice than in hospital outpatient departments. When discussing follow-up with breast cancer patients, they should be provided with complete and accurate information about the goals, expectations, and limitations of the follow-up programme so that they can make an informed choice.
Parkinson's disease is a common degenerative neurological condition. A number of general instruments exist to measure quality of life, but these were not designed to address areas salient to a specific disease. This contribution reviews the development and validation of the PDQ-39, a short 39-item quality of life questionnaire for Parkinson's disease. PDQ-39 data can be presented either in profile form or as a single index. This report also reviews the development and validation of a briefer measure (PDQ-8) derived from the PDQ-39, and of two summary indices (PDQ-39SI and PDQ-8SI.
Quality of life is increasingly regarded as an important outcome measure in the evaluation of treatment regimes. The last decade has seen an enormous growth in the application of measures designed to assess quality of life in a vast array of medical specialities. However, the use of such measures in neurology has been limited and is virtually non-existent in amyotrophic lateral sclerosis (ALS). The European ALS Health Profile Study is a longitudinal survey of patients diagnosed with ALS or motor neurone disease in which patients are asked to complete questionnaires concerning their subjective health status. Data from clinical assessments is also collected. It is intended that the information collected will provide more systematic and detailed evidence of the impact of the disease from the perspective of the patient. This paper outlines the purpose and methodology of the project.
Many new hip prostheses are now available for use in total hip replacement. The majority remain untested relative to standard prostheses; however, many new prostheses are substantially more costly. We examine how much more effective new prostheses must be, in terms of reducing the need for revision operations, in order to justify this increased cost.
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OBJECTIVE: To assess the reliability, validity and sensitivity to change of the Derriford Scale, a quality of life instrument designed to assess the distress and dysfunction experienced by people who are self-conscious about their physical appearance. SUBJECTS AND DESIGN: Postal questionnaire survey of 656 cosmetic surgery patients recruited from new referral letters and plastic surgery waiting list reports. A total of 443 subjects completed and returned the questionnaire. Of these respondents, 203 were sent a second questionnaire to assess reproducibility, of whom 155 subjects responded. MAIN OUTCOME MEASURES: Reliability was assessed in terms of internal consistency and reproducibility. Face, content and construct validity were also investigated. RESULTS: The analyses led to mixed results. There was some evidence of internal consistency, but a number of items had low endorsement levels and there may be scope for reducing the overall size of the instrument. When assessed for reproducibility, the level of agreement between scores of individuals completing the questionnaire on two occasions was high, but an important systematic shift in responses was also detected. Correlations between the other health status measures and the Derriford Scale provided some evidence of construct validity. CONCLUSION: In its present form the Derriford Scale has good descriptive value, but there are some measurement problems identified in this report that need to be addressed before the scale is taken up into general use.
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We have developed a 12-item questionnaire for patients having a total knee replacement (TKR). We made a prospective study of 117 patients before operation and at follow-up six months later, asking them to complete the new questionnaire and the form SF36. Some also filled in the Stanford Health Assessment Questionnaire (HAQ). An orthopaedic surgeon completed the American Knee Society (AKS) clinical score. The single score derived from the new questionnaire had high internal consistency, and its reproducibility, examined by test-retest reliability, was found to be satisfactory. Its validity was established by obtaining significant correlations in the expected direction with the AKS scores and the relevant parts of the SF36 and HAQ. Sensitivity to change was assessed by analysing the differences between the preoperative scores and those at follow-up. We also compared change in scores with the patients' retrospective judgement of change in their condition. The effect size for the new questionnaire compared favourably with those for the relevant parts of the SF36. The change scores for the new knee questionnaire were significantly greater (p < 0.0001) for patients who reported the most improvement in their condition. The new questionnaire provides a measure of outcome for TKR that is short, practical, reliable, valid and sensitive to clinically important changes over time.
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OBJECTIVE: To assess the validity and responsiveness of a questionnaire to assess health-related quality of life in Parkinson's disease (PD)--the 39-item Parkinson's Disease Questionnaire (PDQ-39)--and to report problems experienced by patients by means of the questionnaire. METHODS: Patients completed the PDQ-39 and the SF-36 at baseline and 4 months later. At the same assessments, neurologists rated patients with Hoehn and Yahr and Columbia Scales. RESULTS: Evidence for validity of the new questionnaire was observed by agreement of scores with clinical scales at both assessments. Evidence for responsiveness of scales assessing physical function, particularly mobility and activities of daily living, was observed from significant paired t tests for differences between scores at baseline and follow-up, and correlations with patients' retrospective judgments and changes in the SF-36 summary scores. However, there were no significant associations with changes in neurologists' clinical scores. Patients most frequently reported problems of physical function in the PDQ-39. Scores for several dimensions of the PDQ-39 were significantly more favorable than those reported by nonclinic samples of patients with PD. CONCLUSIONS: The PDQ-39 has validity for use among patients attending neurological clinics for treatment of PD. There is also some evidence of responsiveness. The questionnaire identifies problems that are important to patients and that appear to be more commonly experienced by nonclinic attenders.
OBJECTIVE: To assess the stretch of pronator muscles produced by a specifically designed upper-limb Lycra garment that could have a better acceptability than rigid splints in treating upper-limb spasticity. DESIGN: Double-blind comparison among three garments. They were designed to produce a supinating, a pronating, and no torsional force, and were individually manufactured and tested in 10 healthy volunteers. MAIN OUTCOME MEASURE: Angular position and passive rotational stiffness of the forearm were measured with and without each of the garments immediately after the garment was fitted and every hour for 6 hours. RESULTS: When put on by a trained person, the supinator garment supinated the forearm in all subjects (mean, 17 degrees; p < .01; range, 5 degrees to 44 degrees) while the pronator garment pronated the forearm in 8 of 10 subjects (mean, 5 degrees; p < .01). These effects gradually decayed over 6 hours, as garment position was not readjusted. Passive rotational stiffness of the forearm increased by about 30% with each type of garment. The garments designed to produce no torsional force exerted no intrinsic rotational effect. CONCLUSION: Individually made Lycra garments can produce continuous stretch of muscles for several hours and may be useful in the treatment of spasticity. The garments, however, must be put on by a trained person and their position adjusted when necessary.
OBJECTIVES: to briefly outline the development and validation of the Parkinson's Disease Questionnaire (PDQ-39) and then to provide evidence for the use of the measure as either a profile of health status scores or a single index figure. DESIGN: the PDQ-39 was administered in two surveys: a postal survey of patients registered with local branches of the Parkinson's Disease Society of Great Britain (n = 405) and a survey of patients attending neurology clinics for treatment for Parkinson's disease (n = 146). Data from the eight dimensions of the PDQ-39 were factor-analysed. This produced a single factor on the data from both surveys. OUTCOME MEASURES: the eight dimensions of the PDQ-39 and the new single index score-the Parkinson's disease summary index (PDSI), together with clinical assessments (the Columbia rating scale and the Hoehn and Yahr staging score). RESULTS: in the postal survey 227 patients returned questionnaires (58.2%). AH 146 patients approached in the clinic sample agreed to take part. Higher-order principal-components factor analysis was undertaken on the eight dimensions of the PDQ-39 and produced one factor on both datasets. Consequently it was decided that the scores of the eight domains could be summed to produce a single index figure. The psychometric properties of this index were explored using reliability tests and tests of construct validity. The newly derived single index was found to be both internally reliable and valid. DISCUSSION: data from the PDQ-39 can be presented either in profile form or as a single index figure. The profile should be of value in studies aimed at determining the impact of treatment regimes upon particular aspects of functioning and well-being in patients with Parkinson's disease, while the PDSI will provide a summary score of the impact of the illness on functioning and well-being and will be of use in the evaluation of the overall effect of different treatments. Furthermore, the PDSI reduces the number of statistical comparisons and hence the role of chance when exploring data from the PDQ-39.
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A sample of people with Parkinson's disease (PD), identified from a 1992 Northampton Health Authority (NHA) Prevalence Study, and currently registered with a general practitioner, completed a postal survey. People with PD reported considerably worse health status than other individuals of the same age across a wide range of health-related dimensions. Health status was worse in those with longer-standing illness. The Parkinson's disease questionnaire (PDQ-39) identified particular problems associated with PD. The health status for those seen most recently by various health-related professionals was generally worse. Access to various services continues to be a problem for a substantial minority of people with PD. The health status of those reporting dissatisfaction with access to services was generally worse. More research, using feasible and relevant outcome measures, is needed to assess different interventions, and to assist in the determination of appropriate services.
1. The effects of hydrostatic changes in perfusion pressure on performance of working fatigue-resistant muscle fibres in the hand were studied in six normal subjects. 2. Supramaximal stimuli were delivered in trains of 200 ms duration, at 1 train S-1, to the ulnar nerve proximal to the wrist to produce isometric contractions of adductor pollicis. The force of contraction was measured and, after a level contraction force was achieved, the arm was passively raised or lowered. 3. Lifting the hand 45 cm above the heart produced a decline in force output from the muscle within several seconds which, after 4 min, fell by 22% below the steady-state level. Lowering the hand 45 cm below heart level produced an 8% increase in force output. Greater changes in force output occur at higher workloads. 4. It is concluded that in human subjects, muscle performance is sensitive to changes in perfusion pressure that occur across the physiological range. At moderate work levels, force output of the working muscles in the hand can vary by up to 30% over the physiological range of blood pressure. This dependence of muscular force on blood perfusion is of potential importance to motor control during normal activities.