Effect of dystonia and botulinum toxin treatment on health-related quality of life.

In view of the steadily rising demand for treatment of dystonia with botulinum toxin (BT), a relatively expensive neurologic paralytic agent, an exploratory study was undertaken to assess the extent to which dystonia and BT treatment affect the quality of people's lives. One hundred thirty adults with a current diagnosis of dystonia completed two generic measures of health-related quality of life (HRQoL) at regular intervals over a minimum of 6 months. One hundred two participants were receiving regular injections of BT; 28 were not taking BT. The HRQoL instruments used were the EuroQol and the Short Form 36 health survey questionnaire (SF-36). Compared with general population samples, study participants reported greater impairment on all EuroQol and SF-36 dimensions and gave a lower rating to their own health status. Participants with nonfocal dystonia had significantly more problems with usual activities than participants with focal dystonia, and a higher number had problems with mobility and self-care. The groups reported similar levels of pain and emotional well-being. Small improvements in HRQoL were seen after the administration of BT, although few of these were statistically significant. The study results offer further psychometric evidence for the discriminant and construct validity of both the EuroQol and the SF-36.

Measuring patient benefit from botulinum toxin in the treatment of dystonia. Feasibility of cost-utility analysis.

The dystonias are a group of movement disorders arising from CNS dysfunction and characterised by involuntary and prolonged spasms of muscle contraction. Recently there has been increasing demand for treatment with botulinum toxin (BT), a relatively expensive neurological paralytic agent. As there has been no systematic assessment of patient benefit from BT, this study was undertaken to develop and test a methodology for assessing the cost utility of BT therapy for patients with dystonias. A generic health status instrument, the EuroQOL, was completed at regular intervals over at least 6 months by 130 patients with a current diagnosis of dystonia. A general population tariff was used to calculate quality-adjusted life-year (QALY) gains from BT treatment, and relevant cost data were obtained from patients and medical records. The cost-per-QALY estimates ranged considerably, depending on the type of dystonia, the duration of BT treatment, type of health-related quality-of-life (HR-QOL) tariff used and baseline characteristics of participants. The study findings reflect the general clinical impression of BT: that it can benefit patients with dystonia, but the benefit may be small compared with many treatments for other diseases. The nature of the disease and its cyclical treatment caused practical difficulties in recruiting participants, administering questionnaires and in estimating QALY gains.

Health-related quality of life in endstage renal failure.

The objective of this study was to describe the health-related quality of life (HRQOL) of patients on different forms of treatment for endstage renal failure in such a way that the data could be used in a cost-utility analysis of renal failure treatment in Britain. Twenty-four British renal units participated in this study. 997 adult dialysis and transplant patients were randomly selected from these units using the European Dialysis and Transplant (EDTA) Registry Database. The Health Measurement Questionnaire was completed by 705 of the 900 patients who were alive at the time of the survey (response rate of 78%). The HRQOL data were linked with comorbidity data and with clinical data from the EDTA Registry. Compared to the general population, patients with endstage renal failure experienced a lower quality of life. Many factors contributed to this, but uncertainty about the future and lack of energy emerged as key components. Transplant recipients reported better HRQOL than dialysis patients, they reported fewer problems with physical mobility, self-care, social and personal relationships and usual activities. They also experienced significantly less distress, while dialysis patients reported problems with depression, anxiety, pain and uncertainty about the future. These differences remained after controlling for age and comorbidity.

Measuring health status in the community: a comparison of methods.

STUDY OBJECTIVE--The measurement of health outcomes is central to the evaluation of medical treatment and intervention. It is generally acknowledged that such measurement ought to include an assessment of the impact of health care on the quality of life, as well as its quantity. The Health Measurement Questionnaire (HMQ) was developed as a means of identifying respondents in terms of Rosser's classification of illness states. This study examines the extent of convergent validity of the HMQ when used as a self report measure of health status, alongside the General Health Questionnaire (GHQ) and the Nottingham Health Profile (NHP). DESIGN--A randomised survey of residents of Wolverhampton was drawn from the electoral register. Interviews were conducted in the respondents' own homes. The three measurement instruments formed part of an extensive battery of questionnaires aimed at assessing a broad range of health issues. PARTICIPANTS--A total of 430 respondents were interviewed, of whom 407 completed the HMQ. Altogether 210 also completed the GHQ, and a further 207 completed the NHP. Failure in the interview protocol meant that 12 respondents did not complete either the GHQ or the NHP; these respondents did complete their HMQ. MEASUREMENTS AND MAIN RESULTS--At the descriptive level, Rosser distress categories derived from the HMQ seem to correlate well with the GHQ. There is a strong association between weighted Rosser disability/distress states and scores produced using the NHP. All three measures discriminated between "healthy" and "not healthy" subgroups of respondents. CONCLUSIONS--The results indicate strong evidence for convergent validity. There are significant levels of physical and psychological morbidity within the community. The results of this study reinforce the case for the continued measurement of health status within the general population. Low cost techniques such as the HMQ offer the prospect of such measurement.