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IMPORTANCE: Many people with Parkinson disease (PD) develop motor complications that are uncontrolled by levodopa dose adjustment. Among these patients, it is uncertain which drug class is more effective as adjuvant therapy. OBJECTIVE: To compare the long-term effects on patient-rated quality of life of adding a dopamine agonist vs a dopamine reuptake inhibitor (DRI), either a monoamine oxidase type B (MAO-B) inhibitor or a catechol-O-methyltransferase (COMT) inhibitor, to levodopa therapy for the treatment of patients with motor complications of PD. DESIGN, SETTING, AND PARTICIPANTS: This pragmatic semifactorial (2 × 1) randomized clinical trial recruited from 64 neurology and geriatric clinics (62 in the United Kingdom, 1 in the Czech Republic, and 1 in Russia) between February 23, 2001, and December 15, 2009. A total of 500 patients with idiopathic PD who developed uncontrolled motor complications and did not have dementia were randomly assigned on a 1:1:1 basis using a computerized minimization program. Data were analyzed between 2017 and 2020. INTERVENTIONS: Open-label dopamine agonist, MAO-B inhibitor, or COMT inhibitor. MAIN OUTCOMES AND MEASURES: Primary outcomes were scores on the 39-item Parkinson’s Disease Questionnaire (PDQ-39) mobility domain and cost-effectiveness. Outcomes were assessed before study entry, at 6 and 12 months after randomization, and annually thereafter. Repeated-measures and log rank analyses were used in an intention-to-treat approach. RESULTS: Among 500 participants, the mean (SD) age was 73.0 (8.2) years; 314 participants (62.8%) were men. Over a median of 4.5 years (range, 0-13.3 years) of follow-up, the participants in the dopamine agonist group had a mean PDQ-39 mobility score that was 2.4 points (95% CI, −1.3 to 6.0 points) better than that of the combined MAO-B and COMT groups; however, this difference was not significant (P=.20). With regard to DRIs, participants in the MAO-B group had mean PDQ-39 mobility scores that were 4.2 points (95% CI, 0.4-7.9 points; P=.03) better than those of the COMT group and EuroQol 5-dimension 3-level (EQ-5D-3L) utility scores that were 0.05 points (95% CI, 0.003-0.09 points; P=.04) better than the COMT group. Nonsignificant improvements were found in the PDQ-39 summary index (mean difference, 2.2 points; 95% CI, −0.2 to 4.5 points; P=.07) along with nonsignificant reductions in dementia (rate ratio [RR], 0.70; 95% CI, 0.47-1.03; P = .07) and mortality (RR, 0.76; 95% CI, 0.56-1.03; P=.07). When dopamine agonists were compared with MAO-B inhibitors only, the outcomes were similar. CONCLUSIONS AND RELEVANCE: In this study, patient-rated quality of life was inferior when COMT inhibitors were used as adjuvant treatment compared with MAO-B inhibitors or dopamine agonists among people with PD who experienced motor complications that were uncontrolled by levodopa therapy. The MAO-B inhibitors produced equivalent disease control, suggesting that these agents may be underused as adjuvant therapy. TRIAL REGISTRATION: isrctn.org Identifier: ISRCTN69812316; EU Clinical Trials Register Identifier: 2005-001813-16

Background: The PDQ-39 is the most thoroughly validated and widely used self-report instrument for the assessment of health-related quality of life in people with Parkinson's (PwP). Given the breadth of its use and increasing emphasis on electronic data capture, there may be significant benefit in validating an electronic version of the PDQ-39. Objective: Firstly, to migrate the paper-based version of the PDQ-39 to a computer based platform and assess its usability and acceptability to respondents. Secondly, to investigate the impact of implementing non-response options on response rates and data completeness. Methods: Six PwP participated in cognitive interviews in order to assess the usability and acceptability of the electronic version of the PDQ-39, the ePDQ. This was followed by an online survey of 129 PwP, randomly assigned to one of two groups; one required to provide a response to every item and one with the option to skip any item they did not wish to answer. Results: Cognitive interviews indicated that the ePDQ is acceptable to PwP, with positive feedback regarding layout, features and functionality. 125 PwP fully completed the ePDQ. Following randomization 60 participants completed the forced response ePDQ and 65 completed the non-forced version. Response rates of 98.4% were achieved for the forced response ePDQ and 95.6% for the non-forced. Missing value analyses calculated levels of missing data at below 5% in the non-forced sample. Conclusions: The ePDQ is user-friendly and acceptable to respondents. Additionally, there appears little difference when implementing non-response options on response rates and data completeness.

The Stroke Impact Scale (SIS) covers 8 dimensions and a composite disability score. This study evaluates the SIS in the UK context, and develops a single index and an 8-item short form.

Introduction Endometriosis can be difficult to diagnose clinically and models that use symptoms to predict whether the disease is present or not are based on limited patient populations. Endometriosis also influences health-related quality of life, but little is known about its impact across the world. We therefore initiated two integrated multi-center studies to collect prospective, standardized, epidemiological data, to 1) examine the global impact of endometriosis and relative effect of risk-factors, and 2) develop a symptom-based diagnostic tool.Methods The Global Study of Women's Health (GSWH) and the Women's Health Symptom Survey (WHSS) prospectively recruit 18–45 year old women having a laparoscopy across 23 and 19 centers, respectively, worldwide. Women with a previous surgical diagnosis of endometriosis are excluded. Multi-lingual patient questionnaires and a surgical questionnaire, incorporating validated instruments, are used to collect the data. The GSWH aims to recruit >2,000 women by December 2009; the WHSS to recruit 1,000 women in each of the two model-generating and validation stages.Results A six-week pilot study in Oxford, UK, established the feasibility of the study protocols. Of 32 eligible women, 27 participated (response rate - 84.4%); 26% completed the questionnaire online. Endometriosis was found in 47.4%. Extrapolating the recruitment rates from the pilot study, the target sample sizes for the GWSH and WHSS were deemed feasible.Conclusions Using standardized data collection, the GSWH and WHSS will provide insight into the global impact of endometriosis and develop a validated, symptom-based, diagnostic tool. They have the potential to provide the basis for future, longitudinal, follow-up studies and a collaborative Endometriosis Biobank implementing standardized collection of DNA and tissue samples

The purpose of this study was to develop and test a core set of questions to measure patients' experiences of in-patient care. Questions were selected from the bank of items developed for use in in-patient surveys undertaken by the Picker Institute for the purposes of assessing the quality of care.

Objective:To compare the distribution of the domains and summary scores of the SF‐36 health survey by occupation‐related social classes in England and Australia for employed males age 18–65 years. Method:Relative and absolute measures of inequality based on the concentration index were used to examine the distribution of indicators of reported health based on domains and summary scores of the SF‐36 across occupation‐related social classes in both countries. Results:The degree of inequality is most pronounced in the domains and summary scores of the SF‐36 representing aspects of physical health. In regard to comparisons between these countries, there is no significant difference in the distribution of summary scores for physical health. Although there are differences in the summary scores for mental health, there is no evidence of significant inequality in Australia in this aspect of health. Conclusions:There is a similar pattern of occupation‐related health inequality in the physical health dimensions of the SF‐36 health survey for employed males age 18–65 years. Implications:Given the similarities in the distribution of measures of physical health in the two countries, it would be useful to assess recent English policy initiatives aimed at reducing health inequalities when developing interventions to tackle inequalities in Australia.

To describe a new diseasespecific questionnaire with established measurement properties that addresses the dimensions of healthrelated quality of life considered important to women with endometriosis.

The Short Form 12 health survey is a short, easily completed and psychometrically sound generic measure of health status that can be used to measure population health, patient health perceptions and assess treatment effectiveness. Here, for the first time in the Irish context, population norms for two health summary scales for the Irish population are derived from the SF-12 and compared to norms for the English population. Results show that, although the Irish population is in general both physically and mentally healthier than the English population, the difference in physical health is not significant among men and weakens among both men and women with age.

In recent years there has been an increased appreciation of the importance of measuring health status from the patient’s point of view, but until now no attempt has been made to develop an amyotrophic lateral sclerosis (ALS)-specific health status measure. The development of such an instrument is especially relevant now with the introduction of drugs that prolong life in ALS but limited data is available on the impact such treatments have on quality of life. This paper reports on the development of an ALS-specific measure, the forty item ALS assessment questionnaire (ALSAQ-40). The development of the ALSAQ-40 followed three main stages. Stage 1 consisted of in-depth, semi-structured exploratory interviews conducted on a sample of 18 patients to identify areas of salience and concern to patients with ALS. These interviews generated 78 candidate questions. In stage 2, the 78-item questionnaire was used in a postal survey to identify appropriate rephrasing/shortening and to determine the acceptability of the measure. In addition, this exercise helped identify sub-scales of the instrument addressing different dimensions of ALS. Finally in stage 3 the data collected in stage 2 was analysed to areas measured by the instrument and to remove redundant question. The resulting measure contains forty questions measuring five areas of health status: Eating and Drinking, Communication, ADL/ independence, Physical mobility, Emotional Functioning. The measure has high face, internal and construct validity and is likely to prove a useful measure in the evaluation of treatment regimes for ALS/MND.

AbstractThe Parkinson's Disease Questionnaire (PDQ-39) is a disease specific measure of health status which covers eight dimensions of ill-health, and contains 39 questions. The development of the questionnaire has been outlined in full elsewhere and is briefly outlined in this paper. Psychometric techniques are used here to derive a single index figure from the PDQ-39, transformed to a score range from 0 (good health) to 100 (poor health). A sub-set of items is then selected from the PDQ-39 to create a shorter form version, the PDQ-8. Each item selected is the one most highly correlated with the overall domain score to which it contributes: in cases where more than one item correlated equally strongly with the total then the item which received the highest completion rate was selected. The items were summed together and transformed onto a score from 0 to 100. The results gained from the single index gained from the PDQ-39 are compared with that gained from the PDQ-8, and are found to be remarkably similar. The use of the PDQ-8 is recommended over the PDQ-39 where a shorter form measure is required and where a single index measure of overall health status is acceptable or desirable.

The authors compare two generic measures of health status with disease-specific measures in a randomized controlled trial of transurethral resection of the prostate with laser vaporization prostatectomy for benign prostatic hypertrophy.

Objectives: To provide normative data, in the form of percentile scores from a community sample, for the Physical Component and Mental Health Component Summary scores derived from the SF-36, and to provide an example of how to interpret scores on these measures, comparing normative results with data from a clinical sample.Method: Normative data were gained from a postal survey using a questionnaire, containing the SF-36 and a number of other items concerned with lifestyles and illness.The questionnaire was sent to 13 042 randomly selected subjects between the ages of 18 and 64 years, drawn from Family Health Services Authority computerised registers for four English counties. The clinical sample comprised 84 patients aged 18–64 years diagnosed with obstructive sleep apnoea (OSA) who were asked to take part in the study. The Physical Component Summary (PCS) score and Mental Health Component Summary (MCS) score gained from the SF-36 health status measure were the outcome measures.Results: The community survey achieved a response rate of 72% (n = 9332). All 84 patients in the age range 18–64 years approached to take part in the OSA study agreed to do so; complete data were available for 60 patients. Results indicated that, prior to treatment, 75% of OSA patients' scores on the PCS/MCS were less than the standardised mean score of 50 and fell in the lowest 34% of scores in the general population. However, after treatment, over 50% of patients scored above the standardised mean score on both the PCS and MCS and more closely mirrored the distribution of the normative sample.Conclusion: The data provided here should enable a more meaningful presentation of data than is generally provided in research papers presenting SF-36 summary scores.

Objectives: The 36 item short form health survey (SF-36) has proved to be of use in a variety of settings where a short generic health measure of patient-assessed outcome is required. This measure can provide an eight dimension profile of health status, and two summary scores assessing physical function and mental well-being. The developers of the SF-36 in America have developed algorithms to yield the two summary component scores in a questionnaire containing only one-third of the original 36 items, the SF-12. This paper documents the construction of the UK SF-12 summary measures from a large-scale dataset from the UK in which the SF-36, together with other questions on health and lifestyles, was sent to randomly selected members of the population. Using these data we attempt here to replicate the findings of the SF-36 developers in the UK setting, and then to assess the use of SF-12 summary scores in a variety of clinical conditions.Methods: Factor analytical methods were used to derive the weights used to construct the physical and mental component scales from the SF-36. Regression methods were used to weight the 12 items recommended by the developers to construct the SF-12 physical and mental component scores. This analysis was undertaken on a large community sample (n= 9332), and then the results of the SF-36 and SF-12 were compared across diverse patient groups (Parkinson's disease, congestive heart failure, sleep apnoea, benign prostatic hypertrophy).Results: Factor analysis of the SF-36 produced a two factor solution. The factor loadings were used to weight the physical component summary score (PCS-36) and mental component summary score (MCS-36). Results gained from the use of these measures were compared with results gained from the PCS-12 and MCS-12, and were found to be highly correlated (PCS: ? = 0.94, p< 0.001; MCS: ? = 0.96, p< 0.001), and produce remarkably similar results, both in the community sample and across a variety of patient groups.Conclusions: The SF-12 is able to produce the two summary scales originally developed from the SF-36 with considerable accuracy and yet with far less respondent burden. Consequently, the SF-12 may be an instrument of choice where a short generic measure providing summary information on physical and mental health status is required. Crispin Jenkinson DPhil, Deputy Director

The growing demand for subjective measurements of health in clinical studies has encouraged the rapid creation of many new scales, leading to compromises in the quality of some instruments. Quality control standards are desirable to guide the development of measures and subsequent data interpretation. The limitations in existing measures may be classified under three broad topic areas: 1) shortcomings in the design of the instrument, 2) methodological limitations in the process of its development, and 3) shortcomings in the way that the instrument is described following its initial development. We outline examples of common problems in these areas and propose guidelines for the design, development and presentation of health measurement methods. The guidelines represent a preliminary step in formalizing a discipline of health measurement.

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.

Treatment of obstructive sleep apnoea (OSA) with nasal continuous positive airway pressure (NCPAP) has become a standard treatment since its introduction in 1981. Following such treatment the apnoeas disappear, sleep quality improves as apparently do daytime symptoms of sleepiness. Sleepiness is an unusual symptom and its impact on conventional indices of quality of life has rarely been measured. To allow comparison of NCPAP therapy with treatments for other conditions, measurements of quality of life before and after treatment using standard techniques are required. It is not clear which of the standard measures is most suited to measuring the health gain from nasal NCPAP, and indeed whether the disability of excessive sleepiness is included in all such measures. This study looks at three well recognized quality of life measures in OSA, before and after NCPAP therapy; the Short Form 36 (SF‐36), Functional Limitations Profile (FLP), and the EuroQol (EQ‐5D). The results were compared with data from normal populations. One hundred and eight patients with OSA undergoing a therapeutic assessment of NCPAP completed the three quality of life questionnaires before and 5 weeks after commencing treatment. Over 90 subjects completed all sections of the three measures on both occasions. The SF‐36 revealed substantial adverse effects on subjective health of OSA and that NCPAP treatment produced dramatic positive effects. For example, the effect sizes (difference in score, divided by SD of baseline score) in the Energy/Vitality dimension was 0.98 and for the overall Mental and Physical Component Scores, 0.76 and 0.57, respectively: an effect size over 0.5 is considered moderate and over 0.8 as large. The FLP data showed similar pre treatment decrements in quality of life and substantial improvements following NCPAP. The changes with treatment in the majority of the dimensions from both the SF‐36 and FLP were statistically significant (P<0.01). In contrast the EQ‐5D did not show significant improvements with therapy, presumably because of its failure to measure the aspects of quality of life related to severe sleep fragmentation and daytime sleepiness. In conclusion, this study has clearly shown considerable decrements in quality of life in patients with OSA, similar to other chronic disabling conditions. Furthermore, NCPAP therapy returns patients to a quality of life similar to the normal population.

Many health care organizations want to improve their responses to patients who suffer medical injuries. Their ability to understand how well they meet patients’ needs is limited by the lack of suitable instruments for assessing injured patients’ experiences.

OBJECTIVE:s To systematically assess the type of outcomes selected and the prevalence of patient reported outcomes in contemporary cardiovascular trials and to quantify any misuse or underuse of patient reported outcomes using a specially developed tool that would allow estimation of the relevance of such outcomes to clinical decision making. Design Systematic review. DATA SOURCES: Medline and Embase. STUDY SELECTION: Randomised controlled trials of the treatment for or prevention of cardiovascular disease published in 10 leading general medical and cardiology journals from January 2005 to December 2008. RESULTS: Primary outcomes were patient important (death, morbidity, or patient reported outcomes) in only 93 of 413 trials (23%, SE 2%), whereas another 92 (22%, SE 2%) combined these outcomes with other less important ones into a composite. Sixty five trials (16%; SE 2%) used at least one instrument to measure patient reported outcomes, mostly in trials where such information would have been important or crucial for clinical decision making (52 trials). Patient reported outcomes were judged to be of little incremental value to a large number of, mostly explanatory, cardiovascular trials (152 trials). However, many trials in which patient reported outcomes would have been important or crucial for clinical decision making did not report such outcomes (122 of 174 trials, 70%). These included several trials that primarily aimed to improve symptoms or functional status, trials that tested interventions with a considerable potential for causing harm (mainly bleeding) that were not meaningfully measured, and trials with composite outcomes that were dominated by outcomes of questionable importance to patients. CONCLUSIONS: Despite a continued rise in the reporting of patient reported outcomes with no evidence for their misuse in more recent cardiovascular trials, they seem to be still underused once their relevance to clinical decision making has been taken into account. This was largely explained by inappropriate use of composite outcomes and inadequate measurement of harms.

The use of patient reported outcome measures might seem to be quite straightforward; however, a number of pitfalls await clinicians with limited expertise. Jill Dawson and colleagues provide a guide for individuals keen to use patient reported outcome measures at a local level

To compare alternative Rasch-based approaches to the assessment of change over time through the example of an outcome measure used in total hip replacement surgery.

Background The SF-36 is a generic health status measure which has gained popularity as a measure of outcome in a wide variety of patient groups and social surveys. However, there is a need for even shorter measures, which reduce respondent burden. The developers of the SF-36 have consequently suggested that a 12-item sub-set of the items may accurately reproduce the two summary component scores which can be derived from the SF-36 [the Physical Component Summary Score (PCS) and Mental Health Component Summary Score (MCS)]. In this paper, we adopt scoring algorithms for the UK SF-36 and SF-12 summary scores to evaluate the picture of change gained invarious treatment groups. Methods The SF-36 was administered in three treatment groups (ACE inhibitors for congestive heart failure, continuous positive airways therapy for sleep apnoea, and open vs laparoscopic surgery for inguinal hernia). Results PCS and MCS scores calculated from the SF-36 or a sub-set of 12 items (the‘SF-12’) were virtually identical, and indicated the same magnitude of ill-health and degree of change overtime. Conclusions The results suggest that where two summary scores of health status are adequate then the SF-12 may be the instrument of choice.

Jenkinson C and Fitzpatrick R. Measurement of health status in patients with chronic illness: comparison of the Nottingham health profile and the general health questionnaire. Family Practice 1990; 7:121–124. The results of two commonly used instruments for measuring health status were compared in patients with chronic illness. The Nottingham health profile (NHP) is a measure of perceived health, while the general health questionnaire (GHQ) is a measure of non-psychotic psychiatric disturbance. The questionnaires were completed by patients suffering either rheumatoid arthritis or migraine. The results provide evidence that, despite some specific problems in the measurement of pain and emotional reactions, the NHP and GHQ may be used to assess the impact of illness upon sufferers lives, not only in severely disabling disorders such as rheumatoid arthritis, but in health problems such as migraine which have often been considered as relatively minor ailments.

This prospective cohort study of patients who consulted general practitioners complaining of excessive menstrual bleeding measured changes in quality of life and patients satisfaction following different forms of treatment for menorrhagia. Three hundred and forty-eight patients were followed-up for 18 months using self-completion questionnaires which included generic measurements of health-related quality of life (SF-36) and a disease-specific questionnaire to measure the social impact of menstrual symptoms. Only 15 patients (4%) received no active treatment, 132 (38%) underwent surgical treatment (hysterectomy or endometrial resection), and the remainder were prescribed drugs. Those in the surgical group with both moderate and severe symptoms experienced significant improvements in their quality of life. Patients with moderate symptoms who did not undergo surgery improved in the social functioning and energy dimensions of the SF-36, but those with severe symptoms who received drug treatment only experienced no significant quality-of-life benefits. Patients who had not had surgery were significantly more likely to be dissatisfied with their treatment (21%) than those in the surgical group (5%). Since menorrhagia can have adverse effects on many aspects of a patients daily life, it is important to measure the effects of treatment on quality of life. This study has demonstrated the feasibility of doing so.