Does Knowledge of Treatment Assignment Affect Patient Report of Symptoms,Function, and Health Status? An Evaluation Using Multiple Myeloma Trials

ObjectivesUnblinded trials are common in oncology, but patient knowledge of treatment assignment may bias response to questionnaires. We sought to ascertain the extent of possible bias arising from patient knowledge of treatment assignment.MethodsThis is a retrospective analysis of data from 2 randomized trials in multiple myeloma, 1 double-blind and 1 open label. We compared changes in patient reports of symptoms, function, and health status from prerandomization (screening) to baseline (pretreatment but postrandomization) across control and investigational arms in the 2 trials. Changes from prerandomization scores at ~2 and 6 months on treatment were evaluated only across control arms to avoid comparisons between 2 different experimental drugs. All scores were on 0- to 100-point scales. Inverse probability weighting, entropy balancing, and multiple imputation using propensity score splines were used to compare score changes across similar groups of patients.ResultsMinimal changes from screening were seen at baseline in all arms. In the control arm, mean changes of <7 points were seen for all domains at 2 and 6 months. The effect of unblinding at 6 months in social function was a decline of less than 6 points (weighting: ?3.09; 95% confidence interval ?8.41 to 2.23; balancing: ?4.55; 95% confidence interval ?9.86 to 0.76; imputation: ?5.34; 95% confidence interval ?10.64 to ?0.04).ConclusionIn this analysis, we did not find evidence to suggest that there was a meaningful differential effect on how patients reported their symptoms, function or health status after knowing their treatment assignment.     

Measuring quality of life in early HIV disease: the modular approach

Rationale: to examine the reliability and validity of the General Health Self-assessment, a modular questionnaire for self-assessment of quality of life (QoL) in human immunodeficiency virus (HIV) clinical trials and to describe the baseline QoL of participants in a large HIV clinical trial. Design: the domains assessed include health perceptions, physical, psychological and role/social functioning, health care utilization and symptom distress. Method: 1,694 subjects with early HIV infection enrolled in the AIDS Clinical Trials Group Protocol 175 completed the scale at baseline. Results: the domains demonstrated reliability, construct and discriminant validity. A worse QoL was associated with recent hospitalization and symptomatic status. Prior antiretroviral therapy was associated with higher health perceptions and well-being. The presence of symptom distress was related to lower QoL on the other scales. There was no relationship between QoL scales and the baseline CD4 count. Women showed a lower QoL than men on all scales, while ethnicity was related to differences in health perceptions and physical and psychological functioning. Conclusions: the General Health Self-assessment shows excellent potential as a measure of QoL for HIV-infected patients in clinical trials. Further research is necessary to determine the responsiveness of the scale to clinical and immunological changes in HIV-infected individuals.     

The Course of Quality of Life and Its Predictors in Nursing Home Residents With Young-Onset Dementia

ObjectiveTo explore the course of quality of life (QoL) and possible resident-related predictors associated with this course in institutionalized people with young-onset dementia (YOD).DesignAn observational longitudinal study.Setting and ParticipantsA total of 278 residents with YOD were recruited from 13 YOD special care units in the Netherlands.MethodsSecondary analyses were conducted with longitudinal data from the Behavior and Evolution in Young-ONset Dementia (BEYOND)-II study. QoL was assessed with proxy ratings, using the Quality of Life in Dementia (QUALIDEM) questionnaire at 4 assessment points over 18 months. Predictors included age, gender, dementia subtype, length of stay, dementia severity, neuropsychiatric symptoms, and psychotropic drug use at baseline. Multilevel modeling was used to adjust for the correlation of measurements within residents and clustering of residents within nursing homes.ResultsThe total QUALIDEM score (range: 0-111) decreased over 18 months with a small change of 0.65 (95% confidence interval −1.27, −0.04) points per 6 months. An increase in several domains of QoL regarding care relationship, positive self-image, and feeling at home was seen over time, whereas a decline was observed in the subscales positive affect, social relations, and having something to do. Residents with higher levels of QoL and more advanced dementia at baseline showed a more progressive decline in QoL over time. Sensitivity analyses indicated a more progressive decline in QoL for residents who died during the follow-up.Conclusion and ImplicationsThis study shows that although overall QoL in nursing home residents with YOD was relatively stable over 18 months, there were multidirectional changes in the QoL subscales that could be clinically relevant. Higher levels of QoL and more advanced stages of dementia at baseline predicted a more progressive decline in QoL over time. More longitudinal studies are needed to verify factors influencing QoL in YOD.     

Listen to their answers! Response behaviour in the measurement of physical and role functioning

Background Quality of life (QoL) is considered to be an indispensable outcome measure of curative and palliative treatment. However, QoL research often yields findings that raise questions about what QoL measurement instruments actually assess and how the scores should be interpreted. Objective To investigate how patients interpret and respond to questions on the EORTC-QLQ-C30 over time and to find explanations to account for counterintuitive findings in QoL measurement. Methods Qualitative investigation was made of the response behaviour of small-cell lung cancer patients (n = 23) in the measurement of QoL with the European Organization for Research and Treatment of Cancer Core Quality of Life Questionnaire (EORTC QLQ-C30). Focus was on physical functioning (PF, items 1 to 5), role functioning (RF, items 6 and 7), global health and QoL rating (GH/QOL, items 29 and 30). Interviews were held at four points: at the start of the chemotherapy, 4 weeks later, at the end, and 6 weeks after the end of chemotherapy. Patients were asked to ‘think aloud’ when filling in the questionnaire. Results Patients used various response strategies when answering questions about problems and limitations in functioning, which impacted the accuracy of the scale. Patients had scores suggesting they were less limited than they actually were by taking the wording of questions literally, by guessing their functioning in activities that they did not perform, and by ignoring or excluding certain activities that they could not perform. Conclusion Terminally ill patients evaluate their functioning in terms of what they perceive to be normal under the circumstances. Their answers can be interpreted in terms of change in the appraisal process (Rapkin and Schwartz 2004; Health and Quality of Life Outcomes, 2, 14). More care should be taken in assessing the quality of a set of questions about physical and role functioning.     

Insufficient evidence to determine the impact of patient preferences on clinical outcomes in acupuncture trials: a systematic review

ObjectiveTo review reporting of preferences in acupuncture studies and their effect on clinical outcomes.Study Design and SettingSystematic review of published randomized and quasi-randomized controlled trials of acupuncture reporting participant preferences for randomization or treatment or using a preference design.ResultsOf the 31 included trials, 5 reported on randomization preference, 18 on treatment preference, and 1 reported on both. Seven used a preference design. Four out of seven trials noted that the group with preferences had different baseline characteristics (less education, worse baseline measure score, and greater or fewer years with pain). There was a tendency for greater attrition in nonpreference arms at 6 months but not earlier. Around three-quarters of participants turned down randomization in favor of nontrial treatment, and preference for acupuncture was around 20% when offered multiple treatments. Questions used to elicit preferences varied across trials and were poorly reported. Ten trials reported the effects of preferences on outcomes; only one detected a statistically (but not clinically) significant difference.ConclusionThere is little evidence that preferences cause detectable effects on outcomes in acupuncture trials; however, trials use inconsistent methods and poorly report these data. Monitoring the level and effect of preferences in trials is recommended.     

Quality of life findings from a multicenter, multinational, observational study of patients with metastatic hormone-refractory prostate cancer

Background The assessment of health-related quality of life (HRQL) for patients with hormone-refractory prostate cancer (HRPC) is of paramount importance because new treatments have a modest impact on survival but side effects of treatment and disease symptoms can significantly impact HRQL. Methods This was an observational, non-interventional, multi-center, multi-national cohort study of patients with metastatic HRPC. Health-related quality of life was assessed using the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ C30), the Functional Assessment of Cancer Therapy-Prostate (FACT-P) and the EQ-5D index. Mean changes from baseline to month 3, 6, and 9 were computed and tested using paired t-tests. Results FACT-P PCS, EQ-5D index and 10 of 14 EORTC domains were statistically significantly lower (P < 0.05) than the baseline scores at the 3, 6 and 9 month visits. The domains that did not reach statistical significance were cognitive functioning, insomnia, diarrhea and financial difficulties. Conclusions These data demonstrate that patients with metastatic HRPC experience rapid, significant deterioration in HRQL, highlighting the need for effective palliative therapy for men with HRPC.     

Using Multiple Anchor- and Distribution-Based Estimates to Evaluate Clinically Meaningful Change on the Functional Assessment of Cancer Therapy-Biologic Response Modifiers (FACT-BRM) Instrument

OBJECTIVE: The interpretation of health-related quality of life (HRQL) data from clinical trials can be enhanced by understanding the degree of change in HRQL scores that is considered meaningful. Our objectives were to combine distribution-based and two anchor-based approaches to identify minimally important differences (MIDs) for the 27-item Trial Outcome Index (TOI), the seven-item Social Well-Being (SWB) subscale, and the six-item Emotional Well-being (EWB) subscale from the Functional Assessment of Cancer Therapy-Biological Response Modifiers (FACT-BRM) instrument. METHODS: Distribution-based MIDs were based on the standard error of measurement. Anchor-based approaches utilized patient-reported global rating of change (GRC) and change in physician-reported performance status rating (PSR). Correlations and weighted kappa statistics were used to assess association and agreement between the two anchors. FACT-BRM changes were evaluated for three time periods: baseline to month 1, month 2 to month 3, and month 5 to month 6. RESULTS: Association between GRC and change in PSR was poor. Correlation between the anchors and HRQL change scores was largest at month 1 and decreased through month 6. Combining results from all approaches, the MIDs identified were 5-8 points for the TOI, 2 points for the SWB subscale, and 2-3 points for the EWB subscale. CONCLUSIONS: We combined patient-reported estimates, physician-reported estimates, and distribution-based estimates to derive MIDs for HRQL outcomes from the FACT-BRM. These results will enable interpretation of treatment group effects in a clinical trial setting, and they can be used to estimate sample size or power when designing future studies.     

A Randomized Clinical Trial of Vitamin D Supplementation in Healthy Adolescents

PurposeThe most safe and effective dose of vitamin D supplementation for healthy adolescents is currently unknown. The aim of this study was to compare the efficacy of 200 IU versus 1,000 IU of daily vitamin D₃ for supplementation in healthy adolescents with baseline vitamin D sufficiency.MethodsWe conducted a double-blind, randomized clinical trial. Fifty-six subjects, ages 11–19 years, with baseline vitamin D sufficiency received 1,000 IU or 200 IU of daily vitamin D₃ for 11 weeks. Compliance was assessed using MEMS6 Trackcaps and pill counts.ResultsFifty-three subjects completed the clinical trial. Subjects in the two treatment arms were similar in terms of age, race, gender, body mass index, and dietary calcium and vitamin D intake. Serum 25(OH)D level in the 200 IU treatment arm was 28.1 ± 6.2 ng/mL at baseline (mean ± SD) and 28.9 ± 7.0 ng/mL at follow-up. In the 1,000 IU treatment arm, 25(OH)D levels were 29.0 ± 7.3 and 30.1 ± 6.6 at baseline and follow-up, respectively. Mean change in 25(OH)D level did not differ significantly between treatment arms (p = .87), nor did mean change in parathyroid hormone, calcium, phosphate, bone turnover markers, fasting glucose, or fasting insulin.ConclusionsIn healthy adolescents with baseline vitamin D sufficiency, supplementation with vitamin D₃ doses of 200 and 1,000 IU for 11 weeks did not increase serum 25(OH)D levels, with no significant difference observed between treatment arms.     

The Impact of Body Mass Index and Waist Circumference on Health-related Quality of Life Among Colorectal Cancer Survivors: Results from the PROFILES Registry

Background: We aimed to assess the association of waist circumference (WC) and body mass index (BMI) with health-related quality of life (HRQL) among colorectal cancer (CRC) survivors. Methods: CRC survivors diagnosed between 2000 and 2009 completed questionnaires in August 2013 (with self-reported weight, height, and self-assessed WC) and January 2014 (with HRQL using the EORTC-QLQ-C30). Clinical characteristics were retrieved from the Netherlands Cancer Registry. In multivariable linear regression analyses associations of BMI only, WC only and both BMI and WC with HRQL outcomes were assessed. Results: 1,111 CRC survivors were included of whom 34% had a normal weight (18.5 ≤ BMI < 25 kg/m²), 49% had overweight (25 ≤ BMI < 30 kg/m²), 17% had obesity (BMI ≥ 30 kg/m²), and 44% had an increased WC (i.e., >102 and >88 cm for men and women, respectively). Both BMI and WC were separately associated with worse global health status, functioning, and more symptoms of fatigue. Increased WC was associated with lower physical, role and emotional functioning, regardless of BMI, with average differences ranging between 3 and 5 points. Conclusion: Future research on HRQL among CRC survivors should consider both BMI and WC. Furthermore, weight reduction trials should not only focus on general weight loss but also on the loss of abdominal fat.     

Meta-analysis shows clinically relevant and long-lasting deterioration in health-related quality of life after esophageal cancer surgery

Purpose

The purpose of the study is to (1) estimate the direction, clinical relevance, and duration of health-related quality of life (HRQL) change in the first year following esophageal cancer surgery and (2) to assess the robustness of the estimates by subgroup and sensitivity analyses, and an exploration of publication bias.

Methods

A systematic literature search in MEDLINE, EMBASE, CINAHL, PsychINFO, and CENTRAL to identify randomized and non-randomized studies was performed. We compared the baseline HRQL data with 3-, 6-, 9-, or 12-month follow-ups to estimate the magnitude and duration of HRQL change. These estimates were then classified as trivial, small, medium, or large. Primary outcomes were role functioning, eating, and fatigue. Secondary outcomes were physical and social functioning, dysphagia, pain, and coughing problems. We conducted subgroup analysis for open surgery, open surgery preceded by neo-adjuvant therapy, and minimally invasive surgery. Sensitivity analyses assessed the influence of study design, transformation/imputation of the data, and HRQL questionnaire used.

Results

We included data from 15 studies to estimate the change in 28 HRQL outcomes after esophageal cancer surgery. The main analysis showed that patients’ social functioning deteriorated. Symptoms of fatigue, pain, and coughing problems increased. These changes lasted for 9–12 months, although some symptoms persisted beyond the first year after surgery. For many other HRQL outcomes, estimates were only robust after subgroup or sensitivity analyses (e.g., role and physical functioning), or remained too heterogeneous to interpret (e.g., eating and dysphagia).

Conclusions

Patients will experience a clinically relevant and long-lasting deterioration in HRQL after esophageal cancer surgery. However, for many HRQL outcomes, more and better quality evidence is needed.     

Quality of Life and Pneumonia in Nursing Home Residents: A 1-Year Observational Study

ObjectivesTo compare the evolution of quality of life (QoL) in nursing home residents (NHRs) with and without (hospitalization for) pneumonia.DesignTwelve-month prospective, observational study.Setting and ParticipantsParticipants of the Incidence of pNeumonia and related ConseqUences in nursing home Resident (INCUR) study were included. The INCUR study included 800 NHRs in France for which comprehensive assessments were performed at baseline, 6 months, and 12 months in 2012-2013.MethodsParticipants’ health related QoL was assessed at 3 time points: baseline, 6 months, and 12 months. NHRs with or without pneumonia and hospitalizations for this condition at any time during follow-up were compared using adjusted mixed effects linear regressions on the QoL outcome. Pre- and postpneumonia QoL were compared using a Wilcoxon signed-rank test.ResultsA total of 622 NHRs (mean age 86.2 years; 73.3% women) were included; 13.8% (n = 86) died, 19.9% (n = 124) developed at least 1 episode of pneumonia and 6.4% (n = 40) were hospitalized for pneumonia. Median QoL was 70 at baseline [n = 436, interquartile range (IQR) = 50-90], 80 at 6 months (n = 546, IQR = 50-90), and 76 at 12 months (n = 468, IQR = 50-80). QoL in NHRs with pneumonia showed a 2-point decrease during the 12-month follow-up, whereas QoL in NHRs without pneumonia showed an 8-point increase during follow-up. QoL in NHR hospitalized for pneumonia showed a 16-point decrease during the 12-month follow-up, whereas QoL in NHRs in the control group showed a 6-point increase. In linear regressions, neither pneumonia nor hospitalization for pneumonia were significantly associated with the evolution of QoL during follow-up. No significant difference was found between pre- and postpneumonia QoL.Conclusions and ImplicationsQoL in NHRs remains stable over 12 months regardless of pneumonia events but seems to decline in NHRs hospitalized for pneumonia. Uncaptured short-term variations of QoL after pneumonia and/or related hospitalizations may occur.     

Differences in Patient-Reported Outcomes That Are Most Frequently Detected in Randomized Controlled Trials in Patients With Solid Tumors: A Pooled Analysis of 229 Trials

ObjectivesPatient-reported outcome (PRO) measurements used in cancer research can assess a number of health domains. Our primary objective was to investigate which broad types of PRO domains (namely, functional health, symptoms, and global quality of life [QoL]) most frequently yielded significant differences between treatments in randomized controlled trials (RCTs).MethodsA total of 229 RCTs published between January 2004 and February 2019, conducted on patients diagnosed with the most common solid malignancies and assessed using the European Organization for the Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ)-C30, were considered. Studies were identified systematically using literature searches in key electronic databases. Unlike other PRO measurements typically used in RCTs, the scoring algorithm of the multidimensional EORTC QLQ-C30 allowed us to clearly distinguish the 3 broad types of PRO domains.ResultsIn total, 134 RCTs (58.5%) reported statistically significant differences between treatment arms for at least 1 of the QLQ-C30 domains. Most frequently, differences were reported for 2 or all 3 broad types of PRO domains (78 of 134 trials; 58.2%). In particular, 35 trials (26.1%) found significant differences for symptoms, functional health, and global QoL, 24 trials (17.9%) for symptoms and functional health, 11 trials (8.2%) for functional health and global QoL, and 8 trials (6.0%) for symptoms and global QoL. The likelihood of finding a statistically significant difference between treatment arms was not associated with key study characteristics, such as study design (ie, open-label vs blinded trials) and industry support.ConclusionsOur findings emphasize the importance of a multidimensional PRO assessment to most comprehensively capture the overall burden of therapy from the patients’ standpoint.     

Measuring and reporting quality of life outcomes in clinical trials in cystic fibrosis: a critical review

Good quality clinical trials are essential to inform the best cystic fibrosis (CF) management and care, by determining and comparing the effectiveness of new and existing therapies and drug delivery systems. The formal inclusion of quality of life (QoL) as an outcome measure in CF clinical trials is becoming more common. Both an appropriate QoL measure and sound methodology are required in order to draw valid inferences about treatments and QoL. A review was undertaken of randomised controlled trials in cystic fibrosis where QoL was measured. EMBASE, MEDLINE and ISI Web of Science were searched to locate all full papers in the English language reporting randomised controlled trials in cystic fibrosis, published between January 1991 and December 2004. All Cochrane reviews published before December 2004 were hand searched. Papers were included if the authors had reported that they had measured QoL or well being in the trial. 16 trials were identified. The interventions investigated were: antibiotics (4); home versus hospital administration of antibiotics (1); steroids (1); mucolytic therapies (6); exercise (3) and pancreatic enzymes (1). Not one trial evaluated in this review provided conclusive results concerning QoL. This review highlights many of the pitfalls of QoL measurement in CF clinical trials and provides constructive information concerning the design and reporting of trials measuring QoL.     

Randomised controlled trial of an automated, interactive telephone intervention (TLC Diabetes) to improve type 2 diabetes management: Baseline findings and six-month outcomes

ABSTRACT: BACKGROUND: Effective self-management of diabetes is essential for the reduction of diabetes-related complications, as global rates of diabetes escalate. METHODS: Randomised controlled trial. Adults with type 2 diabetes (n=120), with HbA1c greater than or equal to 7.5%, were randomly allocated (4x4 block randomised block design) to receive an automated, interactive telephone-delivered management intervention or usual routine care. Baseline sociodemographic, behavioural and medical history data were collected by self-administered questionnaires and biological data were obtained during hospital appointments. Health-related quality of life (HRQL) was measured using the SF-36. RESULTS: The mean age of participants was 57.4 (SD 8.3), 63% of whom were male. There were no differences in demographic, socioeconomic and behavioural variables between the study arms at baseline. Over the six-month period from baseline, participants receiving the Australian TLC (Telephone-Linked Care) Diabetes program showed a 0.8% decrease in geometric mean HbA1c from 8.7% to 7.9%, compared with a 0.2% HbA1c reduction (8.9% to 8.7%) in the usual care arm (p=0.002). There was also a significant improvement in mental HRQL, with a mean increase of 1.9 in the intervention arm, while the usual care arm decreased by 0.8 (p=0.007). No significant improvements in physical HRQL were observed. CONCLUSIONS: These analyses indicate the efficacy of the Australian TLC Diabetes program with clinically significant post-intervention improvements in both glycaemic control and mental HRQL. These observed improvements, if supported and maintained by an ongoing program such as this, could significantly reduce diabetes-related complications in the longer term. Given the accessibility and feasibility of this kind of program, it has strong potential for providing effective, ongoing support to many individuals with diabetes in the future.     

A two-year prospective study of the health-related quality of life of children with chronic illness – the parents’ perspective

The aim of this study was to assess prospectively changes in the health-related quality of life (HRQL) of children and adolescents with diabetes, asthma or cystic fibrosis (CF). One hundred and twenty-two parents of children aged 10–16 years with asthma, diabetes, or CF were recruited from specialist paediatric clinics. Parents described their childrens HRQL using the Child Health Questionnaire (PF98) at baseline, 6, 12, 18 and 24 months post-baseline. They reported that the general health of children with CF was significantly worse than that of children with asthma and diabetes at baseline. In other domains there were few differences between the HRQL of children in the three groups. In several domains, the HRQL of children with asthma or diabetes improved over the 2years of the study. This improvement was less evident for children with CF.     

Quality of Life and Pain Medication Use in Persons With Advanced Dementia Living in Long-Term Care Facilities

ObjectivesIn residents with dementia living in a long-term care facility (LTCF), un(der)treated pain may trigger behavioral disturbances, mood syndromes, and deterioration of physical functioning and self-maintenance. Because these factors can have considerable impact on the quality of life (QoL), this study aimed to (1) compare characteristics of persons with advanced dementia living in LTCFs with and without pain medication; (2) compare QoL in these persons with and without pain, stratified by type of pain medication use; and (3) explore associations between the use of paracetamol and QoL in persons with advanced dementia living in LTCFs.Design and settingThis study analyzed baseline data from the Communication, Systematic Assessment and Treatment of Pain, Medication Review, Occupational Therapy, and Safety Study; a multicenter, cluster-randomized effectiveness-implementation clinical hybrid trial in 67 Norwegian LTCF clusters.ParticipantsIn total, 407 LTCF residents (rural and urban areas) aged ≥65 years, with Functional Assessment Staging scores of 5–7 (ie, moderate to advanced dementia).Main outcome measureQoL as assessed by the 6 QUALIDEM (validated questionnaire to measure QoL in persons with dementia living in LTCF) domains applicable to persons with moderate to severe dementia. The association between QoL and paracetamol was estimated using linear mixed-effect models, adjusting for confounding variables.Results62.0% used pain medication (paracetamol, opioids, or both). QoL was lower in residents using pain medication, compared with those without pain medication [mean QUALIDEM score 68.8 (standard deviation 17.4 vs) 75.5 (standard deviation 14.6), respectively, P < .001). Multilevel analysis showed that paracetamol use was not associated with QoL.Conclusions and ImplicationsPersons with advanced dementia living in LTCF using pain medication have a lower QoL compared with those not using pain medication. These results are of key importance for the clinician because they stress the need for regular medication review and pain management. When measured cross-sectionally, use of paracetamol is not associated with increased QoL.     

Convergent discriminitive, and predictive validity of the Prostate Cancer Specific Quality of Life Instrument (PROSQOLI) assessment and comparison with analogous scales from the EORTC QLQ-C30 and a trial-specific module. European Organisation for Research and Treatment of Cancer. Core Quality of Life Questionnaire.

The Prostate Cancer Specific Quality of Life Instrument (PROSQOLI) is a measure of health-related quality of life (HRQL) that was designed to be an outcome measure for clinical trials in advanced hormone-resistant prostate cancer. The cross-sectional validity of the PROSQOLI was assessed using baseline data from a randomized trial in which HRQL was also assessed with the European Organisation for Research and Treatment of Cancer (EORTC) Core Quality of Life Questionnaire (QLQ-C30) and a trial-specific quality of life module (QLM-P14). Convergent validity was assessed with the multitrait-multimethod matrix approach; discriminative validity was assessed according to conventional clinical criteria; and predictive validity was assessed by the ability to predict survival duration. These assessments provided strong support for the validity of all PROSQOLI scales except those for family/marriage relationships and passing urine; modifications of these two scales are under evaluation. The strength, consistency, and independence of the prognostic information provided by the HRQL scales were striking. Differences between the instruments were generally subtle. These data support validity of the PROSQOLI and the analogous scales from the QLQ-C30 and QLM-P14 in symptomatic men with advanced hormone resistant prostate cancer. The PROSQOLI is a short, simple, and valid measure of HRQL in this setting.     

Qualify of life and palliation predict survival in patients with chronic alveolar hypoventilation and nocturnal ventilatory support

Objectives  Non-invasive positive pressure ventilation (NPPV) improves health-related quality of life (HRQL) in patients with chronic alveolar hypoventilation (CAH). We studied the prognostic impact of HRQL on survival in relation to clinical factors. Patients  Forty-four patients with CAH due to post-polio (12), scoliosis (11), post-tb (17) or other diagnoses (4) who received nocturnal NPPV were prospectively studied during 6–10 years. Measurements  Blood gases and HRQL were analysed at baseline and after 9 months and after 8 years. HRQL was evaluated with measures of functioning (SIP), emotional well-being (HADS and MACL), and global QL. Results  Blood gases and HRQL measures improved during NPPV. The overall 5-year survival rate was 73%. In multivariate survival analysis, a diagnosis of post-polio and low baseline SIP physical index scores, indicating low levels of physical dysfunction, predicted longer survival (P = 0.02, respectively). Similarly, palliation of physical dysfunction and preserved or improved global QL by 9 months were associated with longer overall survival (P = 0.009 and P = 0.001, respectively; multivariate Cox regression). Conclusion  Seventy-three percent of patients treated for CAH with NPPV survived more than 5 years. Diagnosis and self-rated physical functioning at pre-treatment were related to survival, as were major improvements in physical functioning and global QL during NPPV.