Quality Of Life In Clinical Trials Research Articles

The Inconsistent Assessment of Quality of Life in Patients Treated for Head and Neck Cancer with Anti-EGFR Inhibitors: A Systematic Scoping Review.

In patients receiving treatment for head and neck cancer (HNC), there is a correlation between quality of life (QoL) scores and treatment outcomes. Higher QoL scores have been associated with improved survival. Despite this, the assessment of QoL in clinical trials varies considerably. Three databases (Scopus, PubMed, and Cinahl) were queried for articles published in English between 2006 and 2022. Two reviewers (SRS and ANT) performed study screening, data extraction, and risk of bias assessment. The authors identified 21 articles that met the inclusion criteria. A total of 5961 patients were evaluated. QoL was reported as average scores for specific variables across five different surveys in 12 included articles. Supplemental QoL data were available in 10 included studies. Critical appraisal of studies indicated a high risk of bias due to the inclusion of trials. There is no standard method for reporting QoL data in clinical trials for HNC patients undergoing treatment with anti-EGFR inhibitors. Future clinical trials should standardize their method for assessing and reporting quality-of-life data to increase patient-centered care and refine treatment choices to optimize survival.

Using Qualitative Interviews to Improve Quantitative Assessments of Quality of Life in Patients with Neurofibromatosis 2 (P7-13.006)

<h3>Objective:</h3> To use qualitative interviews to elucidate seemingly discordant quantitative ratings in patient reported outcomes for quality of life (QOL). <h3>Background:</h3> Neurofibromatosis 2 (NF2) is a progressive, neurogenetic disease. Assessing QOL is important in demonstrating that new drugs improve how NF2 patients feel and function. The NFTI-QOL is the currently recommended measure to assess NF2-specific QOL in clinical trials. <h3>Design/Methods:</h3> We interviewed 16 participants enrolled in stage one of the brigatinib arm of INTUITT-NF2 (NCT04374305) –a multicenter, adaptive platform-basket trial targeting progressive NF2-related tumors – after one year of treatment or at time of trial discontinuation. Interviews included cognitive debriefings of the NFTI-QoL and global impression of change (GIC) scale. We also analyzed NFTI-QoL scores collected over the same time period, using a MCID of ±2 points to classify improvement or worsening in QOL. <h3>Results:</h3> 11/16 (69%) participants reported a GIC rating discordant with their change in NFTI-QoL scores. 8/13 (62%) of participants who improved on their GIC had stable (N=6) or worsened (N=2) NFTI-QoL scores. This group cited tumor stability as a major contributor to improved GIC ratings and also had lower baseline NFTI-QoL scores (median=8) than those with concordant ratings (median=12). Participants with lower NFTI-QoL scores disproportionally reported that a meaningful improvement in their symptoms would not lead to an accompanying score improvement due to inadequate NFTI-QOL response options. <h3>Conclusions:</h3> Qualitative interviews revealed two reasons for conflicting patient reported outcome results. In the setting of a progressive disease, participants can view tumor volume stability as an improvement, even with stable or worsening symptoms. Also, the NFTI-QoL may not be capturing improvement in people with a lower symptom burden. In order for the NFTI-QoL and GIC to better reflect changes in NF2 patients QOL, we recommend adding another response option to the NFTI-QoL and altering the instructions of the GIC. <b>Disclosure:</b> Ms. Von Imhof has nothing to disclose. Dr. Plotkin has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for NFlection Therapeutics. Dr. Plotkin has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Akouos. Dr. Plotkin has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for SonALAsense. Dr. Plotkin has stock in NFlection Therapeutics. Dr. Plotkin has stock in NF2 Therapeutics. Dr. Plotkin has stock in SonALAsense. Dr. Plotkin has received research support from Children’s Tumor Foundation. Dr. Plotkin has received research support from Department of Defense. Dr. Plotkin has received research support from National Institutes of Health. Dr. Plotkin has received intellectual property interests from a discovery or technology relating to health care. Dr. Yohay has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Astra Zeneca. Dr. Yohay has received publishing royalties from a publication relating to health care. An immediate family member of Dr. Yohay has received publishing royalties from a publication relating to health care. Dr. Nghiemphu has received research support from Erasca. Dr. Nghiemphu has received research support from Springswork. Dr. Nghiemphu has received research support from Children’s Tumor Foundation. Dr. Nghiemphu has received research support from NIH-NCI . Dr. Nghiemphu has received research support from Genentech. Dr. Nghiemphu has received research support from DOD. Dr. Nghiemphu has received research support from Millenium. Dr. Nghiemphu has received research support from GCAR. Dr. Nghiemphu has received research support from Novartis. Dr. Nghiemphu has received research support from BMS. Dr. Nghiemphu has received personal compensation in the range of $500-$4,999 for serving as a advisory board with Alexion. Dr. Babovic-Vuksanovic has received personal compensation for serving as an employee of AstraZeneka. Dr. Babovic-Vuksanovic has received personal compensation for serving as an employee of Alexion. Dr. Babovic-Vuksanovic has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AstraZeneca. Dr. Babovic-Vuksanovic has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Dr. Babovic-Vuksanovic has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for AstraZeneca . Dr. Babovic-Vuksanovic has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Loeb&amp;Loeb. The institution of Dr. Babovic-Vuksanovic has received research support from DOD. Dr. Babovic-Vuksanovic has received publishing royalties from a publication relating to health care. The institution of Vanessa Merker has received research support from Children’s tumor foundation . The institution of Vanessa Merker has received research support from NF Northeast. Vanessa Merker has received personal compensation in the range of $10,000-$49,999 for serving as a science writer with Neurofibromatosis Network.

HL-210: Assessment and Reporting of Quality of Life Measures in Pivotal Clinical Trials of Lymphoma

Context Clinical trials are integral to improve treatment outcomes for patients with lymphoma. The importance of assessing and reporting of health-related quality of life (QoL) has been increasingly recognized in the field of oncology. QoL measures, which reflect patient's perceived benefits and satisfaction, might be especially important in lymphoma clinical trials, where the intervention may not result in cure. Objective To investigate assessment of health-related QoL in clinical trials that resulted in a subsequent approval of drugs to treat lymphoma by Food and Drug Administration (FDA) and to determine if ensuing published studies reported QoL as part of their results. Design Data on health-related QoL were obtained from studies' protocols and product labeling available publicly at Drugs@FDA. Drugs approved in the period of 2016 till 2020 were analyzed. Setting On the basis of publicly available study protocols and FDA reviews, the authors reviewed the assessment of health-related QoL in 19 clinical trials supporting 17 drug approvals. Those trials resulted in approval of medications to treat classic Hodgkin lymphoma, follicular lymphoma, marginal zone lymphoma, mantle cell lymphoma, diffuse large B cell lymphoma, and T cell lymphoma. Patients or other participants A total of 5292 patients were assessed in the 5-year period after exclusion of unpublished studies. Main outcome measures We calculated the frequency of assessment of health-related QoL and if QoL measures were reported in the subsequent publications. Results After exclusion of 2 unpublished studies, we analyzed 17 clinical trials for reporting of health-related QoL. Eighteen percent of the protocols included assessment of health-related QoL with only 33% of them (6% of all the analyzed clinical trials) reported the results of health-related QoL. The results were consistent across all lymphoma subtypes and were similar regardless of the studied primary endpoint. Conclusions Health-related QoL measures are under studied in clinical trials of lymphoma. Studies that measured QoL didn't report the results in more than half of the time. Improvement in assessing and reporting of QoL will help to incorporate QoL measures in patient care and therapy choice decision.

Utility analysis and calibration of QOL assessment in disease management

ABSTRACTIn clinical trials, the assessment of health-related quality of life (QOL) (or patient-reported outcome [PRO] measure) has become very popular especially for clinical studies conducted for evaluating clinical benefits of patients with chronic, severe, and/or life threatening diseases. Health-related QOL information and PRO measures are useful for disease management for achieving best clinical practice. In this article, we will focus on health-related QOL assessment. The concept, design, and analysis of health-related QOL in clinical trials are reviewed. Validation of the use of health-related QOL instrument in terms of some key performance characteristics such as accuracy, reliability, sensitivity, and responsibility for assuring quality, integrity, and validity of collected QOL data are discussed. The concept of utility analysis and calibration (e.g., with respect to life events) for achieving the optimization of disease management are proposed. The change of the QOL could be translated into different life events for effective disease management. These translations could evaluate the treatment effect by more directly displaying the change of the QOL.

A Systematic Review of Health-Related Quality of Life Reporting in Ovarian Cancer Phase III Clinical Trials: Room to Improve.

Epithelial ovarian cancer (OC) remains a significant cause of morbidity and mortality for women worldwide. Patients may experience a multitude of disease- and treatment-related symptoms that can impact quality of life (QOL) and should be measured and reported in clinical trials. This systematic review investigated the adequacy of reporting of QOL in randomized phase III trials in OC in both the first-line and recurrent disease setting. A systematic review of MEDLINE and EMBASE identified randomized clinical trials of systemic therapy in OC from 1980 to 2014. The adequacy of reporting QOL was evaluated with respect to adherence to established guidelines on reporting QOL in clinical trials and the recent recommendations on the inclusion of patient-reported outcomes in clinical trials from the Fifth Ovarian Cancer Consensus Conference. Of 3,247 abstracts, 35 studies, including 24,664 patients, met inclusion criteria. Twenty-two trials (63%) were in the first-line setting, with 13 (37%) in the recurrent setting. The inclusion of QOL assessments increased from 2% (1980s) to 62% (2010+). Quality of life was a co-primary endpoint in only one trial.Minimal clinically important differences in QOL were defined in eight trials (23%), with results included in the abstract in 37% and article in 86%. Compliance was reported in 26 trials (74%), with 13 trials (37%) reporting specifically how they dealt with missing data. Only seven trials reported the reasons for missing data (20%).Group results were published in 29 trials (83%), with 6 (17%) reporting individual patient results. Results were more commonly reported as a mean overall score (21 trials; 60%), with specific domain scores in only 9 trials (26%). No studies reported QOL beyond progression or included predefined context-specific endpoints based on objectives of treatment (i.e., palliation/cure/maintenance) and the patient population. Duration of benefit of palliative chemotherapy was reported in only one study. Inclusion and reporting of QOL as a trial endpoint has improved in phase III trials in OC, but there are still significant shortfalls that need to be addressed in future trials. The impact of treatment on quality of life (QOL) is an important consideration in patients with ovarian cancer for whom treatment is often given with palliative intent. Both the disease and treatment impact a patient's QOL and require careful evaluation in clinical trials. Matching the QOL questions to the patient population of interest is critical. Similar rigor to that used to assess progression-based endpoints is essential to guide clinical decisions. This systematic review demonstrated that although the inclusion and reporting of QOL as a trial endpoint has improved in phase III trials there are still significant shortfalls that need to be addressed in future trials.

Assessment of quality of life using FACT-G survey in a phase I trial in cancer patients

e20709 Background: Quality of life (QoL) assessment in clinical trials has been gaining more attention. FACT-G surveys have been validated to assess QoL in clinical trials involving oncology patient (Cella DF et al, J Clin Oncol 11:570–579, 1993). However, there is paucity of evaluation of QoL in patients with advanced cancer participating in Phase I clinical trials. Methods: FACT-G surveys were conducted within the context of a Phase I trial to identify a safe dose and potential drug-drug interations of capecitabine and irinotecan combination (Goel, S et al, Invest New Drugs 25:237–245, 2007). The FACT-G survey consists of 28 questions in 5 sections, namely, physical well-being, social/family well-being, emotional well-being, relationship with doctor, and functional well-being). Patients were requested to complete the FACT-G surveys at baseline and every two cycles thereafter (each cycle of 3 weeks duration). Results: Forty-one of 47 patients with advanced solid tumors who participated in the clinical trial completed FACT-G surveys. Mean scores were calculated for each time point. The mean QoL scores at baseline and post cycle 2 were 53 and 58, respectively (p = 0.1). Post cycle 4, the mean QoL score was 62 [p = 0.01, (vs. baseline)]. Following cycle 4, the number of respondents decreased to the extent where we were unable to ascertain any further changes in the QoL scores. Conclusions: It is feasible to use FACT-G survey as a tool to assess QoL in patients participating in an oncology phase I clinical trial. Although the sample size of the patient population was not powered for any statistical significance, there was a trend toward improving QoL based on FACT-G survey scores. This suggests that phase I clinical trials may provide improvement of QoL for some patients. FACT-G is a useful tool in assessing QoL in oncology phase I trial study population. No significant financial relationships to disclose.

Social functioning and quality of life as measures of effectiveness in the treatment of schizophrenia

From the perspective of health care practitioners, the most useful clinical trials are those that assess the effectiveness of a drug, that is, determine how well the drug works under conditions of actual clinical practice. Clinical trials designed to determine effectiveness should use outcome measures that have the greatest clinical significance for practitioners, with the goal of maximizing generalizability and addressing practical questions about the risks, benefits, and costs of an intervention in routine clinical practice 1. Here we would like to emphasize the importance of social functioning and quality of life (QOL) as outcome variables in clinical trials in patients with schizophrenia. Recent research has shown that atypical antipsychotics improve QOL in patients with schizophrenia 2. However, studies that incorporate QOL in the assessment of long-term effectiveness are limited and inconsistent. Fleischhacker and Goodwin summarize the results of the Cost Utility of the Latest Antipsychotic Drugs in Schizophrenia Study (CUtLASS) 3, which employed the Quality of Life Scale 4 as the primary outcome measure for evaluating the effectiveness of treatment. The CUtLASS found no disadvantage of first-generation antipsychotics in comparison to non-clozapine second-generation antipsychotics. Similarly, in a double-blind, randomized controlled trial, Rosenheck et al 5 reported that measures of effectiveness demonstrated no advantage for olanzapine compared with haloperidol in overall QOL. In contrast, a naturalistic observational study on patients with schizophrenia undergoing usual care indicated that ziprasidone treatment resulted in improved satisfaction with general activity as measured by the Quality of Life Enjoyment and Satisfaction Questionnaire 6. When measuring QOL in patients taking antipsychotics, it is important to acknowledge that a variety of factors may influence QOL outcomes: these include side effects and daily dosage of the antipsychotic, depressive and negative symptoms, duration of treatment, and subjective tolerability. In a naturalistic comparative study, Ritsner et al 7 found no significant difference in general QOL between patients using atypical vs. typical antipsychotics. However, after adjusting for daily dosage, duration of treatment, and subjective tolerability, QOL measures indicated that atypical antipsychotics (olanzapine and risperidone) were superior to typical ones. Some randomized controlled trials of antipsychotics have used a social functioning scale as an outcome measure, such as the Social and Occupational Functioning Assessment Scale 8 - 9, the Medical Outcomes Study Short-Form 36 Health Survey 10, or the Personal and Social Performance scale (PSP) 11. However, these trials have largely involved short-term interventions, limiting their ability to detect meaningful changes in the social functioning of patients. We are currently undertaking a randomized, controlled, open-label clinical trial in Korea to evaluate improvement in social functioning in patients with schizophrenia or schizoaffective disorder. The study compares long-acting injectable vs. oral risperidone using a hybrid model to assess both efficacy and effectiveness after one year of treatment. Primary outcome measures in this study are the PSP and the Social Functioning Scale. The scales used to assess social functioning and QOL in clinical trials must be appropriate to the study population and the clinical condition, and should measure several dimensions of social functioning and QOL. Furthermore, we need to develop scales that measure functioning independently from symptoms, and that are sensitive to changes over the course of the illness.

Prospective assessment of the reliability, validity, and sensitivity to change of the Functional Assessment of Cancer Therapy-Melanoma questionnaire.

The authors previously developed a melanoma-specific module for the Functional Assessment of Cancer Therapy (FACT-Melanoma), a tool for the assessment of quality of life (QOL) in patients with melanoma. The reliability and validity of the FACT-Melanoma was examined in this study. Patients with melanoma (N = 273; stages I-IV) completed a battery of questionnaires at the time of enrollment. The validity of the instrument was examined by comparing FACT-Melanoma scores with performance status, disease stage, treatment status, and other scales, including the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Melanoma Module, the Profile of Mood States, and the Marlowe-Crowne Social Desirability Scale. Patients were assessed after 1 week to assess test-retest reliability and at 3 months to determine the sensitivity of the instrument to change in performance status. The internal consistency and test-retest reliability (r) of the melanoma subscale (Cronbach alpha = .85; r = .81) and the total FACT-Melanoma (alpha = .95; r = .90) were excellent. Overall, the scales were correlated with other measures, as anticipated. Total FACT-Melanoma scores, along with scores for physical well-being, emotional well-being, functional well-being, and melanoma-specific scales, were lower for patients with advanced (stage III/IV) melanoma, poor performance status, and patients who were receiving active treatment. The FACT-Melanoma total score and the score for physical well-being were sensitive to changes in performance status (P = .0012 and P = .004, respectively). The results of the current study indicated that the FACT-Melanoma questionnaire is a reliable and valid instrument for patients with melanoma that can be used for the assessment of QOL in clinical trials.

Quality-of-Life Assessment in Rheumatoid Arthritis

Rheumatoid arthritis (RA) is a chronic and lifelong autoimmune disorder that results in significant pain, disability and excess mortality if untreated or inadequately treated. Quality-of-life (QOL) assessments are particularly important in the absence of a cure for RA. Generic and disease-specific patient-reported QOL instruments, such as the Health Assessment Questionnaire (HAQ) Disability Index and the SF-36, have proven validity and sensitivity for assessment of changes in QOL in clinical trials of disease-modifying anti-rheumatic drugs (DMARDs). However, these instruments are rarely utilized in clinical practice, and patients have reported that the actual clinical assessments alone do not address important parameters, such as fatigue and disturbed sleep, which significantly affect QOL.New biological DMARDs have shown significant efficacy in improving clinical and QOL parameters in randomized controlled trials. However, the high cost of biological DMARDs compared with non-biological DMARDs is a factor in the increasing health costs associated with the treatment of RA. Generic health utility instruments that measure QOL parameters enable calculation of the increased QALYs associated with more costly treatment in patients with RA. The costs per QALY associated with biological DMARDs in RA appear to be comparable to those of other accepted medical interventions. Interest in incorporating QOL parameters in formulary and public health decision making concerning the use of new agents for RA is increasing.

Quality of life and breast cancer – state of the art

The quality of life (QoL) impairments of patients receiving a diagnosis and treatment of breast cancer can be substantial. These include early menopause, chemotherapy induced toxicity, loss of body image, limitations of sexual activity and fertility. When assessing the value of a particular anticancer treatment, it is important to consider the impact it may have not only on survival but also on QoL. In clinical trials, the standard endpoints are response rates, progression free survival, overall survival, and toxicity. In recent years, QoL has become an additional secondary trial endpoint. The current state of evaluating the QoL of breast cancer patients, employ a multidimensional approach involving validated self-reported questionnaires. Among the QoL instruments, there are some measures specifically developed for breast cancer such as the European Organisation for Research and Treatment (EORTC) BR-23 module. Up to now, several randomized EORTC trials have evaluated QoL using the EORTC QLQ-BR23 module. Results from randomized clinical breast cancer trials in which two treatments of comparable efficacy could be evaluated and chosen on the basis of their impact on QoL parameters is important to clinical decision-making. After a general Introduction: to the conceptualisation of QoL, methodological issues, measurements specific for breast cancer and guidelines for assessing QoL in clinical trials will be presented. Aspects affecting the QoL of breast cancer patient in relation to different treatment regimens will be discussed.

Development of the US PSORIQoL: a psoriasis‐specific measure of quality of life

Several instruments are available for assessing impairment and disability associated with psoriasis. The first true psoriasis-specific quality of life (QoL) questionnaire suitable for use in clinical trials, the Psoriasis Index of Quality of Life (PSORIQoL), has recently been developed in the UK. The aim of the current study was to produce and validate a conceptually equivalent US version of the PSORIQoL with equally good psychometric properties. A lay translation panel was employed to ensure that the wording of the questionnaire was appropriate for a US population. Semi-structured interviews conducted with 37 patients tested the instrument's ease of completion, relevance and comprehensiveness. Finally, a test-retest validation mail survey was conducted with 72 patients to determine reliability, internal consistency and construct validity. Few changes were necessary to the wording of the questionnaire. Interviewees found the questionnaire easy to complete (requiring an average of 4 min) and the content relevant and comprehensive. The adapted measure had comparable psychometric properties to the original, with a test-retest reliability coefficient of 0.90, indicating excellent reproducibility. Internal consistency and initial indications of construct validity were also good, with scores on the measure related as expected to perceived general health and severity of psoriasis and the presence of visible lesions. The US PSORIQoL is a practical, reliable and valid instrument for measuring the impact of psoriasis and its treatment on QoL in clinical trials and in routine practice. It remains necessary to establish the instrument's responsiveness to changes in QoL associated with effective interventions.

Association between health-related quality of life and clinical efficacy endpoints in rheumatoid arthritis patients after four weeks treatment with anti-inflammatory agents

Improvement of health-related quality of life (QoL) is increasingly recognized as a maj or treatment goal for patients with rheumatoid arthritis (RA). There are several measures of general health status and of physical functioning for assessing treatment effects on QoL in patients with RA, however, the relationship between QoL outcomes and conventional clinical efficacy endpoints is not completely understood. To describe the association between changes in QoL and changes in other efficacy measures, among patients with RA after four weeks of treatment with etoricoxib, naproxen or placebo, and to explore differences in the association of changes in efficacy and changes in QoL parameters across treatment groups. The study used data from 1684 patients with RA enrolled in two identical clinical trials (one US and one multinational). Patients were randomized to placebo, etoricoxib 90 mg once daily, or naproxen 500 mg twice daily in a 2 : 2: 1 allocation ratio. Primary efficacy endpoints were tender joint count, swollen joint count, patient global assessment of disease activity (100 mm VAS), and investigator global assessment of disease activity (0 - 4 Likert scale). QoL assessments were based on the Health Assessment Questionnaire (HAQ) and the Medical Outcomes Survey Short Form 36 (SF-36). Mean differences between baseline and week four were calculated for each parameter studied. Linear regression analysis was performed to assess the association between changes in clinical efficacy and changes in QoL parameters, adjusted for covariates. The degree of association between changes in tender or swollen joint counts and changes in QoL variables was low, explaining less than 10% of the variability for most QoL variables, except bodily pain (SF-36). In contrast, changes in patient global assessment of disease activity explained 33% of the variability in the overall HAQ score, and in the physical component score (SF-36; adjusted regression models). Values for investigator global assessment of disease activity were below those for patient global assessment but above joint count measures. Results were similar between the etoricoxib, naproxen and placebo groups in the degree of association between changes in efficacy and QoL variables. Currently used efficacy endpoints are less than ideal predictors of change in QoL. There is no evidence from this study that the association between changes in CE endpoints and QoL was different across treatments. Our results highlight the need to assess both conventional efficacy measures and QoL in clinical trials of RA treatments.

Quality of life

Sinusitis is one of the most frequent diseases in humans. Despite severe complications sinusitis may be considered a non-serious disease compared to other illnesses. Therefore, sinusitis is sometimes erroneously regarded as a banal disorder. It is mostly unknown that patients suffering from sinusitis may have a marked impairment of their quality of life (QOL). The term health-related QOL comprises physical and psychological well-being as well as aspects of daily life and social life from the patient's perspective. Validated measures (questionnaires) are able to quantify the impairment of QOL as well as the influence of therapy on QOL. Especially in the treatment of sinusitis, QOL becomes a relevant additional parameter due to the fact that clinical and subjective, patient-based criteria do not correlate sufficiently. This article provides an overview of available QOL questionnaires on sinusitis, highlights results of QOL studies in sinusitis, and elucidates the relevance of QOL in clinical trials as well as in the relation between the physician and the patient.

International development of the Parents’ Index of Quality of Life in Atopic Dermatitis (PIQoL-AD)

The international development of the Parents' Index of Quality of Life in Atopic Dermatitis (PIQoL-AD), a quality of life (QoL) instrument specific to parents of children with atopic dermatitis (AD) is described. The instrument was developed simultaneously in several countries. Its content was derived from 65 qualitative interviews with parents in the UK, Netherlands and Italy. The measure was then produced for the UK, Netherlands, Italy, Germany, France, US and Spain. Field-test interviews were conducted with approximately 20 patients in each country to assess face and content validity. A two time-point survey was conducted with between 45 and 328 parents in each country to finalise the instrument through application of the Rasch model and to evaluate the psychometric properties of the final instrument. Application of the Rasch model to the survey data identified the final 28-item version. All language versions had good item fit, test-retest reliability (above 0.85), internal consistency and promising validity. The PIQoL-AD is a valuable instrument for inclusion in clinical trials and routine clinical practice. It provides distinct and complementary information to that of existing dermatology-specific measures and has been shown to be responsive to changes in QoL in clinical trials.

Development of a questionnaire module to supplement the EORTC QLQ-C30 to assess quality of life in patients with hepatocellular carcinoma, the EORTC QLQ-HCC18

Measurement of quality of life (QoL) in hepatocellular carcinoma (HCC) requires assessment of factors related to chronic liver disease, as well as issues related to the primary tumour and its treatment. This study describes the development of a questionnaire module in patients from Europe, as well as Taiwan and Hong Kong. The questionnaire was developed according to the European Organisation for Research and Treatment of Cancer (EORTC) QoL Group guidelines. Twenty nine QoL issues were identified from a literature search. Semi-structured interviews with patients ( n = 32) and health-care professionals ( n = 10) reduced the issues to 22 items forming a provisional questionnaire. This was tested in 158 patients from three countries. Descriptive statistics and clinical judgement reduced the module to 18 items conceptualised as containing six scales and two single item. This study recommends the EORTC QLQ-HCC18 to accompany the QLQ-C30 to measure QoL in clinical trials in HCC.

The psychometric properties of four quality of life instruments used in cardiovascular populations.

The psychometric properties of four quality of life instruments used in cardiovascular populations.

A review of measures of quality of life for children with chronic illness

AIMSTo identify currently available generic and disease specific measures of quality of life (QoL) for work with children; and make recommendations about the future development and application of QoL measures.METHODSSystematic...

Quality of life assessment in clinical trials--guidelines and a checklist for protocol writers: the U.K. Medical Research Council experience. MRC Cancer Trials Office.

Many clinical trials groups now routinely consider including Quality of Life (QoL) assessment in trials. Indeed, several have policies stating that QoL should be considered as a potential endpoint in all new trials and that if it is not to be evaluated the applicants should justify not doing so. However, inclusion of QoL in clinical trials presents a number of difficult organisational issues, and serious problems in compliance have frequently been reported. Thus, in multicentre clinical trials many of the expected QoL questionnaires fail to be successfully completed and returned, although a few groups have claimed high success rates. However, it is well recognised that if questionnaires are missing, there may be bias in the interpretation of trial results, and the estimates of treatment differences and the overall level of QoL may be inaccurate and misleading. Hence it is important to seek methods of improving compliance, at the level of both the participating institution and the patient. We describe a number of methods for addressing these issues, which we suggest should be considered by all those writing clinical trial protocols involving QoL assessment. These are based upon over a decade of experience with assessing QoL in Medical Research Council (MRC) cancer clinical trials. In particular, we provide a checklist for points that should be covered in protocols. Examples are given from a range of current MRC Cancer Trials Office protocols, which it is proposed might act as templates when writing new protocols.

Quality-of-life considerations in the adjuvant setting: critical review.

This session combined theoretical and practical considerations relevant to the measurement of quality of life (QL) in the setting of adjuvant therapy and preventive trials in breast cancer. Perhaps more importantly, by its very inclusion it underlined the importance attached to the concept of QL in clinical trials and clinical practice. In adjuvant therapy, and still more in preventive interventions, the woman who undertakes more or less toxic treatment does so in the hope of future gain. In neither case does the patient have discernible disease at the time that therapy is used; thus any morbidity incurred can be compensated only by delay of disease or death. Where alternative strategies for the pursuit of such benefits are being compared, it is important to measure the impact of each on QL.