Stefan J. Cano

Development of a new patient-reported outcome measure for breast surgery: the BREAST-Q.

Background: Measuring patient-reported outcomes has become increasingly important in cosmetic and reconstructive breast surgery. The objective of this study was to develop a new patient-reported outcome measure to assess the unique outcomes of breast surgery patients. Methods: Patient interviews, focus groups, expert panels, and a literature review were used to develop a conceptual framework and a list of questionnaire items. Three procedure-specific questionnaires (augmentation, reduction, and reconstruction) were developed and cognitive debriefing interviews used to pilot each questionnaire. Revised questionnaires were field tested with 1950 women at five centers in the United States and Canada (response rate, 72 percent); 491 patients also completed a test-retest questionnaire. Rasch measurement methods were used to construct scales, and traditional psychometric analyses, following currently recommended procedures and criteria, were performed to allow for comparison with existing measures. Results: The conceptual framework included six domains: satisfaction with breasts, overall outcome, and process of care, and psychosocial, physical, and sexual well-being. Independent scales were constructed for these domains. This new patient-reported outcome measure “system” (the BREAST-Q) contains three modules (augmentation, reconstruction, and reduction), each with a preoperative and postoperative version. Each scale fulfilled Rasch and traditional psychometric criteria (including person separation index 0.76 to 0.95; Cronbach’s alpha 0.81 to 0.96; and test-retest reproducibility 0.73 to 0.96). Conclusions: The BREAST-Q can be used to study the impact and effectiveness of breast surgery from the patient’s perspective. By quantifying satisfaction and important aspects of health-related quality of life, the BREAST-Q has the potential to support advocacy, quality metrics, and an evidence-based approach to surgical practice.

Rating scales as outcome measures for clinical trials in neurology: problems, solutions, and recommendations

Have state-of-the-art clinical trials failed to deliver treatments for neurodegenerative diseases because of shortcomings in the rating scales used? This Review assesses two methodological limitations of rating scales that might help to answer this question. First, the numbers generated by most rating scales do not satisfy the criteria for rigorous measurements. Second, we do not really know which variables most rating scales measure. We use clinical examples to highlight concerns about the limitations of rating scales, examine their underlying rationales, clarify their implications, explore potential solutions, and make some recommendations for future research. We show that improvements in the scientific rigour of rating scales can improve the chances of reaching the correct conclusions about the effectiveness of treatments.

Improving the evaluation of therapeutic interventions in multiple sclerosis: the role of new psychometric methods.

OBJECTIVES In this monograph we examine the added value of new psychometric methods (Rasch measurement and Item Response Theory) over traditional psychometric approaches by comparing and contrasting their psychometric evaluations of existing sets of rating scale data. We have concentrated on Rasch measurement rather than Item Response Theory because we believe that it is the more advantageous method for health measurement from a conceptual, theoretical and practical perspective. Our intention is to provide an authoritative document that describes the principles of Rasch measurement and the practice of Rasch analysis in a clear, detailed, non-technical form that is accurate and accessible to clinicians and researchers in health measurement. REVIEW METHODS A comparison was undertaken of traditional and new psychometric methods in five large sets of rating scale data: (1) evaluation of the Rivermead Mobility Index (RMI) in data from 666 participants in the Cannabis in Multiple Sclerosis (CAMS) study; (2) evaluation of the Multiple Sclerosis Impact Scale (MSIS-29) in data from 1725 people with multiple sclerosis; (3) evaluation of test-retest reliability of MSIS-29 in data from 150 people with multiple sclerosis; (4) examination of the use of Rasch analysis to equate scales purporting to measure the same health construct in 585 people with multiple sclerosis; and (5) comparison of relative responsiveness of the Barthel Index and Functional Independence Measure in data from 1400 people undergoing neurorehabilitation. RESULTS Both Rasch measurement and Item Response Theory are conceptually and theoretically superior to traditional psychometric methods. Findings from each of the five studies show that Rasch analysis is empirically superior to traditional psychometric methods for evaluating rating scales, developing rating scales, analysing rating scale data, understanding and measuring stability and change, and understanding the health constructs we seek to quantify. CONCLUSIONS There is considerable added value in using Rasch analysis rather than traditional psychometric methods in health measurement. Future research directions include the need to reproduce our findings in a range of clinical populations, detailed head-to-head comparisons of Rasch analysis and Item Response Theory, and the application of Rasch analysis to clinical practice.

Measuring quality of life in cosmetic and reconstructive breast surgery: a systematic review of patient-reported outcomes instruments.

Background: Patient-reported outcomes in cosmetic and reconstructive breast surgery are increasingly important for clinical research endeavors. Traditional surgical outcomes, centered on morbidity and mortality, remain important but are no longer sufficient on their own. Quality of life has become a crucial research topic augmenting traditional concerns focused on complications and survival. Given this, reliable and valid patient questionnaires are essential for aesthetic and reconstructive breast surgeons. Methods: The authors performed a systematic literature review to identify patient-reported outcome measures developed and validated for use in cosmetic and reconstructive breast surgery patients. Qualifying instruments were assessed for adherence to international guidelines for health outcomes instrument development and validation. Results: The authors identified 227 health outcomes questionnaires used in breast surgery studies. After 135 generic instruments, 65 ad hoc instruments, seven oncologic instruments, 11 education questionnaires, and two non-English-language questionnaires were excluded, seven measures remained. Detailed analysis revealed that six of the seven measures had undergone limited development and validation. Only one measure, the Breast-Related Symptoms Questionnaire, demonstrated adequate development and validation in its target population. It had, nevertheless, significant content limitations. Conclusions: Valid, reliable, and responsive instruments to measure patient-reported outcomes in cosmetic and reconstructive breast surgery are lacking. To demonstrate the benefits of aesthetic and reconstructive breast surgery, future research to rigorously develop and validate new cosmetic and reconstructive breast surgery-specific instruments is needed.

Measuring Quality of Life in Oncologic Breast Surgery: A Systematic Review of Patient‐Reported Outcome Measures

Abstract:  Multiple randomized trials demonstrate equivalent survival between BCT and mastectomy, but clinical outcomes research must also evaluate patient satisfaction and quality of life. This review analyzes existing patient‐reported outcome (PRO) measures in oncologic breast surgery to assess utility and make recommendations for future research. We performed a systematic literature review to identify PRO measures used in oncologic breast surgery patients. After applying inclusion and exclusion criteria, qualifying instruments were assessed for adherence to international guidelines for health outcomes instrument development and validation. Ten measures underwent development and psychometric evaluation in an oncologic breast surgery population. Five of ten measures (EORTC QLQ BR‐23, FACT‐B, HBIS, BIBCQ, and BREAST‐Q) reported an adequate development and validation process. Three of these 5 measures (EORTC QLQ BR‐23, FACT‐B, HBIS) focused on non‐surgical treatment issues. A fourth instrument (BIBCQ) did not address aesthetic concerns after breast reconstruction. The fifth instrument (BREAST‐Q) was developed for use in patients undergoing mastectomy ± reconstruction, but did not address breast‐conserving therapy. Overall, two key limitations were noted: 1) surgery‐specific issues of breast‐conserving surgery patients were not well represented and 2) measures were largely developed without the aid of newer psychometric methods that may improve their clinical utility. Reliable and valid PRO measures in breast cancer patients exist, but even the best instruments do not address all important surgery‐specific and psychometric issues of oncologic breast surgery patients. Newer psychometric methods would facilitate development of scales for use in individual patient care as well as group level comparisons.

Measuring Quality of Life in Dysphonic Patients: A Systematic Review of Content Development in Patient-Reported Outcomes Measures

To review existing patient reported outcomes measures (PROMs) used in dysphonic populations to assess the procedures used in their development and the extent to which these meet current development standards for content generation and psychometric evaluation. The study is a systematic review. A systematic review of Medline, Cumulative Index to Nursing & Allied Health, and Health and Psychosocial Instruments databases was completed using voice, quality of life, and PROMs as keywords. We identified all patient or parent-reported questionnaires measuring quality of life associated with voice disorders from the review findings. Questionnaires were appraised for adherence to international guidelines for the development and evaluation of PROMs as outlined by the Scientific Advisory Committee of the Medical Outcome Trust. Nine PROMs fulfilled the inclusion criteria. The quality of these questionnaires was variable with regard to instrument development and none met all of the current, recommended criteria. Of the nine questionnaires, the Voice Symptom Scale underwent the most rigorous development process. Furthermore, many instruments have been augmented to allow for proxy administration, failing to address quality of life-related issues specific to the target population. Instrument development is often overlooked when attempting to quantify patient reported outcomes in dysphonic patients. Careful instrument development procedures are required to ensure that PROMs are valid, reliable, and responsive. Our review suggests that the deficits in psychometric properties of the current voice-related PROMs may be, at least in part, due to deficits in the development process. Furthermore, these data suggest the potential utility of a novel PROM adhering to rigorous international standards to better ensure that clinicians appreciate the variables most relevant to patients with voice disorders and address some of the psychometric shortcomings of the currently used questionnaires.

Patient satisfaction with postmastectomy breast reconstruction: a comparison of saline and silicone implants.

At a time when the safety and effectiveness of breast implants remains under close scrutiny, it is important to provide reliable and valid evidence regarding patient outcomes. In the setting of postmastectomy reconstruction, patient satisfaction and quality of life may be the most significant outcome variables when evaluating surgical success. The objective of the current study was to identify predictors of patient satisfaction with breast appearance, including implant type, in a large sample of women who underwent breast reconstruction surgery using implants.

The ADAS-cog in Alzheimer's disease clinical trials: psychometric evaluation of the sum and its parts

Background The Alzheimers Disease Assessment Scale Cognitive Behavior Section (ADAS-cog), a measure of cognitive performance, has been used widely in Alzheimers disease trials. Its key role in clinical trials should be supported by evidence that it is both clinically meaningful and scientifically sound. Its conceptual and neuropsychological underpinnings are well-considered, but its performance as an instrument of measurement has received less attention. Objective To examine the traditional psychometric properties of the ADAS-cog in a large sample of people with Alzheimers disease. Methods Data from three clinical trials of donepezil (Aricept) in mild-to-moderate Alzheimers disease (n=1421; MMSE 10–26) were analysed at both the scale and component level. Five psychometric properties were examined using traditional psychometric methods. These methods of examination underpin upcoming Food and Drug Administration recommendations for patient rating scale evaluation. Results At the scale-level, criteria tested for data completeness, scaling assumptions (eg, component total correlations: 0.39–0.67), targeting (no floor or ceiling effects), reliability (eg, Cronbachs α: = 0.84; test-retest intraclass correlations: 0.93) and validity (correlation with MMSE: −0.63) were satisfied. At the component level, 7 of 11 ADAS-cog components had substantial ceiling effects (range 40–64%). Conclusions Performance was satisfactory at the scale level, but most ADAS-cog components were too easy for many patients in this sample and did not reflect the expected depth and range of cognitive performance. The clinical implication of this finding is that the ADAS-cogs estimate of cognitive ability, and its potential ability to detect differences in cognitive performance under treatment, could be improved. However, because of the limitations of traditional psychometric methods, further evaluations would be desirable using additional rating scale analysis techniques to pinpoint specific improvements.

The Sino-Nasal Outcome Test (SNOT) : Can we make it more clinically meaningful?

OBJECTIVE: To test whether subscales should be used when analyzing the Sino-Nasal Outcome Test (SNOT). STUDY DESIGN AND SETTING: Prospective cohort study involved 87 acute NHS hospitals in England and Wales. RESULTS: With the use of exploratory factor analysis we found evidence for the existence of four unique constructs within the SNOT. Two constructs address symptoms (rhinologic and ear/facial) and two address aspects of health-related quality of life (psychological issues and sleep function). Subscales of the SNOT that correspond to these constructs provided clinically meaningful information over and above that provided by the SNOT total score on the type of surgical benefits gained by patients with different sino-nasal conditions. CONCLUSION: The SNOT is not unidimensional and a SNOT total score will mask variation in the true underlying constructs. SIGNIFICANCE: The SNOT would have greatly improved clinical use if it was scored with appropriate subscales. Such a measure would allow us to tease out the differential impact of sino-nasal conditions, in addition to allowing greater understanding of treatment effects.

Quality of life among breast cancer patients with lymphedema: a systematic review of patient-reported outcome instruments and outcomes.

PurposeLymphedema following breast cancer surgery remains a common and feared treatment complication. Accurate information on health-related quality of life (HRQOL) outcomes among patients with lymphedema is critically needed to inform shared medical decision making and evidence-based practice in oncologic breast surgery. Our systematic review aimed to (1) identify studies describing HRQOL outcomes in breast cancer-related lymphedema (BCRL) patients, (2) assess the quality of these studies, and (3) assess the quality and appropriateness of the patient-reported outcome (PRO) instruments used.MethodsUsing the PRISMA statement, we performed a systematic review including studies describing HRQOL outcomes among BCRL patients. Studies were classified by levels of evidence and fulfillment of the Efficace criteria. PRO instruments were assessed using the COSMIN criteria.ResultsThirty-nine studies met inclusion criteria, including 8 level I and 14 level II studies. Sixteen of 39 studies were compliant with the Efficace criteria. Seventeen HRQOL instruments were used, two specific to lymphedema patients. Exercise and complex decongestive therapy treatment interventions were associated with improved HRQOL.ConclusionsHigh-quality data on HRQOL outcomes is required to inform surgical decisions for breast cancer management and survivors. Of the lymphedema-specific PRO instruments, the Upper Limb Lymphedema 27 (ULL-27) was found to have strong psychometric properties. Future studies should strive to use high-quality condition- specific PRO instruments, follow existing guidelines for HRQOL measurement and to consider economic burdens of BCRL.Implications for Cancer SurvivorsAs lymphedema may develop many years after breast cancer surgery, the ULL-27 may offer greater content validity for use in survivorship research.