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Heart failure (HF) is a common and morbid condition impacting multiple health domains. We previously reported the development of the PROMIS®-Plus-HF (PROMIS+HF) profile measure, including universal and HF-specific items. To facilitate use, we developed shorter, PROMIS+HF profiles intended for research and clinical use.Candidate items were selected based on psychometric properties and symptom range coverage. HF clinicians (n = 43) rated item importance and clinical actionability. Based on these results, we developed the PROMIS+HF-27 and PROMIS+HF-10 profiles with summary scores (0-100) for overall, physical, mental, and social health. In a cross-sectional sample (n = 600), we measured internal consistency reliability (Cronbach's alpha and Spearman-Brown), test-retest reliability (intraclass coefficient; n = 100), known-groups validity via New York Heart Association (NYHA) class, and convergent validity with Kansas City Cardiomyopathy Questionnaire (KCCQ) scores. In a longitudinal sample (n = 75), we evaluated responsiveness of baseline/follow-up scores by calculating mean differences and Cohen's d and comparing with paired t-tests. Internal consistency was good to excellent (α 0.82-0.94) for all PROMIS+HF-27 scores and acceptable to good (α/Spearman-Brown 0.60-0.85) for PROMIS+HF-10 scores. Test-retest intraclass coefficients were acceptable to excellent (0.75-0.97). Both profiles demonstrated known-groups validity for the overall and physical health summary scores based on NYHA class, and convergent validity for nearly all scores compared with KCCQ scores. In the longitudinal sample, we demonstrated responsiveness for PROMIS+HF-27 and PROMIS+HF-10 overall and physical summary scores. For the PROMIS+HF overall summary scores, a group-based increase of 7.6-8.3 points represented a small to medium change (Cohen's d = 0.40-0.42). For the PROMIS+HF physical summary scores, a group-based increase of 5.0-5.9 points represented a small to medium change (Cohen's d = 0.29-0.35).The PROMIS+HF-27 and PROMIS+HF-10 profiles demonstrated good psychometric characteristics with evidence of responsiveness for overall and physical health. These new measures can facilitate patient-centred research and clinical care, such as improving care quality through symptom monitoring, facilitating shared decision-making, evaluating quality of care, assessing new interventions, and monitoring during the initiation and titration of guideline-directed medical therapy.

Background In clinical trials and clinical practice, patient-reported outcomes are almost always assessed using multiple patient-reported outcome measures at the same time. This raises concerns about whether patient responses are affected by the order in which the patient-reported outcome measures are administered. Methods This questionnaire-based study of order effects included adult cancer patients from five cancer centers. Patients were randomly assigned to complete questionnaires via paper booklets, interactive voice response system, or tablet web survey. Linear Analogue Self-Assessment, Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events, and Patient-Reported Outcomes Measurement Information System assessment tools were each used to measure general health, physical function, social function, emotional distress/anxiety, emotional distress/depression, fatigue, sleep, and pain. The order in which the three tools, and domains within tools, were presented to patients was randomized. Rates of missing data, scale scores, and Cronbach’s alpha coefficients were compared by the order in which they were assessed. Analyses included Cochran–Armitage trend tests and mixed models adjusted for performance score, age, sex, cancer type, and curative intent. Results A total of 1830 patients provided baseline patient-reported outcome assessments. There were no significant trends in rates of missing values by whether a scale was assessed earlier or later. The largest order effect for scale scores was due to a large mean score at one assessment time point. The largest difference in Cronbach’s alpha between the versions for the Patient-Reported Outcomes Measurement Information System scales was 0.106. Conclusion The well-being of a cancer patient has many different aspects such as pain, fatigue, depression, and anxiety. These are assessed using a variety of surveys often collected at the same time. This study shows that the order in which the different aspects are collected from the patient is not important.

Background Treatment burden refers to the work involved in managing one’s health and its impact on well-being and has been associated with nonadherence in patients with chronic illnesses. No kidney transplant (KT)-specific measure of treatment burden exists. The aim of this study was to develop a KT-specific supplement to the Patient Experience with Treatment and Self-Management (PETS), a general measure of treatment burden. Methods After drafting and pretesting KT-specific survey items, we conducted a cross-sectional survey study involving KT recipients from Mayo Clinic in Minnesota, Arizona, and Florida. Exploratory factor analysis (EFA) was used to identify domains for scaling the KT-specific supplement. Construct and known-groups validity were determined. Results Survey respondents (n = 167) had a mean age of 61 years (range 22–86) and received a KT on average 4.0 years ago. Three KT-specific scales were identified (transplant function, self-management, adverse effects). Higher scores on the KT-specific scales were correlated with higher PETS treatment burden, worse physical and mental health, and lower self-efficacy (p Conclusions We developed a KT-specific supplement to the PETS general measure of treatment burden. Scores may help providers identify recipients at risk for nonadherence.

Background In primary care there is a need for more quality measures of person-centered outcomes, especially ones applicable to patients with multiple chronic conditions (MCCs). The aim of this study was to derive and validate a short-form version of the Patient Experience with Treatment and Self-management (PETS), an established measure of treatment burden, to help fill the gap in quality measurement. Methods Patient interviews (30) and provider surveys (30) were used to winnow items from the PETS (60 items) to a subset targeting person-centered care quality. Results were reviewed by a panel of healthcare providers and health-services researchers who finalized a pilot version. The Brief PETS was tested in surveys of 200 clinic and 200 community-dwelling MCC patients. Surveys containing the Brief PETS and additional measures (e.g., health status, medication adherence, quality of care, demographics) were administered at baseline and follow-up. Correlations and t-tests were used to assess validity, including responsiveness to change of the Brief PETS. Effect sizes (ES) were calculated on mean differences. Results Winnowing and panel review resulted in a 34-item Brief PETS pilot measure that was tested in the combined sample of 400 (mean age = 57.9 years, 50% female, 48% white, median number of conditions = 5). Reliability of most scales was acceptable (alpha > 0.70). Brief PETS scores were associated with age, income, health status, and quality of chronic illness care at baseline (P P P Conclusions The Brief PETS (final version: 32 items) is a reliable and valid tool for assessing person-centered care quality related to treatment burden. It holds promise as a means of giving voice to patient concerns about the complexity of disease management.

PURPOSE: The purpose of this study was to test the known-groups validity and responsiveness to change of the Patient Experience with Treatment and Self-management (PETS, vs. 2.0), a measure of treatment burden. METHODS: The PETS and other standard measures were mailed at baseline and 12-month follow-up to adults living with multiple chronic conditions in southeast Minnesota (USA). A sample of 365 people (mean age = 62.1 years) completed both surveys. Baseline, 12-month, and changes in PETS burden scores were examined. Clinical anchors used to test validity included number of diagnoses (2-4 vs. 5+), mental health diagnosis (yes/no), medication adherence and health literacy (suboptimal/optimal), and changes in self-efficacy, global physical and global mental health (worsening/improving). Independent samples t-tests were used to compare scores. RESULTS: PETS scales showed good internal consistency (alphas ≥ 0.80). There were few differences across number of diagnoses, but having a mental health diagnosis was associated with higher baseline PETS burden scores (Ps < .05). Suboptimal medication adherence and health literacy over time were associated with worse 12-month PETS burden scores (Ps < .05). Compared with improvements, declines over time in self-efficacy, global physical health, and global mental health were each associated with worsening change scores on PETS impact summary, medical expenses, and bother due to medication reliance and medication side effects (Ps < .05). CONCLUSION: Among multi-morbid adults, the PETS demonstrated evidence of known-groups validity and responsiveness to change across both objective (e.g., mental health diagnoses) and subjective anchors (e.g., changes in self-efficacy, global physical and mental health).

INTRODUCTION: New hypertension guidelines in the United States may require more people to take multiple medications or implement lifestyle changes. Increased treatment burden may be an unintended consequence and lead to worse health outcomes. Our study examined whether treatment burden is associated with factors related to self-management in those with hypertension. METHODS: We conducted a cross-sectional mailed survey of patients from two medical centers in Minnesota. Participants with 2 or more medical conditions completed the Patient Experience with Treatment and Self-management (PETS), a validated treatment burden questionnaire, as well as measures of confidence in self-management ability, health literacy, healthcare-related financial difficulties, and perception of provider interpersonal skills. We used Partial correlation analyses, controlling for age, sex, race, and education, to test relationships among study variables. RESULTS: Of 254 respondents who had a diagnosis of hypertension, mean age was 67, 54% were female, and 74% were non-Hispanic White. People with hypertension who reported having lower confidence in self-management ability, lower health literacy, more financial difficulties, and healthcare providers with poorer interpersonal skills, reported higher treatment burden scores (PETS scales correlation magnitude range 0.09–0.62, p < 0.05 on all but four). The strongest associations were observed for medical information and physical/mental exhaustion with self-management (correlation magnitudes from 0.25 to 0.54, p

Background The purpose of this study was to investigate whether patient-reported knee function and health status before and after primary total knee replacement (TKR) at an academic medical center differs among patients from diverse geographic regions. Methods We assessed patient-reported outcomes (PROs) as measured by the Oxford Knee Score (OKS) and EuroQoL-5D (EQ-5D) in 2855 TKR patients at preprimary and one year postprimary TKR procedure between January 1, 2012 and June 30, 2014. We compared the demographic characteristics, response rates, and changes in OKS, EQ-5D, and EQ visual analog scale among local, regional, and national patient groups. Patient- or hospital-related predictors of the postoperative scores were identified after controlling for preoperative scores on the PRO measures. Results Local patients had more comorbid conditions. Groups were similar in clinical outcomes such as length of stay at hospital, complication, and reoperation rates. Local, regional, and national patients had similar response rates and reported a similar level of knee function and health-related quality of life before and after TKR. Eighty nine percent had clinically important improvement on OKS, 69% on EQ-5D index, and 28% EQ visual analog scale, and there were no differences among groups in the proportions of clinically meaningful change. Better postoperative PRO scores were associated with older age, shorter hospital stay, fewer comorbidities, nonsmoking status, fewer complications, and private health insurance. Conclusion Some patient characteristics such as comorbidities were greater in the local patient cohort versus the national cohort. Nevertheless, clinical outcomes and PROs were comparable across all geographic tiers.

Patient-reported outcome measures (PROMs), which are generic or condition-specific, are used for a number of reasons, including clinical care, clinical trials, and in national-level efforts to monitor the quality of health care delivery. Creating PROMs that meet different purposes without overburdening patients, healthcare systems, providers, and data systems is paramount. The objective of this study was to test a generalizable method to incorporate condition-specific issues into generic PROM measures as a first step to producing PROMs that efficiently provide a standardized score. This paper outlines the method and preliminary findings focused on a PROM for osteoarthritis of the knee (OA-K). We used a mixed-methods approach and PROMIS® measures to test development of a combined generic and OA-K-specific PROM. Qualitative methods included patient focus groups and provider interviews to identify impacts of OA-K important to patients. We then conducted a thematic analysis and an item gap analysis: identified areas covered by existing generic PROMIS measures, identified “gap” areas not covered, compared gap areas to legacy instruments to verify relevance, and developed new items to address gaps. We then performed cognitive testing on new items and drafted an OA-K-specific instrument based on findings. We identified 52 existing PROMIS items and developed 24 new items across 14 domains. We developed a process for creating condition-specific instruments that bridge gaps in existing generic measures. If successful, the methodology will create instruments that efficiently gather the patient’s perspective while allowing health systems, researchers, and other interested parties to monitor and compare outcomes over time, conditions, and populations.

Background. Extensi ve family coping research has been conducted among breast cancer, prostate cancer and melanoma with lesser emphasis on the coping experiences of colorectal cancer (CRC) patients and their family members. Objective. T o examine ways in which patients and their family members cope with the diagnosis of CRC. Methods. A total of 73 par ticipants (21 patients, 52 family members) from 23 families described their experiences during and after a CRC diagnosis, including their coping experiences with the diagnosis. Data from semi-structured interviews were audio recorded and transcribed. The data were analyzed utilizing content analysis with inductive coding methods. Results. Eight major themes were identified: positi ve reframing, holding on to a sense of normalcy, religion and spirituality, joining a group, creating awareness of CRC, lifestyle change, seeking information and alternative treatments. Maintaining an emotional sense of normalcy through positive thinking, engaging in activities to take one’s mind off the diagnosis and believing that there is a higher authority which has control over the diagnosis and life were vital for the patients and their family members. Patients and family members used similar coping strategies. Conclusion. Findings from this study have implications for under standing how families blend emotion-based and problem-focused coping strategies in the face of a CRC diagnosis. Further developing evidence-based interventions that target coping and well-being in cancer patients and extending them to family members is necessary and holds great promise for providers who care for patients with familial cancers.

Objectives To examine the concordance between clinicians and men diagnosed with prostate cancer on a clinician-derived pathophysiological classification of the following self-reported urinary complications: storage (irritative), voiding (obstructive), and leakage/incontinence. Materials and methods Fourteen urology experts classified 37 urinary function questionnaire items into 3 primary conceptual dimensions (e.g., storage [irritative], voiding [obstructive] and urinary leakage/incontinence) that would best reflect each item's content. In addition, 218 patient participants provided responses to the 37 items. Using classifications by experts to develop the conceptual framework, the structure was tested using confirmatory factor analyses with patient data. Results Expert consensus was achieved in the classification of 31 out of 37 items. Using the 3-factor conceptual framework and patient data, the fit indices for the overall correlated factor model suggested an acceptable overall model fit. The analyses of the separate domains showed acceptable fit for the storage/irritative domain and the leaking/incontinence domain. The dimensionality of the voiding/obstructive domain was too difficult to estimate. Conclusions Our analysis found items that conceptually and psychometrically support 2 constructs (leaking/incontinence and storage/irritative). The consistency of this support between the groups suggests a clinical relevance that is useful in treating patients. We have conceptual support for a third hypothesis (voiding/obstructive), although there were too few items to assess this psychometrically. Relative motivating factors of bother and urinary complaints were not addressed and remain an unmet need in this field.

ObjectivesTreatment burden is the workload of healthcare for people with long-term conditions (LTC) and its impact on well-being. A method of measurement is required to identify those experiencing high burden and to measure intervention efficacy. Our aim was to identify, examine and appraise validated patient-reported measures (PRMs) of treatment burden in stroke. Here, stroke serves as an exemplar LTC of older adults.DesignA systematic review of published studies that describe the development and validation of PRMs measuring treatment burden in stroke survivors.Data sourcesWe searched MEDLINE, Embase, CINAHL and PsycINFO electronic databases.Eligibility criteriaStudies published between January 2000 and 12 April 2019 inclusive, in English language. No restrictions were set based on clinical setting or geographical location.Data extraction and synthesisScreening, data extraction and quality appraisal were conducted by two independent reviewers. Content of the PRMs was compared with a published taxonomy of treatment burden. Quality appraisal was conducted using International Society for Quality of Life Research standards.ResultsFrom 3993 articles, 6 relevant PRMs were identified: 3 were stroke specific: The Satisfaction with Stroke Care questionnaire; The Stroke Patient-Reported Outcome Measure and The Barriers to Physical Activity after Stroke scale. Three were generic but validated in stroke: The WHO Quality of Life-100; The Patient’s Questionnaire on Participation in Discharge Planning and The Chao Perception of Continuity scale. None comprehensively measured treatment burden. Examples of omitted burdens included developing coping strategies, managing finances and returning to driving. The most notable issue regarding quality appraisal was that three PRMs lacked any underpinning qualitative research relevant to the sample.ConclusionThere is a need to develop a comprehensive PRM of treatment burden for use in stroke, with potential for use in other older populations.

PURPOSE: The purpose of this study was to develop and validate a new comprehensive patient-reported measure of treatment burden-the Patient Experience with Treatment and Self-management (PETS). METHODS: A conceptual framework was used to derive the PETS with items reviewed and cognitively tested with patients. A survey battery, including a pilot version of the PETS, was mailed to 838 multi-morbid patients from two healthcare institutions for validation. RESULTS: A total of 332 multi-morbid patients returned completed surveys. Diagnostics supported deletion and consolidation of some items and domains. Confirmatory factor analysis supported a domain model for scaling comprised of 9 factors: medical information, medications, medical appointments, monitoring health, interpersonal challenges, medical/healthcare expenses, difficulty with healthcare services, role/social activity limitations, and physical/mental exhaustion. Scales showed good internal consistency (α range 0.79-0.95). Higher PETS scores, indicative of greater treatment burden, were correlated with more distress, less satisfaction with medications, lower self-efficacy, worse physical and mental health, and lower convenience of healthcare (Ps

The Patient-Reported Outcomes Measurement Information System (PROMIS) was a National Institutes of Health-funded initiative to develop measures of symptoms and function. Responsiveness is the degree to which a measure can detect underlying changes over time. The objective of the current study was to document the responsiveness of 8 PROMIS measures in a large, population-based cancer cohort.The Measuring Your Health study recruited 2968 patients who were diagnosed with 1 of 7 cancers between 2010 and 2012 through 4 Surveillance, Epidemiology, and End Results registries. Participants completed a baseline survey (6-13 months after diagnosis) and a 6-month follow-up survey. Changes in 8 PROMIS scores were compared with global ratings of transition, changes in performance status, and clinical events.Measures were responsive to 6-month declines and improvements in performance status with small to large effect sizes (ES) (Cohen d = 0.34-0.71; P .01). Mean changes and effect sizes were larger for participants who reported declines compared with those who reported improvements. Small-to-medium ES were observed in patients who reported being "a little" worse (d = 0.31-0.56), and medium-to-large ES were observed in those who reported being "a lot" worse (d = 0.53-0.72). Hospitalized participants reported significant score increases, resulting in worsening of pain (d = 0.51), fatigue (d = 0.35), and depression (d = 0.57; all P .01). Cancer recurrence and progression were associated with smaller increases in pain, fatigue, and sleep disturbance (d = 0.22-0.27).The current results indicated that all 8 PROMIS measures were sensitive to patient-perceived worsening and improvement and to major clinical events. These findings will be able to inform the design and interpretation of future research studies and clinical initiatives administering PROMIS measures. Cancer 2017;123:327-335. © 2016 American Cancer Society.

Background Childhood diseases, such as atopic dermatitis, have a negative impact on quality of life (QoL) of parents. How pediatric psoriasis affects a parent's QoL is unknown. Objective To explore the impact of childhood psoriasis on the lives of the parents. Methods Semistructured interviews were conducted with 31 parents of children with psoriasis. Narrative data were analyzed and a conceptual framework of the effects of childhood psoriasis on parents was developed. Results All parents reported that their child's psoriasis caused a substantial, negative impact on their own QoL. A conceptual framework showed a negative impact on health and self-care, emotional well-being, family and social function, personal well-being, and life pursuits. Emotional well-being was the most affected domain. Limitations It was not possible to correlate psoriasis severity with parental QoL. Conclusion Childhood psoriasis alters the QoL of parents in multiple ways. Information from this study can be used to develop a QoL instrument to explore treatment and support strategies for families affected by pediatric psoriasis.

Objective A conceptual framework for patient-reported outcomes (PROs) is a structured representation of outcome concepts and issues. Our aim was to develop a conceptual framework of PROs for hormone-refractory prostate cancer (HRPC) to support measurement clarity. Methods Relevant outcome issues were identified from review of recent clinical trials. This provided content for an interview with 15 metastatic HRPC patients and a survey of 10 practitioners. All participants were asked about the relevance and importance of 26 outcomes and were allowed to nominate new outcomes. Practitioners were also asked to determine which outcomes endorsed by patients were attributable to the disease (symptoms) versus treatment (side effects). Analyses of archived clinical trial data were used to verify and augment the interview and survey results. Results Patients endorsed 11 concerns as relevant and important to HRPC including general pain, bone pain, urinary problems, fatigue, appetite loss, constipation, erectile dysfunction, peripheral neuropathy, diarrhea, PSA anxiety, and changes in self image. Practitioner judgments helped classify each concern into one of four categories, disease symptom, treatment side effect, both symptom and side effect, or psychological concern. Additionally, patients endorsed (and practitioners confirmed) the relevance and importance of several general domains of quality of life. Analyses of archived data confirmed the importance of these issues and suggested two additional concerns. Conclusion Findings were used to propose a conceptual framework of PROs for metastatic HRPC. Such frameworks can be used to help specify targets for assessment in clinical studies such as treatment trials.

Objective To compare Cohen's guidelines for small (0.2), medium (0.5), and large (0.8) effect sizes with empirical estimates for a cancer-specific health-related quality-of-life questionnaire (HRQOL), the Functional Assessment of Cancer Therapy - General (FACT-G). Methods Seventy-one papers satisfied inclusion criteria for meta-analysis. Blinded to the HRQOL results, three “experts” (with expertise in interpreting the FACT-G questionnaire and managing cancer patients), predicted the relative magnitude of HRQOL mean differences. Size classes (small, medium, large) were defined in terms of relevance to clinical decision making. The experts worked independently and based their predictions on patient characteristics and clinical circumstances. Their judgments were linked with FACT-G results and inverse-variance–weighted mean effect sizes calculated for each size class. Results At least two experts were perfectly concordant and up to one was discordant by at most one size category for 833 of the mean differences; for these, weighted kappas were generally in the “substantial” range (0.60–0.79). Of these mean differences, 617 were cross-sectional; small, medium, and large mean effect sizes were physical well-being 0.42, 0.87, 1.6; functional well-being 0.37, 0.71, 1.6; emotional well-being 0.32, 0.40, no large differences; and social well-being 0.14, 0.23, no large differences. Two hundred and sixteen longitudinal mean differences yielded small and medium effect sizes: physical well-being 0.26, 0.34; functional well-being 0.14, 0.28; emotional well-being 0.27, 0.23; and social well-being 0.08, 0.01. There was virtually no evidence for large longitudinal effects. Conclusion These results provide specific, evidence-based alternatives to Cohen's generic guidelines, for use in sample-size calculations for the FACT-G and interpretation of the clinical significance of effects measured with FACT-G.

Because bone metastases cause significant pain, we developed a questionnaire to evaluate its nature, severity, and impact. This 16-item questionnaire is the Functional Assessment of Cancer Therapy-Bone Pain (FACT-BP). We also developed a 13/18-item questionnaire, the Functional Assessment of Chronic Illness Therapy-Treatment Satisfaction-Bone Treatment Convenience and Satisfaction Questionnaire (FACIT-TS-BTCSQ), to evaluate patients' expectations and acceptance of bone-specific therapies. We evaluated the performance of these scales in two clinical trials. In both trials, we enrolled patients with metastatic breast cancer, who had progressive bone metastases despite first-line therapy with pamidronate or clodronate. We administered intravenous zoledronic acid to 31 patients in one trial and oral ibandronate to 30 patients in the other. Patients completed the FACT-BP questionnaire and FACIT-TS-BTCSQ at baseline, then at Weeks 4, 8, and 12. The FACT-BP scale showed good internal consistency reliability [Cronbach's alpha (alpha)=0.93-0.96]. There was evidence of construct validity, and known-group validity was supported by score shifts in the anticipated direction (Cohen's d effect size=0.36). The FACT-BP score reflected clinical change as evidenced by differences in performance status. This cross-sectional anchor-based criterion suggested reasonable clinically important differences (effect size=0.36). The FACIT-TS-BTCSQ showed good internal consistency reliability for treatment expectation (alpha=0.87) and treatment experience (alpha's=0.89-0.92). The FACT-BP scale is meaningful and appears appropriate for broader use. The assessment of satisfaction (FACIT-TS-BTCSQ) raised questions that will require further research.

Objective To determine clinically meaningful changes (CMCs) for the Functional Assessment of Cancer Therapy–Prostate (FACT–P). Methods We obtained data from a Phase III trial of atrasentan in metastatic hormone-refractory prostate cancer patients (n = 809). We determined anchor-based differences using Karnofsky Performance Status (KPS), bone alkaline phosphatase (BAP), hemoglobin, time to disease progression (TTP), adverse events (AE), and survival. One-third and one-half standard deviation and standard error of measurement (SEM) were used as distribution-based criteria for CMCs. Comparison across baseline FACT–P domains and derived scales [FACT–P total score, Trial Outcome Index (TOI) score, prostate cancer subscale (PCS) score, pain-related score, and FACT Advanced Prostate Symptom Index (FAPSI)] were conducted for KPS, BAP, and hemoglobin using Student's t tests. Twelve-week change scores were compared for TTP, AE, and survival using ANCOVA. Results CMCs were estimated as 6 to 10 for FACT–P total score, 5 to 9 for FACT–P TOI score, 2 to 3 for FACT–P PCS, 1 to 2 for the 4 PCS pain-related questions, and 2 to 3 for FAPSI. CMCs were also estimated using distribution-based criteria. Kappa statistics were computed to determine the degree of correspondence between the recommended guideline of 1.0 SEM and empirically derived standards. Most of the kappas for health-related quality of life domains and SEM standards had "substantial" to "almost perfect" concordance. Conclusions The significant relationship between clinical and quality of life data provides support for the use of CMCs to increase interpretability of FACT–P scores.

We previously developed a multidomain, self-report outcome measure relevant to a wide range of complementary and alternative medical (CAM) therapies. We report the results of a pilot study to validate the measure.Fifty-two patients (60% female, mean age, 53 years) seeking services from CAM providers completed a battery of measures prior to a scheduled visit. The battery included the Positive and Negative Affect Schedule, items from the Brief Pain and Fatigue Inventories, ratings of global quality of life (QOL) and change in physical health, and our pilot measure consisting of six hypothesized subscales (pain, fatigue, physical/functional ability, personal control, existential issues, and general QOL). Internal reliability, item convergence and discrimination, construct, and concurrent validity were assessed.Pilot measure domains appeared to be internally reliable with five of six alpha coefficients exceeding 0.70. Multitrait scaling analyses demonstrated that most items converged on the domains that they were hypothesized to represent, although item discrimination was demonstrated for the pain domain only. Correlations with standardized assessments of pain, fatigue, and global QOL showed evidence of construct validity. All domain scores differentiated patients classified high versus low in global QOL (P.01) and four (pain, fatigue, physical/functional ability, and general QOL) differentiated patients who perceived recent changes in their physical health (improvement vs decline; P.05).These results provide preliminary psychometric data for a pilot measure of CAM outcomes, though further testing is warranted.

We tested the reliability and validity of a brief symptom index for use with patients in the advanced stages of lung cancer. The Functional Assessment of Cancer Therapy--Lung Symptom Index-12 (FLSI-12) is a brief self-report measure that combines seven items addressing symptoms common in advanced-stage lung cancer with five symptoms or concerns that are relevant to most people with advanced-stage cancer. The index was administered prospectively to 92 advanced-stage lung cancer patients beginning at the initiation of chemotherapy and for 12 consecutive weeks. Reliability, convergent and concurrent validities, and responsiveness to change were determined and a minimally important difference (MID) was estimated. The index had good internal consistency (all Cronbach's alpha's>0.70), moderate to high item-to-total correlations (93% rho's> or =0.30), and correlated highly with a measure of overall quality of life (rho's> or =0.50). Baseline scores differentiated patients with better versus worse clinical features (p's

Background: To test the reliability, sensitivity to change in biomarkers associated with disease progression and response to treatment, and clinical meaningfulness of the Functional Assessment of Cancer Therapy-Hepatobiliary (FACT-Hep) in patients with hepatobiliary carcinoma. Patients and methods: One hundred and fifty-eight patients diagnosed with hepatobiliary carcinoma were prospectively studied. Health-related quality of life (HRQL) was assessed at baseline (prior to treatment), 3-month follow-up (n = 55) and 6-month follow-up (n = 27). Results: The internal consistency of all the scales of the FACT-Hep were adequate at all time points (>0.75). The FACT-Hep was found to be sensitive to changes in clinical indicators (alkaline phosphate, alpha-fetoprotein, hemoglobin and survival) that reflect disease progression and response to treatment. Combined results from distribution-based and cross-sectional anchor-based analyses provide the following minimally important difference (MID) estimates: FACT-General (FACT-G) subscales = 2–3; FACT-G = 6–7; Hepatobiliary Cancer Subscale = 5–6; FACT-Hep = 8–9; Trial Outcome Index = 7–8; and FACT-Hepatobiliary Symptom Index = 2–3 points. Conclusions: The FACT-Hep is a reliable instrument that is responsive to clinical indicators of disease progression and response to treatment. The MID estimates can aid interpretation of HRQL data and facilitate sample size

The authors examined whether 3 individual difference variables--self-esteem, self-efficacy, and depressive symptoms--interacted with psychoeducational group interventions for men with prostate cancer (n = 250) to predict general and prostate-specific quality of life. Men with nonmetastatic prostate cancer were randomly assigned to an education intervention, an education plus group discussion intervention, or usual care and followed for 12 months. Eight groups of 10 to 12 men were convened in each condition. Men who began the study with lower self-esteem, lower prostate-specific self-efficacy, and higher depressive symptoms benefited the most from the interventions. Of these 3 moderator variables, the most consistent results emerged for self-esteem. That is, the benefits of the intervention were strongest for men with low self-esteem.

BACKGROUND. To develop and validate a quality of life subscale for patients with esophageal cancer to be used with the Functional Assessment of Cancer Therapy—General (FACT-G). METHODS. Prospective cohort study of patients with esophageal cancer treated with surgery alone or neoadjuvant chemoradiotherapy and surgery evaluating the validity, internal consistency, and responsiveness to change of the FACT-Esophageal (FACT-E) when comparing it with the European Organization for the Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ 30) and esophageal (OES 24) as well as clinical factors. RESULTS. The FACT-E demonstrated very good convergent and divergent validity when compared with the EORTC QLQ30 and OES 24 and clinical variables. Internal consistency was also good with coefficient α >0.70 for all subscales and individual items. Stability coefficients were >0.80. Changes in clinical status were reflected in changes in FACT-E scores demonstrating responsiveness to change, particularly in patients receiving neoadjuvant chemoradiotherapy before surgery. CONCLUSIONS. The FACT-E met or exceeded all standards for validity, providing an option to measure health-related quality of life for different treatment strategies for esophageal cancer. Cancer 2006. © 2006 American Cancer Society.

Objective To estimate minimally important differences (MIDs) on the Functional Assessment of Cancer Therapy–Colorectal (FACT-C) instrument using anchor- and distribution-based methods. Study Design and Setting Preliminary MIDs were generated for FACT-C scores based on published results for two samples (n = 60 and n = 63) from the FACT-C validation study. Preliminary MIDs were confirmed using data from a Phase II randomized controlled clinical trial (n = 104) and a population-based observational study (n = 568). MIDs were estimated for the colorectal cancer subscale (CCS); the FACT-C Trial Outcome Index (TOI-C), which is the sum of the CCS, physical well-being, and functional well-being subscales; and the FACT-C total score. Both cross-sectional and longitudinal analyses were used. Results MIDs were stable across the different patient samples. The recommended MIDs ranged from 2 to 3 points for the CCS, 4 to 6 points for the TOI-C, and 5 to 8 points for the FACT-C total score. Conclusions MIDs can enhance the interpretability of FACT-C scores, and they can be used to provide a basis for sample size estimation and to determine clinical benefit in combination with other measures of efficacy. General guidelines for estimating MIDs for other FACT instruments are suggested.

Health-related quality of life (HRQOL) is an important endpoint in cancer clinical trials and in cancer treatment in general; however, the meaningfulness of HRQOL scores may not be apparent to clinicians or researchers. Minimally important differences (MIDs) can enhance the interpretability of HRQOL scores by identifying differences likely to be meaningful to patients and clinicians. This article’s objective is to describe and provide examples of approaches we have used to identify MIDs for instruments in the Functional Assessment of Chronic Illness Therapy (FACIT) measurement system. Distribution- and anchor-based approaches are described and illustrated. We also discuss the importance of assessing the appropriateness of anchors, and we provide suggestions for combining results into a single range of plausible MIDs. MIDs for FACIT instruments established to date are summarized, and general guidelines that can be used to estimate MIDs for other FACIT instruments are provided. Applications of MIDs in research are illustrated.

We assessed the reliability, validity, and responsiveness to change of the 6-item Functional Assessment of Cancer Therapy-Breast Symptom Index (FBSI) in a sample of 615 metastatic breast cancer patients. The FBSI is a brief, clinically relevant, and psychometrically sound instrument that can be used to measure symptoms in patients with breast cancer.

This article discusses quality of life in lung cancer: the validity and cross-cultural applicability of the Functional Assessment of Cancer Therapy-Lung scale.

To determine distribution- and anchor-based minimal important difference (MID) estimates for four scores from the Functional Assessment of Cancer Therapy-Breast (FACT-B): the breast cancer subscale (BCS), Trial Outcome Index (TOI), FACT-G (the general version), and FACT-B.We used data from a Phase III clinical trial in metastatic breast cancer (ECOG study 1193; n=739) and a prospective observational study of pain in metastatic breast cancer (n=129). One third and one half of the standard deviation and 1 standard error of measurement were used as distribution-based criteria. Clinical indicators used to determine anchor-based differences included ECOG performance status, current pain, and response to treatment.FACT-B scores were responsive to performance status and pain anchors, but not to treatment response. By combining the results of distribution- and anchor-based methods, MID estimates were obtained: BCS=2-3 points, TOI=5-6 points, FACT-G=5-6 points, and FACT-B=7-8 points.Distribution- and anchor-based estimates of the MID do show convergence. These estimates can be used in combination with other measures of efficacy to determine meaningful benefit and provide a basis for sample size estimation in clinical trials.

Purpose: To determine the ability of longitudinal patient-reported health (PRH) scores to enhance prediction of clinical outcomes beyond baseline scores. Patients and Methods: In 573 advanced non–small-cell lung cancer patients enrolled in a phase III clinical trial, we used baseline and 6-week follow-up PRH scores to predict best response to treatment, disease progression, and survival. Using regression analyses, we tested the predictive ability of the five subscales of the Functional Assessment of Cancer Therapy–Lung (physical, functional, social/family, emotional well-being, and the lung cancer subscale) as well as the trial outcome index (TOI) aggregate score. Results: After clinical factors were controlled for, baseline physical well-being (PWB) and TOI scores predicted all three clinical outcomes. A higher baseline PWB score was associated with a better response to treatment (odds ratio, 1.09; P < .001) and lower risk of death (risk ratio, 0.95; P < .001). Higher baseline TOI score was associated with a lower risk of disease progression (risk ratio, 0.98; P < .001). These two baseline predictors (PWB and TOI) were then used along with 6-week change scores to classify patients into four groups: low baseline-declined, low baseline-improved, high baseline-declined, and high baseline-improved. Patients with low baseline-declined PWB scores showed the worst responses to treatment and survived the shortest duration. Patients with low baseline-declined TOI scores had the shortest time to progression. Conclusion: The physical aspects of baseline PRH and PRH change during chemotherapy are significant predictors of clinical outcomes in lung cancer. This has implications for patient stratification in clinical trials and may aid decision-making in clinical practice.

With the established effectiveness of diverse treatments for prostate cancer, identification of the physical and psychosocial consequences of the disease and various treatments becomes critical. We review the literature on the effects of prostate cancer and its treatment on health-related quality of life (HRQoL). Studies show that prostate cancer and its treatment affect both disease-specific HRQoL (i.e. urinary, sexual, and bowel function) as well as general HRQoL (i.e. energy/vitality, performance in physical and social roles). Yet, these effects appear to differ across stage of disease and type of treatment. We outline evidence from three sources: (1) studies that compare men with the disease with an age-matched sample of men without the disease, (2) studies that assess men with the disease across time, and (3) cross-sectional studies that highlight predictors of HRQoL. Future research directions are discussed.

Background A compelling ethical rationale supports patient engagement in healthcare research. It is also assumed that patient engagement will lead to research findings that are more pertinent to patients’ concerns and dilemmas. However; it is unclear how to best conduct this process. In this systematic review we aimed to answer 4 key questions: what are the best ways to identify patient representatives? How to engage them in designing and conducting research? What are the observed benefits of patient engagement? What are the harms and barriers of patient engagement? Methods We searched MEDLINE, EMBASE, PsycInfo, Cochrane, EBSCO, CINAHL, SCOPUS, Web of Science, Business Search Premier, Academic Search Premier and Google Scholar. Included studies were published in English, of any size or design that described engaging patients or their surrogates in research design. We conducted an environmental scan of the grey literature and consulted with experts and patients. Data were analyzed using a non-quantitative, meta-narrative approach. Results We included 142 studies that described a spectrum of engagement. In general, engagement was feasible in most settings and most commonly done in the beginning of research (agenda setting and protocol development) and less commonly during the execution and translation of research. We found no comparative analytic studies to recommend a particular method. Patient engagement increased study enrollment rates and aided researchers in securing funding, designing study protocols and choosing relevant outcomes. The most commonly cited challenges were related to logistics (extra time and funding needed for engagement) and to an overarching worry of a tokenistic engagement. Conclusions Patient engagement in healthcare research is likely feasible in many settings. However, this engagement comes at a cost and can become tokenistic. Research dedicated to identifying the best methods to achieve engagement is lacking and clearly needed.

To determine the association between patient assessment of healthcare delivery and publicly reported asthma performance measures.We identified individuals 5-50 years old who had asthma quality measures reported to the Minnesota Community Measurement© (MCM©) by their clinic and sent them a postal survey which included within it the Patient Assessment of Chronic Illness care (PACIC), a validated measure of patient perception of the quality of healthcare delivery. We performed a multivariable analysis to examine the association between PACIC scores and achievement of asthma care quality measures.The response rate for the ACS was 102/367 (28%); a non-response bias analysis revealed no differences between responders and non-responders for age, gender and asthma control. Most responders (73%) reported taking asthma medications daily and most (71%) had poorly controlled asthma. The PACIC score was not associated with any of the asthma quality measures based on the data reported to MCM© by the clinic. A higher PACIC score was, however, associated with having an asthma action plan based on patient-reported data in the ACS (p 0.0001) but not with patient-reported asthma control or emergency department/hospitalizations for asthma.Patient assessment of high quality asthma care delivery was associated with patient self-report of having an asthma action plan but was not associated with any of the publicly reported asthma performance measures.

In a systematic review of qualitative research, Katie Gallacher and colleagues examine the evidence related to treatment burden after stroke from the patient perspective. Please see later in the article for the Editors' Summary, Background Patients with chronic disease may experience complicated management plans requiring significant personal investment. This has been termed ‘treatment burden’ and has been associated with unfavourable outcomes. The aim of this systematic review is to examine the qualitative literature on treatment burden in stroke from the patient perspective. Methods and Findings The search strategy centred on: stroke, treatment burden, patient experience, and qualitative methods. We searched: Scopus, CINAHL, Embase, Medline, and PsycINFO. We tracked references, footnotes, and citations. Restrictions included: English language, date of publication January 2000 until February 2013. Two reviewers independently carried out the following: paper screening, data extraction, and data analysis. Data were analysed using framework synthesis, as informed by Normalization Process Theory. Sixty-nine papers were included. Treatment burden includes: (1) making sense of stroke management and planning care, (2) interacting with others, (3) enacting management strategies, and (4) reflecting on management. Health care is fragmented, with poor communication between patient and health care providers. Patients report inadequate information provision. Inpatient care is unsatisfactory, with a perceived lack of empathy from professionals and a shortage of stimulating activities on the ward. Discharge services are poorly coordinated, and accessing health and social care in the community is difficult. The study has potential limitations because it was restricted to studies published in English only and data from low-income countries were scarce. Conclusions Stroke management is extremely demanding for patients, and treatment burden is influenced by micro and macro organisation of health services. Knowledge deficits mean patients are ill equipped to organise their care and develop coping strategies, making adherence less likely. There is a need to transform the approach to care provision so that services are configured to prioritise patient needs rather than those of health care systems. Systematic Review Registration International Prospective Register of Systematic Reviews CRD42011001123 Please see later in the article for the Editors' Summary, Editors' Summary Background Every year, 15 million people have a stroke. About 5 million of these people die within a few days, and another 5 million are left disabled. Stroke occurs when the blood supply of the brain is suddenly interrupted by a blood vessel in the brain being blocked by a blood clot (ischemic stroke) or bursting (hemorrhagic stroke). Deprived of the oxygen normally carried to them by the blood, the brain cells near the blockage die. The symptoms of stroke depend on which part of the brain is damaged but include sudden weakness or paralysis along one side of the body, vision loss in one or both eyes, and confusion or trouble speaking or understanding speech. Anyone experiencing these symptoms should seek immediate medical attention because prompt treatment can limit the damage to the brain. In the longer term, post-stroke rehabilitation can help individuals overcome the physical disabilities caused by stroke, and drugs that thin the blood, reduce blood pressure and reduce cholesterol (major risk factors for stroke) alongside behavioral counseling can reduce the risk of a second stroke. Why Was This Study Done? Treatment for, and rehabilitation from, stroke is a lengthy process that requires considerable personal investment from the patient. The term “treatment burden” describes the self-care practices that patients with stroke and other chronic diseases must perform to follow the complicated management strategies that have been developed for these conditions. Unfortunately, treatment burden can overwhelm patients. They may be unable to cope with the multiple demands placed on them by health-care providers and systems for their self-care, a situation that leads to poor adherence to therapies and poor outcomes. For example, patients may find it hard to complete all the exercises designed to help them regain full movement of their limbs after a stroke. Treatment burden has been poorly examined in relation to stroke. Here, the researchers identify and describe the treatment burden in stroke by undertaking a systematic review (a study that uses predefined criteria to identify all the literature on a given topic) of qualitative studies on the patient experience of stroke management. Qualitative studies collect non-quantitative data so, for example, a qualitative study on stroke treatment might ask people how the treatment made them feel whereas a quantitative study might compare clinical outcomes between those receiving and not receiving the treatment. What Did the Researchers Do and Find? The researchers identified 69 qualitative studies dealing with the experiences of stroke management of adult patients and analyzed the data in these papers using framework synthesis—an approach that divides data into thematic categories. Specifically, the researchers used a coding framework informed by normalization process theory, a sociological theory of the implementation, embedding and integration of tasks and practices; embedding is the process of making tasks and practices a routine part of everyday life and integration refers to sustaining these embedded practices. The researchers identified four main areas of treatment burden for stroke: making sense of stroke management and planning care; interacting with others, including health care professionals, family and other patients with stroke; enacting management strategies (including enduring institutional admissions, managing stroke in the community, reintegrating into society and adjusting to life after stroke); and reflecting on management to make decisions about self-care. Moreover, they identified problems in all these areas, including inadequate provision of information, poor communication with health-care providers, and unsatisfactory inpatient care. What Do These Findings Mean? These findings show that stroke management is extremely demanding for patients and is influenced by both the micro and macro organization of health services. At the micro organizational level, fragmented care and poor communication between patients and clinicians and between health-care providers can mean patients are ill equipped to organize their care and develop coping strategies, which makes adherence to management strategies less likely. At the macro organizational level, it can be hard for patients to obtain the practical and financial help they need to manage their stroke in the community. Overall, these findings suggest that care provision for stroke needs to be transformed so that the needs of patients rather than the needs of health-care systems are prioritized. Further work is required, however, to understand how the patient experience of treatment burden is affected by the clinical characteristics of stroke, by disability level, and by other co-existing diseases. By undertaking such work, it should be possible to generate a patient-reported outcome measure of treatment burden that, if used by policy makers and health-care providers, has the potential to improve the quality of stroke care. Additional Information Please access these Web sites via the online version of this summary at http://dx.doi.org/10.1371/journal.pmed.1001473. The US National Institute of Neurological Disorders and Stroke provides information about all aspects of stroke (in English and Spanish); its Know Stroke site provides educational materials about stroke prevention, treatment, and rehabilitation including personal stories (in English and Spanish); the US National Institutes of Health SeniorHealth website has additional information about stroke The Internet Stroke Center provides detailed information about stroke for patients, families, and health professionals (in English and Spanish) The UK National Health Service Choices website also provides information about stroke for patients and their families, including personal stories MedlinePlus has links to additional resources about stroke (in English and Spanish) The UK not-for-profit website Healthtalkonline provides personal stories about stroke Wikipedia provides information on the burden of treatment and on the normalization process theory (note: Wikipedia is a free online encyclopedia that anyone can edit; available in several languages)

Quality of life (QoL) is an important outcome in patients with non-Hodgkin lymphoma (NHL). We assessed the validity of administering the Functional Assessment of Cancer Therapy - General (FACT-G) at 12-month intervals over 3 years in a longitudinal study of 611 prospectively enrolled, newly diagnosed patients with NHL. We evaluated corrected item-total correlation and percent missing to identify items that may be less useful in certain NHL patient subgroups. The FACT-G subscales and total score demonstrated good internal consistency reliability, convergent validity and known-groups validity. Most scores also demonstrated good responsiveness to change. Questions that could be problematic included GE3 (losing hope) and GP2 (nausea) for patients in remission, and GP5 (bothered by side effects) for patients being observed. Overall, the FACT-G was a valid measure for monitoring QoL over time in patients with NHL. However, sensitivity analyses based on subscale scoring that excludes potentially problematic items may be warranted.

Background Patients experience an increasing treatment burden related to everything they do to take care of their health: visits to the doctor, medical tests, treatment management and lifestyle changes. This treatment burden could affect treatment adherence, quality of life and outcomes. We aimed to develop and validate an instrument for measuring treatment burden for patients with multiple chronic conditions. Methods Items were derived from a literature review and qualitative semistructured interviews with patients. The instrument was then validated in a sample of patients with chronic conditions recruited in hospitals and general practitioner clinics in France. Factor analysis was used to examine the questionnaire structure. Construct validity was studied by the relationships between the instrument's global score, the Treatment Satisfaction Questionnaire for Medication (TSQM) scores and the complexity of treatment as assessed by patients and physicians. Agreement between patients and physicians was appraised. Reliability was determined by a test-retest method. Results A sample of 502 patients completed the Treatment Burden Questionnaire (TBQ), which consisted of 7 items (2 of which had 4 subitems) defined after 22 interviews with patients. The questionnaire showed a unidimensional structure. The Cronbach's α was 0.89. The instrument's global score was negatively correlated with TSQM scores (rs = -0.41 to -0.53) and positively correlated with the complexity of treatment (rs = 0.16 to 0.40). Agreement between patients and physicians (n = 396) was weak (intraclass correlation coefficient 0.38 (95% confidence interval 0.29 to 0.47)). Reliability of the retest (n = 211 patients) was 0.76 (0.67 to 0.83). Conclusions This study provides the first valid and reliable instrument assessing the treatment burden for patients across any disease or treatment context. This instrument could help in the development of treatment strategies that are both efficient and acceptable for patients.

Background Early mitral valve (MV) repair of degenerative mitral regurgitation is associated with superior clinical outcomes compared with prosthetic replacement and restores normal life expectancy, even in those without symptoms. Although current guidelines recommend prompt referral for effective MV repair in those with severe mitral regurgitation, some are reluctant to pursue early correction due to the perception that short-term quality of life (QOL) may be adversely affected by the operation. Methods Between January 2008 and November 2009, 202 patients underwent conventional transsternotomy or minimally invasive port-access robot-assisted MV repair, with or without patent foramen ovale closure or left Maze, and were mailed a postsurgical QOL survey. Results Unadjusted QOL scores for patients undergoing MV repair were excellent early after the operation using both approaches. Robotic repair was associated with slightly improved scores on the Duke Activity Status Index, the Short Form-12 Item Health Survey Physical domain, and the Linear Analogue Self-Assessment frequency of chest pain and fatigue indices during the first postoperative year; however, differences between treatment groups became indistinguishable after 1 year. Robotic repair patients returned to work slightly quicker (median, 33 vs 54 days, p Conclusions Functional QOL outcomes within the first 2 years after early MV repair are excellent using open and robotic platforms. A robotic approach may be associated with slightly improved early QOL and return to employment-based activities. These results may have implications regarding future evolution of clinical guidelines and economic health care policy.

Patient-reported outcome (PRO) measures are frequently used to assess therapeutic efficacy and effectiveness of complementary and alternative medicine (CAM). Although CAM investigators are increasingly making use of valid, self-report instruments to assess patient-relevant outcomes, the sheer number of available instruments poses challenges. Two of the more pressing issues are discussed: the lack of guidance for selecting measures and the limited attention paid to the clinical meaningfulness of PRO results. Solutions are offered that may support selection and standardization of outcome measures for future CAM studies. This includes testing of tools from the National Institutes of Health's Patient-Reported Outcomes Measurement Information System (PROMIS). Increased standardization of outcome measurement in CAM studies will allow for more cross-study comparisons and facilitate the statistical pooling of results, ultimately leading to a more informative evidence base.

Interest in the efficacy and potential deleterious consequences of treatment with erythropoietic-stimulating agents (ESAs) is very high. Recently, the ESAs have come under intense scrutiny as several clinical trials have shown their use to be associated with an increased risk of thrombosis, and a concern for increased mortality risk in oncology. In this context, attention to the effect of ESAs upon fatigue and other aspects of quality of life has tended to be lost. To aid inclusion of this endpoint in the important consideration of risks and benefits of ESA therapy, we summarize the many reviews that have been conducted on this topic. The ten selected reviews were all conducted systematically or were otherwise comprehensive. While these reviews acknowledge an overall positive fatigue or quality-of-life effect, some were equivocal about the meaningfulness or magnitude of the benefit. The overall evidence from these reviews supports a fatigue and overall quality-of-life benefit from treatment with ESAs that is unlikely to be due to chance. This information should be included in the risk/benefit consideration of these controversial agents.

PURPOSE Our objective was to identify which symptoms of advanced lung cancer are most likely to change with objective tumor measurements (progressions and responses) or changes in performance status (PS). PATIENTS AND METHODS Eighty patients with advanced non–small-cell lung cancer were studied during the first 12 weeks of chemotherapy. Symptoms were assessed weekly through telephone administration of the Functional Assessment of Cancer Therapy–Lung Symptom Index-12. Data on PS were collected from patients every 3 weeks. Symptom reports were mapped onto clinical events (progression or response as determined by clinicians) and PS assessments. RESULTS Disease progression and declining PS were associated with worsening of several symptoms. Pain, shortness of breath, cough, weight loss, and appetite loss worsened most from before to after progression. Patients with an objective response to chemotherapy reported more fatigue and difficulty breathing at response than before response. However, unlike patients who experienced progression, patients responding to chemotherapy never or rarely complained of clinically significant pain, weight loss, cough, chest tightness, nausea, or confusion before, during, or after response. With the exception of bother with side effects of treatment, confusion, and difficulty breathing, symptoms tracked fairly closely over the 12 weeks with changes in PS. Declining PS was associated with considerably more symptom worsening than unchanged or improved PS, independent of treatment response. CONCLUSION These data can help the clinician identify symptoms of lung cancer most reliably associated with objective responses and perceived changes in functional status during chemotherapy. Symptom self-reports could be used by clinicians to monitor patient status and possibly inform treatment modification.

Ian Thompson (Chair),* James Brantley Thrasher (Co-Chair),† Gunnar Aus,‡ Arthur L. Burnett,§ Edith D. Canby-Hagino, Michael S. Cookson,¶ Anthony V. D’Amico, Roger R. Dmochowski, David T. Eton, Jeffrey D. Forman, S. Larry Goldenberg, Javier Hernandez, Celestia S. Higano, Stephen R. Kraus,** Judd W. Moul†† and Catherine M. Tangen (Prostate Cancer Clinical Guideline Update Panel) From the American Urological Association Education and Research, Inc.

In conjunction with the assignment to update the Guidelines for Management of Clinically Localized Prostate Cancer, the American Urological Association Prostate Cancer Guideline Update Panel performed a side analysis of the reporting of erectile function outcomes in this clinical context as published in the medical literature.Four National Library of Medicine PubMed(R) Services literature searches targeting articles published from 1991 through early 2004 were done to derive outcome reporting (efficacy or side effects) for the treatment of clinical stage T1 or T2 N0M0 prostate cancer. A database was constructed containing descriptions relating to erectile function as well as numerical frequency rates of complete erectile dysfunction, and partial and intact erectile function for various treatments. A literature review was also done, consisting of a PubMed Services search of current measures and protocols used for assessing erectile function outcomes and a survey of consensus opinion sources on the management of male sexual dysfunctions.Based on inclusion criteria 436 articles were selected. Of these articles database extraction from 100 pertaining to radical prostatectomy garnered various characterizations of erectile function, including qualitative descriptions, generic terminology and rating systems. Database extraction from 31 articles, in which results for at least 50 patients were reported, yielded ranges of rates for complete erectile dysfunction, partial erectile function and intact erectile function that were 26% to 100%, 16% to 48% and 9% to 86% for radical prostatectomy, 8% to 85%, 21% to 47% and 36% to 63% for external beam radiation, and 14% to 61%, 21% and 18% for interstitial radiation, respectively. The literature review showed an evolution in standards for studying and reporting erectile function outcomes.Clinical studies reporting erectile function outcomes after localized prostate cancer treatment often demonstrate poorly interpretable and inconsistent manners of assessment as well as widely disparate rates of erectile dysfunction and erectile function. Future studies must apply scientifically rigorous methodology and standard outcomes measures to advance this field of study.

The American Urological Association Prostate Guideline Update Panel was charged with updating the Guidelines for Clinically Localized Prostate Cancer. In assessing outcomes with treatment, it became apparent that a highly variable number of definitions exist with respect to biochemical recurrence. Herein, we review the variability in published definitions of biochemical recurrence and make recommendations directed toward improving this terminology by recommending a standard definition in patients treated with radical prostatectomy.Four PubMed literature searches were performed between May 2001 and April, 2004 and covered articles published from 1991 through early 2004. The search terms included the MeSH major headings of prostate cancer and prostatic neoplasm. All potentially relevant articles were retrieved and a more detailed screen for relevance was performed. An article was considered relevant if it reported treatment outcomes of patients with clinical T1 or T2N0M0 prostate cancer. Data extractors recorded the definition of biochemical recurrence and definitions were then collapsed into categories representing the same criteria. The results of biochemical failure were subcategorized by initial treatment.Of 13,800 citations, a total of 436 articles were selected. Among these, a total of 145 articles contained 53 different definitions of biochemical recurrence for those treated with radical prostatectomy. Of these, the most common definition (35) was a prostate specific antigen of0.2 ng/mL or a slight variation thereof. In addition, a total of 208 articles reported 99 different definitions of biochemical failure among those treated with radiation therapy. Of these, the American Society for Therapeutic Radiology and Oncology definition (70) and/or a variation thereof was the most commonly reported. In total, 166 different definitions of biochemical failure were identified. Following radical prostatectomy, the Panel recommends defining biochemical recurrence as an initial serum prostate specific antigen ofor =0.2 ng/mL, with a second confirmatory level of prostate specific antigen of0.2 ng/mL. The Panel recommends the use of the American Society for Therapeutic Radiology and Oncology criteria for patients treated with radiation therapy and acknowledges that these criteria will soon be updated although not yet published.A high degree of variability in the definition of biochemical recurrence exists following treatment for localized prostate cancer. Strict definitions for biochemical recurrence are necessary to identify men at risk for disease progression and to allow meaningful comparisons among patients treated similarly. The Panel acknowledges the American Society for Therapeutic Radiology and Oncology criteria and future modifications thereof for those receiving radiation therapy and recommends the newly developed American Urological Association criteria for those treated with radical prostatectomy. The purpose for the establishment of this standard is for data reporting purposes and for comparison of similarly treated patients. It is not intended to represent a threshold value for which to initiate treatment. The Panel acknowledges that the clinical decision to initiate treatment will be dependent on multiple factors including patient and physician interaction rather than a specific prostate specific antigen threshold value.

Recent advances in cancer cell biology have led to the development of therapeutic agents that target pathways critical to the development and progression of disease. These so-called “targeted therapies” might offer patients a more tolerable alternative to traditional systemic chemotherapy that often achieves therapeutic benefit at the cost of debilitating side effects. Several targeted agents have been recently tested in clinical trials of advanced-stage lung cancer. As interest in these therapies grows, an understanding of their impact on the patient's well-being will be important. Patient-reported outcome measures such as formal assessments of health-related quality of life and disease symptoms provide a useful means for addressing the impact of therapy from the perspective of the patient. We summarize the most commonly used measures of health-related quality of life in clinical trials of advanced-stage lung cancer. Although existing measurement systems do provide adequate coverage of many important patient-related outcome issues, the advent of targeted therapies in advanced-stage lung cancer does expose several measurement gaps. We highlight some of these gaps by reviewing a representative sample of recently conducted clinical trials of targeted lung cancer therapy and offer insight as to how these gaps can be filled. We also briefly discuss a set of issues unique to patients receiving targeted therapy (eg, perceptions of treatment efficacy, treatment compliance, patient satisfaction, and treatment convenience) and consider a few methodologic concerns unique to patients with advanced cancer.

Objective Our objective was to develop a pilot measure of subjective outcomes of complementary and alternative medicine (CAM). Current options for assessing subjective outcomes in CAM are either too burdensome or fail to represent diverse outcomes. A single measure specifying common, patient-reported outcomes of CAM would be of value. Design We conducted a three-phase instrument development study. In phase I, 30 CAM-receiving patients and 12 CAM practitioners completed a battery of standardized measures. Participants identified those standardized items relevant to CAM outcome and suggested additional outcome issues. In phase II, 20 CAM-receiving patients completed a Q-sort to determine which items from phase I were most relevant to CAM outcome. In phase III, five experts reviewed the items from phase II for content validity. Setting An integrative medicine clinic at a private, Midwest US hospital. Participants CAM patients, practitioners, and researchers and an outcomes measurement specialist. Results In phase I, 30 standardized items were judged relevant to CAM outcome, and 8 additional items were suggested. In phase II, 29 of the 38 phase I items were deemed at least "moderately relevant" to CAM outcome. In phase III, experts added 15 items, dropped 9 items, and altered 3 items. The 35 resulting items were classified into content domains, forming a testable, pilot version of the instrument.

BACKGROUND The authors examined levels and predictors of psychological distress in the wives of men treated for early-stage prostate carcinoma (PCa). METHODS Patients with PCa (N = 165) and spouses were interviewed to assess general and cancer-specific distress. Social and intrapersonal factors of spouses as well as clinical characteristics and quality of life of patients were assessed as potential predictors of spouses' distress. RESULTS Spouses reported more cancer-specific distress than did patients (P < 0.001), but did not differ from patients in general distress. Several spouse-reported factors predicted higher spouses' distress, including less education (P < 0.005), worse marriage quality and less social support (Ps < 0.005), more negative social interaction with the patient (Ps < 0.001), lower self-esteem (Ps < 0.001), less positive coping (Ps < 0.005), searching for meaning (P < 0.001), not finding meaning (P < 0.005), and greater illness uncertainty (Ps < 0.001). Patients' bowel function and mental health also predicted greater spouses' distress (Ps < 0.005). CONCLUSIONS The findings indicated that overall distress in spouses of early-stage patients with PCa was modest, and it was more likely to be predicted by psychosocial than medical factors. Cancer 2005. © 2005 American Cancer Society.

Assessing bowel function (BF) in prostate cancer can help determine therapeutic trade-offs. We determined the components of BF commonly assessed in prostate cancer studies as an initial step in creating an item bank for clinical and research application. We analyzed six archived data sets representing 4,246 men with prostate cancer. Thirty-one items from validated instruments were available for analysis. Items were classified into domains (diarrhea, rectal urgency, pain, bleeding, bother/distress, and other) then subjected to conventional psychometric and item response theory (IRT) analyses. Items fit the IRT model if the ratio between observed and expected item variance was between 0.60 and 1.40. Four of 31 items had inadequate fit in at least one analysis. Poorly fitting items included bleeding (2), rectal urgency (1), and bother/distress (1). A fifth item assessing hemorrhoids was poorly correlated with other items. Our analyses supported four related components of BF: diarrhea, rectal urgency, pain, and bother/distress.

Men who were recently treated for prostate cancer (N = 250) were randomly assigned to a control group, a group education intervention (GE), or a group education-plus-discussion intervention (GED). Both GE and GED increased prostate cancer knowledge. In the year postintervention, men in the GED condition were less bothered by sexual problems than men in the control condition, and they were more likely to remain steadily employed (93.0%) than men in the GE (75.6%) or control (72.5%) conditions. Among noncollege graduates, GED and GE resulted in better physical functioning than the control condition, and GED resulted in more positive health behaviors than the control or GE condition. Among college graduates, controls were comparable with the GE and GED groups in physical functioning and positive health behaviors.

Magnitude differences in scores on a measure of quality of life that correspond to differences in function or clinical course are called clinically important differences (CIDs). Anchor-based and distribution-based methods were used to provide ranges of CIDs for five targeted scale scores of the Functional Assessment of Cancer Therapy-Anemia (FACT-An) questionnaire. Three samples of cancer patients were used: Sample 1 included 50 patients participating in a validation study of the FACT-An; Sample 2 included 131 patients participating in a longitudinal study of chemotherapy-induced fatigue; sample 3 included 2,402 patients enrolled in a community-based clinical trial evaluating the effectiveness and safety of a treatment for anemia. Three clinical indicators (hemoglobin level; performance status; response to treatment) were used to determine anchor-based differences. One-half of the standard deviation and 1 standard error of measurement were used as distribution-based criteria. Analyses supported the following whole number estimates of a minimal CID for these five targeted scores: Fatigue Scale = 3.0; FACT-G total score = 4.0; FACT-An total score = 7.0; Trial Outcome Index-Fatigue = 5.0; and Trial Outcome Index-Anemia = 6.0. These estimates provide a basis for sample size estimation when planning for a clinical trial or other longitudinal study, when the purpose is to ensure detection of meaningful change over time. They can also be used in conjunction with more traditional clinical markers to assist investigators in determining treatment efficacy.