Predicting Survival in Patients With Pulmonary Arterial Hypertension: The REVEAL Risk Score Calculator 2.0 and Comparison With ESC/ERS-Based Risk Assessment Strategies.

BACKGROUND: Pulmonary arterial hypertension is a progressive, fatal disease. Published treatment guidelines recommend treatment escalation on the basis of regular patient assessment with the goal of achieving or maintaining low-risk status. Various strategies are available to determine risk status. This analysis describes an update of the Registry to Evaluate Early and Long-Term PAH Disease Management (REVEAL) risk calculator (REVEAL 2.0) and compares it with recently published European Society of Cardiology/Respiratory Society guideline-derived risk assessment strategies. METHODS: A subpopulation from the US-based registry REVEAL that survived ≥ 1 year postenrollment (baseline for this cohort) was analyzed. For REVEAL 2.0, point values and cutpoints were reassessed, and new variables were evaluated. The Kaplan-Meier method was used to estimate survival at 12 months postbaseline; discrimination was quantified using the c-statistic. Mortality estimates and discrimination were compared between REVEAL 2.0 and Comparative, Prospective Registry of Newly Initiated Therapies for Pulmonary Hypertension (COMPERA) and French Pulmonary Hypertension Registry (FPHR) risk assessment strategies. For this comparison, a three-category REVEAL 2.0 score was computed in which patients were classified as low-, intermediate-, or high-risk. RESULTS: REVEAL 2.0 demonstrated similar discrimination as the original calculator in this subpopulation (c-statistic = 0.76 vs 0.74), provided excellent separation of risk among the risk categories, and predicted clinical worsening as well as mortality in patients who were followed ≥ 1 year. The REVEAL 2.0 three-category score had greater discrimination (c-statistic = 0.73) than COMPERA (c-statistic = 0.62) or FPHR (c-statistic = 0.64). Compared with REVEAL 2.0, COMPERA and FPHR both underestimated and overestimated risk. CONCLUSIONS: REVEAL 2.0 demonstrates greater risk discrimination than the COMPERA and FPHR risk assessment strategies in patients enrolled in REVEAL. After external validation, the REVEAL 2.0 calculator can assist clinicians and patients in making informed treatment decisions on the basis of individual risk profiles. TRIAL REGISTRY: ClinicalTrials.gov; No. NCT00370214; URL: www.clinicaltrials.gov.

Medical Expenditures and Earnings Losses Among US Adults With Arthritis in 2013.

OBJECTIVE: We estimated the economic impact of arthritis using 2013 US Medical Expenditure Panel Survey (MEPS) data. METHODS: We calculated arthritis-attributable and all-cause medical expenditures for adults age ≥18 years and arthritis-attributable earnings losses among those ages 18-64 years who had ever worked. We calculated arthritis-attributable costs using multistage regression-based methods, and conducted sensitivity analyses to estimate costs for 2 other arthritis definitions in MEPS. RESULTS: In 2013, estimated total national arthritis-attributable medical expenditures were $139.8 billion (range $135.9-$157.5 billion). Across expenditure categories, ambulatory care expenditures accounted for nearly half of arthritis-attributable expenditures. All-cause expenditures among adults with arthritis represented 50% of the $1.2 trillion national medical expenditures among all US adults in MEPS. Estimated total national arthritis-attributable earning losses were $163.7 billion (range $163.7-$170.0 billion). The percentage with arthritis who worked in the past year was 7.2 percentage points lower than those without arthritis (76.8% [95% confidence interval (95% CI)] 75.0-78.6 and 84.0% [95% CI 82.5-85.5], respectively, adjusted for sociodemographics and chronic conditions). Total arthritis-attributable medical expenditures and earnings losses were $303.5 billion (range $303.5-$326.9 billion). CONCLUSION: Total national arthritis-attributable medical care expenditures and earnings losses among adults with arthritis were $303.5 billion in 2013. High arthritis-attributable medical expenditures might be reduced by greater efforts to reduce pain and improve function. The high earnings losses were largely attributable to the substantially lower prevalence of working among those with arthritis compared to those without, signaling the need for interventions that keep people with arthritis in the workforce.

Effectiveness of triple therapy with direct-acting antivirals for hepatitis C genotype 1 infection: application of propensity score matching in a national HCV treatment registry.

BACKGROUND: Observational studies are used to measure the effectiveness of an intervention in non-experimental, real world scenarios at the population level and are recognised as an important component of the evidence pyramid. Such data can be accrued through prospective cohort studies and a patient registry is a proven method for this type of study. The national hepatitis C (HCV) registry was established in Ireland in 2012 with the aim of monitoring the clinical and economic outcomes from new, high cost regimens for the treatment of HCV infection. A sustained virological response (SVR) 24 weeks following completion of therapy with interferon-containing regimens is considered a cure. Non-randomisation in these studies can result in confounding or selection bias. Propensity score (PS) matching is one of a number of statistical tools that can be used to mitigate the effects of confounding in observational studies. METHODS: We analysed the data of 309 patients who underwent triple therapy treatment with telaprevir (TPV) in combination with pegylated-interferon and ribavirin (PR) or boceprevir (BOC)/PR between June 2012 and December 2014. The decision to initiate treatment and the selection of the treatment regimen was at the discretion of the physician. To adjust for confounding, three approaches to propensity score matching were assessed Adjusted sustained-virological response rates (SVR), odds ratios, p-values and 95% confidence intervals were calculated from the three PS matched dataset. RESULTS: Prior to matching, the unadjusted sustained virological response rates 24 weeks after treatment complete (SVR24) were 74% (n = 158/215) and 61% (n = 57/94) for telaprevir/PR and boceprevir/PR, respectively. After matching, adjusted SVR24 rates were between 73-74% and 60-61% for telaprevir/PR and boceprevir/PR, respectively. CONCLUSION: Efficacy rates were comparable with those reported in pivotal clinical trials and real world studies. After adjusting for confounding, we conclude that there was no difference in treatment effect after PS matching. The small sample size limits the conclusions that can be made about the effect of PS matching. Propensity score adjustment remains a tool that can be applied to future analysis, however, we suggest, where possible, using a larger sample size in order to reduce the uncertainty around the outcomes.

Assessment of safety and efficacy of long-term treatment with combination lumacaftor and ivacaftor therapy in patients with cystic fibrosis homozygous for the F508del-CFTR mutation (PROGRESS): a phase 3, extension study.

BACKGROUND: The 24-week safety and efficacy of lumacaftor/ivacaftor combination therapy was shown in two randomised controlled trials (RCTs)-TRAFFIC and TRANSPORT-in patients with cystic fibrosis who were aged 12 years or older and homozygous for the F508del-CFTR mutation. We aimed to assess the long-term safety and efficacy of extended lumacaftor/ivacaftor therapy in this group of patients in PROGRESS, the long-term extension of TRAFFIC and TRANSPORT. METHODS: PROGRESS was a phase 3, parallel-group, multicentre, 96-week study of patients who completed TRAFFIC or TRANSPORT in 191 sites in 15 countries. Patients were eligible if they were at least 12 years old with cystic fibrosis and homozygous for the F508del-CFTR mutation. Exclusion criteria included any comorbidity or laboratory abnormality that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering the study drug to the participant, history of drug intolerance, and history of poor compliance with the study drug. Patients who previously received active treatment in TRANSPORT or TRAFFIC remained on the same dose in PROGRESS. Patients who had received placebo in TRANSPORT or TRAFFIC were randomly assigned (1:1) to receive lumacaftor (400 mg every 12 h)/ivacaftor (250 mg every 12 h) or lumacaftor (600 mg once daily)/ivacaftor (250 mg every 12 h). The primary outcome was to assess the long-term safety of combined therapy. The estimated annual rate of decline in percent predicted FEV1 (ppFEV1) in treated patients was compared with that of a matched registry cohort. Efficacy analyses were based on modified intention-to-treat, such that data were included for all patients who were randomly assigned and received at least one dose of study drug. This study is registered with ClinicalTrials.gov, number NCT01931839. FINDINGS: Between Oct 24, 2013, and April 7, 2016, 1030 patients from the TRANSPORT and TRAFFIC studies enrolled in PROGRESS, and 1029 received at least one dose of study drug. 340 patients continued treatment with lumacaftor 400 mg every 12 h/ivacaftor 250 mg every 12 h; 176 patients who had received placebo in the TRANSPORT or TRAFFIC studies initiated treatment with lumacaftor 400 mg every 12 h/ivacaftor 250 mg every 12 h, the commercially available dose, for which data are presented. The most common adverse events were infective pulmonary exacerbations, cough, increased sputum, and haemoptysis. Modest blood pressure increases seen in TRAFFIC and TRANSPORT were also observed in PROGRESS. For patients continuing treatment, the mean change from baseline in ppFEV1 was 0·5 (95% CI -0·4 to 1·5) at extension week 72 and 0·5 (-0·7 to 1·6) at extension week 96; change in BMI was 0·69 (0·56 to 0·81) at extension week 72 and 0·96 (0·81 to 1·11) at extension week 96. The annualised pulmonary exacerbation rate in patients continuing treatment through extension week 96 (0·65, 0·56 to 0·75) remained lower than the placebo rate in TRAFFIC and TRANSPORT. The annualised rate of ppFEV1 decline was reduced in lumacaftor/ivacaftor-treated patients compared with matched controls (-1·33, -1·80 to -0·85 vs -2·29, -2·56 to -2·03). The efficacy and safety profile of the lumacaftor 600 mg once daily/ivacaftor 250 mg every 12 h groups was generally similar to that of the lumacaftor 400 mg every 12 h/ivacaftor 250 mg every 12 h groups. INTERPRETATION: The long-term safety profile of lumacaftor/ivacaftor combination therapy was consistent with previous RCTs. Benefits continued to be observed with longer-term treatment, and lumacaftor/ivacaftor was associated with a 42% slower rate of ppFEV1 decline than in matched registry controls. FUNDING: Vertex Pharmaceuticals Incorporated.

Socioeconomic status and the likelihood of antibiotic treatment for signs and symptoms of pulmonary exacerbation in children with cystic fibrosis.

OBJECTIVE: To determine whether socioeconomic status (SES) influences the likelihood of antibiotic treatment of pulmonary exacerbations in patients with cystic fibrosis (CF). STUDY DESIGN: We used data on 9895 patients ≤ 18 years old from the Epidemiologic Study of CF. After establishing an individual baseline of clinical signs and symptoms, we ascertained whether antibiotics were prescribed when new signs/symptoms suggested a pulmonary exacerbation, adjusting for sex, presence of Pseudomonas aeruginosa, the number of new signs/symptoms, and baseline disease severity. RESULTS: In a 12-month period, 20.0% of patients <6 years of age, 33.8% of patients 6 to 12 years of age, and 41.4% of patients 13 to 18 years of age were treated with any (oral, intravenous (IV), or inhaled) antibiotics; the percentage receiving IV antibiotics was 7.3%, 15.2%, and 20.9%, respectively. SES had little effect on treatment for pulmonary exacerbation with any antibiotics, but IV antibiotics were prescribed more frequently for patients with lower SES. CONCLUSIONS: SES-related disparities in CF health outcomes do not appear to be explained by differential treatment of pulmonary exacerbations.

Longitudinal assessment of health-related quality of life in an observational cohort of patients with cystic fibrosis.

BACKGROUND: Patient-reported outcomes (PROs) are increasingly used to evaluate the efficacy of new treatments and the progression of chronic diseases. The Cystic Fibrosis Questionnaire-Revised (CFQ-R) is a disease-specific, PRO measure of health-related quality of life (HRQOL). We evaluated associations between changes in health status over time and HRQOL in a national CF database. METHODS: Using the Epidemiologic Study of Cystic Fibrosis (ESCF) data, we identified participants who had completed age-appropriate CFQ-R assessments on two occasions separated by 9-15 months. Next, we developed multivariate regression models to test whether associations existed between (1) changes in respiratory signs/symptoms and changes in the respiratory health domains of the CFQ-R, (2) changes in nutritional health status and changes in the nutritional health domains of the CFQ-R, and (3) changes in treatment complexity and changes in the Treatment Burden scale of the CFQ-R. RESULTS: We analyzed 1,947 pairs of assessments: 337 child (mean age 8.9, range 6-13 years), 581 parent (mean age of child 8.8, range 6-13 years), 398 adolescent (mean age 15.3, range 14-17 years), and 631 adult (mean age 26.9, range 18-73 years). On average, we found little change in both health status indicators and CFQ-R domain scores over 1 year. Significant associations over time, however, were found between increases in respiratory symptoms and worse CFQ-R Respiratory Symptom scores, declining weight and worsening scores on CFQ-R nutritional health domains, and increases in treatment complexity and worsening CFQ-R Treatment Burden scores for parent respondents. CONCLUSION: Health status and HRQOL changes were small over a 1-year period in this CF population. However, changes in respiratory symptoms and weight were associated with significant changes on relevant CFQ-R scores, indicating that this PRO is sensitive to changes in health status over time.

Impact of socioeconomic status, race, and ethnicity on quality of life in patients with cystic fibrosis in the United States.

BACKGROUND: Patient-reported outcomes are increasingly used in clinical trials to assess the natural history of chronic diseases and the efficacy of new treatments. Understanding the effects of socioeconomic and minority status on health-related quality of life (HRQOL) will facilitate interpretation of the results of clinical trials and suggest targets for interventions to improve patient care and outcomes. The objective of this study was to examine the effects of socioeconomic and minority status on HRQOL in patients with cystic fibrosis (CF) from childhood through adulthood in a large, comprehensive database containing medical and HRQOL data for patients with CF. METHODS: A cross-sectional study was performed using data obtained from the Epidemiologic Study of Cystic Fibrosis on 4,751 patients and 1,826 parents who were non-Hispanic white, African-American, or Hispanic and who completed the Cystic Fibrosis Questionnaire-Revised (CFQ-R), a disease-specific HRQOL measure, during a stable clinic visit. RESULTS: Multivariate models assessed the main effects of socioeconomic and minority status on clinical and HRQOL outcomes. Regression models that controlled for disease severity identified the contributions of these two variables to HRQOL. Low socioeconomic status was associated with significantly lower CFQ-R scores for children, parents, and adults on the majority of domains. After controlling for disease severity and socioeconomic status, African-American and Hispanic patients reported worse emotional and social functioning. CONCLUSIONS: Low socioeconomic and minority status may affect important clinical and patient-reported outcomes for patients with CF across their life span.

Trends in medical care expenditures of US adults with arthritis and other rheumatic conditions 1997 to 2005.

OBJECTIVE: To examine trends in annual medical expenditures from 1997 to 2005 among adults with arthritis and other rheumatic conditions (denoted Arthritis group). METHODS: We analyzed annual medical expenditures (2005 US dollars) among adults with Arthritis using the Medical Expenditure Panel Survey (MEPS), a nationally representative survey of the US civilian, noninstitutionalized population. Expenditures were stratified by Arthritis and comorbidity status. RESULTS: The Arthritis population increased by 22% (36.8 to 44.9 million) during this period, attributable entirely to the subpopulation with at least one comorbid condition (31.8 to 40.3 million). The overall, inflation-adjusted annual mean medical expenditures for adults with Arthritis increased from $6,848 in 1997 to $7,854 in 2005. In 1997, inpatient care was the most expensive component of overall expenditures (mean $2,702), but beginning in 2001, mean inpatient and ambulatory expenditures were almost identical. Mean prescription expenditures increased nearly every year, almost doubling from $970 in 1997 to $1,811 in 2005. Aggregate total expenditures for the Arthritis population increased markedly during this period, from $252.0 to $353.0 billion (+40%). Most of this increase was attributable to the population increase in the Arthritis and comorbid condition subgroup. CONCLUSION: Mean annual ambulatory and prescription expenditures for adults with Arthritis increased far above the rate of medical inflation, offsetting a relative decline in inpatient expenditures. Increases in overall mean and aggregate total expenditures are attributable to the increasing number of adults with Arthritis and at least one comorbid chronic condition. Projected increases in this population suggest that these expenditures will continue to rise.

Comparison of levalbuterol and racemic albuterol in hospitalized patients with acute asthma or COPD: a 2-week, multicenter, randomized, open-label study.

BACKGROUND: The National Heart, Lung, and Blood Institute guideline recommends that dosing racemic albuterol be administered every 1 to 4 hours for treating patients with asthma or chronic obstructive pulmonary disease (COPD) in the hospital. Previously published preliminary and retrospective studies suggested that levalbuterol can be administered every 8 hours for the treatment of bronchoconstriction in hospitalized patients. However, it is unclear how the different dosing regimens affect the total number of nebulizations (scheduled plus as-needed treatments) and the costs of treatment of bronchoconstriction in a hospital setting. Moreover, it is not clear how the different dosing regimens affect symptom outcomes and health status in hospitalized patients with asthma or COPD. OBJECTIVE: The aim of this study was to evaluate these issues in hospitalized patients with acute asthma or COPD. METHODS: In this prospective, multicenter, randomized, open-label study, hospitalized patients aged > or = 18 years were randomly assigned to receive 14-day treatment with levalbuterol 1.25 mg q6-8h or racemic albuterol 2.5 mg q1-4h, administered per routine hospital practice at each institution. The primary efficacy end point was total number of nebulizations during hospitalization. Pulmonary function, symptom evaluation (subject general well-being score [SGWB], disease symptom assessment [DSA], and beta-mediated adverse effect scores), hospital costs (excluding medication costs) and hospital length of stay (LOS) were also evaluated. RESULTS: In the intent-to-treat population (n = 479; levalbuterol, 241;racemic albuterol, 238), the mean (SE) age was 55.3 (16.9) years, the majority of patients were white (57.8%), and the mean (SE) weight was 80.9 (24.5) kg. Demographic characteristics were similar between the 2 treatment groups, except that there were more females with COPD in the levalbuterol treatment group (63.88%) compared with the racemic albuterol treatment group (45.5%) (P = 0.005). Patients treated with levalbuterol required significantly fewer median total nebulizations (10 vs 12; P = 0.031) and scheduled nebulizations (9 vs 11; P = 0.009) compared with those in the racemic albuterol group. The 2 treatment groups required 0 rescue nebulizations. Mean (SD) forced expiratory volume in 1 second improved from baseline with both levalbuterol and racemic albuterol (0.06 [0.43] and 0.10 [0.37] L, respectively); these improvements were maintained throughout the hospital stay (0.11 [0.48] and 0.16 [0.52] L). DSA and SGWB scores improved significantly from baseline in both treatment groups, and beta-mediated adverse effects mean scores were significantly greater with levalbuterol versus racemic albuterol (P < 0.001). In the levalbuterol and racemic albuterol treatment groups, hospital LOS (70.6 and 65.7 hours, respectively), time to discharge (66.0 and 62.8 hours), and total hospital costs (least squares mean [SE], US $4869.30 [$343.58] and $4899.41 [$343.20]) were similar. CONCLUSIONS: In these hospitalized patients with acute asthma or COPD treated with levalbuterol every 6 to 8 hours or racemic albuterol every 1 to 4 hours, significantly fewer total nebulizations were required with levalbuterol, without an increased need for rescue nebulizations during 14 days of hospitalization. Both treatments were associated with improvements from baseline in symptoms and health status. The costs of treating bronchoconstriction in hospitalized patients were similar between the levalbuterol and racemic albuterol groups.

Insurance and quality of life in men with prostate cancer: data from the Cancer of the Prostate Strategic Urological Research Endeavor.

OBJECTIVE: To evaluate the effect of medical insurance coverage on health-related quality of life (HRQoL) outcomes in men newly diagnosed with prostate cancer, as insurance status has been shown to be related to clinical presentation, and types of treatments received for localized prostate cancer, but the relationship of insurance and QoL has not been explored sufficiently. PATIENTS AND METHODS: Data from the Cancer of the Prostate Strategic Urological Research Endeavor (CaPSURE), a national longitudinal database registry of men with prostate cancer, were used for this study. Men who were newly diagnosed at entry to CaPSURE and completed one questionnaire before treatment, and one or more afterwards, were included. Insurance groups specific to age distribution of the study population were assessed, i.e. Medicare, preferred provider organizations (PPOs), health maintenance organizations (HMOs), fee for service (FFS), and the Veterans Administration (VA) for the younger group, and Medicare only, Medicare plus supplement (+S), and HMO/PPO for the older group. Associations between patients' clinical and sociodemographic characteristics and insurance status were evaluated by chi-square and analysis of variance. Relationships between insurance status and HRQoL outcomes over time were evaluated by multivariate mixed model. RESULTS: Of 2258 men who met the study criteria, 1259 were younger and 999 were older than 65 years. More than half of the younger patients belonged to an HMO or PPO (42.2% and 32.5%, respectively), with the remainder distributed between Medicare, FFS and VA. In the older group most men belonged to Medicare only and the Medicare +S groups (22.4% and 58.8%, respectively). There was greater variation in clinical risk categories at presentation by insurance groups in the younger group. In the multivariate analysis, insurance status was significantly associated with changes in most HRQoL outcomes over time in the younger group, while in the older patients the effect of insurance diminished. Men in the VA and Medicare systems had lower scores at baseline and a steeper decline in Physical Function, Role Physical, Role Emotional, Social Function, Bodily Pain, Vitality, and General Health domains over time, controlling for type of initial treatment received, timing of HRQoL assessment, number of comorbidities, clinical risk at presentation, and income. CONCLUSION: Insurance was independently related to changes in a wide range of HRQoL outcomes in men aged <65 years treated for prostate cancer. With the latest advances in early diagnosis and treatment of prostate cancer, clinicians and researchers should be aware of the specific groups of patients who are more vulnerable to the adverse effects of treatment and subsequent decline in functioning. The present findings could provide important tools for understanding the process of recovery after treatment for prostate cancer, and identifying needs for specific services.

Medical care expenditures and earnings losses among persons with arthritis and other rheumatic conditions in 2003, and comparisons with 1997.

OBJECTIVE: To obtain estimates of medical care expenditures and earnings losses associated with arthritis and other rheumatic conditions and the increment in such costs attributable to arthritis and other rheumatic conditions in the US in 2003, and to compare these estimates with those from 1997. METHODS: Estimates for 2003 were derived from the Medical Expenditures Panel Survey (MEPS), a national probability sample of households. We tabulated medical care expenditures of adult MEPS respondents, stratified by arthritis and comorbidity status, and used regression techniques to estimate the increment of medical care expenditures attributable to arthritis and other rheumatic conditions. We also estimated the earnings losses sustained by working-age adults with arthritis and other rheumatic conditions. Estimates for 2003 were compared with those from 1997, inflated to 2003 terms. RESULTS: In 2003, there were 46.1 million adults with arthritis and other rheumatic conditions (versus 36.8 million in 1997). Adults with arthritis and other rheumatic conditions incurred mean medical care expenditures of $6,978 in 2003 (versus $6,346 in 1997), of which $1,635 was for prescriptions ($899 in 1997). Expenditures for adults with arthritis and other rheumatic conditions totaled $321.8 billion in 2003 ($233.5 billion in 1997). In 2003, the mean increment in medical care expenditures attributable to arthritis and other rheumatic conditions was $1,752 ($1,762 in 1997), for a total of $80.8 billion ($64.8 billion in 1997). Persons with arthritis and other rheumatic conditions ages 18-64 years earned $3,613 less than other persons (versus $4,551 in 1997), for a total of $108.0 billion (versus $99.0 billion). Of this amount, $1,590 was attributable to arthritis and other rheumatic conditions (versus $1,946 in 1997), for a total of $47.0 billion ($43.3 billion in 1997). CONCLUSION: Our findings indicate that the increase in medical care expenditures and earnings losses between 1997 and 2003 is due more to an increase in the number of persons with arthritis and other rheumatic conditions than to costs per case.

Multiinstitutional validation of the UCSF cancer of the prostate risk assessment for prediction of recurrence after radical prostatectomy.

BACKGROUND: The University of California, San Francisco (UCSF) Cancer of the Prostate Risk Assessment (CAPRA) is a novel preoperative index which predicts the risk of biochemical recurrence after radical prostatectomy. The performance of the index is at least as good as the best available instruments based on clinical variables, and the 0 to 10 score is simple to calculate for both clinical and research purposes. This study used a large external dataset to validate CAPRA. METHODS: Data were abstracted from the Shared Equal Access Regional Cancer Hospital (SEARCH) database, a registry of men who underwent radical prostatectomy at 4 Veterans Affairs and 1 active military medical center. Of 2096 men in the database, 1346 (64%) had full data available to calculate the CAPRA score. Performance of the CAPRA score was assessed with proportional hazards regression, survival analysis, and the concordance (c) index. RESULTS: Of the studied patients, 41% were non-Caucasian, and their mean age was 62 years. Twenty-six percent suffered recurrence; median follow-up among patients who did not recur was 34 months. The hazard ratio (HR) for each 1-point increase in CAPRA was 1.39 (95% CI [confidence interval], 1.31-1.46). The 5-year recurrence-free survival rate ranged from 86% for CAPRA 0-1 patients to 21% for CAPRA 7-10 patients. Increasing CAPRA scores were significantly associated with increasing risk of adverse pathologic outcomes. The c-index for CAPRA for the validation set was 0.68, compared with 0.66 for the original development set. CONCLUSIONS: The UCSF-CAPRA accurately predicted both biochemical and pathologic outcomes after radical prostatectomy among a large, diverse, cohort of men. These results validated the effectiveness of this powerful and straightforward instrument.

The University of California, San Francisco Cancer of the Prostate Risk Assessment score: a straightforward and reliable preoperative predictor of disease recurrence after radical prostatectomy.

PURPOSE: Multivariate prognostic instruments aim to predict risk of recurrence among patients with localized prostate cancer. We devised a novel risk assessment tool which would be a strong predictor of outcome across various levels of risk, and which could be easily applied and intuitively understood. MATERIALS AND METHODS: We studied 1,439 men diagnosed between 1992 and 2001 who had undergone radical prostatectomy and were followed in the Cancer of the Prostate Strategic Urologic Research Endeavor (CaPSURE) database, a longitudinal, community based disease registry of patients with prostate cancer. Disease recurrence was defined as prostate specific antigen (PSA) 0.2 ng/ml or greater on 2 consecutive occasions following prostatectomy or a second cancer treatment more than 6 months after surgery. The University of California, San Francisco-Cancer of the Prostate Risk Assessment (UCSF-CAPRA) score was developed using preoperative PSA, Gleason score, clinical T stage, biopsy results and age. The index was developed and validated using Cox proportional hazards and life table analyses. RESULTS: A total of 210 patients (15%) had recurrence, 145 by PSA criteria and 65 by second treatment. Based on the results of the Cox analysis, points were assigned based on PSA (0 to 4 points), Gleason score (0 to 3), T stage (0 to 1), age (0 to 1) and percent of biopsy positive cores (0 to 1). The UCSF-CAPRA score range is 0 to 10, with roughly double the risk of recurrence for each 2-point increase in score. Recurrence-free survival at 5 years ranged from 85% for a UCSF-CAPRA score of 0 to 1 (95% CI 73%-92%) to 8% for a score of 7 to 10 (95% CI 0%-28%). The concordance index for the UCSF-CAPRA score was 0.66. CONCLUSIONS: The UCSF-CAPRA score is a straightforward yet powerful preoperative risk assessment tool. It must be externally validated in future studies.

Demographics, insurance coverage, and utilization of medical services in newly diagnosed prostate cancer: data from caPSURE.

This study was conducted to identify factors that might influence access to medical services by patients with prostate cancer. The sample comprised 1882 patients who underwent radical prostatectomy and received at least six months of posttreatment follow-up. The investigators found that the type of insurance coverage significantly influenced the number of visits to general practitioners, mental health professionals, and oncologists. However, visits to urologists, nurse practitioners, or alternative-therapy providers were not influenced by type of insurance coverage. Moreover, race, risk profile, and education were significantly correlated with utilization of health services by these patients. The influence of demographic features and insurance coverage on health care utilization by patients with prostate cancer should be further explored.

Longitudinal assessment of changes in sexual function and bother in patients treated with external beam radiotherapy or brachytherapy, with and without neoadjuvant androgen ablation: data from CaPSURE.

PURPOSE: To evaluate the effects of external beam radiotherapy (EBRT), with or without brachytherapy (BT) boost or brachytherapy monotherapy with and without short-term androgen ablation (<==6 months; STAD) on sexual function (SF) and sexual bother (SB) in men treated for localized prostate cancer. METHODS AND MATERIALS: A total of 992 men with newly diagnosed prostate cancer enrolled in the Cancer of the Prostate Strategic Urological Research Endeavor database were studied to assess treatment-related changes in SF and SB. Six treatment subgroups (EBRT - STAD, EBRT + STAD, BT - STAD, BT + STAD, EBRT + BT - STAD, EBRT + BT + STAD) were compared. RESULTS: The greatest reported changes in SF occurred during the first 2 posttreatment years. Patients receiving BT reported greater SF and the least change in SF overall; those receiving EBRT + BT reported the greatest decline in SF. SF scores associated with STAD were initially lower than in patients without STAD; however by 1 year no statistically significant difference in SF or SB was noted. CONCLUSION: Each treatment for prostate cancer can negatively affect SF and SB. Initial differences among treatment subgroups exist, but diminish with time. SF changes associated with EBRT +/- BT were statistically significant and those for BT were not. STAD appeared to confer only temporary and recoverable impairment of erectile function.

Medical care expenditures and earnings losses of persons with arthritis and other rheumatic conditions in the United States in 1997: total and incremental estimates.

OBJECTIVE: To provide estimates of the total medical care expenditures and earnings losses associated with arthritis and other rheumatic conditions (AORC), as well as the increment in such costs specifically attributable to these conditions, in the US in 1997. METHODS: The estimates were derived from the 1997 Medical Expenditures Panel Survey (MEPS), a national probability sample of 14,147 households including 34,551 persons, of whom 4,776 self-reported arthritis. After weighting, those who self-reported AORC represent 38.4 million persons. We tabulated all medical care expenditures of the adult MEPS respondents, stratified by arthritis and comorbidity status, and then used regression techniques to estimate the increment in health care expenditures attributable to AORC, after taking comorbidity, demographic characteristics, and insurance status into account. Using the same methods, we also estimated the magnitude of the earnings losses sustained by persons of working ages (18-64 years) who had AORC. RESULTS: Persons with AORC incurred mean total medical care expenditures of 4,865 dollars (total 186.9 billion dollars). The largest components of these expenditures were inpatient care (39%), ambulatory care (29%), and prescriptions (14%). The mean increment in medical care expenditures specifically attributable to AORC among those ages 18 years and older was 1,391 dollars(total approximately 51.1 billion dollars). Persons with AORC ages 18-64 years earned 3,812 dollars less on average than did other persons of these ages (total 82.4 billion dollars). Of this average, 1,579 dollars was attributable to the AORC (total 35.1 billion dollars). CONCLUSION: In 1997, persons with AORC incurred direct and indirect costs of 269.3 billion dollars, of which 86.2 billion dollars was attributable to these conditions.

Fear of cancer recurrence in patients undergoing definitive treatment for prostate cancer: results from CaPSURE.

PURPOSE: Contemporary cancer treatments have resulted in patients living longer but with the risk of disease recurrence. Studies suggest that fear of recurrence is a significant burden. We described fear of cancer recurrence in patients with prostate cancer undergoing treatment with radical prostatectomy (RP), radiation (XRT) or brachytherapy (BT). MATERIALS AND METHODS: A total of 519 patients (326 RP, 53 XRT, 140 BT) were identified from CaPSURE (Cancer of the Prostate Strategic Urologic Research Endeavor), a national longitudinal registry of men with prostate cancer. To be included in the study patients had to complete at least 1 pretreatment and 2 posttreatment health related quality of life questionnaires and have complete clinical information. Fear of cancer recurrence was assessed with a validated 5-item scale, and was described at baseline and up to 2 years after treatment. Multivariate linear regression was performed to determine significant predictors of fear of cancer recurrence. RESULTS: Men receiving XRT were older and had worse clinical disease characteristics than patients treated with RP or BT. For all groups fear of cancer recurrence was more severe before treatment and improved after treatment but did not change substantially in the 2 years thereafter. Regression revealed that only general health and mental health were important predictors of fear of cancer recurrence. No other general or disease specific health related quality of life domains or clinical characteristics contributed appreciable explanatory power. CONCLUSIONS: Fear of prostate cancer recurrence imposes a substantial burden in patients before and after treatment. Understanding the fear of cancer recurrence associated with different treatments can help physicians better counsel patients and promote psychological well-being.

Telephone assessment of functioning and well-being following stroke: is it feasible?

Stroke can affect the physical, emotional, and social aspects of patients' lives. The purpose of this study was to assess the feasibility and psychometric properties of a telephone-administered version of the Health Utilities Index Mark 2 and 3 (HUI2/3). Subjects included patients who had had an ischemic stroke within the prior 12 months and their unpaid caregivers (n = 76 pairs) and an additional 33 unpaid caregivers of patients who were generally aphasic or severely affected. Complete response rates, test-retest reliability, and convergent, divergent, and known-groups validity were determined. For patient-caregiver pairs, 27% had no complete Health Utilities Index Mark 2 (HUI2) responses (i.e., had missing responses for at least 1 item of each assessment), 51% had partial responses (i.e., had complete responses for at least 1, but not all of the assessments), and 22% had complete responses. For the Health Utilities Mark 3 (HUI3), the percentages were 19%, 52%, and 29%. Test-retest reliability for patients intraclass correlation coefficient (ICC = 0.76 for HUI2; 0.75 for HUI3) and caregivers (ICC = 0.91 and 0.89, respectively) were excellent. There were generally high levels of both convergent and divergent validity. There was limited known-groups validity (mild v moderately and mild v severely affected patients reported different overall HUI2 and HUI3 scores; there was no difference between those with moderate and severe disabilities). The same pattern was found for caregivers. We conclude that the telephone-administered HUI2/3 appears to be reliable and have at least limited validity. However, the proportions of missing data for patient/caregivers administered the HUI2/3 were surprisingly high. This high proportion of missing data would limit the use of the telephone-administered HUI2/3 in the context of stroke trials.