The impact of lower urinary tract symptoms on health-related quality of life among patients with multiple sclerosis.

AIMS: Lower urinary tract symptoms are commonly experienced among patients with multiple sclerosis (MS), however, their impact on health-related quality of life (HRQOL) has not been well characterized. Herein the incremental impact of lower urinary tract symptoms on HRQOL among patients with MS has been evaluated. METHODS: A cross-sectional online survey was administered to US residents with a self-reported MS diagnosis. Data pertaining to demographics, disease history, urinary symptoms, and HRQOL, including the Short Form 36, version 2 (SF-36v2), were collected. Patients were stratified into four urinary symptom groups: no/minimal urinary symptoms, urinary urgency (UU), urinary urgency incontinence (UUI), and other lower urinary tract symptoms. Multiple linear regression models evaluated the impact of these symptoms. RESULTS: Out of the 1,052 respondents, mean age was 47.8 ± 10.6 years; mean time since MS diagnosis was 8.5 ± 7.8 years. UUI and UU subgroups showed the greatest adjusted HRQOL decrement compared with the no/minimal urinary symptoms group, scoring 2.8 (SE ± 0.7, UUI) and 3.5 (SE ± 0.8, UU) points lower on SF-36v2 Physical Component Summary, respectively, and 3.7 (SE ± 1.0, UUI) and 5.0 (SE ± 1.2, UU) points lower on SF-36v2 Mental Component Summary (P < 0.001 for all), respectively. CONCLUSIONS: Both UU and UUI symptoms contribute to a decrement in HRQOL among patients with MS.

Asthma control in the United States, 2008-2010: indicators of poor asthma control.

BACKGROUND: An estimated 23 million Americans have asthma, of whom at least 12 million experience an asthma exacerbation every year. Clinical practice guidelines focus on asthma control, with an emphasis on reducing both impairment and risk. OBJECTIVE: We sought to explore broad patterns of asthma prevalence, self-reported medication use, and indicators of control in a nationally representative sample. METHODS: The 2008, 2009, and 2010 Medical Expenditure Panel Surveys were used to examine the national prevalence of self-reported asthma, trends in medication use, and demographic characteristics of asthmatic patients. History of lifetime asthma and current diagnosis were ascertained based on self-report. Asthma management and control were examined by using patient-reported medication use. RESULTS: Of the 102,544 subjects asked about an asthma diagnosis, 9,782 reported lifetime asthma, and 8,837 reported current asthma. Five thousand five subjects (4.8% of the population) reported experiencing an asthma exacerbation in the previous year. Four thousand five hundred twenty-one subjects used a quick-relief inhaler for asthma symptoms, and 14.6% used more than 3 canisters of this type of medication in the past 3 months. Of this group, 60% were using daily long-term control medication but still required significant use of quick-relief inhalers, whereas 28% had never used long-term control medication. Of those who had a recent exacerbation, 29% were using daily preventive medication, whereas 54% had never used long-term control medication. CONCLUSIONS: Improvement of asthma control continues to be a US public health concern. Results suggest suboptimal asthma control with underuse of long-term control medications, overuse of quick-relief inhalers, and a significant number of self-reported asthma exacerbations.

The relationship between asthma, asthma control and economic outcomes in the United States.

OBJECTIVE: Asthma, a serious chronic lung disease affecting approximately 26 million Americans, remains clinical and economic burdens on the healthcare system. Although associations between uncontrolled asthma and poor health outcomes is known, the extent of this impact of uncontrolled asthma on economic outcomes in the United States (US) is unknown. We sought to determine the relationship between asthma, asthma control and economic outcomes in the US. METHODS: The 2008-2010 Medical Expenditure Panel Surveys were used to estimate the impact of uncontrolled asthma (asthma-related emergency department [ED] visit, use of >3 canisters of quick-relief inhaler in past 3 months or asthma attack in past 12 months) on medical expenditures, utilization and productivity. Estimates were generated using multivariate regression controlling for sociodemographics and comorbidity. RESULTS: Medical expenditures attributable to asthma were up to $4423 greater for those with markers of uncontrolled asthma compared with those who did not have asthma. Frequency of hospital discharges were up to 4.6-fold greater for those with uncontrolled asthma than those without asthma (p < 0.01), while all others with asthma did not have significantly more discharges. ED visits were up to 1.8-fold greater for those with uncontrolled asthma compared with those without asthma (p < 0.01). Productivity was significantly (p < 0.01) decreased (more likely to be unemployed, more days absent from work and more activity limitations) for those with uncontrolled asthma. CONCLUSIONS: In recent national data, individuals with asthma and markers of uncontrolled asthma had higher medical expenditures, greater utilization and decreased productivity.

Asthma in USA: its impact on health-related quality of life.

OBJECTIVES: Given the growing prevalence of asthma in USA, it is important to understand its national burden from the patient's perspective. The objective of this research is to examine the national burden of asthma and poor asthma control on health function, health perception and preference-based health-related quality of life (HRQL). METHODS: The Medical Expenditure Panel Survey (MEPS), a nationally representative survey, was used to estimate the impact of asthma and indicators of poor asthma control on health function, self-rated health perception and preference-based HRQL using multivariate regression methods controlling for socioeconomic, clinical and demographic characteristics. Two HRQL instruments were used: SF-12v2 Physical Component Scale (PCS-12) and Mental Component Scale (MCS-12); EQ-5D-3L index and visual analogue scale (VAS). Two multivariate regression methods were used, Censored Least Absolute Deviation [EQ-5D-3L and VAS (due to censoring)] and Ordinary Least Squares (OLS) (PCS-12 and MCS-12). RESULTS: After controlling for covariates, asthma resulted in a statistically significant reduction in preference-based HRQL, health perception and physical and mental function (EQ-5D -0.023; VAS -2.21; PCS-12 -2.36; MCS-12 -0.96). Likewise, experiencing an exacerbation in the previous year and using more than three canisters of quick-relief medication in the previous 3 months were both associated with a statistically significant and clinically meaningful reduction in all four measures. CONCLUSIONS: Asthma itself and especially indicators of poor asthma control were associated with a deleterious effect on health function, preference-based HRQL and self-perceived health status. Given the prevalence of asthma, poorly controlled asthma constitutes a significant national burden in USA.

Economic and humanistic consequences of preventable bladder tumor recurrences in nonmuscle invasive bladder cancer cases.

PURPOSE: Perioperative intravesical chemotherapy following transurethral resection of bladder tumor has been underused despite level 1 evidence supporting its performance. The primary objective of this study was to estimate the economic and humanistic consequences associated with preventable recurrences in patients initially diagnosed with nonmuscle invasive bladder cancer. MATERIALS AND METHODS: Using population based estimates of nonmuscle invasive bladder cancer incidence, a 2-year model was developed to estimate the number of preventable recurrences in eligible patients untreated with perioperative intravesical chemotherapy. Therapy utilization rates were obtained from a retrospective database analysis and a chart review study of 1,010 patients with nonmuscle invasive bladder cancer. Recurrence rates of nonmuscle invasive bladder cancer were obtained from a randomized clinical trial comparing transurethral resection of bladder tumor with or without perioperative mitomycin C. Costs were estimated using prevailing Medicare reimbursement rates. Quality adjusted life-year estimates and disutilities for complications were obtained from the literature. RESULTS: The model estimated that 7,827 bladder recurrences could be avoided if all patients received immediate intravesical chemotherapy. It estimated an economic savings of $3,847 per avoidable recurrence, resulting in an aggregate savings of $30.1 million. The model also estimated that 1,025 quality adjusted life-years are lost every 2 years due to preventable recurrences, resulting in 0.13 quality adjusted life-years (48 quality adjusted days) lost per avoidable recurrence. This translates into 0.02 quality adjusted life-years (8.1 quality adjusted days) lost per patient not receiving immediate intravesical chemotherapy. CONCLUSIONS: Greater use of immediate intravesical chemotherapy in the United States has the potential to substantially decrease the economic and humanistic burdens of nonmuscle invasive bladder cancer.

Health-related quality of life of pediatric patients with moderate to severe plaque psoriasis: comparisons to four common chronic diseases.

Health-related quality of life (HRQOL) is an important indicator of the burden of illness in moderate-to-severe plaque psoriasis. This study evaluated self-reported generic HRQOL among pediatric patients with moderate-to-severe plaque psoriasis based on pooled baseline clinical trial data and compared them to four common chronic diseases and to a healthy sample. The Pediatric Quality of Life Inventory Version 4.0 (PedsQL™ 4.0) Generic Core Scales was administered to 208 patients ages 4 to 17 years with stable, moderate-to-severe plaque psoriasis. Patients with moderate-to-severe plaque psoriasis were compared using one-sample t-tests to published PedsQL™ ( http://www.pedsql.org ) data on healthy children and pediatric patients with arthritis, psychiatric disorders, asthma, and diabetes. Pediatric patients with moderate-to-severe plaque psoriasis demonstrated significantly impaired physical, emotional, social, and school functioning in comparison to healthy children. The PedsQL™ Emotional and School Functioning Scales demonstrated the largest mean difference between the two groups (12.1, 11.1 points, respectively). In general, patients with plaque psoriasis demonstrated significantly more impaired generic HRQOL compared to patients with diabetes, comparable HRQOL to arthritis and asthma, and better HRQOL than psychiatric patients. In conclusion, the findings indicate that pediatric patients with moderate-to-severe plaque psoriasis have significantly impaired generic HRQOL in comparison to healthy children, and HRQOL generally comparable to other serious chronic diseases. These results demonstrate the significant negative impact of plaque psoriasis on the daily lives of these children from the patients' perspective.

The burden of adult asthma in the United States: evidence from the Medical Expenditure Panel Survey.

BACKGROUND: It is important to have an accurate picture of the sources and extent of medical expenditures and productivity loss to understand the nature and scope of the burden of asthma in the United States (US). OBJECTIVE: The current study aims to provide recent nationally representative estimates of direct and productivity-related costs attributable to asthma in adults in the US. METHODS: The 2003 and 2005 Medical Expenditure Panel Surveys were used to estimate the effect of asthma on medical expenditures, use, productivity, and chronic comorbidity among adults (≥ 18 years). Productivity-related outcome variables included employment, annual wages, missed work days, days spent sick in bed, and activity limitations. Multivariate regression was conducted, controlling for sociodemographics and comorbidity. RESULTS: Of 44,795 adults, 1,935 reported an encounter for asthma [corrected]. Compared with those without, subjects with asthma were significantly less likely to be employed (odds ratio, 0.78), spent 1.4 more days sick in bed annually, and were significantly more likely to have activity limitations or to be unable to work. Adults with asthma incurred an additional $1,907 (2008 US dollars) annually and experienced higher health care use and comorbidity. The total national medical expenditure attributable to adult asthma was $18 billion. Adults with asthma were more likely to be covered by Medicaid (30%) than the general adult population (10%). The largest contributors to medical expenditures for adults with asthma were prescription drugs, followed by inpatient hospitalizations and home health care. CONCLUSIONS: In recent national data adult asthma is associated with a significant deleterious effect on direct and indirect costs in the US.

Measuring asthma control is the first step to patient management: a literature review.

Asthma control is recognized as a critical aspect of the evaluation and management of the disease. Here we evaluate and compare existing instruments for measuring asthma control in an attempt to evaluate their clinical utility. Based on a literature review, we identified validated instruments used to assess asthma control in adults. We examined the specific measurement properties and the strengths and weaknesses of each instrument, and evaluated a single instrument, the Asthma Control Questionnaire (ACQ), more closely as an example, evaluating its applicability in the clinical setting. Our review identified five validated instruments designed to measure asthma control: the Asthma Control Questionnaire (ACQ), Asthma Control Scoring System (ACSS), Asthma Control Test (ACT), Asthma Therapy Assessment Questionnaire (ATAQ), and the Lara Asthma Symptom Scale (LASS). None of the instruments covered all relevant control characteristics, but most were aligned with guideline definitions of control. All instruments demonstrated validity and responsiveness, with some measure of reliability. All instruments were short and easily administered, easy to interpret, and all had evidence to support their use in clinical decision making.

A feasibility study comparing two chiropractic protocols in the treatment of patellofemoral pain syndrome.

OBJECTIVE: The purpose of this pilot study was to determine the feasibility of (1) using the existing clinic, clinicians, interns, faculty, and staff from our college in conducting all components of a planned randomized controlled clinical trial; (2) successfully recruiting patients with patellofemoral pain syndrome (PFPS); and (3) consistently, effectively, and safely implementing the study protocols and therapy. METHODS: Diagnostic, treatment, and blind assessment procedures were carried out while recruitment and administrative techniques for managing long-term storage of data and files were developed. Thirty-one patients were randomized into a local manipulative group (group A) or to a full kinetic chain manipulative therapy group (group B), each combined with exercise and soft tissue treatment. The Anterior Knee Pain Scale, visual analog scale, and Patient Satisfaction Scale were used. RESULTS: All phases of the feasibility study including use of the clinic, staff, recruitment techniques, treatment protocols, data collection, input, and analysis were effectively and safely carried out. CONCLUSIONS: A feasibility study investigating the ability to conduct a randomized controlled trial of a manipulative therapy protocol for PFPS using available chiropractic college infrastructure was accomplished. A fully powered PFPS trial is feasible and merited.

Prevalence and correlates of fatigue in long-term survivors of childhood leukemia.

PURPOSE: To estimate the prevalence of fatigue, identify the factors associated with fatigue, and to explore the relationship between fatigue and quality of life (QOL) in long-term survivors of childhood acute lymphoblastic leukemia (ALL). METHODS: One hundred sixty-one ALL survivors diagnosed at Childrens Hospital Los Angeles (Los Angeles, CA) before age 18 years and between January 1, 1975 and December 31, 1995, participated in a structured telephone interview. Participants were aged 18 to 41 years and off treatment for an average of 14 years. Four measures of fatigue, including the Revised-Piper Fatigue Scale, were used to assess fatigue; depression was assessed using the Center for Epidemiological Studies Depression Scale. Multivariate logistic regression models were developed to identify factors associated with fatigue and depression. RESULTS: Prevalence of fatigue (30%) fell within the general population normal limits. Fatigue and depression were highly correlated (Pearson r = 0.75). Fatigue was associated with marriage (OR = 0.11; 95% CI, 0.02 to 0.50), having children (OR = 5.80; 95% CI, 1.30 to 25.82), sleep disturbances (OR = 6.15; 95% CI, 2.33 to 16.22), pain (OR = 5.56; 95% CI, 2.13 to 14.48), obesity (OR = 3.80; 95% CI, 1.41 to 10.26), cognitive impairment (OR = 2.56; 95% CI, 1.02 to 6.38), and exercise-induced symptoms (OR = 2.98; 95% CI, 1.11 to 8.02). Four factors associated with fatigue were also associated with depression: sleep disturbances, pain, obesity, and cognitive impairment. Fatigue was inversely related to QOL. CONCLUSION: Some survivors of childhood ALL experience fatigue many years after treatment. Fatigued survivors represent a high-risk subgroup as they report more depression and poorer QOL than non-fatigued survivors and their peers.

Validation and test characteristics of a 10-item neuro-ophthalmic supplement to the NEI-VFQ-25.

PURPOSE: To determine whether a 10-Item Neuro-Ophthalmic Supplement increases the capacity of the 25-Item National Eye Institute Visual Function Questionnaire (NEI-VFQ-25) to capture self-reported visual dysfunction in patients with neuro-ophthalmologic disorders. DESIGN: A cross-sectional survey to examine the characteristics of a 10-Item Neuro-Ophthalmic Supplement to the 25-Item NEI-VFQ-25 in a cohort of patients with neuro-ophthalmologic disorders. METHODS: The 10-Item Neuro-Ophthalmic Supplement was designed previously by our research group by survey and focus-group methods. In the present study, the NEI-VFQ-25 and 10-Item Supplement were administered concurrently to patients and disease-free control subjects. High-contrast visual acuities with patient usual distance correction were measured with the use of Early Treatment Diabetic Retinopathy Study (ETDRS) charts. RESULTS: Diagnoses for patients (n = 215) included optic neuritis, multiple sclerosis, idiopathic intracranial hypertension, ischemic optic neuropathy, stroke, ocular myasthenia gravis, ocular motor palsies, and thyroid eye disease. Scores for the 10-Item Supplement had a significant capacity to distinguish patients vs disease-free control subjects that was independent of the NEI-VFQ-25 composite score (odds ratio in favor of patient vs control status for 10-point worsening in Supplement scores: 2.7 [95% confidence interval [CI], 1.6, 4.6]; P < .001, logistic regression models that account for NEI-VFQ-25 composite score, age, and gender). Patients with visual dysfunction (binocular Snellen equivalents worse than 20/20) had significantly lower mean scores (9-21 points lower); these differences remained significant after accounting for age and gender (P >or= .001, linear regression). Supplement items and composite scores demonstrated appropriate degrees of internal consistency reliability. CONCLUSION: The 10-Item Neuro-Ophthalmic Supplement demonstrates a capacity to capture self-reported visual dysfunction beyond that of the NEI-VFQ-25 alone, which supports validity for this new scale. The use of the 10-Item Supplement in clinical trials and epidemiologic studies will examine its capacity to demonstrate treatment effects in longitudinal cohorts.

Self-reported comorbidities and visual function in a population-based study: the Los Angeles Latino Eye Study.

OBJECTIVE: To assess the association of self-reported systemic and ocular comorbid disease and visual function in Latino subjects. METHODS: National Eye Institute 25-item Visual Function Questionnaire (NEI-VFQ-25) and eye examination data were obtained from 5380 participants in the Los Angeles Latino Eye Study, a population-based prevalence study of eye disease in Latino subjects 40 years and older. We developed and contrasted 5 comorbidity measures. One-way analysis of variance was used to assess the association between comorbidity and visual impairment and self-reported visual function. Regression analyses determined the association of sociodemographic variables, clinical variables, and the best measure of comorbidity with the NEI-VFQ-25 composite score. The main outcome measure was self-reported visual function as assessed by the NEI-VFQ-25 composite score. RESULTS: On average, visual function subscale scores were lowest for those participants with the most systemic comorbid conditions (P<.05). This was more evident in participants with moderate or severe visual impairment compared with those with mild or no visual impairment (P<.05). CONCLUSIONS: Self-reported systemic comorbidities were associated with self-reported visual function. This association was greater at more severe levels of visual impairment. Of the 5 comorbidity measures assessed, the measure that summed the number of self-reported systemic comorbidities correlated most with self-reported visual function.

Lower urinary tract symptom prevalence and management among patients with multiple sclerosis.

BACKGROUND: This study was conducted to assess self-reported prevalence and management of lower urinary tract symptoms (LUTS), along with drivers of treatment seeking, among patients with multiple sclerosis (MS). METHODS: An online, cross-sectional survey was administered to US-residing participants with self-reported MS to assess presence of LUTS, including urinary incontinence (UI). Participants experiencing LUTS were asked additional questions related to management and current therapies. Multivariate logistic regression identified drivers of treatment-seeking behavior. RESULTS: A total of 1052 participants completed the survey; 1047 were included in the analysis. Nine hundred sixty-six participants (92%) reported at least one LUTS, the most common being post-micturition dribble (64.9%), urinary urgency (61.7%), and a feeling of incomplete emptying (60.7%). Eight hundred twenty-six (79%) reported having some type of UI. Of those with any type of LUTS, 70% (n = 680) had previously discussed urinary symptoms with a health-care provider (HCP), while only 32% (n = 311) had seen an HCP in the past year. Logistic regression found urgency (odds ratio [OR] 1.20 [95% confidence interval (CI), 1.00-1.44]), intermittent urine stream (OR 1.40 [95% CI, 1.15-1.69]), and urgency UI (OR 1.78 [95% CI, 1.22-2.60]) to be significant predictors of seeking treatment. Of those who had discussed LUTS with an HCP, 480 (70.6%) were currently receiving at least one LUTS treatment; the most common treatments were reducing fluid intake, pelvic exercises, oral anticholinergic medications, and avoiding certain foods/alcohol. CONCLUSIONS: LUTS are commonly experienced among people with MS but are largely untreated. Proper LUTS assessment and work-up is warranted in MS patients.

Relationship between self-reported depression and self-reported visual function in Latinos.

OBJECTIVE: To validate and assess the relationship between self-reported depression as measured by a single item on the Medical Outcomes Study Short-Form 12 (SF-12) and self-reported visual function. METHODS: The Los Angeles Latino Eye Study is population-based and designed to assess the prevalence of visual impairment, ocular disease, and visual functioning in Latinos. Both the 25-item National Eye Institute-Visual Function Questionnaire (NEI VFQ-25) (self-reported visual function) and the SF-12 (health-related quality of life) were administered. A single item from the SF-12 was used to measure self-reported depression and validated against the Center for Epidemiologic Studies-Depression measure of depression. Covariate-adjusted NEI VFQ-25 subscale scores were contrasted across the 6 response choices of the SF-12, as well as across 3 combined response categories of the SF-12 using analysis of covariance. Covariate-adjusted regression analyses assessed the contribution of self-reported depression in explaining self-reported visual function. RESULTS: The sensitivity and specificity of the SF-12 single item with the Center for Epidemiologic Studies-Depression measure was 0.96 and 0.50, respectively. Using the 3 combined response categories of the SF-12 single item, it was found that (1) all covariate-adjusted subscales of the NEI VFQ-25 were statistically significantly different across the self-reported depression categories (P<.001) and (2) covariate-adjusted self-reported depression was a significant predictor of self-reported visual function (P<.001). CONCLUSIONS: A single SF-12 item may be used as a measure of self-reported depression. In addition, self-reported depression is an important covariate to consider when assessing self-reported visual function in Latinos.

Using focus groups to develop a culturally sensitive methodology for epidemiological surveys in a Latino population: findings from the Los Angeles Latino Eye Study (LALES).

BACKGROUND: Many standardized survey instruments are initially developed in English-speaking populations. These instruments may not be culturally appropriate for other ethnic groups, such as Latinos. METHODS: The Los Angeles Latino Eye Study (LALES) was designed to determine the prevalence of blindness, visual impairment, and ocular diseases; risk factors for these diseases; and general and ocular healthcare use among non-institutionalized adult Latinos in suburban Los Angeles County, California. The LALES investigators used focus groups to develop questionnaires and to devise culturally sensitive methods for implementing this epidemiologic study in a Latino population. Eighty-four respondents were administered the in-home questionnaire and then scheduled into one of 6 in-home focus groups, allowing participants to reflect in groups about the study, their participation, and the state of the instruments and the translations. FINDINGS: Overall, respondents reported that the questionnaire was well written, easy to understand, and very clear. Recommendations from participants included: 1) keep questions brief; 2) minimize the number of response choices; 3) keep phrasing clear; 4) simplify technical terms; 5) provide test results immediately after the clinical examination; and 6) hire Spanish-speaking staff to increase participants' trust and confidence. CONCLUSION: Focus groups assisted in yielding a culturally appropriate survey research tool and methodology for this population-based study in a Latino community.

Psychometric performance of the incontinence quality-of-life questionnaire among patients with overactive bladder and urinary incontinence.

BACKGROUND: The Incontinence Quality-of-Life Instrument (I-QOL) is a condition-specific questionnaire that assesses the health-related QOL impact of urinary incontinence, but it has not been validated in patients with overactive bladder (OAB) who have been inadequately managed by anticholinergic therapy. OBJECTIVE: This study assessed the reliability and validity of the I-QOL among patients with OAB with urinary incontinence. METHODS: I-QOL scores were analyzed from a Phase II study that compared the efficacy and tolerability of onabotulinumtoxinA and placebo. Conceptual framework was confirmed via confirmatory factor analysis. Reliability was assessed using Cronbach's alpha and intraclass correlation coefficients (ICCs). Validity was tested by comparing I-QOL scores to tertiles of urinary symptom severity. Effect size statistics estimated the ability of the I-QOL to detect change. Responder analysis with cumulative distribution function was plotted to show differentiation between treatment groups with respect to I-QOL scores. RESULTS: Comparative fit indices ranged from 0.87 to 0.99 on the confirmatory factor analysis. I-QOL scores showed high internal consistency (0.86 ≤ Cronbach's α ≤ 0.93), good test-retest reliability (0.68 ≤ ICC ≤ 0.84), and good differentiation between tertiles of increasing urinary symptom severity (all, P ≤ 0.002). Significant differences in I-QOL change scores were noted between responders and nonresponders across all responder definitions (all, P < 0.001) and corresponded with large effect sizes among responders to treatment (1.34 ≤ effect size ≤ 2.82). CONCLUSION: This study demonstrated that OAB with urinary incontinence affects health-related QOL and that the I-QOL reliably and validly measures these impacts.

OnabotulinumtoxinA improves quality of life in patients with neurogenic detrusor overactivity.

OBJECTIVE: To evaluate the effects of onabotulinumtoxinA on patient-reported outcomes including health-related quality of life (HRQOL), treatment satisfaction, and treatment goal attainment in patients with urinary incontinence (UI) due to neurogenic detrusor overactivity (NDO). METHODS: In this multicenter, double-blind, randomized, placebo-controlled, phase III, 52-week study (ClinicalTrials.gov NCT00311376), patients with UI due to NDO who were not adequately managed with anticholinergic therapy were treated with intradetrusor injections of onabotulinumtoxinA (200 or 300 U) or placebo (0.9% saline). HRQOL measures included the Incontinence Quality of Life (I-QOL) Questionnaire total score, and the 3 domain scores (avoidance and limiting behavior, psychosocial, and social embarrassment), the modified Overactive Bladder Patient Satisfaction with Treatment Questionnaire (OAB-PSTQ), and Patient Global Assessment. Assessments were made at baseline, posttreatment week 6 (primary time point), week 12, and at 12-week intervals. RESULTS: Patients (mean age of 46 years with 30.5 weekly UI episodes at baseline) were randomized to receive placebo (n = 149) or onabotulinumtoxinA (200 U [n = 135] or 300 U [n = 132]). At week 6, improvements from baseline in I-QOL Questionnaire total score were greater (p < 0.001) in both onabotulinumtoxinA-treated groups vs placebo. Responses to the OAB-PSTQ also demonstrated greater mean improvements from baseline (p < 0.001) in both onabotulinumtoxinA-treated groups vs placebo at week 6. Patients who received onabotulinumtoxinA also reported greater improvement in the Patient Global Assessment than those in the placebo group (p ≤ 0.001 vs placebo). CONCLUSIONS: Patients with UI due to NDO reported greater improvement in HRQOL and treatment satisfaction with onabotulinumtoxinA than with placebo consistently across several patient-reported outcome instruments. CLASSIFICATION OF EVIDENCE: This study provides Class I evidence that onabotulinumtoxinA intradetrusor injections (200 or 300 U) can improve quality of life measures in patients with NDO not adequately managed with anticholinergic therapy.

Estimating the cost-effectiveness of onabotulinumtoxinA for neurogenic detrusor overactivity in the United States.

BACKGROUND: Urinary incontinence (UI) secondary to a neurogenic pathology, including spinal cord injury and multiple sclerosis, is termed neurogenic detrusor overactivity (NDO). Patients with NDO experience decreased quality of life and are at risk for upper urinary tract damage. Two recent trials demonstrated that onabotulinumtoxinA significantly reduced UI, improved urodynamic parameters, and improved quality of life relative to placebo. However, the economic impact of onabotulinumtoxinA treatment for UI due to NDO in the United States remains unknown. OBJECTIVE: The objective of this analysis was to evaluate whether the benefit observed in NDO patients receiving onabotulinumtoxinA provides good value for money. METHODS: We developed a Markov state transition model to estimate population outcomes and costs for anticholinergic-refractory NDO patients who received either onabotulinumtoxinA or best supportive care (use of incontinence pads with either an anticholinergic drug, clean intermittent self-catheterization, or both). Nonresponding patients (<50% reduction in UI episodes at 6 weeks) were eligible to receive invasive procedures, including augmentation cystoplasty or sacral neuromodulation. Patients could transition through 6 health states, 3 defined based on response to initial treatment, 2 capturing patients who underwent invasive procedures, and death. Time in each health state was adjusted for patient quality of life and summed to estimate quality-adjusted life-years (QALYs). The model included direct medical costs related to initial and subsequent drug and invasive treatments, physician visits, and catheterization. Outcomes and costs were summed and compared across intervention groups by using the incremental cost-effectiveness ratio (ICER; cost per QALY). The time horizon of the model was 3 years, and results were discounted at 3%. Scenario, 1-way, and probabilistic sensitivity analyses were performed to test the robustness of the model results. RESULTS: In the base case, onabotulinumtoxinA increased QALYs by 0.059 and costs by $1466 compared with best supportive care, which resulted in an estimated ICER of $24,720/QALY. OnabotulinumtoxinA also decreased mean UI episodes per person-year by 398, resulting in a cost of $4 per UI episode avoided. Model results were most sensitive to the probability of treatment response. The probabilistic sensitivity analysis indicated that at a willingness to pay of $50,000/QALY, onabotulinumtoxinA has a 97% probability of being cost-effective. In subgroup analyses of each etiology, onabotulinumtoxinA yielded an ICER of $32,268/QALY in multiple sclerosis and $2182 in spinal cord injury. CONCLUSION: OnabotulinumtoxinA seems to be a cost-effective intervention for UI due to NDO compared with best supportive care.

Time trends in 30 day case-fatality following hospitalisation for asthma in adults in Scotland: a retrospective cohort study from 1981 to 2009.

BACKGROUND: The risk of case-fatality following hospitalisation for asthma has not been well characterised. We describe trends in 30 day case-fatality following hospitalisation for asthma in adults in Scotland from 1981 to 2009. METHODS: Using the Scottish Morbidity Record Scheme (SMR01) with all asthma hospitalisations for adults (≥18 years) with ICD9 493 and ICD10 J45-J46 in the principal diagnostic position at discharge (1981-2009). These data were linked to mortality data from the General Register Office for Scotland (GROS), with asthma case-fatality defined as death within 30 days of asthma admission (in or out of hospital). Logistic regression was used to explore the impact of age, sex, previous asthma admission (in the 12 months prior to hospitalisation), socioeconomic deprivation, year of admission and co-morbidity on 30-day case-fatality. RESULTS: There were a total of 116,457 asthma hospitalisations; a total of 1000 (0.9%) hospitalisations resulted in a post-admission death (within 30 days of admission). Odds ratios for unadjusted and adjusted case-fatality showed a decreased risk of case-fatality from the mid-1990s onwards when compared to case-fatality in 1981. Advancing age and co-morbid diagnoses of respiratory failure, cancer, renal failure, cor pulmonale, coronary heart disease and respiratory infection were associated with increased likelihood of death. CONCLUSIONS: 30 day case-fatality has declined over the last three decades, comparable to case-fatality reported in other parts of the U.K. This decline may be in part due to improved guidelines, protocols and disease management for asthma over the last 30 years. The likelihood of death 30 days following an asthma admission increased with age group and was associated with respiratory failure, renal failure and cancer.

Surveillance and treatment of non-muscle-invasive bladder cancer in the USA.

Seventy percent of newly diagnosed bladder cancers are classified as non-muscle-invasive bladder cancer (NMIBC) and are often associated with high rates of recurrence that require lifelong surveillance. Currently available treatment options for NMIBC are associated with toxicities that limit their use, and actual practice patterns vary depending upon physician and patient characteristics. In addition, bladder cancer has a high economic and humanistic burden in the United States (US) population and has been cited as one of the most costly cancers to treat. An unmet need exists for new treatment options associated with fewer complications, better patient compliance, and decreased healthcare costs. Increased prevention of recurrence through greater adherence to evidence-based guidelines and the development of novel therapies could therefore result in substantial savings to the healthcare system.