Neuromodulation as a treatment for overactive bladder syndrome

- Neuromodulation is used as a treatment for overactive bladder syndrome, if conservative management is ineffective or only partially effective.- Sacral neuromodulation is now a minimally invasive treatment as a result of improvements in surgical technique, and it has good long-term results. Nevertheless the risk of complications such as pain and decreasing effect is still present.- Developments in alternative neuromodulation treatments, such as posterior tibial nerve stimulation, pudendal nerve stimulation and intravesical injection therapy with botulinum neurotoxin, have extended the role of neuromodulation.- Intravesical injection therapy with botulinum toxin, along with sacral neuromodulation, has been included in the treatment algorithm of overactive bladder syndrome if this does not respond to conservative therapy.- When choosing for a neuromodulation therapy, the side-effects and complications of the treatments should be considered alongside the specific patient situation and evidence for effectiveness and long-term effectiveness.     

Prevalence of overactive bladder and urinary incontinence

We conducted a cross-sectional study in Italy among men at least 50 years old and women at least 40 years old who consecutively visited their general practitioners. Patients were asked about the frequency of symptoms of overactive bladder and urinary incontinence. A total of 9613 men (mean age, 64.8 years; range, 50-98 years) and 13,365 women (mean age, 60.3 years; range, 40-98 years) were identified by 774 general practitioners. The frequencies of overactive bladder were 3.0% (95% confidence interval, 2.7-3.5) in men and 1.1% (95% confidence interval, 0.9-1.3) in women. The corresponding frequencies for urinary incontinence were 8.3% (95% confidence interval, 7.7-8.9) in men and 10.2% (95% confidence interval, 9.6-10.8) in women.     

Using item response theory improved responsiveness of patient-reported outcomes measures in carpal tunnel syndrome

ObjectiveTo compare responsiveness based on item response theory (IRT) with that based on conventional scoring for two patient-reported outcomes measures in carpal tunnel syndrome (CTS); the short disabilities of the arm, shoulder, and hand (QuickDASH) measure, and the 6-item CTS symptoms scale (CTS-6).Study Design and SettingProspective cohort study of patients with CTS undergoing carpal tunnel release at one orthopedic department. Of 455 consecutive patients, 343 completed the QuickDASH and the CTS-6 before and within 1 year after surgery. IRT-based and conventional scores were compared in subgroups according to global rating of change in hand status and treatment satisfaction. The effect size (ES) and the area under the receiver operating characteristic (ROC) curve were used as measures of responsiveness.ResultsThe mean value for the IRT-based QuickDASH estimate was ?0.09 (standard deviation [SD] = 1.13) preoperatively and ?2.14 (SD = 1.79) postoperatively (ES = ?1.8) and for the CTS-6 estimate was 0.29 (SD = 1.36) preoperatively and ?3.87 (SD = 2.3) postoperatively (ES = ?3.1), indicating very large improvement. The ES for the QuickDASH and CTS-6 were very large (?2.4 and ?3.8), respectively, in the group with the largest perceived improvement and decreased with lower perceived improvement. The ES was consistently larger with IRT-based scoring than conventional scoring. The AUC for the QuickDASH and CTS-6 exceeded 0.85.ConclusionIRT-based scoring showed high responsiveness for the QuickDASH and CTS-6, and the ES were larger than those estimated using conventional scoring.     

The causes and consequences of overactive bladder

Overactive bladder (OAB) is a syndrome characterized by symptoms of urinary urgency with or without urgency urinary incontinence (UUI), usually with frequency and nocturia. OAB affects approximately 17% of women in the United States and Europe. The causes of OAB, as with many bladder disorders, are multifactorial and are not completely understood. The primary functions of the lower urinary tract (bladder and bladder outlet mechanism) are storage and evacuation of urine. The bladder and the micturition cycle are under complex neural control involving both the sympathetic and parasympathetic nervous systems. Micturition may occur in response to the activation of receptors in the bladder muscle and detection of chemical stimuli by receptors within the bladder lining. Neurogenic or myogenic bladder dysfunction can lead to the symptoms of urgency, frequency, and UUI that characterize OAB. The consequences of this condition are far-reaching and include direct medical consequences and coping strategies that adversely affect quality of life. Although the prevalence of OAB increases with age, it is not a normal consequence of aging. Antimuscarinic agents (e.g., oxybutynin, tolterodine, trospium, solifenacin, and darifenacin) have demonstrated efficacy for the treatment of OAB symptoms in multiple clinical trials. This review explores the physiological basis for OAB, the effects of OAB on health-related quality of life, and the pharmacotherapies that may provide relief to patients with this distressing condition.     

Incorporating patient-reported outcomes in dose-finding clinical trials

Oncology dose-finding clinical trials determine the maximum tolerated dose (MTD) based on toxicity outcomes captured by clinicians. With the availability of more rigorous instruments for measuring toxicity directly from patients, there is a growing interest to incorporate patient-reported outcomes (PRO) in clinical trials to inform patient tolerability. This is particularly important for dose-finding trials to ensure the identification of a well-tolerated dose. In this paper, we propose three extensions of the continual reassessment method (CRM), termed PRO-CRMs, that incorporate both clinician and patient outcomes. The first method is a marginal modeling approach whereby clinician and patient toxicity outcomes are modeled separately. The other two methods impose a constraint using a joint outcome defined based on both clinician and patient toxicities and model them either jointly or marginally. Simulation studies show that while all three PRO-CRMs select well-tolerated doses based on clinician's and patient's perspectives, the methods using a joint outcome perform better and have similar performance. We also show that the proposed PRO-CRMs are consistent under robust model assumptions.     

患者报告结局的国内外研究进展

患者报告结局是指直接来自患者的有关患者健康状态的任何方面的信息,广泛应用于健康相关生活质量、临床试验、不良事件监测及批准药物等.患者自主报告比医生报告对患者自身状态改变更敏感,可在治疗早期就发现异常症状.患者自主报告主观性不良事件与整体健康状况一致性远远高于医生报告.将患者报告的不良事件及症状感受纳入到临床试验结局指标评价体系是近几年癌症研究领域的热点.本文就患者报告结局的概念、内容、测评工具及应用的研究进展进行综述,以期为未来开展相关研究提供指导.     

Prospects and challenges in using patient-reported outcomes in clinical practice

Background  Patient-reported measures include preferences and reports about care received, health behaviors, and outcomes of care (patient satisfaction and health-related quality of life). These measures are a core aspect of health care, but there is much to be learned about how to use them to improve clinical practice. Method  We specify linkages among different patient-reported measures and focus upon the prospects and challenges for use of patient-reported outcomes in clinical practice. Results  Patient-reported measures are important throughout the continuum of patient care. At the initial visit, patient-reported outcomes provide information about what is important to the patient, the patient’s current behaviors, and the patient’s baseline health-related quality of life. At subsequent visits, patient-reported outcomes help evaluate disease progression or regression as well as treatment effects. Conclusions  Patient-reported measures can help clinicians target interventions that will improve patient outcomes of care. However, there are a number of challenges in using patient-reported outcomes in clinical practice.     

Using patient-reported outcomes in clinical practice: challenges and opportunities

Purpose

Introduce and explore issues at an international conference about the use of patient-reported outcomes (PROs) in clinical practice.

Methods

Review of salient literature, clinical and personal experiences, conference presentations and discussions, and post-conference comments from outside experts.

Results

PROs (information from patients about a health condition and its management) have been assessed through self-reports for at least four decades. Traditional applications are in clinical and health services research. Uses in clinical practice, although increasing, are less common and more challenging. PROs can enhance the understanding of patients’ experiences and responses to therapy and inform clinical practice.

Conclusions

We pose and discuss four main questions: (1) Will clinicians accept PRO measures? (2) Will clinicians use PRO measures? (3) Will measuring PROs actually improve those outcomes? (4) Will PROs be perceived as having other, less salutary purposes? A patient-centered perspective on PRO measurement presents issues about the extent to which PROs can accurately capture patient experiences and assess psychosocial and environmental factors that influence communication with clinicians and eventual outcomes. We end with comments about the intersection of PROs and bioethics, noting contributions that PROs may make to beneficence, patient autonomy, and distributive justice.     

Drug compliance, co-payment and health outcomes: evidence from a panel of Italian patients

This paper studies the relationship between medical compliance and health outcomes - hospitalization and mortality rates - using a large panel of patients residing in a local health authority in Italy. These data allow us to follow individual patients through all their accesses to public health care services until they either die or leave the local health authority. We adopt a disease specific approach, concentrating on hypertensive patients treated with ACE-inhibitors. Our results show that medical compliance has a clear effect on both hospitalization and mortality rates: health outcomes clearly improve when patients become more compliant to drug therapy. At the same time, we are able to infer valuable information on the role that drug co-payment can have on compliance, and as a consequence on health outcomes, by exploiting the presence of two natural experiments during the period of analysis. Our results show that drug co-payment has a strong effect on compliance, and that this effect is immediate.     

The importance of rating scales in measuring patient-reported outcomes

ABSTRACT: BACKGROUND: A critical component that influences the measurement properties of a patient-reportedoutcome (PRO) instrument is the rating scale. Yet, there is a lack of general consensusregarding optimal rating scale format, including aspects of question structure, the number andthe labels of response categories. This study aims to explore the characteristics of ratingscales that function well and those that do not, and thereby develop guidelines forformulating rating scales. METHODS: Seventeen existing PROs designed to measure vision-related quality of life dimensions weremailed for self-administration, in sets of 10, to patients who were on a waiting list for cataractextraction. These PROs included questions with ratings of difficulty, frequency, severity, and global ratings. Using Rasch analysis, performance of rating scales were assessed byexamining hierarchical ordering (indicating categories are distinct from each other and followa logical transition from lower to higher value), evenness (indicating relative utilization ofcategories), and range (indicating coverage of the attribute by the rating scale). RESULTS: The rating scales with complicated question format, a large number of response categories, orunlabelled categories, tended to be dysfunctional. Rating scales with five or fewer responsecategories tended to be functional. Most of the rating scales measuring difficulty performedwell. The rating scales measuring frequency and severity demonstrated hierarchical orderingbut the categories lacked even utilization. CONCLUSION: Developers of PRO instruments should use a simple question format, fewer (four to five) andlabelled response categories.     

The importance of rating scales in measuring patient-reported outcomes

Background

Discrepancy between self-reports and parent-proxy reports of adolescent health-related quality of life (HRQoL) has been repeatedly acknowledged in the literature as the proxy problem. However, little is known about the extent and direction of this discrepancy. The purpose of this study is to explore to what extent and in what direction HRQoL self-reports of adolescents with chronic conditions and those of their parents differ.

Methods

A cross-sectional survey was conducted among adolescents suffering from chronic conditions and their parents. Socio-demographic and disease-related characteristics were collected and information about consequences of the chronic condition was assessed. HRQoL was measured with KIDSCREEN-10 and DISABKIDS condition generic measure (DCGM-10). Agreement was analysed through defining a threshold of agreement based on half of the standard deviation of the HRQoL score with the highest variance. Agreement occurred if the difference between adolescent and parent scores was less than or equal to half of the standard deviation. Intra-class correlation coefficients and Bland-Altman plots were also computed. The characteristics associated with direction of disagreement were statistically tested with one-way ANOVA and Chi-square tests.

Results

584 paired HRQoL scores were obtained. Ratings from both adolescents and parents were high, compared to European norm data. Differences between adolescents and parents were statistically significant, yet relatively small. Disagreement existed in both directions: in 24.5% (KIDSCREEN-10) and 16.8% (DCGM-10) of the cases adolescents rated their HRQoL lower than did their parent, while in 32.2% (KIDSCREEN-10) and 31.7% (DCGM-10) of the cases the opposite was true. Adolescent's age, educational level and type of education, parent's educational level, number of hospital admissions and several other disease-related factors influenced direction of disagreement.

Conclusions

In a reasonable proportion of cases the adolescent and parent agreed on the adolescent's HRQoL (43-51% of the cases) and most disagreement tended to be minor. Thus, the proxy problem may be smaller than presented in the literature and its extent may differ per population. As adolescents are expected to become partners in their own health care, it is recommended to focus on adolescents' own perceptions of HRQoL.     

Patient data work: filtering and sensing patient-reported outcomes

With digital patient-reported outcome (PRO) tools in clinical practice, patients are given new tasks of providing data that aim at supporting and individualising care, simultaneously reducing unnecessary clinical visits. While the innovative potential of mobilising PRO data for care is increasingly explored, little attention is given to the efforts that the provision of PRO data rests on – that of the patients. Based on ethnographic fieldwork carried out among cancer patients receiving PRO-based follow-up care, we argue that with the increased reliance on patient-generated health data, we need to consider patients’ data work. Drawing on emerging literature on healthcare professionals’ data work and the rich work in sociology and Science and Technology Studies (STS) on patients’ active engagement in shaping and managing care, we conceptualise PRO patient data work as two simultaneous processes: the process of data filtering – patients filter information to fit the envisaged recipient and purpose; and the process of data sensing – patients evaluate their embodied experiences. By doing so, we show that patients’ data work has implications beyond simply providing data that represent their experiences.     

Electronic patient-reported outcomes and toxicities during radiotherapy for head-and-neck cancer

Purpose

To characterize quality of life (QOL) using real-time, electronic patient-reported outcomes (ePROs) and to evaluate adverse events (AEs) and supportive care during head-and-neck radiotherapy (RT) and concurrent chemoradiotherapy (CCRT).

Methods

Sixty-five patients undergoing head-and-neck RT completed electronic, real-time, 12-item linear analog self-assessments (LASA) at baseline, before biweekly appointments, and at the last week of RT. Changes in QOL domains between time points were calculated. Clinical data were collected from the institutional medical record. AEs were recorded at the same time points as the LASA and graded.

Results

During head-and-neck RT, most patients had clinically meaningful decreases in all QOL domains except level of support, financial concerns, and legal concerns. QOL domains with the most prevalent, clinically meaningful decreases were fatigue (75.4% of patients; 95% CI, 62.9–84.9%), social activity (70.8%; 95% CI, 58.0–81.1%), and overall QOL (70.8%; 95% CI, 58.0–81.1%). All patients had grade 2 AEs; 35.4% had grade 3 (50.0%, CCRT; 12.0%, RT; P?=?.002). Weight loss averaged 5.5 kg (6.9 kg, CCRT; 2.8 kg, RT; P?<?.001). Intravenous hydration was needed in 52.3% (77.5%, CCRT; 12.0%, RT; P?<?.001); feeding tube placement 40.0% (57.5%, CCRT; 12.0%, RT; P?=?.001); emergency department visits without hospitalization, 10.8%; and emergent hospitalization, 27.7% (37.5%, CCRT; 12.0%, RT; P?=?.04).

Conclusions

Head-and-neck RT, particularly CCRT, negatively impacts patients’ overall QOL, social activity, and fatigue, with frequent grade 3 AEs, weight loss, intravenous hydration, feeding tube placement, ED visits, and hospitalization. Real-time ePROs allow providers to monitor QOL at multiple time points during RT, potentially allowing early intervention to improve QOL and mitigate AEs.

     

Item response theory and its applications to patient-reported outcomes measurement

This article provides an overview of item response theory (IRT) models and how they can be appropriately applied to patient-reported outcomes (PROs) measurement. Specifically, the following topics are discussed: (a) basics of IRT, (b) types of IRT models, (c) how IRT models have been applied to date, and (d) new directions in applying IRT to PRO measurements.     

Ambulatory and diary methods can facilitate the measurement of patient-reported outcomes

Purpose

Ambulatory and diary methods of self-reported symptoms and well-being have received increasing interest in recent years. These methods are a valuable addition to traditional strategies for the assessment of patient-reported outcomes (PROs) in that they capture patients’ recent symptom experiences repeatedly in their natural environments. In this article, we review ways that incorporating diary methods into PRO measurement can facilitate research on quality of life.

Methods

Several diary methods are currently available, and they include “real-time” (Ecological Momentary Assessment) and “near-real-time” (end-of-day assessments, Day Reconstruction Method) formats. We identify the key benefits of these methods for PRO research.

Results

(1) In validity testing, diary assessments can serve as a standard for evaluating the ecological validity and for identifying recall biases of PRO instruments with longer-term recall formats. (2) In research and clinical settings, diaries have the ability to closely capture variations and dynamic changes in quality of life that are difficult or not possible to obtain from traditional PRO assessments. (3) In test construction, repeated diary assessments can expand understanding of the measurement characteristics (e.g., reliability, dimensionality) of PROs in that parameters for differences between people can be compared with those for variation within people.

Conclusions

Diary assessment strategies can enrich the repertoire of PRO assessment tools and enhance the measurement of patients’ quality of life.

     

Issues in the design of Internet-based systems for collecting patient-reported outcomes

Background and objectives Although there is a growing interest in using patient-reported outcomes (PRO) to monitor disease progression and/or therapeutic response, to improve care, and to screen for physical or psychosocial problems in routine clinical practice, PRO instruments can be difficult to administer, score, and interpret in this setting. Internet-based approaches to PRO collection may help overcome these obstacles. This paper discusses the rationale for using the Internet for routine PRO collection, summarizes relevant literature and ongoing projects, and raises several key design and development issues that should guide further efforts in this area. Major findings A small number of Internet-based PRO collection applications have been or are currently being developed. The major characteristics of several of these projects are reviewed and summarized. Successful Internet-based PRO collection applications must address patient and clinician-specific needs related to workflow and to the way in which results are presented. A growing number of instruments have been adapted for and evaluated in a web-based format. Conclusions Collecting PROs via the Internet has the potential to overcome many of the challenges associated with efforts to routinely use PROs in the clinical encounter. James B. Jones, Claire F. Snyder, and Albert W. Wu for the “Website for Outpatient QOL Assessment” Research Network.     

A review of patient-reported outcomes for children and adolescents with obesity

Introduction

Obesity is a chronic condition that can impact the physical, emotional, mental and social elements that encompass a child’s life. The objectives of this study were to identify which generic and obesity-specific patient-reported outcome (PRO) instruments are used in obesity literature, as well as review their conceptual approach, health and health-related content, ethical content and psychometric properties.

Method

PubMed, CINAHL, EMBASE and PsycINFO were searched from the inception of each database to May 2012 to identify all studies using multi-dimensional PRO instruments with children who are overweight or obese. The most common generic and all obesity-specific instruments were analyzed according to the study objectives.

Results

From 4,226 articles identified by our search, 70 articles used 6 generic and 4 obesity-specific PRO instruments. While the most commonly used PRO instrument was the generic PedsQL 4.0 (used in 53 studies), many health domains were found in the obesity-specific instruments that are not measured by the PedsQL 4.0. Summary of the development and psychometric properties of the generic and obesity PROs identified that no one instrument meets all the guideline criteria for instrument development and validation, e.g., only one instrument included qualitative input from children with obesity in the content development phase.

Discussion

This comprehensive review provides information to aid in selecting multi-dimensional PRO instruments in children with obesity according to various aspects of content as well as psychometric properties. The conceptual analysis shows that the reviewed PRO instruments contain inconsistencies in their conceptual approaches. Also, certain relevant health domains to children and youth with obesity were not included in the most commonly used generic instrument. The obesity-specific instruments require further validation before they can be used in intervention studies.