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Gait Problems in Cerebral Palsy focuses on the surgical care and postoperative rehabilitation of children with cerebral palsy as they grow through early and late childhood, and adolescence. The book describes the neurologic and musculoskeletal issues that are frequently treated in children in each age group. It describes how to evaluate the children, create problem lists, make treatment choices, carry out interventions, and provide rehabilitation therapies. It: Emphasizes the critical importance of dialogue with children and parents, and age-related data derived from the GOAL questionnaire is provided to focus insight on the priorities of patients and families. Presents descriptions of patient and family priorities, decision-making and surgical techniques, and rehabilitation therapies for each age group. Offers consistent chapter structures that emphasize clarity and brevity, supplemented by figures and tables. Highlights the importance of building collaborative multidisciplinary clinical teams. Includes access to numerous videos which demonstrate clinical teams at work with patients, surgical techniques, and rehabilitation activities.   Essential reading for orthopaedic surgeons, physiatrists, physical therapists, kinesiologists, gait analysis experts, and other members of the interdisciplinary team involved.  

Purpose An essential aspect of patient-centered outcomes research (PCOR) and comparative effectiveness research (CER) is the integration of patient perspectives and experiences with clinical data to evaluate interventions. Thus, PCOR and CER require capturing patient-reported outcome (PRO) data appropriately to inform research, healthcare delivery, and policy. This initiative’s goal was to identify minimum standards for the design and selection of a PRO measure for use in PCOR and CER. Methods We performed a literature review to find existing guidelines for the selection of PRO measures. We also conducted an online survey of the International Society for Quality of Life Research (ISOQOL) membership to solicit input on PRO standards. A standard was designated as “recommended” when >50 % respondents endorsed it as “required as a minimum standard.” Results The literature review identified 387 articles. Survey response rate was 120 of 506 ISOQOL members. The respondents had an average of 15 years experience in PRO research, and 89 % felt competent or very competent providing feedback. Final recommendations for PRO measure standards included: documentation of the conceptual and measurement model; evidence for reliability, validity (content validity, construct validity, responsiveness); interpretability of scores; quality translation, and acceptable patient and investigator burden. Conclusion The development of these minimum measurement standards is intended to promote the appropriate use of PRO measures to inform PCOR and CER, which in turn can improve the effectiveness and efficiency of healthcare delivery. A next step is to expand these minimum standards to identify best practices for selecting decision-relevant PRO measures.

Purpose To assess the impact of providing healthcare professionals with feedback on patient-reported outcome measures (PROMs). Methods This is a systematic review including controlled studies investigating the effectiveness of PROMs feedback, specifically examining the impact at a group-level and a patient-level. Results Only one study provided feedback at a group-level as a measure of professional performance, which found no intervention effect. At a patient-level, sixteen studies were identified and only one study found an overall significant difference in the PROM score. However, an additional six studies found a significant result favouring the intervention group for a particular subgroup or domain. The studies which demonstrated the greatest impact primarily used PROMs as a management tool in an outpatient setting on a specialised patient population. In contrast, there was weak evidence supporting with the use of PROMs as a screening tool. The studies which found a positive effect had a lower quality score on average. Conclusions The effectiveness of PROMs feedback seems to be related to the function of the PROM. However, the evidence regarding the impact of PROMs feedback on patient outcomes is weak, and methodological issues with studies are frequent. The use of PROMs as a performance measure is not well investigated. Future research should focus on the appropriate application of PROMs by testing specific hypothesis related to cause and effect. Qualitative research is required to provide a deeper understanding of the practical issues surrounding the implementation of PROMs and the methodological issues associated with the effective use of the information.

AbstractObjectivesTo use the estimates from the Global Burden of Disease Study 2016 to describe patterns of suicide mortality globally, regionally, and for 195 countries and territories by age, sex, and Socio-demographic index, and to describe temporal trends between 1990 and 2016.DesignSystematic analysis.Main outcome measuresCrude and age standardised rates from suicide mortality and years of life lost were compared across regions and countries, and by age, sex, and Socio-demographic index (a composite measure of fertility, income, and education).ResultsThe total number of deaths from suicide increased by 6.7% (95% uncertainty interval 0.4% to 15.6%) globally over the 27 year study period to 817 000 (762 000 to 884 000) deaths in 2016. However, the age standardised mortality rate for suicide decreased by 32.7% (27.2% to 36.6%) worldwide between 1990 and 2016, similar to the decline in the global age standardised mortality rate of 30.6%. Suicide was the leading cause of age standardised years of life lost in the Global Burden of Disease region of high income Asia Pacific and was among the top 10 leading causes in eastern Europe, central Europe, western Europe, central Asia, Australasia, southern Latin America, and high income North America. Rates for men were higher than for women across regions, countries, and age groups, except for the 15 to 19 age group. There was variation in the female to male ratio, with higher ratios at lower levels of Socio-demographic index. Women experienced greater decreases in mortality rates (49.0%, 95% uncertainty interval 42.6% to 54.6%) than men (23.8%, 15.6% to 32.7%).ConclusionsAge standardised mortality rates for suicide have greatly reduced since 1990, but suicide remains an important contributor to mortality worldwide. Suicide mortality was variable across locations, between sexes, and between age groups. Suicide prevention strategies can be targeted towards vulnerable populations if they are informed by variations in mortality rates.

Background Neoadjuvant chemoradiotherapy (nCRT) plus surgery is a standard treatment for locally advanced oesophageal cancer. With this treatment, 29% of patients have a pathologically complete response in the resection specimen. This provides the rationale for investigating an active surveillance approach. The aim of this study is to assess the (cost-)effectiveness of active surveillance vs. standard oesophagectomy after nCRT for oesophageal cancer. Methods This is a phase-III multi-centre, stepped-wedge cluster randomised controlled trial. A total of 300 patients with clinically complete response (cCR, i.e. no local or disseminated disease proven by histology) after nCRT will be randomised to show non-inferiority of active surveillance to standard oesophagectomy (non-inferiority margin 15%, intra-correlation coefficient 0.02, power 80%, 2-sided α 0.05, 12% drop-out). Patients will undergo a first clinical response evaluation (CRE-I) 4–6 weeks after nCRT, consisting of endoscopy with bite-on-bite biopsies of the primary tumour site and other suspected lesions. Clinically complete responders will undergo a second CRE (CRE-II), 6–8 weeks after CRE-I. CRE-II will include 18F–FDG-PET-CT, followed by endoscopy with bite-on-bite biopsies and ultra-endosonography plus fine needle aspiration of suspected lymph nodes and/or PET- positive lesions. Patients with cCR at CRE-II will be assigned to oesophagectomy (first phase) or active surveillance (second phase of the study). The duration of the first phase is determined randomly over the 12 centres, i.e., stepped-wedge cluster design. Patients in the active surveillance arm will undergo diagnostic evaluations similar to CRE-II at 6/9/12/16/20/24/30/36/48 and 60 months after nCRT. In this arm, oesophagectomy will be offered only to patients in whom locoregional regrowth is highly suspected or proven, without distant dissemination. The main study parameter is overall survival; secondary endpoints include percentage of patients who do not undergo surgery, quality of life, clinical irresectability (cT4b) rate, radical resection rate, postoperative complications, progression-free survival, distant dissemination rate, and cost-effectiveness. We hypothesise that active surveillance leads to non-inferior survival, improved quality of life and a reduction in costs, compared to standard oesophagectomy. Discussion If active surveillance and surgery as needed after nCRT leads to non-inferior survival compared to standard oesophagectomy, this organ-sparing approach can be implemented as a standard of care.

This book defends medical nihilism, which is the view that we should have little confidence in the effectiveness of medical interventions. If we consider the frequency of failed medical interventions, the extent of misleading evidence in medical research, the thin theoretical basis of many interventions, and the malleability of empirical methods in medicine, and if we employ our best inductive framework, then our confidence in the effectiveness of medical interventions ought to be low. Part I articulates theoretical and conceptual groundwork, which offers a defense of a hybrid theory of disease, which forms the basis of a novel account of effectiveness, and this is applied to pharmacological science and to issues such as medicalization. Part II critically examines details of medical research. Even the very best methods in medical research, such as randomized trials and meta-analyses, are malleable and suffer from various biases. Methods of measuring the effectiveness of medical interventions systematically overestimate benefits and underestimate harms. Part III summarizes the arguments for medical nihilism and what this position entails for medical research and practice. To evaluate medical nihilism with care, the argument is stated in formal terms. Medical nihilism suggests that medical research must be modified, that clinical practice should be less aggressive in its therapeutic approaches, and that regulatory standards should be enhanced.

Background Precisely defining the different applications of patient-reported outcome measures (PROs) in clinical practice can be difficult. This is because the intervention is complex and varies amongst different studies in terms of the type of PRO used, how the PRO is fed back, and to whom it is fed back. Methods A theory-driven approach is used to describe six different applications of PROs in clinical practice. The evidence for the impact of these applications on the process and outcomes of care are summarised. Possible explanations for the limited impact of PROs on patient management are then discussed and directions for future research are highlighted. Results The applications of PROs in clinical practice include screening tools, monitoring tools, as a method of promoting patient-centred care, as a decision aid, as a method of facilitating communication amongst multidisciplinary teams (MDTs), and as a means of monitoring the quality of patient care. Evidence from randomised controlled trials suggests that the use of PROs in clinical practice is valuable in improving the discussion and detection of HRQoL problems but has less of an impact on how clinicians manage patient problems or on subsequent patient outcomes. Many of the reasons for this may lie in the ways in which PROs fit (or do not fit) into the routine ways in which patients and clinicians communicate with each other, how clinicians make decisions, and how healthcare as a whole is organised. Conclusions Future research needs to identify ways in with PROs can be better incorporated into the routine care of patients by combining qualitative and quantitative methods and adopting appropriate trial designs.

Purpose To develop expert consensus on a suite of reporting standards for HRQL outcomes of RCTs. Methods A Task Force of The International Society of Quality of Life Research (ISOQOL) undertook a systematic review of the literature to identify candidate reporting standards for HRQL in RCTs. Subsequently, a web-based survey was circulated to the ISOQOL membership. Respondents were asked to rate candidate standards on a 4-point Likert scale based on their perceived value in reporting studies in which HRQL was a study outcome (primary or secondary). Results were synthesized into draft reporting guidelines, which were further reviewed by the membership to inform the final guidance. Results Forty-six existing candidate standards for reporting HRQL results in RCTs were synthesized to produce a 40 item survey that was completed electronically by 161 respondents. The majority of respondents rated all 40 items to be either 'essential' or 'desirable' when HRQL was a primary RCT outcome. Ratings changed when HRQL was a secondary study outcome. Feedback on the survey findings resulted in the Task Force generalizing the guidance to include patient-reported outcomes (PROs). The final guidance, which recommends standards for use in reporting PROs generally, and more specifically, for PROs identified as primary study outcomes, was approved by the ISOQOL Board of Directors. Conclusions ISOQOL has developed a suite of recommended standards for reporting PRO results of RCTs. Improved reporting of PROs will enable accurate interpretation of evidence to inform patient choice, aid clinical decision making, and inform health policy.

Artificial Intelligence for Improved Patient Outcomes provides new, relevant, and practical information on what AI can do in healthcare and how to assess whether AI is improving health outcomes.  With clear insights and a balanced approach, this innovative book offers a one-stop guide on how to design and lead pragmatic real-world AI studies that yield rigorous scientific evidence-all in a manner that is safe and ethical. Daniel Byrne, Director of Artificial Intelligence Research at AVAIL (the Advanced Vanderbilt Artificial Intelligence Laboratory) and author of landmark pragmatic studies published in leading medical journals, shares four decades of experience as a biostatistician and AI researcher. Building on his first book, Publishing Your Medical Research, the author gives the reader the competitive advantage in creating reproducible AI research that will be accepted in prestigious high-impact medical journals.

Lack of standardization of outcome measures limits the usefulness of clinical trial evidence to inform health care decisions. This can be addressed by agreeing on a minimum core set of outcome measures per health condition, containing measures relevant to patients and decision makers. Since 1992, the Outcome Measures in Rheumatology (OMERACT) consensus initiative has successfully developed core sets for many rheumatologic conditions, actively involving patients since 2002. Its expanding scope required an explicit formulation of its underlying conceptual framework and process. Literature searches and iterative consensus process (surveys and group meetings) of stakeholders including patients, health professionals, and methodologists within and outside rheumatology. To comprehensively sample patient-centered and intervention-specific outcomes, a framework emerged that comprises three core “Areas,” namely Death, Life Impact, and Pathophysiological Manifestations; and one strongly recommended Resource Use. Through literature review and consensus process, core set development for any specific health condition starts by identifying at least one core “Domain” within each of the Areas to formulate the “Core Domain Set.” Next, at least one applicable measurement instrument for each core Domain is identified to formulate a “Core Outcome Measurement Set.” Each instrument must prove to be truthful (valid), discriminative, and feasible. In 2012, 96% of the voting participants (n=125) at the OMERACT 11 consensus conference endorsed this model and process. The OMERACT Filter 2.0 explicitly describes a comprehensive conceptual framework and a recommended process to develop core outcome measurement sets for rheumatology likely to be useful as a template in other areas of health care.