Patient-reported outcomes of periacetabular osteotomy from the prospective ANCHOR cohort study

BACKGROUND: Current literature describing the periacetabular osteotomy (PAO) is mostly limited to retrospective case series. Larger, prospective cohort studies are needed to provide better clinical evidence regarding this procedure. The goals of the current study were to (1) report minimum 2-year patient-reported outcomes (pain, hip function, activity, overall health, and quality of life), (2) investigate preoperative clinical and disease characteristics as predictors of clinical outcomes, and (3) report the rate of early failures and reoperations in patients undergoing contemporary PAO surgery. METHODS: A large, prospective, multicenter cohort of PAO procedures was established, and outcomes at a minimum of 2 years were analyzed. A total of 391 hips were included for analysis (79% of the patients were female, and the average patient age was 25.4 years). Patient-reported outcomes, conversion to total hip replacement, reoperations, and major complications were documented. Variables with a p value of ≤0.10 in the univariate linear regressions were included in the multivariate linear regression. The backward stepwise selection method was used to determine the final risk factors of clinical outcomes. RESULTS: Clinical outcome analysis demonstrated major clinically important improvements in pain, function, quality of life, overall health, and activity level. Increasing age and a body mass index status of overweight or obese were predictive of improved results for certain outcome metrics. Male sex and mild acetabular dysplasia were predictive of lesser improvements in certain outcome measures. Three (0.8%) of the hips underwent early conversion to total hip arthroplasty, 12 (3%) required reoperation, and 26 (7%) experienced a major complication. CONCLUSIONS: This large, prospective cohort study demonstrated the clinical success of contemporary PAO surgery for the treatment of symptomatic acetabular dysplasia. Patient and disease characteristics demonstrated predictive value that should be considered in surgical decision-making. LEVEL OF EVIDENCE: Therapeutic Level IV. See Instructions for Authors for a complete description of levels of evidence

Myocardial ischemia after orthotopic liver transplantation

A hypercoagulable state exists after orthotopic liver transplantation. This hematologic abnormality may predispose patients to coronary thrombosis and unstable angina. The incidence of post-operative myocardial ischemia in such patients is unknown. Suitable electrocardiograms and clinical events of consecutive patients undergoing orthotopic liver transplantation (n = 45) and major intraabdominal surgery (n = 28) during a 3-month period at a major university teaching hospital and transplant center were examined retrospectively. Clinical myocardial ischemia or ischemic electrocardiographic changes, or both, occurred in 6 transplant patients compared with no patient in the nontransplant or comparison group. In 4 of the 6 patients with dramatic electrocardiographic changes and ischemic events, coronary arteriography failed to demonstrate significant obstructive disease. It is concluded that severe myocardial ischemia may occur in patients after orthotopic liver transplantation in the absence of significant coronary disease. A hypercoagulable state may predispose to coronary thrombosis in this setting, providing insight (and a future model for study) into the development of unstable angina. © 1994

Keeping company with hope and despair. Family therapists' reflections and experience of working with childhood depression

The BIOMED international outcome study on childhood depression offered a unique opportunity for the systematic treatment of children and families with major depression using systemic psychotherapy. This paper describes the experiences of clinicians working with the families referred and the theoretical and clinical models that evolved during the treatment process. The concept of 'keeping company with hope and despair' emerged as an overarching framework for thinking about the quality of the therapeutic relationship in this developing area of clinical practice. We illustrate our systemically informed interventions with case examples and discuss the role played by therapists' 'use of self' in engaging and fostering change in families gripped by depression

Exploring Meaningful Patient Engagement in ADAPTABLE (Aspirin Dosing: A Patient-centric Trial Assessing Benefits and Long-term Effectiveness).

BackgroundGenuine patient engagement can improve research relevance, impact and is required for studies using the National Patient-Centered Clinical Research Network including major multicenter research projects. It is unclear, however, how best to integrate patients into governance of such projects.MethodsADAPTABLE (Aspirin Dosing: A Patient-centric Trial Assessing Benefits and Long-term Effectiveness) is the first major multicenter research project to be conducted in National Patient-Centered Clinical Research Network. Here, we provide a description of how we implemented patient engagement in ADAPTABLE thus far, including a description of committee structures and composition, first-hand patient testimonials, specific contributions, and lessons learned during the planning and early implementation of ADAPTABLE.ResultsWe recruited 1 patient leader from 6 of the 7 enrolling networks to serve on a Patient Review Board for ADAPTABLE, supported the Board with an experienced patient engagement team including an "investigator-advocate" not otherwise involved in the trial, and facilitated bidirectional communication between the Board and ADAPTABLE Coordinating Center. The Board has reviewed and provided substantial input on the informed consent procedure, recruitment materials, patient portal design, and study policy including compensation of participants. Although it was "too late" for some suggested modifications, most modifications suggested by the patient leaders have been implemented, and they are enthusiastic about the study and their role. The patient leaders also attend Steering and Executive Committee calls; these experiences have been somewhat less productive.ConclusionsWith adequate support, a cadre of committed patient leaders can provide substantial value to design and implementation of a major multicenter clinical trial

Comparative efficacy of deferiprone, deferoxamine and combination of deferiprone and deferoxamine on serum ferritin value in Beta-Thalassemia patients

Background: Iron overload is a predictable and life-threatening complication in patients with thalassemia. Effective and convenient iron chelation remains one of the main targets of clinical management of thalassemia major. The development of a safe and effective chelator has been the goal for many years. Aims and Objective: It was aimed to compare the effect of deferiprone, deferoxamine and combination of deferiprone and deferoxamine on serum ferritin value in beta-thalassemia patients. Material and Methods: This controlled clinical trial was conducted on 46 major beta-thalassemic patients. Fifteen patients in deferiprone group received deferiprone 75mg/kg/day three times a day orally. Nineteen patients in deferoxamine group received deferoxamine 30-50 mg/kg/day subcutaneously for 8-12 hours/day and 5 days per week. Twelve patients in combined therapy group received deferiprone 75 mg/kg/day three times a day orally with deferoxamine 30–50 mg/kg subcutaneously every other day. Serum ferritin value was measured at the beginning and at the end of 6th and 12th months of study. Results: The mean of serum ferritin value in deferiprone group insignificantly increased from 2731± 1398.5 μg/L at the beginning to 2788.5 ± 978.6 μg/L and to 3331.8 ± 1833.9 μg/L at the end of 6th and 12th months of study, respectively. The mean of serum ferritin value in deferoxamine group insignificantly increased from 2883.5 ± 1598.1 μg/L at the beginning to 2935.3 ± 1258.2 μg/L at the end of 6th month of study and decreased to 2773.8 ± 1216.1 μg/L and 12th month of study. The mean of serum ferritin level in combined therapy group significantly decreased from 7498.7 ± 3512.9 μg/L at the beginning to 4839.9 ± 2698.2 μg/L (P < 0.001) and to 4298.2 ± 2288.7 μg/L (P < 0.001) at the end of 6th and 12th months of study, respectively. Conclusion: Combined therapy significantly decreases serum ferritin level. Study suggests deferiprone as a significant addition to support therapy in patients with betathalassemia major on regular transfusion regimens. © 2015 Journal of Krishna Institute of Medical Sciences University

Frailty and elderly in urology: Is there an impact on post-operative complications?

INTRODUCTION: Frailty used as predictive tool is still not carried out in daily practice, although many studies confirm the great clinical importance of the frailty syndrome in surgical outcomes. There is no standardized method of measuring the physiological reserves of older surgical patients. The aim of this study was to analyze a cohort of older urological patients according to various frailty indices, in order to evaluate whether they are predictors of post-operative complications after urological procedures. MATERIAL AND METHODS: This is a prospective observational study on 78 consecutive older ( 6570 years) patients, subjected to major urological (both endoscopic and 'open surgical') procedures. Frailty was defined according to the Edmonton Frail Scale. Several risk models and biochemical parameters were evaluated. Post-operative outcomes were surgical and medical complications, mortality and rehospitalisation within 3 months. RESULTS: An overall prevalence of frailty of 21.8% was found. Patients with complications were frailer than those without complications (univariate analysis), considering both total patients (p = 0.002) and endoscopic (p = 0.04) and 'open surgical' patients (p = 0.013). However, in multivariate analysis, a significant correlation was not found between all frailty indices tested and the risk of major complications. Limitation of the study: the small sample size (lack of statistical power), although this is a prospective study focused on older urological patients. CONCLUSIONS: New urology-tailored pre-operative assessment tools may prove beneficial when calculating the risks/benefits of urological procedures, so that objective data can guide surgical decision- making and patient counselling. Further large clinical studies specifically focusing on elderly in urology will be needed

Mirroring everyday clinical practice in clinical trial design: a new concept to improve the external validity of randomized double-blind placebo-controlled trials in the pharmacological treatment of major depression

Background: Randomized, double-blind, placebo-controlled trials constitute the gold standard in clinical research when testing the efficacy of new psychopharmacological interventions in the treatment of major depression. However, the blinded use of placebo has been found to influence clinical trial outcomes and may bias patient selection. Discussion: To improve clinical trial design in major depression so as to reflect clinical practice more closely we propose to present patients with a balanced view of the benefits of study participation irrespective of their assignment to placebo or active treatment. In addition every participant should be given the option to finally receive the active medication. A research agenda is outlined to evaluate the impact of the proposed changes on the efficacy of the drug to be evaluated and on the demographic and clinical characteristics of the enrollment fraction with regard to its representativeness of the eligible population. Summary: We propose a list of measures to be taken to improve the external validity of double-blind, placebocontrolled trials in major depression. The recommended changes to clinical trial design may also be relevant for other psychiatric as well as medical disorders in which expectations regarding treatment outcome may affect the outcome itself

Applying a Dynamical Systems Model and Network Theory to Major Depressive Disorder

Mental disorders like major depressive disorder can be seen as complex dynamical systems. In this study we investigate the dynamic behaviour of individuals to see whether or not we can expect a transition to another mood state. We introduce a mean field model to a binomial process, where we reduce a dynamic multidimensional system (stochastic cellular automaton) to a one-dimensional system to analyse the dynamics. Using maximum likelihood estimation, we can estimate the parameter of interest which, in combination with a bifurcation diagram, reflects the expectancy that someone has to transition to another mood state. After validating the proposed method with simulated data, we apply this method to two empirical examples, where we show its use in a clinical sample consisting of patients diagnosed with major depressive disorder, and a general population sample. Results showed that the majority of the clinical sample was categorized as having an expectancy for a transition, while the majority of the general population sample did not have this expectancy. We conclude that the mean field model has great potential in assessing the expectancy for a transition between mood states. With some extensions it could, in the future, aid clinical therapists in the treatment of depressed patients.Comment: arXiv admin note: text overlap with arXiv:1610.0504

Development and validation of a risk score for chronic kidney disease in HIV infection using prospective cohort data from the D:A:D study.

Chronic kidney disease (CKD) is a major health issue for HIV-positive individuals, associated with increased morbidity and mortality. Development and implementation of a risk score model for CKD would allow comparison of the risks and benefits of adding potentially nephrotoxic antiretrovirals to a treatment regimen and would identify those at greatest risk of CKD. The aims of this study were to develop a simple, externally validated, and widely applicable long-term risk score model for CKD in HIV-positive individuals that can guide decision making in clinical practice