Adopting a multidisciplinary telemedicine intervention for fall prevention in Parkinson’s disease. Protocol for a longitudinal, randomized clinical trial

Background Approximately 40–70% of people with Parkinson’s disease (PD) fall each year, causing decreased activity levels and quality of life. Current fall-prevention strategies include the use of pharmacological and non-pharmacological therapies. To increase the accessibility of this vulnerable population, we developed a multidisciplinary telemedicine program using an Information and Communication Technology (ICT) platform. We hypothesized that the risk for falling in PD would decrease among participants receiving a multidisciplinary telemedicine intervention program added to standard office-based neurological care. Objective To determine the feasibility and cost-effectiveness of a multidisciplinary telemedicine intervention to decrease the incidence of falls in patients with PD. Methods Ongoing, longitudinal, randomized, single-blinded, case-control, clinical trial. We will include 76 non-demented patients with idiopathic PD with a high risk of falling and limited access to multidisciplinary care. The intervention group (n = 38) will receive multidisciplinary remote care in addition to standard medical care, and the control group (n = 38) standard medical care only. Nutrition, sarcopenia and frailty status, motor, non-motor symptoms, health-related quality of life, caregiver burden, falls, balance and gait disturbances, direct and non-medical costs will be assessed using validated rating scales. Results This study will provide a cost-effectiveness assessment of multidisciplinary telemedicine intervention for fall reduction in PD, in addition to standard neurological medical care. Conclusion In this challenging initiative, we will determine whether a multidisciplinary telemedicine intervention program can reduce falls, as an alternative intervention option for PD patients with restricted access to multidisciplinary care. Trial registration ClinicalTrials.gov Identifier: NCT04694443.

Cost-effectiveness of mechanical thrombectomy for acute ischemic stroke in Brazil: Results from the RESILIENT trial

Background The RESILIENT trial demonstrated the clinical benefit of mechanical thrombectomy in patients presenting acute ischemic stroke secondary to anterior circulation large vessel occlusion in Brazil. Aims This economic evaluation aims to assess the cost-utility of mechanical thrombectomy in the RESILIENT trial from a public healthcare perspective. Methods A cost-utility analysis was applied to compare mechanical thrombectomy plus standard medical care (n = 78) vs. standard medical care alone (n = 73), from a subset sample of the RESILIENT trial (151 of 221 patients). Real-world direct costs were considered, and utilities were imputed according to the Utility-Weighted modified Rankin Score. A Markov model was structured, and probabilistic and deterministic sensitivity analyses were performed to evaluate the robustness of results. Results The incremental costs and quality-adjusted life years gained with mechanical thrombectomy plus standard medical care were estimated at Int$ 7440 and 1.04, respectively, compared to standard medical care alone, yielding an incremental cost-effectiveness ratio of Int$ 7153 per quality-adjusted life year. The deterministic sensitivity analysis demonstrated that mRS-6 costs of the first year most affected the incremental cost-effectiveness ratio. After 1000 simulations, most of results were below the cost-effective threshold. Conclusions The intervention's clear long-term benefits offset the initially higher costs of mechanical thrombectomy in the Brazilian public healthcare system. Such therapy is likely to be cost-effective and these results were crucial to incorporate mechanical thrombectomy in the Brazilian public stroke centers.

Efficacy of Non-pharmacologic Auxiliary Treatments in Improving Defecation Function in Children With Chronic Idiopathic Constipation: A Systematic Review and Network Meta-analysis

Background: Non-pharmacologic auxiliary treatments have been considered crucial therapies for treating chronic idiopathic constipation (CIC) during the past decades worldwide. Several treatment patterns are available, but their relative efficacy is obscure because there are no head-to-head randomized controlled trials, especially in children. We conducted this network meta-analysis to evalute the effectiveness of these therapies in improving defecation function based on their direct comparisons with standard medical care.Methods: Medline, Embase, and Cochrane Central were searched for randomized controlled trials (RCTs) published in English from inception to October 2020, assessing the efficacy of auxiliary therapies (behavior therapy, physiotherapy, biofeedback, or anorectal manometry) in children with CIC. We extracted data for endpoints, risk of bias, and evidence quality. Eligible studies in the meta-analysis reported the data of a dichotomous assessment of overall response to treatment (response or not) or defecation frequency per week after treatment. The hierarchical Bayesian network meta-analysis was used in the study. We chose a conservative methodology, random effects model, to pool data which could handle the heterogeneity well. The relative risk (RR) with 95% confidence intervals (CIs) was calculated for dichotomous outcomes. For continuous results, weighted mean difference (WMD) with related CIs was calculated. The included treatments were ranked to define the probability of being the best treatment.Results: Seven RCTs (838 patients) met inclusion and endpoint criteria. Based on an endpoint of the absence of constipation (Rome criteria) with laxatives allowed, physiotherapy plus standard medical care (SMC) had the highest probability (84%) to bethe most effective therapy. When the treatment response was defined as an absence of constipation with not laxatives allowed, biofeedback plus SMC ranked first (probability 52%). Physiotherapy plus SMC ranked first when the endpoint was based on defecation frequency per week with laxatives allowed (probability 86%).Conclusion: Almost all auxiliary therapies are effective complementary therapies for treating CIC, but they needed to be used simultaneously with SMC. Nevertheless, because of the small number of eligible studies and their small sample sizes, the differences in treatment duration and the endpoints, large sample RCTs with long-term follow-up are required for further investigation.

How health literacy can enhance the design and conduct of clinical trials from consent to conclusion

Health literacy research and interventions have provided multiple tools to improve communication between professionals and patients in clinical contexts for many years. Despite the reality that many patients participate in clinical trials in conjunction with standard medical care, only recently have efforts extended to address and improve the health literacy of both clinical trial researchers and participants. To date, the primary focus of health literacy activities in clinical trials has centered on communicating trial results to trial participants. This report describes the opportunities and strategies necessary to layer health literacy activities across the clinical trial process from consent to conclusion.

Effects and Implementation of a Mindfulness and Relaxation App for Cancer Patients: Mixed-Methods Feasibility Study (Preprint)

BACKGROUND A cancer diagnosis and cancer treatment can cause high levels of distress, which is often not sufficiently addressed in standard medical care. Therefore, a variety of supportive non-pharmacological treatments have been suggested to reduce cancer patients’ distress. However, not all patients use those interventions because of limited access or being unaware. To overcome these barriers, mHealth might be a promising way to deliver respective supportive treatments. OBJECTIVE The aim of this study was to evaluate effects and the implementation of a mindfulness and relaxation app intervention for cancer patients as well as patients’ adherence to such an intervention. METHODS In this observational feasibility study with a mixed-methods approach, cancer patients were recruited online and through hospitals in Switzerland. All enrolled patients received access to a mindfulness and relaxation app. Patients completed self-reported outcomes (general health, health-related quality of life, anxiety, depression, distress, mindfulness, and fear of progression) at baseline, week 4, 10, and 20. The frequency of app exercise usage was gathered directly through the app in order to assess the adherence of patients. In addition, we conducted interviews with 5 health professionals for their thoughts on the implementation of the app intervention into standard medical care. We analyzed patients’ self-reported outcomes with linear mixed models (LMM) and qualitative data with content analysis. RESULTS A total of 100 cancer patients (74 female) with a mean age of 53.2 (SD 11.6) participated in the study, of which 25 patients used the app regularly until week 20. LMM analyses revealed an improvement in anxiety (P=.04), distress (P<.001), fatigue (P=.01), sleep disturbance (P=.02), quality of life (P=.03), and mindfulness (P<.001) over the course of 20 weeks. Further LMM analyses revealed a larger improvement of distress (P<.001), a moderate improvement of anxiety (P=.001), and depression (P=.03) in patients with high symptom levels at baseline in the respective domains. The interviews revealed that the health professionals perceived the app as a helpful addition to standard care. They also made suggestions for improvements, which could facilitate the implementation and adherence to such an app. CONCLUSIONS This study indicates that a mindfulness and relaxation app for cancer patients can be a feasible and effective way to deliver a self-care intervention, especially for highly distressed patients. Future studies should investigate if the appeal of the app can be increased with more content and the effectiveness of such an intervention needs to be tested in a randomized controlled trial. CLINICALTRIAL

Psychological Outcomes of a Cognitive Behavioral Therapy for Youth with Inflammatory Bowel Disease: Results of the HAPPY-IBD Randomized Controlled Trial at 6- and 12-Month Follow-Up

Youth with inflammatory bowel disease (IBD) often experience psychological difficulties, such as anxiety and depression. This randomized controlled study tested whether a 3-month disease-specific cognitive behavioral therapy (CBT) in addition to standard medical care versus standard medical care only was effective in improving these youth’s psychological outcomes. As this study was aimed at prevention, we included 70 youth (10–25 years) with IBD and symptoms of subclinical anxiety and/or depression, and measured psychological outcomes at 6- and 12-month follow-up. In general, participants in both groups showed improvements in anxiety, depression, health-related quality of life, social functioning, coping, and illness perceptions, sustained until 12 months follow-up. Overall, we found no differences between those receiving additional CBT and those receiving standard medical care only. We assume that this can be explained by the perceived low burden (both somatically and psychologically) or heightened awareness of psychological difficulties and IBD. ClinicalTrials.gov NCT02265588.

Health-related Quality of Life of Macedonian Families Experiencing Cystic Fibrosis in Pediatric Practice

Background: : Health-related quality of life (HRQoL) is a parameter that is examined in the area of clinical effectiveness. Like other chronic health conditions, paediatric cystic fibrosis (CF) impacts not only children but also their families. Aim: The present study investigates for the first time the HRQoL of children and parents in the Republic of North Macedonia. Materials and methods: The survey included 22 children (6 to 13 years of age) and their parents and 7 parents of children under 6 years of age by using the CFQ Revised and questions for current medical treatment. Results: Children (6-13 years) reported the highest score for the digestive condition (84.85), while the lowest score was given for social activity (59.74). The highest score for digestive condition was also obtained from the parents of children from 6-13 years and under age of 6. The parents of children (6-13 years) reported the lowest score (60.56) for treatment burden activity, while the lowest score (50.0) for eating condition was obtained from the parents of children under 6 years. Conclusion: Nationality and gender have no significant impact on the HRQoL parameters. The highest scores for the digestive condition, respiratory function and physical condition are in a positive correlation with the fact that enzyme, antibiotic and physical therapy are given as a standard medical care. The lowest scores of the social aspect of the CF patients indicate the need for including a psychological support and support of social workers as a part of the standard medical care of these patients.