Diazoxide choline extended‐release tablet in people with Prader‐Willi syndrome: results from long‐term open‐label study

Objective: This study assessed the effect of 1-year administration of diazoxide choline extended-release tablet (DCCR) on hyperphagia and other complications of Prader-Willi syndrome (PWS). Methods: The authors studied 125 participants with PWS, age ≥ 4 years, who were enrolled in the DESTINY PWS Phase 3 study and who received DCCR for up to 52 weeks in DESTINY PWS and/or its open-label extension. The primary efficacy endpoint was Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score. Other endpoints included behavioral assessments, body composition, hormonal measures, and safety. Results: DCCR administration resulted in significant improvements in HQ-CT (mean [SE] −9.9 [0.77], p < 0.0001) and greater improvements in those with more severe baseline hyperphagia (HQ-CT > 22). Improvements were seen in aggression, anxiety, and compulsivity (all p < 0.0001). There were reductions in leptin, insulin, and insulin resistance, as well as a significant increase in adiponectin (all p < 0.004). Lean body mass was increased (p < 0.0001). Disease severity was reduced as assessed by clinician and caregiver (both p < 0.0001). Common treatment-emergent adverse events included hypertrichosis, peripheral edema, and hyperglycemia. Adverse events infrequently resulted in discontinuation (7.2%). Conclusions: DCCR administration to people with PWS was well tolerated and associated with broad-ranging improvements in the syndrome. Sustained administration of DCCR has the potential to reduce disease severity and the burden of care for families

Resident Clinical Experience in the Emergency Department: Patient Encounters by Postgraduate Year

BackgroundDuring emergency medicine (EM) training, residents are exposed to a wide spectrum of patient complaints. We sought to determine how resident clinical experience changes based on training level in relation to the patient acuity levels, chief complaints, and dispositions.MethodsWe performed a retrospective chart review of patients seen at a safety-net, academic hospital in Los Angeles from July 1, 2015, to June 30, 2016. Resident postgraduate year (PGY) level and specialty, patient acuity (based on the Emergency Severity Index), chief complaint (based on one of 30 categories), and disposition were abstracted. Our primary objective was to examine the progression of EM resident experience throughout the course of training. As a secondary objective, we compared the cases seen by EM and off-service PGY-1s.ResultsA total of 49,535 visits were examined, and of these, 32,870 (66.4%) were in the adult ED (AED) and 16,665 (33.6%) were in the pediatric ED (PED). The median acuity level was 3, and 27.4% of AED patients and 7.3% of PED patients were admitted. Data from 126 residents were analyzed. This included 94 PGY-1 residents (16 EM and 78 off-service), 16 PGY-2 EM, and 16 PGY-3 EM residents. Residents of different training levels evaluated different types of patients. Senior EM residents were more likely to care for higher-acuity patients than junior EM residents. EM PGY-3s saw higher percentages of acuity level 1 and 2 patients (2.3 and 37.8%, respectively, of their total patients) than EM PGY-1s (0.3 and 18.7%, respectively). Conversely, EM PGY-1s saw higher percentages of acuity level 4 and 5 patients (27.9 and 1.6%, respectively) compared to EM PGY-3s (10.7 and 0.7%, respectively). There was a significant linear trend for increasing acuity with training year among EM residents (p < 0.001). EM PGY-1s saw more patients than off-service PGY-1s with slightly higher acuities and admission rates.ConclusionThe clinical experience of EM residents varies based on their level of training. EM residents show a progression throughout residency and are more likely to encounter higher volumes of patients with higher acuity as they progress in their training. When designing EM residency curriculums, this is a model of an EM residency program

Stay in treatment: Predicting dropout from pediatric weight management study protocol

Introduction: Childhood obesity is a serious public health concern. Multidisciplinary pediatric weight management programs have been deemed effective. However, effectiveness of these programs is impacted by attrition, limiting health benefits to children, and inefficiently utilizing scarce resources. Methods: We have developed a model (the Outcomes Forecasting System, OFS) that isolates variables associated with attrition from pediatric weight management, with the potential to forecast participant dropout. In Aim 1, we will increase the power and precision of the OFS and then validate the model through the consistent acquisition of key patient, family, and treatment data, from three different weight management sites. In Aim 2, external validity will be established through the application of the OFS at a fourth pediatric weight management program. Aim 3 will be a pilot clinical trial, incorporating an intervention built on the results of Aims 1 and 2 and utilizing the OFS to reduce attrition. Discussion: A greater understanding of the patient, family, and disease-specific factors that predict dropout from pediatric weight management can be utilized to prevent attrition. The goal of the current study is to refine the OFS to a level of precision and efficiency to be a valuable tool to any weight management program. By identifying the most pertinent factors driving attrition across weight management sites, new avenues for treatment will be identified. This study will result in a valuable forecasting tool that will be applicable for diverse programs and populations, decrease program costs, and improve patient retention, adherence, and outcomes. Clinicaltrials.gov identifier: NCT04364282

Skeletal Muscle Phosphocreatine Recovery after Submaximal Exercise in Children and Young and Middle-Aged Adults

Context: Elderly subjects have reduced mitochondrial function. However, it remains unclear whether the decline in mitochondrial function begins earlier in the life span

Integrating family planning into HIV care in western Kenya: HIV care providers’ perspectives and experiences one year following integration

With high rates of unintended pregnancy in sub-Saharan Africa, integration of family planning (FP) into HIV care is being explored as a strategy to reduce unmet need for contraception. Perspectives and experiences of healthcare providers are critical in order to create sustainable models of integrated care. This qualitative study offers insight into how HIV care providers view and experience the benefits and challenges of providing integrated FP/HIV services in Nyanza Province, Kenya. Sixteen individual interviews were conducted among healthcare workers at six public sector HIV care facilities one year after the implementation of integrated FP and HIV services. Data were transcribed and analyzed qualitatively using grounded theory methods and Atlas.ti. Providers reported a number of benefits of integrated services that they believed increased the uptake and continuation of contraceptive methods. They felt that integrated services enabled them to reach a larger number of female and male patients and in a more efficient way for patients compared to non-integrated services. Availability of FP services in the same place as HIV care also eliminated the need for most referrals, which many providers saw as a barrier for patients seeking FP. Providers reported many challenges to providing integrated services, including the lack of space, time, and sufficient staff, inadequate training, and commodity shortages. Despite these challenges, the vast majority of providers was supportive of FP/HIV integration and found integrated services to be beneficial to HIV-infected patients. Providers' concerns relating to staffing, infrastructure, and training need to be addressed in order to create sustainable, cost-effective FP/HIV integrated service models