Inhaled pirfenidone solution (AP01) for IPF: a randomised, open-label, dose-response trial.

Introduction: Oral pirfenidone reduces lung function decline and mortality in patients with idiopathic pulmonary fibrosis (IPF). Systemic exposure can have significant side effects, including nausea, rash, photosensitivity, weight loss and fatigue. Reduced doses may be suboptimal in slowing disease progression.

Covariation of changing patent ductus arteriosus management and preterm infant outcomes in Pediatrix neonatal intensive care units.

Objective: To test the hypothesis that NICU-specific preterm infant outcomes co-vary with changes in local patent ductus arteriosus (PDA) management.

Study Design: This retrospective multicenter study examined NICU-specific aggregated data for infants born 400-1499 g (VLBW) in the Pediatrix Clinical Data Warehouse. For each NICU and each year 2006-2016 we calculated proportion of infants receiving cyclooxygenase inhibitor (COXI) and/or PDA ligation and determined NICU-specific changes in these therapies between consecutive years.

The Trial to Reduce Antimicrobial Use in Nursing Home Residents With Alzheimer Disease and Other Dementias (TRAIN-AD): A Cluster Randomized Clinical Trial.

Importance: Antimicrobials are extensively prescribed to nursing home residents with advanced dementia, often without evidence of infection or consideration of the goals of care.

Objective: To test the effectiveness of a multicomponent intervention to improve the management of suspected urinary tract infections (UTIs) and lower respiratory infections (LRIs) for nursing home residents with advanced dementia.

Design, Setting, And Participants: A cluster randomized clinical trial of 28 Boston-area nursing homes (14 per arm) and 426 residents with advanced dementia (intervention arm, 199 residents; control arm, 227 residents) was conducted from August 1, 2017, to April 30, 2020.

Assessing the Value to the Patient of New Technologies in Anatomic Total Shoulder Arthroplasty.

Background: Publications regarding anatomic total shoulder arthroplasty (TSA) have consistently reported that they provide significant improvement for patients with glenohumeral arthritis. New TSA technologies that have been introduced with the goal of further improving these outcomes include preoperative computed tomography (CT) scans, 3-dimensional preoperative planning, patient-specific instrumentation, stemless and short-stemmed humeral components, as well as metal-backed, hybrid, and augmented glenoid components. The benefit of these new technologies in terms of patient-reported outcomes is unknown.

Comparison of longitudinal and radial characteristics of intra-anal pressures using 3D high-definition anorectal manometry between children with anoretal malformations and functional constipation.

Background: Pathophysiology of fecal incontinence (FI) in children with anorectal malformations (AM) is not well understood. Standard or high-resolution anorectal manometry (ARM) does not identify radial asymmetry or localize abnormal sphincter function. 3D high-definition anorectal manometry (HDARM) provides detailed topographic and 3D pressure gradient representation of anal canal.

Decreased Incidence of Hepatic Artery Thrombosis in Pediatric Liver Transplantation Using Technical Variant Grafts: Report of the Society of Pediatric Liver Transplantation Experience.

Objective: To evaluate risk factors for hepatic artery thrombosis (HAT) and examine the long-term outcomes of graft and patient survival after HAT in pediatric recipients of liver transplantation.

Study Design: Using multicenter data from the Society of Pediatric Liver Transplantation, Kaplan-Meier and Cox regression analyses were performed on first-time pediatric (aged <18 years) liver transplant recipients (n = 3801) in the US and Canada between 1995 and 2016.

Results: Of children undergoing their first liver transplantation, 7.

Covariate-constrained randomization for cluster randomized trials in the long-term care setting: Application to the TRAIN-AD trial.

Unlabelled: Little has been reported on strategies to ensure key covariate balance in cluster randomized trials in the nursing home setting. Facilities vary widely on key characteristics, small numbers may be randomized, and staggered enrollment is often necessary. A covariate-constrained algorithm was used to randomize facilities in the Trial to Reduce Antimicrobial use In Nursing home residents with Alzheimer's Disease and other Dementias (TRAIN-AD), an ongoing trial in Boston-area facilities (14 facilities/arm).

The trial to reduce antimicrobial use in nursing home residents with Alzheimer's disease and other dementias: study protocol for a cluster randomized controlled trial.

Background: Infections are common in nursing home (NH) residents with advanced dementia but are often managed inappropriately. Antimicrobials are extensively prescribed, but frequently with insufficient evidence to support a bacterial infection, promoting the emergence of multidrug-resistant organisms. Moreover, the benefits of antimicrobials remain unclear in these seriously ill residents for whom comfort is often the goal of care.

Predicting ideal outcome after pediatric liver transplantation: An exploratory study using machine learning analyses to leverage Studies of Pediatric Liver Transplantation Data.

Machine learning analyses allow for the consideration of numerous variables in order to accommodate complex relationships that would not otherwise be apparent in traditional statistical methods to better classify patient risk. The SPLIT registry data were analyzed to determine whether baseline demographic factors and clinical/biochemical factors in the first-year post-transplant could predict ideal outcome at 3 years (IO-3) after LT. Participants who received their first, isolated LT between 2002 and 2006 and had follow-up data 3 years post-LT were included.

Hepatic Venous Pressure Gradient Measurements in Children: Correlation With Hepatic Histology and Clinical Indicators of Portal Hypertension.

Objectives: In adults, elevated hepatic venous pressure gradients (HVPGs) are correlated with the degree of liver fibrosis on histopathology and predict worse outcomes including variceal bleeding and death. We aimed to examine the association between HVPG measurements, histopathologic findings, and clinical indicators of portal hypertension in children.

Methods: Utilizing retrospective data from 2 pediatric centers between 2006 and 2015, we identified children who underwent simultaneous HVPG measurement and transjugular liver biopsy.

Effect of Prophylaxis for Early Adrenal Insufficiency Using Low-Dose Hydrocortisone in Very Preterm Infants: An Individual Patient Data Meta-Analysis.

Objective: To assess the effect of prophylaxis for early adrenal insufficiency using low-dose hydrocortisone on survival without bronchopulmonary dysplasia (BPD) in very preterm infants using an individual patient data meta-analysis.

Study Design: All existing randomized controlled trials testing the efficacy of the prophylaxis of early adrenal insufficiency using low-dose hydrocortisone on survival without BPD were considered for inclusion when data were available. The primary outcome was the binary variable survival without BPD at 36 weeks of postmenstrual age.

Waterfalls and Handoffs: A Novel Physician Staffing Model to Decrease Handoffs in a Pediatric Emergency Department.

Study Objective: Patient handoffs at shift change in the emergency department (ED) are a well-known risk point for patient safety. Numerous methods have been implemented and studied to improve the quality of handoffs to mitigate this risk. However, few have investigated processes designed to decrease the number of handoffs.

Concordance Between Proxy Level of Care Preference and Advance Directives Among Nursing Home Residents With Advanced Dementia: A Cluster Randomized Clinical Trial.

Context: Care consistent with goals is the desired outcome of advance care planning (ACP).

Objectives: The objectives of this study were to examine concordance between advance directives and proxy care preferences among nursing home residents with advanced dementia and to determine the impact of an ACP video on concordance.

Methods: Data were from Educational Video to Improve Nursing home Care in End-stage dementia, a cluster randomized clinical trial conducted in 64 Boston-area facilities (32/arm) from 2013 to 2017.

An Advance Care Planning Video Decision Support Tool for Nursing Home Residents With Advanced Dementia: A Cluster Randomized Clinical Trial.

Importance: Better advance care planning (ACP) can help promote goal-directed care in patients with advanced dementia.

Objectives: To test whether an ACP video (vs usual care) has an effect on documented advance directives, level of care preferences, goals-of-care discussions, and burdensome treatments among nursing home residents with advanced dementia.

Design, Setting, And Participants: The Educational Video to Improve Nursing home Care in End-stage dementia (EVINCE) trial was a cluster randomized clinical trial conducted between February 2013 and July 2017, at 64 Boston-area nursing homes (32 facilities per arm).

Association of Prognostic Estimates With Burdensome Interventions in Nursing Home Residents With Advanced Dementia.

Importance: Prognostication in advanced dementia is challenging but may influence care.

Objectives: To determine the accuracy of proxies' prognostic estimates for nursing home residents with advanced dementia, identify factors associated with those estimates, and examine the association between their estimates and use of burdensome interventions.

Design, Setting, And Participants: Data were combined from 2 studies that prospectively followed 764 residents with advanced dementia and their proxies in Boston-area nursing homes for 12 months: (1) the Study of Pathogen Resistance and Exposure to Antimicrobials in Dementia, conducted from September 2009 to November 2012 (362 resident/proxy dyads; 35 facilities); and (2) the Educational Video to Improve nursing home Care in End-Stage Dementia, conducted from March 2013 to July 2017 (402 resident/proxy dyads; 62 facilities).

Effects of an Antioxidant-enriched Multivitamin in Cystic Fibrosis. A Randomized, Controlled, Multicenter Clinical Trial.

Rationale: Cystic fibrosis (CF) is characterized by dietary antioxidant deficiencies, which may contribute to an oxidant-antioxidant imbalance and oxidative stress.

Objectives: Evaluate the effects of an oral antioxidant-enriched multivitamin supplement on antioxidant concentrations, markers of inflammation and oxidative stress, and clinical outcomes.

Methods: In this investigator-initiated, multicenter, randomized, double-blind, controlled trial, 73 pancreatic-insufficient subjects with CF 10 years of age and older with an FEV between 40% and 100% predicted were randomized to 16 weeks of an antioxidant-enriched multivitamin or control multivitamin without antioxidant enrichment.

Human Herpesvirus-6B Reactivation Is a Risk Factor for Grades II to IV Acute Graft-versus-Host Disease after Hematopoietic Stem Cell Transplantation: A Systematic Review and Meta-Analysis.

Graft-versus-host disease (GVHD) is an important cause of morbidity and mortality after allogeneic hematopoietic cell transplantation (HCT). Many studies have suggested that human herpesvirus-6B (HHV-6B) plays a role in acute GVHD (aGVHD) after HCT. Our objective was to systematically summarize and analyze evidence regarding HHV-6B reactivation and development of aGVHD.

Resolving Malnutrition With Parenteral Nutrition Before Liver Transplant in Biliary Atresia.

Objective: Malnutrition is a common complication of end-stage liver disease (ESLD) associated with poor liver transplant outcomes. Nasogastric feeds are used for nutritional supplementation, but some patients remain malnourished. Parenteral nutrition (PN) can be effective, but has potential complications.

Hope, distress, and later quality of life among adolescent and young adults with cancer.

We aimed to explore the predictive value of screening for distress alone, hope alone, or a combination of both. In a multicenter prospective study, 37 English-speaking adolescents and young adults with cancer and 40 parents completed validated instruments at diagnosis ("baseline") and 3-6 months later ("follow-up"). Correlated regression models described associations.

Associations between Liver Enzyme Levels and Parameters of the Metabolic Syndrome in Obese Children.

Background: Obesity is strongly associated with insulin resistance, hypertension, dyslipidemia, and therefore risk for metabolic syndrome (MetS), which is an increasing problem in youth. The potential role of elevated liver enzyme levels in this context needs to be further investigated.

Methods: This paper provides a post hoc analysis of a cross-sectional study of 77 obese nondiabetic children (51% female; median age 11.

Nutritional Adequacy of the Specific Carbohydrate Diet in Pediatric Inflammatory Bowel Disease.

Introduction: The specific carbohydrate diet (SCD) is an exclusion diet used as a therapy in inflammatory bowel disease. The aim of this study was to evaluate the nutritional adequacy of the SCD.

Methods: Prospective dietary data for 12 weeks were analyzed for pediatric patients on the SCD.

Level of Care Preferences Among Nursing Home Residents With Advanced Dementia.

Context: Delivering goal-directed care is a hallmark of high-quality palliative care, but requires an understanding of preferences.

Objectives: To describe and identify factors associated with level of care preferences among proxies of nursing home (NH) residents with advanced dementia.

Methods: NH residents with advanced dementia and their proxies (N = 402 dyads) were recruited from 62 Boston-area facilities as part of an ongoing randomized clinical trial.

Analysis of Liver Offers to Pediatric Candidates on the Transplant Wait List.

Background & Aims: Approximately 10% of children on the liver transplant wait-list in the United States die every year. We examined deceased donor liver offer acceptance patterns and their contribution to pediatric wait-list mortality.

Methods: We performed a retrospective cohort study of children on the US liver transplant wait-list from 2007 through 2014 using national transplant registry databases.

Reducing Head CT Use for Children With Head Injuries in a Community Emergency Department.

Background And Objective: Clinical decision rules have reduced use of computed tomography (CT) to evaluate minor pediatric head injury in pediatric emergency departments (EDs). CT use remains high in community EDs, where the majority of children seek medical care. We sought to reduce the rate of CT scans used to evaluate pediatric head injury from 29% to 20% in a community ED.