The impact of feedback training on prediction of cancer clinical trial results.

Introduction: Funders must make difficult decisions about which squared treatments to prioritize for randomized trials. Earlier research suggests that experts have no ability to predict which treatments will vindicate their promise. We tested whether a brief training module could improve experts' trial predictions.

A Parent-child yoga intervention for reducing attention deficits in children with congenital heart disease: the Yoga for Little Hearts Feasibility Study Protocol.

Introduction: Preschoolers and school-aged children with congenital heart disease (CHD) are at higher risk of attention deficit hyperactivity disorder (ADHD) compared with the general population. To this day, no randomised controlled trial (RCT) aiming to improve attention has been conducted in young children with CHD. There is emerging evidence indicating that parent-child yoga interventions improve attention and reduce ADHD symptoms in both typically developing and clinical populations.

Relationship between 4-month functional brain network topology and 24-month neurodevelopmental outcome in children with congenital heart disease.

Survivors of complex forms of congenital heart disease (CHD)∗ are at high risk of neurodevelopmental disabilities. Neuroimaging studies have pointed to brain anomalies and immature networks in infants with CHD, yet less is known about their functional network topology and associations with neurodevelopment. To characterize the functional network topology in 4-month-old infants with repaired CHD, we compared graph theory metrics measured using resting-state functional near-infrared spectroscopy (rs-fNIRS) between infants with CHD (n = 22) and healthy controls (n = 30).

Functional brain connectivity after corrective cardiac surgery for critical congenital heart disease: a preliminary near-infrared spectroscopy (NIRS) report.

Patients with congenital heart disease (CHD) requiring cardiac surgery in infancy are at high risk for neurodevelopmental impairments. Neonatal imaging studies have reported disruptions of brain functional organization before surgery. Yet, the extent to which functional network alterations are present after cardiac repair remains unexplored.

The role of parenting stress in anxiety and sleep outcomes in toddlers with congenital heart disease.

Objectives: This retrospective cohort study investigates how parenting stress, measured at 4 months of age by use of a classic three-dimensional parent-reported scale (Parenting Stress Index, 4th Ed. or PSI-4), can predict anxiety symptoms and quality of sleep at 24 months in toddlers with congenital heart disease (CHD).

Study Design: Sixty-six toddlers with CHD followed at our cardiac neurodevelopmental follow-up clinic were included in this study.

Design of placebo-controlled randomized trials of anticancer agents: Ethical considerations based on a review of published trials.

Background: Limited information exists about the design of placebo-controlled cancer trials. Through a systematic review of trials published in 2013, we describe placebo use in randomized trials testing anticancer agents and analyze strategies that increase exposure to the experimental regimen.

Methods: Trials were classified as add-on (placebo in combination with standard treatment) or placebo-only.

Neurodevelopmental Outcome of Children with Congenital Heart Disease: A Cohort Study from Infancy to Preschool Age.

Objective: To characterize the neuropsychological outcome of children with congenital heart disease (CHD) at age 5 years; the stability of cognitive and language abilities across childhood; and to identify early neurodevelopmental markers of neuropsychological outcomes in these children.

Study Design: Five-year-old children (n = 55) with complex CHD were assessed using standardized and comprehensive neuropsychological measures. Stability of language and cognitive performance was assessed by comparing standardized scores between ages 1, 2, and 5 years old.

Utility of the Ages and Stages Questionnaires 3rd Edition for Developmental Screening in Children with Surgically Repaired Congenital Heart Disease.

: This study sought to evaluate the accuracy of the Ages and Stages Questionnaires 3rd Edition (ASQ-3) in identifying developmental delay (DD) in children with congenital heart disease (CHD) born at term who underwent surgical repair.: Participants had to complete ASQ-3 and Bayley Scales of Infant and Toddler Development 3rd Edition (BSID-III) at 12 and 24 months. A child was considered at risk of DD for a ASQ-3 domain when he scored below the cutoff (≤-1SD or ≤-2SD).

Patient Participation in Clinical Trials of Oncology Drugs and Biologics Preceding Approval by the US Food and Drug Administration.

Importance: Several studies have estimated the financial inputs for successful drug development. Such analyses do not capture the large investment that patient study participants commit to drug development.

Objective: To estimate the volume of patients required to achieve a first US Food and Drug Administration (FDA) approval for a new anticancer drug or biologic therapy.

Impacts of an Interdisciplinary Developmental Follow-Up Program on Neurodevelopment in Congenital Heart Disease: The CINC Study.

This study investigates the impact of an early systematic interdisciplinary developmental follow-up and individualized intervention program on the neurodevelopment of children with complex congenital heart disease (CHD) who required cardiac surgery. We prospectively enrolled 80 children with CHD: 41 were already followed at our neurocardiac developmental follow-up clinic from the age of 4 months, while 39 were born before the establishment of the program and therefore received standard health care. We conducted cognitive, motor, and behavioral assessments at 3 years of age.

Patient burden and clinical advances associated with postapproval monotherapy cancer drug trials: a retrospective cohort study.

Objectives: After regulatory approval, drug companies, public funding agencies and academic researchers often pursue trials aimed at extending the uses of a new drug by testing it in new non-approved indications. Patient burden and clinical impact of such research are not well understood.

Design And Setting: We conducted a retrospective cohort study of postapproval clinical trials launched within 5 years after the drug's first approval, testing anticancer drugs in monotherapy in indications that were first pursued after a drug's first Food and Drug Administration (FDA) license, for all 12 anticancer drugs approved between 2005 and 2007.

Gross Motor Development of Children with Congenital Heart Disease Receiving Early Systematic Surveillance and Individualized Intervention: Brief Report.

: This retrospective study aims to describe the gross motor development of children aged 4 to 24 months with congenital heart disease (CHD) enrolled in a systematic developmental follow-up program and to describe the frequency of physical therapy sessions they received between 4 and 8 months of age. : Twenty-nine infants with CHD underwent motor evaluations using the AIMS at 4 months, and the Bayley-III at 12 and 24 months. : Based on AIMS, 79% of 4-month-old infants had a gross motor delay and required physical therapy.

Benefit, burden, and impact for a cohort of post-approval cancer combination trials.

Background: After approval, drug developers often pursue trials aimed at extending the uses of a new drug by combining it with other drugs. Little is known about the risk and benefits associated with such research.

Methods: To establish a historic benchmark of risk and benefit, we searched Medline and Embase for clinical trials testing anti-cancer drugs in combination within 5 years of approval by the Food and Drug Administration of 12 anti-cancer "index" drugs first licensed 2005-2007 inclusive.

Epilepsy and seizures in children with congenital heart disease: A prospective study.

Purpose: Children with complex congenital heart disease (CHD) experience high incidence of perioperative seizures. Population-based studies also report high epilepsy co-morbidity in CHD. Given the increasing survival of patients with CHD and the interference of seizures and epilepsy with the long-term outcomes, characterizing them in this population is of high relevance.

Assessment of Pregabalin Postapproval Trials and the Suggestion of Efficacy for New Indications: A Systematic Review.

Importance: After a drug receives regulatory approval, researchers often pursue small, underpowered trials, called exploratory trials, aimed at testing additional indications. If favorable early findings from exploratory trials are not promptly followed by confirmatory trials, then physicians, patients, and payers can be left uncertain about a drug's clinical value (clinical agnosticism). Such findings may encourage the off-label use of ineffective drugs.

Reporting of stepped wedge cluster randomised trials: extension of the CONSORT 2010 statement with explanation and elaboration.

This report presents the Consolidated Standards of Reporting Trials (CONSORT) extension for the stepped wedge cluster randomised trial (SW-CRT). The SW-CRT involves randomisation of clusters to different sequences that dictate the order (or timing) at which each cluster will switch to the intervention condition. The statement was developed to allow for the unique characteristics of this increasingly used study design.

Superior Performance in Prone in Infants With Congenital Heart Disease Predicts an Earlier Onset of Walking.

Infants with congenital heart disease are at risk of impaired neurodevelopment, which frequently manifests as motor delay during their first years of life. This delay is multifactorial in origin and environmental factors, such as a limited experience in prone, may play a role. In this study, we evaluated the motor development of a prospective cohort of 71 infants (37 males) with congenital heart disease at 4 months of age using the Alberta Infant Motor Scales (AIMS).

Phase I study of axitinib and everolimus in metastatic solid tumours and extension to metastatic renal cell carcinoma: Results of EVAX study.

Purpose: Anti-angiogenic and mammalian target of rapamycin inhibitors have shown efficacy in solid tumours. Reported combination of both drugs was deemed to be too toxic. Due to a potential favourable safety profile of axitinib (AX), a phase I study combining everolimus (EV) and AX for solid tumours was explored.

Organ preservation for rectal cancer (GRECCAR 2): a prospective, randomised, open-label, multicentre, phase 3 trial.

Background: Organ preservation is a concept proposed for patients with rectal cancer after a good clinical response to neoadjuvant chemotherapy, to potentially avoid morbidity and side-effects of rectal excision. The objective of this study was to compare local excision and total mesorectal excision in patients with a good response after chemoradiotherapy for lower rectal cancer.

Methods: We did a prospective, randomised, open-label, multicentre, phase 3 trial at 15 tertiary centres in France that were experts in the treatment of rectal cancer.