Impact of Frailty on Surgical Outcomes of Patients With Cushing Disease Using the Multicenter Registry of Adenomas of the Pituitary and Related Disorders Registry.

Background And Objectives: Despite growing interest in how patient frailty affects outcomes (eg, in neuro-oncology), its role after transsphenoidal surgery for Cushing disease (CD) remains unclear. We evaluated the effect of frailty on CD outcomes using the Registry of Adenomas of the Pituitary and Related Disorders (RAPID) data set from a collaboration of US academic pituitary centers.

Methods: Data on consecutive surgically treated patients with CD (2011-2023) were compiled using the 11-factor modified frailty index.

Can ChatGPT4-vision identify radiologic progression of multiple sclerosis on brain MRI?

Background: The large language model ChatGPT can now accept image input with the GPT4-vision (GPT4V) version. We aimed to compare the performance of GPT4V to pretrained U-Net and vision transformer (ViT) models for the identification of the progression of multiple sclerosis (MS) on magnetic resonance imaging (MRI).

Methods: Paired coregistered MR images with and without progression were provided as input to ChatGPT4V in a zero-shot experiment to identify radiologic progression.

Ocrelizumab dose selection for treatment of pediatric relapsing-remitting multiple sclerosis: results of the OPERETTA I study.

Background: The presented study identified the appropriate ocrelizumab dosing regimen for patients with pediatric-onset multiple sclerosis (POMS).

Methods: Patients with POMS aged 10-17 years were enrolled into cohort 1 (body weight [BW] < 40 kg, ocrelizumab 300 mg) and cohort 2 (BW ≥ 40 kg, ocrelizumab 600 mg) during a 24-week dose-exploration period (DEP), followed by an optional ocrelizumab (given every 24 weeks) extension period.

Primary Endpoints: pharmacokinetics, pharmacodynamics (CD19 B-cell count); secondary endpoint: safety; exploratory endpoints: MRI activity, protocol-defined relapses, Expanded Disability Status Scale (EDSS) score change.

Mutations disrupting the kinase domain of IKKα lead to immunodeficiency and immune dysregulation in humans.

IKKα, encoded by CHUK, is crucial in the non-canonical NF-κB pathway and part of the IKK complex activating the canonical pathway alongside IKKβ. The absence of IKKα causes fetal encasement syndrome in humans, fatal in utero, while an impaired IKKα-NIK interaction was reported in a single patient and causes combined immunodeficiency. Here, we describe compound heterozygous variants in the kinase domain of IKKα in a female patient with hypogammaglobulinemia, recurrent lung infections, and Hay-Wells syndrome-like features.

Assessing Clinical Improvement of Infants Hospitalized for Respiratory Syncytial Virus-Related Critical Illness.

Background: Pediatric respiratory syncytial virus (RSV)-related acute lower respiratory tract infection (LRTI) commonly requires hospitalization. The Clinical Progression Scale Pediatrics (CPS-Ped) measures level of respiratory support and degree of hypoxia across a range of disease severity, but it has not been applied in infants hospitalized with severe RSV-LRTI.

Methods: We analyzed data from a prospective surveillance registry of infants hospitalized for RSV-related complications across 39 U.

Challenges in determining the global burden of non-malignant central nervous system tumors: an analysis of international incidence and mortality data sources.

Background: Non-malignant tumors of the CNS contribute substantially to the morbidity and mortality from CNS tumors. It is critical to understand the epidemiology of non-malignant CNS tumors separately from CNS malignancies to inform resource allocation and policy since treatment and prognosis can differ. High quality international data on non-malignant CNS tumor burden are needed to accomplish this goal.

Critical Issues in the Management of CRMS/CFSPID Children: A National Real-World Survey.

Background: Notwithstanding guidance from the European Cystic Fibrosis (CF) Society (ECFS) neonatal screening (NBS) working group, significant variation persists in the evaluation and management of Cystic Fibrosis Screen Positive, Inconclusive Diagnosis (CFSPID) subjects, leaving many aspects of care under debate. This study reports the results of a national survey investigating management and treatment approaches of pre-school CFSPIDs in Italy.

Methods: In February 2024, a comprehensive questionnaire was distributed to all Italian CF centers.

Trochleoplasty in Pediatric Patients - A Systematic Review.

Background: Trochleoplasty is a surgical consideration for the treatment of high-grade trochlear dysplasia. The safety profile of this procedure remains particularly unclear in the skeletally immature population where concerns exist regarding physeal arrest and the development of premature patellofemoral arthritis. The purpose of this study was to systematically review the literature to evaluate trochleoplasty use, outcomes and complications observed among pediatric patients.

The Albright-Losee Low Profile Pivot Shift Test for ACL Deficiency.

Background: Recognizing ACL injuries on the field and in the office can be very challenging in awake and apprehensive patients. Despite high specificity, many published "pivot-shift" techniques have limited acceptance mainly because of unsatisfactory sensitivity. We describe in detail, four specific modifications and provide a critical review of our clinical experiences to empower the new user's readiness to master a novel screening procedure for ACL disruption.

Trochleoplasty Utilization in the Management of Patellofemoral Instability: Results From an International Survey of Surgeons.

Background: Considerable variability exists in the described clinical and radiographic indications for use, surgical techniques, postoperative management, and risk profile after trochleoplasty for the management of patellofemoral instability (PFI). In areas of clinical uncertainty, a cohesive summary of expert opinion and identification of areas of variation in current practice can be useful in guiding current practice and future research efforts.

Purpose: To assess the current indications for use, surgical techniques, postoperative rehabilitation practices, and observed complication profile for trochleoplasty in the management of PFI among surgeons who perform this procedure.

Use of virtual reality to remotely train healthcare professionals in paediatric emergency tracheostomy skills: protocol for a multi-centre, non-inferiority educational interventional study with historical controls.

Background: The insertion of a tracheostomy is an established technique used to wean patients off ventilatory support, manage secretions in complex conditions, and as a potentially life-saving procedure to bypass upper airway obstruction. Life-threatening complications during aftercare are not uncommon and may be influenced by a lack of education of carers or healthcare providers of children and young people living with a tracheostomy. Education programmes designed and supported by the National Tracheostomy Safety Project are effective, but resources are not available to educate the workforce at scale.

Nonacademic predictors of China medical licensing examination.

Background: National Medical Licensing Examination (NMLE) is the entrance exam for medical practice in China, and its general medical knowledge test (GMKT) evaluates abilities of medical students to comprehensively apply medical knowledge to clinical practice. This study aimed to identify nonacademic predictors of GMKT performance, which would benefit medical schools in designing appropriate strategies and techniques to facilitate the transition from medical students to qualified medical practitioners.

Methods: In 1202 medical students, we conducted the deletion-substitution-addition (DSA) and structural equation model (SEM) analyses to identify nonacademic predictors of GMKT performance from 98 candidate variables including early life events, physical conditions, psychological and personality assessments, cognitive abilities, and socioeconomic conditions.

Reduced Lower Body Muscular Strength and Endurance among Adult Survivors of Childhood Cancer.

Introduction: Impaired physical fitness is a possible late effect among adult survivors of childhood cancer (ASCC). Our study describes lower body muscular strength and endurance among ASCC using the 1-minute sit-to-stand (1-min STS) test, compares them with the general population, identifies risk factors, and describes changes over time.Methods: In a prospective multicenter cohort study, we invited ASCC ≥18 years of age at study, diagnosed between ages 0-20 treated in five pediatric oncology centers across Switzerland from 1976-2017 who survived ≥5 years for a 1-min STS test.

Association Between Language, Interpreter Use, and Pediatric Surgical Outcomes.

Background: Inequities exist in pediatric surgical outcomes. Differential outcomes have been identified across racial groups, geography, and socioeconomic standing. However, the association between preferred language, interpreter use, and surgical outcomes is not well-studied in pediatric surgical literature.

Subclonal TP53 and KRAS variants combined with poor treatment response identify ultra-high-risk pediatric T-ALL patients.

Variations in the TP53 and KRAS genes indicate a particularly adverse prognosis in relapsed pediatric T-ALL. We hypothesized that these variations might be subclonally present at disease onset and contribute to relapse risk. To test this, we examined two cohorts of children diagnosed with T-ALL: one with 81 patients who relapsed and 79 matched non-relapsing controls, and another with 226 consecutive patients, 30 of whom relapsed.

Multi-Modality Imaging in Cardio-Rheumatology.

Purpose Of Review: Our purpose was to discuss the advantages and disadvantages of various noninvasive imaging modalities in the evaluation of cardiovascular disease (CVD) in patients with autoimmune rheumatic diseases (ARDs). The detailed knowledge of imaging modalities will facilitate the diagnosis and follow up of CVD in ARDs.

Recent Findings: Autoimmune Rheumatic Diseases (ARDs) are characterized by alterations in immunoregulatory system of the body.

Perioperative Management of Patients with Preeclampsia: A Comprehensive Review.

Preeclampsia is a common condition of pregnancy characterized by hypertension complicated by cerebral, cardiac, hepatic, renal, hematologic, and placental dysfunction. Patients with preeclampsia frequently undergo cesarean delivery, the most common major surgical procedure in the world. They represent a high-risk perioperative cohort suffering significant preventable morbidity and mortality.

The burden of Alagille syndrome: uncovering the potential of emerging therapeutics - a comprehensive systematic literature review.

Alagille syndrome (ALGS) is a rare, cholestatic multiorgan disease associated with bile duct paucity, leading to cholestasis. Clinical symptoms of cholestasis include debilitating pruritus, xanthomas, fat-soluble vitamin deficiencies, growth failure, renal disease and impaired health-related quality of life (HRQoL). The main objective was to review the current literature on the epidemiological, clinical, psychosocial and economic burden of ALGS in view of the development of ileal bile acid transporter (IBAT) inhibitors.

The role of CNBP in brain atrophy and its targeting in myotonic dystrophy type 2.

Myotonic Dystrophy type 2 (DM2) is a multisystem disease affecting many tissues, including skeletal muscle, heart, and brain. DM2 is caused by unstable expansion of CCTG repeats in an intron 1 of a gene coding for cellular nuclear binding protein (CNBP). The expanded CCTG repeats cause DM2 pathology due to the accumulation of RNA CCUG repeats, which affect RNA processing in patients' cells.

Heterogeneous knowledge of childhood seizures and epilepsy care in Canadian healthcare Providers: Identifying the gaps.

Epilepsy is the most common chronic neurological condition in children. Many barriers exist in early recognition which cause delay in care and impact quality of life. Some of these children require advanced treatments which are underutilized due to lack of education, awareness and referrals.

Do national cancer control plans address care and research for children, adolescents, and young adults? A review of status, priorities, and recommendations across 41 European countries.

Paediatric cancers, although rare, are the leading cause of disease-related mortality in European children above one year. A key pillar of the European Health Union, Europe's Beating Cancer Plan (EBCP) puts a spotlight on childhood cancer. National Cancer Control Plans (NCCPs) have a key role but did not address childhood cancers sufficiently previously.

Enabling participation in community-dwelling children and young people with acquired brain injuries and their families: a theory-based, evidence-based and person-based approach to intervention development.

Objective: To codesign and develop an intervention to promote participation and well-being in children and young people (CYP) with acquired brain injury (ABI) and family caregivers.

Design: A complex intervention development study including a scoping review, mixed-methods study, co-design workshop and theoretical modelling.

Setting: Community-dwelling participants in one geographical region of the UK.

Scoping review of initiation criteria for inhaled nitric oxide in preterm infants (born <34 weeks) after 7 days of age.

Objectives: Inhaled nitric oxide (iNO) is a known treatment for pulmonary hypertension (PH) associated with bronchopulmonary dysplasia in preterm infants after 7 days of age (postacute phase). However, a consensus regarding the optimal criteria for initiating iNO therapy in this population in the postacute phase is currently lacking. This study, therefore, aimed to identify the criteria for initiating iNO therapy, alongside the associated clinical and echocardiographic findings, in this population.

How to Approach a Child About Concerns for Their Mental Health and Seeking Help: A Delphi Expert Consensus Study to Develop Guidelines on Mental Health First Aid for Supporting Children.

Background: Adults who live or work with children are an important source of support and are gateways to professional help when a child is experiencing a mental health problem. This study aimed to develop consensus-based guidelines on how adults such as parents, educators or health professionals should approach a child aged 5-12 years to discuss concerns about the child's mental health and seek help.

Methods: A Delphi consensus method with three rounds was used.