Morgan Stanley Children's Hospital

To assess the complementary value of transverse plane descriptors (orientation of the regional planes of deformation (ORPD) and local apical vertebral rotations (AVR)) integrated into the new modular three-tiered, four-modifier SRS-Lenke-Aubin 3D classification, relative to conventional 2D radiographic parameters and current Lenke 2D classification in adolescent idiopathic scoliosis (AIS). Transverse plane deformities of 285 surgically treated AIS cases reconstructed in 3D were quantified using ORPD and AVR, independently assessed for the proximal thoracic (PT), main thoracic (MT), and thoracolumbar/lumbar (TL/L) regions. Correlation analyses evaluated relationships between standard 2D parameters (Cobb angles, thoracic kyphosis (TK), lumbar lordosis (LL)) and transverse plane indices (ORPD, AVR). The distribution of ORPD and AVR subclasses was examined, as well as the associations between conventional Lenke lumbar and thoracic sagittal profile modifiers, and their corresponding 3D transverse plane modifiers. Complementary analyses also included 3D displacement of the apex relative to the end-vertebrae line (DAEVL). Nearly all ORPD-AVR subclass combinations were observed across regions, confirming the system's ability to capture diverse deformity patterns. ORPD and AVR were independent in PT and MT but correlated in TL/L (r = 0.69). Cobb angle correlated moderately with ORPD in MT (r = 0.43) and strongly in TL/L (r = 0.67), while correlations with AVR were moderate in MT (r = 0.50) and TL/L (r = 0.59). TK correlated negatively with MT ORPD (r = -0.58), whereas LL showed no association with TL/L ORPD. DAEVL correlated strongly with Cobb across all regions but only weakly to moderately with ORPD. Associations between Lenke 2D modifiers and ORPD were strong in TL/L (V = 0.59) and moderate in MT (V = 0.37). Multivariate models showed that Cobb and TK explained ~ 44% of MT ORPD variance, while > 55% of ORPD variability across regions remained unexplained by 2D parameters. ORPD and AVR provide complementary, region-specific 3D information beyond conventional 2D measures and Lenke modifiers. Their integration into the SRS-Lenke-Aubin 3D classification enhances dimensional completeness while preserving usability, laying the groundwork for future outcome-based evaluations.

This article, developed in collaboration with pediatric cardiologists and school nurses, reviews common student complaints that may have a cardiac etiology including palpitations, chest pain, and syncope. We discuss the initial steps that a school nurse should perform for each of these complaints, including relevant history taking and physical exam. We identify red flags which should raise suspicion for a cardiac etiology and outline the initial steps a school nurse should take in managing these students. We discuss which cases require assessment with a bedside automatic external defibrillator and emergent contact of emergency medical services (EMS), less-emergent cases requiring EMS contact and transfer to the emergency department, as well as those occasions where the assessment suggests it is appropriate for a parent to pursue less-urgent outpatient evaluation by a primary healthcare provider or pediatric cardiologist.

Children with inflammatory bowel diseases (IBD) have limited access to the available advanced therapies, given the lengthy gap between adult and pediatric approval. We aimed to review key hurdles for pediatric trials and recommend practical solutions. This position paper was developed jointly by the European and North American Societies for Pediatric Gastroenterology Hepatology and Nutrition (ESPGHAN and NASPGHAN), in consultation with patient representatives. A systematic review was performed for identified topics, and two voting rounds with two group meetings led to agreement on 24 statements. The systematic review (reviewing 4366 manuscripts, of which 123 were included in tables of evidence and 213 in support of 23 statements) found similar biologic pathogenesis, and similar or better effectiveness and safety in children older than 2 years compared to adults. Pharmacokinetics were similar in adolescents but dissimilar in younger children. The review also found sufficiently accurate noninvasive endpoints to reflect post-induction treatment response. There was no significant added benefit for ileocolonoscopy over sigmoidoscopy in ulcerative colitis. Drugs should be approved in children >12 years and ≥40 kg based on extrapolation from adult and real-world data. While efficacy may be extrapolated to children <40 kg, pharmacokinetics cannot and thus one open-label single-arm study should be performed to establish a dose that matches adult exposure-response from the adult trial in which adolescents may be enrolled if not exposed to placebo. Full colonoscopies should be minimized in the pediatric dosing trial. Efficacy in patients with infantile IBD cannot be extrapolated from adult data.

Outcomes in pediatric heart transplantation have steadily improved since the first pediatric heart transplant was performed by Dr. Kantrowitz and his team in 1967; however, there is still progress to be made. Patients with congenital heart diseases (CHD) consistently have worse waitlist mortality, post-transplant survival, infection rates, and post-transplant lymphoproliferative disease (PTLD) risk, with single ventricle disease (SVD) patients being at particularly high risk for rejection and rejection with severe hemodynamic compromise (RSHC). Furthermore, there has been little improvement in the development of cardiac allograft vasculopathy (CAV) and PTLD over the decades, and survival following diagnosis continues to remain poor for all patients regardless of diagnosis. Hopefully, as new advances in immunosuppression, mechanical support, and surgical techniques emerge, we will continue to develop a deeper understanding of the co-morbidities associated with pediatric heart transplant to further improve the survival and quality of life of pediatric heart transplant recipients.

BackgroundIn the FUEL (Fontan Udenafil Exercise Longitudinal) trial, a positive treatment effect was identified for outcomes at the ventilatory anaerobic threshold but not for the primary outcome, oxygen consumption (Vo2) at peak exercise. This disparate response may be explained by the physiologic challenge of improving peak Vo2 in participants with near‐normal baseline exercise performance.MethodsParticipants were divided into subgroups by baseline predicted peak Vo2 (<80% versus ≥80%). Treatment effect was evaluated in those with a baseline peak Vo2 <80% predicted and linear regression was performed to examine the interaction between subgroup and response to therapy for the primary and secondary outcomes.ResultsOf the 379 participants with paired exercise data, 302 (80%) had a baseline peak Vo2 <80% predicted. In this subgroup, the primary outcome of peak Vo2 improved significantly after 6 months of udenafil treatment, when compared with placebo (0.23±4.17 mL/kg per min versus −0.90±3.74 mL/kg per min; P=0.021). Secondary outcome measures, including Vo2 at ventilatory anaerobic threshold (P=0.023), work at ventilatory anaerobic threshold (P=0.032), and the myocardial performance index (P=0.007) were all significantly improved as well. A significant interaction was found between exercise subgroups and response to udenafil for peak Vo2 (P=0.036) but not for other outcomes.ConclusionsIn this post hoc subgroup analysis, after exclusion of patients with Fontan circulation with near‐normal baseline peak Vo2, a positive treatment effect of udenafil was identified for the primary outcome of peak Vo2. The interaction between baseline peak Vo2 and treatment may have influenced the outcome of the FUEL trial.RegistrationURL: https://clinicaltrials.gov; Unique identifier: NCT0274115.