Norton Hospital

Long-term outcomes after Fontan vary widely. Although pre-Fontan hemodynamics predict early failure, their association with long-term outcomes remains unclear. We hypothesized that pre-Fontan hemodynamics predict long-term risk of death or transplantation. We analyzed data from the Pediatric Cardiac Care Consortium, a US-based multicenter registry including patients undergoing first-time Fontan with pre-Fontan catheterization and long-term follow-up. Patients undergoing a Fontan procedure before 18 years of age at any time between 1982 and 2011 were included in the study. Outcomes of interest were in-hospital Fontan failure (death or takedown) and postdischarge death or transplantation, identified through matching with the National Death Index and the Organ Procurement and Transplantation Network through 2022. Associations between pre-Fontan hemodynamics and long-term risk for death or transplantation were assessed with Kaplan-Meier survival curves and extended Cox regression. Among 1175 patients (736 [62.6%] male, 626 [53.3%] with systemic left ventricle), 1111 were discharged with Fontan physiology. Over a median postdischarge follow-up of 20.6 years (interquartile range, 18.2-24.4 years), 85 deaths and 49 transplantations occurred. Pre-Fontan mean pulmonary arterial pressure was the strongest hemodynamic predictor of postdischarge outcomes with a continuous association and no clear inflection point; 25-year transplantation-free survival declined from 83.7% (95% CI, 77.6-88.3) in the low mean pulmonary arterial pressure tertile to 73.7% (95% CI, 65.5-80.3) in the highest tertile (log-rank P=0.02). Each 1-SD increase in mean pulmonary arterial pressure was associated with 1.33-fold higher odds of in-hospital failure (adjusted odds ratio, 1.33 [95% CI, 1.00-1.77]; P=0.05) and a 2.2-fold higher hazard of death or transplantation (adjusted hazard ratio, 2.20 [95% CI, 1.62-3.00]; P<0.01) estimated at discharge. This hazard declined 3% per year after discharge (adjusted hazard ratio per year, 0.97 [95% CI, 0.95-0.99]; P<0.01) and resolved by 17 years in patients with systemic right ventricle and by 23 years in those with systemic left ventricle. Additional independent risk factors included systemic right ventricle versus systemic left ventricle (adjusted hazard ratio, 2.39 [95% CI, 1.65-3.46]; P<0.01) and delayed Fontan completion (>4 years of age versus 2 to 4 years of age; adjusted hazard ratio, 1.80 [95% CI, 1.25-2.60]; P=0.02). Elevated pre-Fontan mean pulmonary arterial pressure is a strong predictor of in-hospital and long-term post-Fontan risk of death or transplantation. Systemic right ventricle and delayed Fontan completion (>4 years of age) further increased risk. These findings support early Fontan consideration and ongoing hemodynamic surveillance to optimize long-term outcomes.

Background: The best treatment for children with KD who fail to respond to the first dose of IVIG (refractory KD) is currently unknown. The purpose of this study was to determine treatment practices of pediatric rheumatologists in North America who manage IVIG-refractory KD. Methods: A 34-item web-based survey was sent to 102 randomly selected members of the Childhood Arthritis and Rheumatology Research Alliance (CARRA). The anonymous survey addressed the use of primary intensification as well as the treatment of IVIG-refractory KD. Results: The response rate was 82%; 56% (all pediatric rheumatologists) completed the survey. Primary intensification was used for macrophage activation syndrome (MAS), KD shock, and those at high risk for coronary artery aneurysms (CAAs) by 84%, 76% and 52% of responders, respectively, with corticosteroids (CSs) used most frequently. For IVIG-refractory KD without CAA, a second dose of IVIG was used most often (63% alone; 23% plus CS). With non-giant CAAs, only 15% used a second IVIG alone, 40% used IVIG plus CS, and 35% took infliximab, usually with CS/IVIG. With giant CAA, treatments used most frequently were CS, a second IVIG, and infliximab (91%, 69%, and 58%, respectively), usually as combinations of two or more medications. Conclusions: Treatment of IVIG-refractory KD varies significantly among North American pediatric rheumatologists, particularly in the presence of CAAs. Our findings emphasize the need for research to identify the most effective therapy for this KD subgroup. The current use of primary intensification and the presence and size of the CAA will need to be considered as consensus treatment plans are developed.

Characterizing the modern person living with sickle cell disease (SCD) in the United States has been limited without a well-curated longitudinal registry. To address this, the Globin Research Network for Data and Discovery (GRNDaD) registry strives to collect clinical outcomes and quality of life metrics from Institutional Review Board-approved centres across the United States. Here, we examined the use of different disease-modifying therapies in (actively consented) adults and children with HgbSS and HgbS-β0 thalassaemia (SCA) from 38 sites. Of the 3169 active patients in GRNDaD, about 65% of subjects were on hydroxyurea (hydroxycarbamide; HU), and 2130 had SCA. As predicted, the absolute neutrophil counts were lower and mean corpuscular volumes were higher for patients on HU. However, there was a lower proportion of patients on HU in older age groups. In contrast, chronic RBC transfusion utilization was nearly twice as high in the 18- to 29-year-old age group than in the 11- to 17-year-old age group. For novel therapeutics, we examined use prior to voxelotor's removal from the market and prior to publication of the negative phase III trial of crizanlizumab. Voxelotor utilization in this cohort was three times that reported by claims data while crizanlizumab usage was nearly double, suggesting high-quality comprehensive sickle cell care could increase utilization of novel therapies.

To address the unmet need for a smaller pacemaker for babies, a specially modified implantable pulse generator was developed containing a Medtronic Micra subassembly in a polymer header connecting to a bipolar epicardial lead. The aim of this study was to report midterm follow-up data and outcomes of patients who underwent implantation of this device. Deidentified data were collected from 12 of 15 sites in the United States implanting the pediatric implantable pulse generator between March 2022 and February 2024. All 29 patients at these 12 sites within this timeframe were included in the analysis. The median age at implant was 15 days (range, 0 days to 3 years, including 1 outlier). The median weight was 2.3 kg (range, 1.3-11.4 kg). Gestational age was 28.5 weeks to term, with 23 (79%) patients born prematurely. Of those with anatomic information, 25% had congenital heart disease. The average duration of implant was 325 days (73-808 days). The most recent lead impedance mean was 612 ohms (450-840 ohms), ventricular capture threshold mean was 1 V @ 0.4 ms (range, 0.38-2.75 V), and R-wave sensing mean was 12.5 mV (3.6-20 mV). There were 7 generator explants (24%), removed at 6.5 to 31 months of age. The pediatric implantable pulse generator can be safely implanted in neonates and infants. This multicenter report demonstrates that the devices remain stable, with effective pacing, normal electrical parameters, and battery longevity aligned with projections. This novel pediatric pacemaker provides a viable alternative to standard-size generators and addresses a vital unmet need for these small patients.