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Objective Multicenter longitudinal outcome data for Fontan patients surviving into adulthood are lacking. The aim of this study was to better understand contemporary outcomes in Fontan survivors by collecting follow-up data in a previously well-characterized cohort. Design Baseline data from the Fontan Cross-Sectional Study (Fontan 1) were previously obtained in 546 Fontan survivors aged 11.9 ± 3.4 years. We assessed current transplant-free survival status in all subjects 6.8 ± 0.4 years after the Fontan 1 study. Anatomic, clinical, and surgical data were collected along with socioeconomic status and access to health care. Results Thirty subjects (5%) died or underwent transplantation since Fontan 1. Subjects with both an elevated (>21 pg/mL) brain natriuretic peptide and a low Child Health Questionnaire physical summary score (<44) measured at Fontan 1 were significantly more likely to die or undergo transplant than the remainder, with a hazard ratio of 6.2 (2.9–13.5). Among 516 Fontan survivors, 427 (83%) enrolled in this follow-up study (Fontan 2) at 18.4 ± 3.4 years of age. Although mean scores on functional health status questionnaires were lower than the general population, individual scores were within the normal range in 78% and 88% of subjects for the Child Health Questionnaire physical and psychosocial summary score, and 97% and 91% for the SF-36 physical and mental aggregate score, respectively. Since Fontan surgery, 119 (28%) had additional cardiac surgery; 55% of these (n = 66) in the interim between Fontan 1 and Fontan 2. A catheter intervention occurred in 242 (57%); 32% of these (n = 78) after Fontan 1. Arrhythmia requiring treatment developed in 118 (28%) after Fontan surgery; 58% of these (n = 68) since Fontan 1. Conclusions We found 95% interim transplant-free survival for Fontan survivors over an average of 7 years of follow-up. Continued longitudinal investigation into adulthood is necessary to better understand the determinants of long-term outcomes and to improve functional health status.

Objectives We sought to identify factors associated with timing and surgical outcomes of the superior cavopulmonary anastomosis (SCPA). Methods The Pediatric Heart Network’s Infant Single Ventricle trial database identified participants who underwent SCPA. Factors potentially associated with age at SCPA, length of stay (LOS) and death by 14 months of age were evaluated. Factors included subject demographics, cardiac anatomy, measures from neonatal hospitalization and pre-SCPA visit, adverse events, echocardiographic variables, intraoperative variables, SCPA type, and the number of concurrent cardiac surgical procedures. Age at SCPA was analyzed by Cox proportional hazards regression. Natural log LOS was analyzed by multiple linear regression. Results SCPA was performed in 193 subjects at 5.2 (IQR 4.2, 6.2) months of age and weight of 5.9 (IQR 5.3, 6.6) kg. Median LOS was 7 days (IQR 6, 10). There were 3 deaths and 1 transplant during SCPA hospitalization and 3 deaths and 3 transplants between discharge and 14 months of age. Age at SCPA was associated with center and interstage adverse events. Longer LOS was associated with younger age and greater case complexity. SCPA type, valve regurgitation, ventricular ejection fraction and ventricular end-diastolic pressure were not independently associated with age at SCPA or LOS. Conclusions Greater case complexity and more frequent interstage adverse events are associated with earlier age at SCPA. Significant variation in age at SCPA among centers, independent of subject factors, highlights a lack of consensus regarding optimal timing. Factors associated with LOS may offer insights for improving pre-SCPA care and surgical outcome.

Objective: CHAMPION-NMOSD (NCT04201262) is a phase 3, open-label, externally controlled interventional study evaluating the efficacy and safety of the terminal complement inhibitor ravulizumab in adult patients with antiaquaporin-4 antibody-positive (AQP4+) neuromyelitis optica spectrum disorder (NMOSD). Ravulizumab binds the same complement component 5 epitope as the approved therapeutic eculizumab but has a longer half-life, enabling an extended dosing interval (8 vs 2 weeks). Methods: The availability of eculizumab precluded the use of a concurrent placebo control in CHAMPION-NMOSD; consequently, the placebo group of the eculizumab phase 3 trial PREVENT (n = 47) was used as an external comparator. Patients received weight-based intravenous ravulizumab on day 1 and maintenance doses on day 15, then once every 8 weeks. The primary endpoint was time to first adjudicated on-trial relapse. Results: The primary endpoint was met; no patients taking ravulizumab (n = 58) had an adjudicated relapse (during 84.0 patient-years of treatment) versus 20 patients with adjudicated relapses in the placebo group of PREVENT (during 46.9 patient-years; relapse risk reduction = 98.6%, 95% confidence interval = 89.7%-100.0%, p < 0.0001). Median (range) study period follow-up time was 73.5 (11.0-117.7) weeks for ravulizumab. Most treatment-emergent adverse events were mild/moderate; no deaths were reported. Two patients taking ravulizumab experienced meningococcal infections. Both recovered with no sequelae; one continued ravulizumab treatment. Interpretation: Ravulizumab significantly reduced relapse risk in patients with AQP4+ NMOSD, with a safety profile consistent with those of eculizumab and ravulizumab across all approved indications.