Spectrum of Disease in Hospitalized Newborns with Congenital Micrognathia: A Cohort of 3,236 Infants at North American Tertiary-Care Intensive Care Units.

OBJECTIVE: To describe the spectrum of disease and burden of care in infants with congenital micrognathia from a multicenter cohort hospitalized at tertiary care centers. STUDY DESIGN: The Children's Hospitals Neonatal Database was queried from 2010 through 2020 for infants diagnosed with micrognathia. Demographics, presence of genetic syndromes, and cleft status were summarized. Outcomes included death, length of hospitalization, neonatal surgery, and feeding and respiratory support at discharge. RESULTS: Analysis included 3,236 infants with congenital micrognathia. Cleft palate was identified in 1266 (39.1%). A genetic syndrome associated with micrognathia was diagnosed during the neonatal hospitalization in 256 (7.9%). Median (IQR) length of hospitalization was 35 (16, 63) days. Death during the hospitalization (n = 228, 6.8%) was associated with absence of cleft palate (4.4%, P < .001) and maternal Black race (11.6%, P < .001). During the neonatal hospitalization, 1289 (39.7%) underwent surgery to correct airway obstruction and 1059 (32.7%) underwent gastrostomy tube placement. At the time of discharge, 1035 (40.3%) were exclusively feeding orally. There was significant variability between centers related to length of stay and presence of a feeding tube at discharge (P < .001 for both). CONCLUSIONS: Infants hospitalized with congenital micrognathia have a significant burden of disease, commonly receive surgical intervention, and most often require tube feedings at hospital discharge. We identified disparities based on race and among centers. Development of evidence-based guidelines could improve neonatal care.

Current Trends in Invasive Closure of Patent Ductus Arteriosus in Very Low Birth Weight Infants in United States Children's Hospitals, 2016-2021.

OBJECTIVE: To describe the current practices in invasive patent ductus arteriosus (PDA) closure (surgical ligation or transcatheter occlusion) in very low birth weight (VLBW) infants and changes in patient characteristics and outcomes from 2016 to 2021 among US children's hospitals. STUDY DESIGN: We evaluated a retrospective cohort of VLBW infants (birth weight 400-1499 g and gestational age 22-31 weeks) who had invasive PDA closure within 6 months of age from 2016 to 2021 in children's hospitals in the Pediatric Health Information System. Changes in patient characteristics and outcomes over time were evaluated using generalized linear models and generalized linear mixed models. RESULTS: 2418 VLBW infants (1182 surgical ligation; 1236 transcatheter occlusion) from 42 hospitals were included. The proportion of infants receiving transcatheter occlusion increased from 17.2% in 2016 to 84.4% in 2021 (P < .001). In 2021, 28/42 (67%) hospitals had performed transcatheter occlusion in > 80% of their VLBW infants needing invasive PDA closure, compared with only 2/42 (5%) in 2016. Although median postmenstrual age (PMA) at PDA closure did not change for the overall cohort, PMA at transcatheter occlusion decreased from 38 weeks in 2016 to 31 weeks by 2020, P < .001. Among those infants not intubated prior to PDA closure, extubation within 3 days postprocedure increased over time (yearly adjusted odds ratios of 1.26 [1.08-1.48]). Length of stay and mortality did not change over time. CONCLUSION: We report rapid adoption of transcatheter occlusion for PDA among VLBW infants in US children's hospitals over time. Transcatheter occlusions were performed at younger PMA over time.

Healthcare Costs of Major Morbidities Associated with Prematurity in US Children's Hospitals.

OBJECTIVE: To evaluate the healthcare costs attributed to major morbidities associated with prematurity, namely, bronchopulmonary dysplasia (BPD), intraventricular hemorrhage, necrotizing enterocolitis (NEC), retinopathy of prematurity (ROP), and nosocomial infections. STUDY DESIGN: This was a retrospective analysis of infants born at 24-30 weeks of gestation, admitted to children's hospitals in the Pediatric Health Information System between 2009 and 2018. Charges were adjusted by geographical price index, converted to costs using cost-to-charge ratios, inflated to 2018 US$, and total costs were accumulated for the initial hospitalization. Quantile regressions, which are less prone to bias from extreme outliers, were used to examine the incremental costs attributed to each morbidity across the entire cost distribution, including the median. RESULTS: There were 19 232 patients from 30 children's hospitals who were eligible. Higher costs were seen in lower gestational age, more severe morbidity, and those with higher number of comorbidities. Patients with surgical NEC, severe ROP, and severe BPD were the costliest with median total costs of $430 860, $413 825, and $399 495, respectively. Quantile regressions showed surgical NEC had the highest adjusted median incremental total cost ($48 621; 95% CI, $39 617-$57 626) followed by severe BPD ($35 773; 95% CI, $32 018-$39 528) and severe ROP ($22 561; 95% CI, $16 699-$28 423). Quantile regressions also revealed that surgical NEC, severe BPD, and severe ROP had increasing incremental costs at higher total cost percentiles, indicating these morbidities have a greater cost impact on the costliest patients. CONCLUSIONS: Severe BPD, surgical NEC, and severe ROP are the costliest morbidities and contribute the most incremental costs especially for the higher costs patients.

Severe hypertriglyceridemia associated with everolimus drug-eluting stent placement in an infant.

We report a case of severe hypertriglyceridemia associated with an everolimus drug-eluting stent in an infant with pulmonary vein stenosis. We review from current literature the mechanisms by which everolimus may cause dyslipidaemia, pharmacokinetics of everolimus in drug-eluting stents, and treatments of hypertriglyceridemia. This case demonstrates the need to closely monitor serum triglyceride levels after everolimus drug-eluting stent placement in infants.

Early recovery of T-cell function predicts improved survival after T-cell depleted allogeneic transplant.

Infection, relapse, and GVHD can complicate allogeneic hematopoietic stem cell transplantation (allo-HSCT). Although the effect of poor immune recovery on infection risk is well-established, there are limited data on the effect of immune reconstitution on relapse and survival, especially following T-cell depletion (TCD). To characterize the pattern of immune reconstitution in the first year after transplant and its effects on survival and relapse, we performed a retrospective study in 375 recipients of a myeloablative TCD allo-HSCT for hematologic malignancies. We noted that different subsets recover sequentially, CD8 + T cells first, followed by total CD4 + and naïve CD4 + T cells, indicating thymic recovery during the first year after HSCT. In the multivariate model, a fully HLA-matched donor and recovery of T-cell function, assessed by PHA response at 6 months, were the only factors independently associated with OS and EFS. In conclusion, T-cell recovery is an important predictor of outcome after TCD allo-HSCT.

Determinants and outcomes of adherence to recommendations from a multidisciplinary tumour conference for hepatocellular carcinoma.

BACKGROUND: The multidisciplinary tumour conference (MTC) represents the standard of care in the management of hepatocellular carcinoma (HCC). Clinical outcomes in relation to adherence and non-adherence to MTC recommendations have not been studied. METHODS: A total of 137 patients with HCC and cirrhosis whose cases were submitted to a first MTC discussion between 1 January 2009 and 31 December 2010 were identified. Clinical data, management recommendations, adherence, treatment regimens and overall survival were reviewed. RESULTS: There were 419 MTC discussions on 137 patients with cirrhosis and HCC. The MTC recommendations made in 145 discussions on 90 separate patients were not followed. Patient-related reasons for deviation from MTC recommendations included failure to attend for follow-up (n = 24, 16.6%), clinical deterioration (n = 19, 13.1%) and patient preference (n = 13, 9.0%). Physician-related reasons for discordance included treating physician preference (n = 43, 29.7%) and finding that the patient was not a candidate for the recommended intervention (n = 37, 25.5%). After the first MTC discussion, 62.0% of patients received the recommended treatment; these patients were more likely to be alive at 1 year compared with those who did not receive the recommended treatment (P = 0.007). More of the patients who followed recommendations underwent liver transplantation (25.6% versus 14.4%; P = 0.10). CONCLUSIONS: There are patient-related as well as physician-related reasons for non-adherence to recommendations. Non-adherence affects clinical outcomes and can be avoided in selected cases.

Estimating utilities for chronic kidney disease, using SF-36 and SF-12-based measures: challenges in a population of veterans with diabetes.

PURPOSE: Using transformations of existing quality-of-life data to estimate utilities has the potential to efficiently provide investigators with utility information. We used within-method and across-method comparisons and estimated disutilities associated with increasing chronic kidney disease (CKD) severity. METHODS: In an observational cohort of veterans with diabetes (DM) and pre-existing SF-36/SF-12 responses, we used six transformation methods (SF-12 to EQ-5D, SF-36 to HUI2, SF-12 to SF-6D, SF-36 to SF-6D, SF-36 to SF-6D (Bayesian method), and SF-12 to VR-6D) to estimate unadjusted utilities. CKD severity was staged using glomerular filtration rate estimated from serum creatinines, with the modification of diet in renal disease formula. We then used multivariate regression to estimate disutilities specifically associated with CKD severity stage. RESULTS: Of 67,963 patients, 22,273 patients had recent-onset DM and 45,690 patients had prevalent DM. For the recent-onset group, the adjusted disutility associated with CKD derived from the six transformation methods ranged from 0.0029 to 0.0045 for stage 2; -0.004 to -0.0009 for early stage 3; -0.017 to -0.010 for late stage 3; -0.023 to -0.012 for stage 4; -0.078 to -0.033 for stage 5; and -0.012 to -0.001 for ESRD/dialysis. CONCLUSION: Disutility did not increase monotonically as CKD severity increased. Differences in disutilities estimated using the six different methods were found. Both findings have implications for using such estimates in economic analyses.

Quality of life implications of bisphosphonate-associated osteonecrosis of the jaw.

PURPOSE: Potentially debilitating, osteonecrosis of the jaw (ONJ) is an emerging complication of bisphosphonates. However, its effect on quality of life (QoL) is unknown. We determined the ONJ-related QoL decline in a cancer patient cohort. PATIENTS AND METHODS: Thirty-four cancer patients with bisphosphonate-associated ONJ completed a telephone survey (October 2007 through May 2008). The Oral Health Impact Profile 14 (OHIP) retrospectively assessed participant oral health-related QoL before and after ONJ. Standardized ONJ descriptions were developed in a multidisciplinary, iterative process and were evaluated with three frequently used preference-based QoL measurement methods on a 0 (death) to 1 (perfect health) scale: Visual Analogue Scale (VAS), Time Trade-Off (TTO), and EQ-5D. RESULTS: ONJ significantly (p < .001) increased OHIP scores (worse QoL) for additive (3.56-16.53) and weighted (7.0-17.5) methods. Seven individual OHIP items significantly increased (Bonferroni correction p < .0035): pain, eating discomfort, self-consciousness, unsatisfactory diet, interrupted meals, irritability, and decreased life satisfaction. Mean preference-based QoL values significantly decreased (p < .001) with worsening ONJ stage (VAS, TTO, and EQ-5D): no ONJ (0.76, 0.86, 0.82), ONJ stage 1 (0.69, 0.82, 0.78), ONJ stage 2 (0.51, 0.67, 0.55), and ONJ stage 3 (0.37, 0.61, 0.32). As ONJ worsened, EQ-5D domain scores significantly increased (p < .001). Pain/discomfort and anxiety/depression contributed most to declining QoL. CONCLUSIONS: ONJ significantly affects QoL, a detriment that increases with worsening ONJ. QoL impairments for ONJ stages 2 and 3 are similar to other treatment side effects that influence decision-making. Bisphosphonate-associated ONJ QoL is an important consideration for patients, clinicians, and policy makers.