Purpose: In October 2018, the OPTN changed adult heart transplant (HT) allocation policy, increasing the number of adult candidates that had higher priority than pediatric candidates, potentially disadvantaging pediatric waitlist registrants.
Methods: To understand the impact of this policy change, we used SRTR data to identify 1469 pre-policy (7/2016-9/2018) and 2901 (10/2018-12/2022) post-policy pediatric (< 18 years) HT registrants. We quantified mortality and transplant risks using weighted cause-specific hazard models, and then using weighted competing risks regression. We further stratified these analyses by age to understand risks for those in direct competition with adults for organs (≥ 12 years).
Results: Post-policy, patients were more likely to need VAD prior to HT. There were no changes in post-policy access to HT (weighted hazard ratio [wHR] = 1.03 , p = 0.43). Mortality risk censoring for transplantation declined by 20% post-policy (wHR = 0.80 , p = 0.05). When accounting for competing risks of transplantation, post policy, mortality decreased by 24% compared to pre-policy (weighted subdistribution HR [wSHR] = 0.76 , p = 0.02). Post policy, 1-year transplant rate did not change in those < 12years (68.2%-71.0%, p = 0.77), but in those ≥ 12years, transplant rate increased (77.3%-81.0%, p = 0.003).
Conclusions: Mortality on the waitlist decreased and access to HT for pediatric registrants did not decline following the 2018 policy change. The decreased mortality rate may reflect changes in patient casemix and/or improved patient care. Continued surveillance is important in ensuring equity in pediatric, and adult, HT.
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http://dx.doi.org/10.1111/petr.14904 | DOI Listing |
Nat Commun
January 2025
Gleiberman Head and Neck Cancer Center, Moores Cancer Center, University of California San Diego Health, La Jolla, CA, 92037, USA.
Tumor initiation represents the first step in tumorigenesis during which normal progenitor cells undergo cell fate transition to cancer. Capturing this process as it occurs in vivo, however, remains elusive. Here we employ spatiotemporally controlled oncogene activation and tumor suppressor inhibition together with multiomics to unveil the processes underlying oral epithelial progenitor cell reprogramming into tumor initiating cells at single cell resolution.
View Article and Find Full Text PDFZhonghua Yi Xue Yi Chuan Xue Za Zhi
January 2025
Department of Pediatric Neurorehabilitation, Zhuhai Maternal and Child Health Care Hospital, Zhuhai, Guangdong 519000, China.
Objective: To investigate the clinical features and genetic variants associated with Multiple mitochondrial dysfunction syndrome (MMDS) type 3 in two children.
Methods: Two children diagnosed with MMDS type 3 at Zhuhai Maternal and Child Health Care Hospital in January 2021 were selected for this study. A retrospective analysis of their clinical data was carried out.
Epilepsy Res
January 2025
Jane and John Justin Institute for Mind Health, Cook Children's Medical Center, Ft Worth, TX, USA.
Lennox-Gastaut syndrome (LGS) is a severe developmental and epileptic encephalopathy characterized by multiple drug-resistant seizure types, cognitive impairment, and distinctive electroencephalographic patterns. Neuromodulation techniques, including vagus nerve stimulation (VNS), deep brain stimulation (DBS), and responsive neurostimulation (RNS), have emerged as important treatment options for patients with LGS who do not respond adequately to antiseizure medications. This review, developed with input from the Pediatric Epilepsy Research Consortium (PERC) LGS Special Interest Group, provides practical guidance for clinicians on the use of these neuromodulation approaches in patients with LGS.
View Article and Find Full Text PDFPediatr Pulmonol
January 2025
Department of Internal Medicine, Division of Pulmonary and Critical Care, University of Virginia, Charlottesville, Virginia, USA.
Introduction: While the diagnosis of cystic fibrosis (CF) is often straightforward and reliant on correlation between genetic testing and clinical signs and symptoms, there is a subset where the distinction is not nearly as clearcut. This has previously been reported in patients identified through newborn screening but not meeting full CF diagnostic criteria, earning the label of CF Screen Positive, Inconclusive Diagnosis (CFSPID) instead. A homologous diagnostic category in adults is named CF Transmembrane Conductance Regulator-Related Disorder (CFTR-RD).
View Article and Find Full Text PDFPediatr Transplant
February 2025
Department of Surgery, NYU Grossman School of Medicine, New York, New York, USA.
Purpose: In October 2018, the OPTN changed adult heart transplant (HT) allocation policy, increasing the number of adult candidates that had higher priority than pediatric candidates, potentially disadvantaging pediatric waitlist registrants.
Methods: To understand the impact of this policy change, we used SRTR data to identify 1469 pre-policy (7/2016-9/2018) and 2901 (10/2018-12/2022) post-policy pediatric (< 18 years) HT registrants. We quantified mortality and transplant risks using weighted cause-specific hazard models, and then using weighted competing risks regression.
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