201. Outcomes of Pediatric Heart Transplantation in Children with Major Genetic Syndromes are Comparable to Those with No Genetic Syndromes
Presented During: Precision Medicine in Congenital Heart Disease*
University of Alabama
Birmingham, AL
United States - Contact Me
Robert Dabal is the section chief of congenital heart surgery at the University of Alabama at Birmingham and Children's of Alabama. His clinical interest is in complex nenonatal repairs. He enjoys medical student and resident education as well.
University of Louisville School of Medicine
Louisville, KY
United States - Contact Me
Professor and Chief of Pediatric Cardiothoracic Surgery
University of Louisville School of Medicine
Co-Director of the Heart Institute
Norton Chidlren's Hospital
Monday, May 8, 2023: 7:15 AM - 7:30 AM
15 Minutes
Los Angeles Convention Center
Room: 403B
Abstract
Objective: Genetic syndromes (GS) are often linked to both congenital heart disease (CHD) and cardiomyopathy (CM). While GS are associated with prolonged recovery and increased morbidity and mortality following surgery for CHD, their effect on survival following pediatric heart transplantation (PHT) is not well described. We aim to compare outcomes following PHT between children with and without GS.
Methods: The United Network for Organ Sharing (UNOS) transplantation database was merged with the Pediatric Health Information System (PHIS) administrative database to identify children with major GS who received PHT from 2009-2019. Characteristics and outcomes were compared between the GS (n=225) and non-GS (n=2204) groups.
Results: Major GS were present in 225/2429 (9%) of PHT recipients. The most common syndromes were DiGeorge (n=28), muscular dystrophy (n=27), Down (n=26) and Turner (n=14). The incidence of CHD was higher in the GS compared to non-GS group, 54% vs. 38%, p<0.1. On the other hand, patient demographics, pulmonary artery pressure, incidence of renal and hepatic dysfunction, and requirement for dialysis, mechanical ventilation, ECMO and mechanical circulatory support on listing were not significantly different. [Table] The waitlist duration was also comparable between the GS (55 days, IQR 20-135) and non-GS (53 days, IQR 20-113) groups, p=0.4. The incidence of post-transplant complications were also similar including dialysis (8% vs. 5%, p=0.38), stroke (3% vs. 4%, p=0.34), primary graft dysfunction (2% vs. 2%, p=0.75), pacemaker need (1% vs. 1%, p=0.84) and rejection (3.4% vs. 3.4%, p=0.96). Post-transplant recovery parameters were likewise comparable. Survival at 10 years following PHT for GS and non-GS groups was 71% and 75%, respectively, p=0.59. This comparable survival was present in those with CM and those with CHD.
Conclusions: Children with major GS and end-stage CM or CHD are expected to have similar waitlist and post-transplant outcomes to those without GS. While early and late post-transplant care is individualized to each patient's characteristics including GS, the presence of GS should not be considered contraindication to PHT in well-selected children.
Methods: The United Network for Organ Sharing (UNOS) transplantation database was merged with the Pediatric Health Information System (PHIS) administrative database to identify children with major GS who received PHT from 2009-2019. Characteristics and outcomes were compared between the GS (n=225) and non-GS (n=2204) groups.
Results: Major GS were present in 225/2429 (9%) of PHT recipients. The most common syndromes were DiGeorge (n=28), muscular dystrophy (n=27), Down (n=26) and Turner (n=14). The incidence of CHD was higher in the GS compared to non-GS group, 54% vs. 38%, p<0.1. On the other hand, patient demographics, pulmonary artery pressure, incidence of renal and hepatic dysfunction, and requirement for dialysis, mechanical ventilation, ECMO and mechanical circulatory support on listing were not significantly different. [Table] The waitlist duration was also comparable between the GS (55 days, IQR 20-135) and non-GS (53 days, IQR 20-113) groups, p=0.4. The incidence of post-transplant complications were also similar including dialysis (8% vs. 5%, p=0.38), stroke (3% vs. 4%, p=0.34), primary graft dysfunction (2% vs. 2%, p=0.75), pacemaker need (1% vs. 1%, p=0.84) and rejection (3.4% vs. 3.4%, p=0.96). Post-transplant recovery parameters were likewise comparable. Survival at 10 years following PHT for GS and non-GS groups was 71% and 75%, respectively, p=0.59. This comparable survival was present in those with CM and those with CHD.
Conclusions: Children with major GS and end-stage CM or CHD are expected to have similar waitlist and post-transplant outcomes to those without GS. While early and late post-transplant care is individualized to each patient's characteristics including GS, the presence of GS should not be considered contraindication to PHT in well-selected children.
Presentation Duration
7 minute presentation; 7 minute discussion