Pediatric waitlist and heart transplant outcomes in patients with syndromic anomalies

Abstract

PurposeWe sought to determine whether the presence of a systemic SA with potential complicating factors affects waitlist and post–HT outcomes in pediatric patients.MethodsThis is a single‐center retrospective review of pediatric patients listed for HT between January 1, 2009, and July 1, 2018. Patients were selected based on the presence of any underlying syndromes, which included chromosomal anomalies, skeletal myopathies, connective tissue disorders, mitochondrial disease,and other systemic disorders. Waitlist and post–HT outcomes were compared to those without SA.ResultsA total of 243 patients were listed for HT, of which 21 (9%) patients had associated SA. Of those, 16 (76%) survived to transplant, 3 (14%) died while on the waitlist, 1 (5%) improved and was removed from the waitlist, and 1 (5%) patient is currently listed. Waitlist survival was not different between those with/without an associated syndrome (P = 1.0). Among those who survived to HT, there was no difference in listing days (70 vs 90, P = .8), survival to hospital discharge [14 (93%) vs 150 (95%), P = .6], post‐HT intubation days (2 vs 2 days, P = .6), or post‐HT hospital length of stay (18 vs 18 days, P = .8). Overall survival during the study period post‐HT was not different between groups (P = .8).ConclusionA SA was present in 9% of pediatric patients wait‐listed for HT, but was not associated with an increased waitlist mortality or post‐HT hospital morbidity or long‐term survival. For several anomalies, HT is safe and feasible.

Heart transplantation in an infant with Williams‐Beuren syndrome and rapidly progressive ischemic cardiomyopathy

“… Our patient had a complex intra‐ and post‐operative course in part related to the preoperative clinical status and complex anatomic repair with long bypass time, but also related to inherent extracardiac comorbidities associated with the syndromic nature of WBS . While recognizing the concerns for the possible progression of systemic and pulmonary vasculopathy, the post‐transplant course to date has been comparable to many other complex and challenging children with syndromic anomalies …”

Heart Transplantation in Children With Down Syndrome

“…This difference highlights potential limitations of administrative data in identification of in‐hospital mortality or identification of patients with DS using International Classification of Diseases, Ninth Revision ( ICD‐9 ) codes as 22 of the patients from our cohort are from centers that participate in PHIS and likely overlap with the patients reported by Broda et al Consistent with our data, 2 previous single‐center reports demonstrate acceptable posttransplant outcomes in patients with DS. 8 , 14 …”

“…Despite the high burden of cardiac disease in patients with DS, reports of heart transplantation (HTx) in this group are exceedingly rare. 8 , 13 , 14 A number of studies have reported lower than expected rates of referral for transplantation in children with DS. 15 , 16 This suggests that there may be an overarching perception that children with DS are not acceptable candidates for transplantation.…”

Jacobsen Syndrome with Hypoplastic Left Heart Syndrome: Outcome after Cardiac Transplantation

“…This case demonstrates that even when detailed genetic knowledge and ramifications of a syndrome are not available at the time major intervention decisions need to be made, a successful outcome can still be achieved. Her post-transplant course has not been dissimilar to many non-JS children and indeed most syndromic children do well after transplantation as a recent study demonstrated, showing no difference concerning waitlist duration, survival to transplant, or post-transplant course [ 24 ] . Of course, our patient’s follow up needs to be strict, considering the possibility of an allograft vasculopathy, which is one of the major factors that affects long-term graft and patient survival after heart transplantation [ 25 ] and was described in a JS patient after cardiac transplantation during the 2010 11q Research and Resource Group Biennial Conference [ 26 ].…”