Waitlist mortality and post‐liver transplant outcomes of pediatric patients with hepatocellular carcinoma and hepatoblastoma in the United States

Abstract

Background: Liver transplantation (LT) is offered in cases of advanced disease for both pediatric patients with hepatoblastoma (HBL) and those with hepatocellular carcinoma (HCC). Current United States organ allocation priorities differ between the two groups. Methods:We retrospectively examined the waitlist and posttransplant outcomes of pediatric LT candidates with HBL and HCC using the United Network for Organ Sharing registry (February 2002 to September 2020). Results: Six hundred sixty-eight children with HBL and 95 children with HCC listed for first LT were identified. Patients with HBL were younger (p < .001), had lower laboratory Model for End-stage Liver Disease (MELD)/Pediatric End-stage Liver Disease (PELD) scores (p < .001), and had lesser proportion with encephalopathy (p = .01).Patients with HCC had an increased risk of waitlist mortality in univariable (unadjusted subdistribution hazard ratio [sHR] = 4.37, 95% confidence interval [CI], 2.01-9.51, p < .001) and multivariable competing risk regression (adjusted sHR = 3.08, 95% CI 1.13-8.37, p = .03) accounting for age and laboratory MELD/PELD score. Five hundred ninety-five children underwent LT for HBL and 76 for HCC. Patients transplanted for HBL had a significantly higher proportion with status 1B exception (71.3% vs. 7.9%, p < .001). No difference was observed in patient (unadjusted log-rank test, p = .52; adjusted hazard ratio [HR] = 0.77, 95% CI, 0.40-1.48, p = .43) or graft survival (unadjusted log-rank test, p = .93; adjusted HR = 0.74, 95% CI 0.42-1.33, p = .32) between HCC and HBL recipients. Conclusion:Waitlist mortality for pediatric LT candidates with HCC is significantly higher than for HBL, while posttransplant patient and graft survival are similar. This highlights an opportunity to improve equitable prioritization for children with HCC who may have reduced access to size-appropriate deceased donor organs and less effective bridge-to-transplant therapies.

Hepatoblastoma in a cirrhotic child with Alagille syndrome

“…Despite multiple comorbidities, post-liver transplant survival for patients with AGS is similar to that with biliary atresia 19. While the waitlist for paediatric liver transplant candidates with HCC is higher as compared with HB, post-transplant survival is the same 20. Although a review by Schindler et al suggested the need for surveillance for hepatocellular carcinoma in adult patients with AGS, there are no such recommendations for children 21.…”

Case Report: ALPPS hepatectomy, an alternative to liver transplantation in central PRETEXT III hepatoblastomas: a case series

“…Pertaining to organ availability, Wu et al reported an average waiting time of approximately 29 days on the transplant list for hepatoblastoma patients in the United States ( 11 ); nevertheless, there is decreased availability in developing countries. An analysis of Colombian data spanning from 2009 to 2017 indicated an all-age mean duration on the liver transplant waiting list of 4.6 months, extending up to a maximum of 8 months ( 12 ).…”

The effect of donor graft type on survival after liver transplantation for hepatoblastoma in children

“…29,30 A recent analysis of HBL LT outcomes showed that the waitlist mortality for HBL is below 5%. 31 Nevertheless, management of these patients the waiting list can be challenging due to the unpredictable nature of deceased donor graft availability and the importance of optimizing receipt of effective chemotherapy in the neoadjuvant and adjuvant setting, hence emphasizing the need to evaluate the impact of donor graft type on post-transplant survival.…”

“…Nowadays, as soon as the diagnosis of unresectable HBL is made, experts suggest that patients are initiated on neoadjuvant chemotherapy and referred for early LT evaluation if tumors remain unresectable, 27,28 provided that disease is limited to the liver or in the case of pulmonary metastases which resolve with resection and/or chemotherapy 29,30 . A recent analysis of HBL LT waitlist outcomes showed that the waitlist mortality for HBL is below 5% 31 . Nevertheless, management of these patients on the waiting list can be challenging due to the unpredictable nature of deceased donor graft availability and the importance of optimizing receipt of effective chemotherapy in the neoadjuvant and adjuvant setting, hence emphasizing the need to evaluate the impact of donor graft type on post‐transplant survival.…”