The higher waiting list mortality for liver transplantation in children with hepatocellular carcinoma (HCC) compared to hepatoblastoma (HBL) indicates that prioritization for children with HCC needs to be improved.
Waiting list mortality for liver transplantation (LT) for pediatric patients with hepatocellular carcinoma (HCC) is significantly higher than that for pediatric patients with hepatoblastoma (HBL), highlighting the need to improve prioritization for children with HCC, according to a study published in Pediatric Leukemia.
LT has a role in the treatment of advanced unresectable disease in pediatric cases of HBL and HCC, but organ allocation policies differ between the 2 groups. The researchers sought to analyze waiting-list mortality and post-transplant outcomes, which have not been evaluated in any study to date.
“Differential allocation in these patients calls for careful analysis of waiting-list-associated morbidity to identify opportunities to optimize equitable access to donor livers,” they explained.
They used data from the United Network for Organ Sharing (UNOS) database, which manages the Organ Procurement and Transplantation Network, to conduct a retrospective cohort study. They identified 688 children with HBL and 95 with HCC listed for the first LT. Of the total 763, 3.5% experienced waiting list mortality.
In 2002, UNOS adopted the MELD/PELD (Model End-Stage Liver Disease/Pediatric End-Stage Liver Disease) scoring systems, and since then, “patients with HBL have undergone several iterations of change in organ prioritization designations.” Since 2013, patients with HBL have progressed to an accelerated status, the authors explain. “In contrast, pediatric HCC has not seen comparable prioritization for organ allocation.”
While children with HBL have effective combination chemotherapy and relapse options beforehand, children with HCC have few treatment alternatives and therapies to lead them to organ transplantation.
In the study, children with HBL were on average younger than children with HCC (2 years vs 12 years) and had fewer days on the waiting list (29 days vs 39 days). Multivariable competitive risk regression showed that children with HCC had a waiting list mortality risk 3 times higher than that of children with HBL (adjusted subdivision HR [sHR], 3.08; 95% CI, 1.13-8.37; P = .03). Children with HCC also had an increased risk of unadjusted waiting list death (sHR 4.37; 95% CI 2.01-9.51; p < 0.001).
Of the 671 patients undergoing the first LT, 595 had HBL and 76 HCC, and children with HBL received the transplant at a younger age than children with HCC (2 years vs 12 years). The median waiting time for children receiving LT was 26.0 days for the HBL group and 35.5 days for the HCC group. Children with HBL had a longer length of stay after LT compared to HCC, but there was no difference in uncorrected patient survival or graft survival between the groups.
The authors found the following:
1-year survival was 89.9% for HBL vs 94.4% for HCC3-year survival was 83.4% for HBL vs 81.6% for HCC5-year survival was 82.0% for HBL vs 78.0% for HCC1 year graft survival was 85.2% for HBL vs 91.9% for HCC 3-year graft survival was 77.6% for HBL vs 78.2% for HCC 5-year graft survival was 75.9% for HBL vs 74.7% for HCC
The authors speculated that the differences in age, size, and graft type between patients with pediatric HCC and HBL may contribute to differential allocation and access to deceased organs.
“These findings indicate an opportunity to further adjust the prioritization of equitable organ allocation in children with HCC, who may have less access to custom deceased donor organs and less effective bridge-to-transplant therapies compared to children with HBL. the authors concluded. .
Wu WK, Ziogas IA, Matsuoka LK, et al. Waiting list mortality and outcomes after liver transplantation of pediatric patients with hepatocellular carcinoma and hepatoblastoma in the United States. Pediatric blood cancer. Published online Nov 4, 2021. doi:10.1002/pbc.29425