This article is more than 5 years old. Information may no longer be current.

HCC liver transplant outcomes better in children with inherited liver disease

Children with inherited liver disease who underwent liver transplantation for hepatocellular carcinoma had superior long-term survival compared with children without inherited liver disease and adults with inherited liver disease, according to a recently published study.

“It has been suggested that HCCs arising in patients with inherited disease form a distinct clinical entity given that the pathophysiology behind these tumors differs considerably from other HCC types; evidence to support this hypothesis is, however, scarce,” Ulrich Baumann, MD, from the Hannover Medical School, Hannover, Germany, and colleagues wrote. “In this study we investigated the hypothesis that children transplanted for HCC with associated inherited liver disease show better LT outcomes than other HCC patients.”

To analyze the differences in survival after LT for HCC between children and adults either with or without inherited liver disease, the researchers enrolled LT recipients from the European Liver Transplant Registry, irrespective of potential or additional underlying diagnoses.

The study included 38 pediatric patients and 79 adult patients with inherited liver disease, and 137 pediatric patients and 316 adult patients without inherited liver disease.

Pediatric patients with underlying inherited liver disease were younger (median, 7 vs. 11 years; P < .001), more likely underwent urgent transplantation (17.7% vs. 4.1%; P = .007) and more likely received a partial graft (68.4% vs. 45.1%; P = .01) than children without an inherited liver disease.

According to multivariate analysis adjusted for sex, year of transplantation and shared frailty term for transplantation center, pediatric patients with inherited liver disease had significantly better long-term survival rates (HR = 0.29; 95% CI, 0.1-0.9). However, once adjusted for age, the protective effect of inherited disease became slightly smaller (HR = 0.45; 95% CI, 0.16-1.31).

Among those without inherited liver disease, survival rates increased with increasing age (HR = 1.11; 95% CI, 1.04-1.18), which the researchers did not observe among cases of inherited disease.

Compared with pediatric patients with inherited liver disease, adult patients with inherited disease were less likely to receive a partial graft (7.6% vs. 68.4%; P < .001), were more often diagnosed with refractory or controlled ascites (P = .048), more likely treated with an extracorporeal or vena cava bypass (P < .001), and were on the waitlist longer (median, 123 vs. 33 days; P < .001).

While there was no significant difference in survival at 2 years posttransplant between pediatric and adult patients with inherited liver disease, pediatric patients with inherited disease had significantly better long-term survival (HR = 0.27; 95% CI, 0.06-1.25).

“The potential long-term benefits observed for pediatric HCC patients with underlying inherited disease in comparison to their adult peers might at least partially be due to the relative survival advantages of children when compared to adults,” Baumann and colleagues concluded. “We suggest that our results are taken into consideration for future transplant organ allocation. However, studies with more detailed information on tumor characteristics, waiting times and disease severity are necessary to fully interpret our findings.” – by Talitha Bennett

Disclosure: The authors report no relevant financial disclosures.