Liver-only transplant leads to better outcomes for kids with advanced CF
Isolated surgery outperforms combined liver-lung procedures for survival: Study
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Children with cystic fibrosis (CF) who undergo a liver transplant alone have significantly better survival rates than those who receive a combined liver-lung transplant, according to a new analysis of U.S. transplant data.
While both procedures are life-saving options for patients with advanced disease, the study highlights a gap in outcomes: children undergoing only a liver transplant had nearly a 90% survival rate after five years, compared to 72% for those who underwent a liver-lung transplant.
The findings also revealed that while liver transplants are effective for CF patients, their overall survival remains slightly lower than that of children receiving transplants for non-CF conditions. Researchers further noted that higher levels of bilirubin, a key indicator of liver damage, at the time of surgery were a major predictor of poorer outcomes.
The study, “Contemporary outcomes of isolated liver and combined liver-lung transplantation for cystic fibrosis in children,” was published in the Journal of Pediatric Gastroenterology and Nutrition.
The impact of CF on liver health
CF is caused by mutations in the CFTR gene, which disrupts the movement of salt and water in and out of cells. Under these conditions, the mucus in the body thickens, impairing the function of multiple organs, including the lungs, liver, and pancreas.
With the widespread use of highly effective CFTR modulator therapies and better multidisciplinary care, lung function and survival have improved significantly. As people with CF live longer, complications outside the lungs are becoming more clinically important.
One of the most serious complications is CF-associated liver disease, which affects roughly 3%-10% of patients and remains a leading cause of death in CF. Liver involvement in CF often begins early, typically within the first two decades of life, although progression varies widely.
For patients with severe liver disease, a liver transplant is the standard treatment. In some cases, especially when both liver and lung disease are advanced, a combined liver and lung transplant may be considered.
In this study, researchers evaluated patient survival and graft outcomes after isolated liver versus combined liver-lung transplant using U.S. transplant registry data. All participants in the study underwent their first transplant between 2002 and 2024.
“Isolated liver transplantation and combined liver-lung transplantation are treatment options for CF-related advanced liver disease, but limited large-scale pediatric data exist comparing outcomes of these approaches,” the team wrote.
The study evaluated 157 children with CF who underwent transplant, 145 of whom received a liver transplant alone, while 12 had a combined liver-lung transplant.
The team also included 10,276 children who underwent a liver transplant for non-CF indications, such as biliary atresia. In this liver disease that affects infants, the bile ducts, the tubes that carry the digestive fluid bile from the liver to the intestines, are blocked or absent at birth, trapping bile and causing liver damage.
The median age of CF children who received a liver transplant was 13 years. Compared to those who had combined liver-lung transplants, liver-only patients had slightly higher markers of liver dysfunction, including total bilirubin, but were otherwise similar.
In CF, higher bilirubin levels were strongly linked to more severe liver disease and were associated with complications such as ascites (fluid buildup), encephalopathy (brain dysfunction), and the need for intensive care or life support.
Survival outcomes favored liver-only transplant. The proportion of CF children alive after liver transplant alone was higher than after combined liver-lung transplant, at one year after the procedure (96.5% vs. 91.7%), three years (93.1% vs. 82.5%), and five years (89.1% vs. 72.2%).
Challenges in graft survival and long-term care
When compared with children who received liver transplants for other conditions, CF patients had slightly worse outcomes. Survival rates in non-CF patients were 94.7% at one year, 92.6% at three years, and 91.5% at five years.
A similar pattern was seen for liver graft survival. In CF patients, graft survival was higher after a liver transplant alone than after a liver-lung transplant at one year (91.7% vs. 83.3%), three years (86.8% vs. 75%), and five years (83.1% vs. 75%). In non-CF patients, graft survival rates were 90.4%, 86.9%, and 84.9% at one, three, and five years, respectively.
In an adjusted statistical analysis, the researchers found that CF children who underwent combined liver-lung transplant had about a threefold higher risk of death compared with those who received a liver transplant alone. Higher total bilirubin at the time of transplant was also associated with increased death risk. Similar graft survival risk differences were observed between the two groups.
In addition, CF patients who received a liver transplant alone had about twice the risk of death and a 54% higher risk of graft loss compared with non-CF patients.
The study shows that while liver transplants in children with CF are generally successful, survival rates are lower than in non-CF patients, and combined liver-lung transplants carry even higher risks, the researchers concluded.
“Further studies incorporating CF‐specific clinical data and longitudinal outcomes are needed to better inform transplant decisions and improve care for this vulnerable pediatric population,” the team added.
