The effect of Kaftrio (Trikafta) on liver problems in cystic fibrosis.

In France, researchers compared rates of serious liver complications before and after starting treatment with Caftrio (elexacaftor, tezacaftor, and ivacaftor), which is sold in the United States under the brand name Tricafta.

The study found that the risk of serious liver problems among patients dropped by more than 70% in the years since the triple combination therapy was approved.

The overall results are impressive:
• The risk of serious liver complications dropped by 72%
• Bleeding from esophageal and gastric varices fell from 8.8 to 0.4.
• The need for liver transplantation fell from 4.1 to 0.04 cases per 1,000 people per year
• Overall mortality fell 15-fold
• Rates of progressive or decompensated progressive cirrhosis fell from 20.1 to 0.9
• Rates of hepatocellular carcinoma, the primary liver cancer, fell from 1.4 to 0.06.

Cystic fibrosis is a genetic disease in which the body produces thick mucus. It accumulates not only in the lungs but also in the liver. In 20-40% of patients, liver problems develop: jaundice, yellowing of the skin and whites of the eyes, itching, and enlargement of the liver or spleen. In some severe cases: liver fibrosis, or scarring, and eventually cirrhosis, where scar tissue replaces healthy tissue and limits liver function.

Cirrhosis can also increase a patient’s risk of liver cancer and increase the need for a liver transplant. Caftrio is a new drug that helps a mutated protein in the body work better. It has been shown primarily to improve lung function. But growing evidence suggests it also protects the liver.

The researchers emphasize that this is not yet conclusive evidence. It is possible that the improvements are due not only to the drug but also to other changes in cystic fibrosis treatment. “More research is needed to determine for sure whether Caftrio is really that good at protecting the liver,” the authors say.

The study, published in the journal JHEP Reports, included more than 10,000 patients aged 14 to 29.