Targeted chemotherapy effective for HCC in woman with Alagille
She's remained free of recurrence 9 years after receiving treatment
A targeted treatment called transarterial chemoembolization (TACE) successfully helped control hepatocellular carcinoma (HCC), the most common type of liver cancer, in a young woman with Alagille syndrome who was not eligible for a liver transplant, a study shows.
TACE involves the delivery of a combination of cancer-fighting drugs and an agent that blocks the tumor’s blood supply.
“Remarkably, she has achieved an exceptional 9-year survival post-treatment with stable imaging and no recurrence, a highly uncommon outcome for HCC in Alagille syndrome,” researchers wrote. “This case underscores the long-term management of HCC in Alagille syndrome, effectively controlled through TACE.”
The study, “Successful long-term survival following lipiodol chemoembolization for hepatocellular carcinoma in Alagille syndrome: A case report,” was published in Radiology Case Reports.
Liver transplant not an option for many patients
In Alagille syndrome, liver problems are caused by too few bile ducts, or the tubes that normally carry bile from the liver to the small intestine to help with digestion. This bile duct paucity leads to slowed bile flow, or cholestasis, as well as bile accumulation in the liver, which can cause damage and cirrhosis, or irreversible liver scarring.
“HCC is an aggressive complication of Alagille syndrome, and treatment options remain limited,” the researchers wrote.
While a liver transplant remains the most effective treatment, many patients can’t get one because of a shortage of donors or the risks of surgery in a liver that’s already damaged.
In the report, a team of researchers in Morocco described the case of a 29-year-old woman with Alagille syndrome who developed HCC that was effectively managed with TACE.
She was diagnosed with Alagille syndrome as a baby and had been monitored closely for chronic cholestasis since.
At age 20, she was found to have infiltrating HCC, meaning the liver cancer had spread and was growing into surrounding tissues. She had cirrhosis due to Alagille, and her blood levels of alpha-fetoprotein (AFP), a biomarker of liver cancer, were high at 282 nanograms (ng)/mL.
A team of doctors from different specialties reviewed her case and decided on lipiodol-based TACE to treat her. Lipiodol is an oily liquid that has blood vessel-blocking properties and is used to deliver chemotherapy directly into the blood vessels feeding a tumor, particularly in the liver.
TACE is usually considered palliative for HCC, meaning it aims to help control the tumor and potentially prolong survival, rather than cure the disease.
Case highlights potential of targeted chemotherapy
The woman had her first TACE session in 2015, followed by a second in 2016 when the cancer came back. In both sessions, anti-cancer drug doxorubicin was mixed with lipiodol and injected into tumor-feeding vessels until blood flow stopped. Imaging showed the mixture stayed inside the tumor, indicating a successful treatment.
Since her last treatment, the woman was regularly monitored, and her condition remained stable. Her blood levels of bilirubin, a biomarker of liver damage, have remained high due to Alagille. However, her AFP levels dropped and remained consistently low, at 13-15 ng/mL, indicating no signs of liver cancer and a good response to treatment over nine years.
A procedure in which a thin tube with a light and a video camera attached is passed through the mouth revealed swollen veins in the esophagus, which is the tube that transports food from the throat to the stomach. This is a sign of portal hypertension, a liver disease complication marked by high blood pressure in the veins that supply the liver.
Beta-blockers were considered to reduce high blood pressure and the risk of bleeding in the esophagus, but were delayed due to missed appointments.
This case highlights the feasibility of long-term disease control with lipiodol chemoembolization, even in patients who are ineligible for liver transplantation.
Despite this, the woman remained in good health, with no signs of advanced liver disease, such as jaundice — the yellowing of the skin and whites of the eyes — or ascites, the buildup of fluid in the abdomen.
“Although adherence to medical appointments has been inconsistent, her liver function has been preserved,” the researchers wrote.
This case provides valuable insight into the potential of TACE as a long-term disease control strategy in such patients, the researchers concluded.
“The patient’s favorable outcome underscores the importance of a tailored, multidisciplinary approach, with regular monitoring and coordinated care among hepatologists [liver specialists], oncologists [cancer specialists], and radiologists [imaging specialists] to optimize outcomes and enhance quality of life in complex cases,” the researchers wrote.
“This case highlights the feasibility of long-term disease control with lipiodol chemoembolization, even in patients who are ineligible for liver transplantation,” they wrote, adding more research is needed to explore this and other local treatments for similar patients, especially when a liver transplant is not possible.
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