Clinical News & Knowledge: Liver, Gallbladder & Biliary Tract Cancer
January 1, 2005
Oncology.
CHAPTER 15
Liver, gallbladder, and biliary tract cancers
Lawrence D. Wagman, MD, John M. Robertson, MD, and Bert O’Neil, MD
HEPATOCELLULAR CANCER
Hepatocellular carcinoma is one of the most common malignancies in the world, with approximately 1 million new cases recorded annually.
Epidemiology
Gender Hepatocellular carcinoma is the most common tumor in males worldwide,
with a male-to-female ratio of 5:1 in Asia and 2:1 in the United States.
Geography Tumor incidence varies significantly, depending on geographical
location. In the United States, hepatocellular carcinoma represents < 2%
of all tumors, whereas in the Far East and sub-Saharan Africa, this neoplasm
occurs at an incidence of 150 per 100,000 population and comprises almost
50% of all diagnosed tumors. A study analyzing SEER (Surveillance, Epidemiology,
and End Results) data has shown that the incidence of hepatocellular
carcinoma is rising in both white and black populations in the United
States, with a current incidence of about 3.4 cases per 100,000 in whites and
5.6 per 100,000 in blacks. Modeling of the spread of hepatitis C virus (HCV)
suggests that this number may continue to increase dramatically.
Age The incidence of hepatocellular cancer increases with age. The mean
age at diagnosis is 53 years in Asia and 67 years in the United States.
Race The incidence of hepatocellular tumors is higher in Asian immigrants
and blacks than in whites.
Survival In patients who undergo curative resection, the 5-year survival rate
is approximately 20%. Recurrence is common, with metastases arising in the
remaining liver, lungs, bone, kidneys, and heart. Most patients present with
unresectable disease. Patients with unimpaired liver function who can undergo
resection may experience significantly longer survival than those whose
disease is not resected.
Etiology and risk factors
Hepatitis B The close geographic relationship between hepatitis B incidence
and hepatocellular carcinoma rates is well recognized. In endemic areas of
hepatitis B, approximately 90% of all patients with hepatocellular carcinoma
are positive for hepatitis B surface antigen (HBsAg). The presence of the hepatitis
B "e" antigen has been found to increase risk ninefold. The most compelling
epidemiologic evidence of a causal relationship between hepatitis B infection
and hepatocellular carcinoma is the observation of a significant decline
in the incidence of childhood hepatocellular carcinoma after the introduction
of a national immunization program in Taiwan. The hepatitis B "x"
gene, which can interact with p53, has been a focus of recent study on the
pathogenesis of hepatocellular carcinoma.
Hepatitis C has also been implicated in hepatocellular carcinoma development.
The molecular mechanisms of HCV infection and carcinogenesis are
poorly understood. Unlike patients with hepatitis B infection, hepatocellular
carcinoma patients infected with hepatitis C usually have cirrhotic livers at
diagnosis; this finding suggests an extended period of infection (or hepatic
damage) before malignancy develops.
Alcohol Patients with alcoholic cirrhosis are at risk for hepatocellular carcinoma,
but the addition of HCV infection increases that risk dramatically.
Other possible etiologies include aflatoxin, hemochromatosis, hepatic
venous obstruction, thorotrast (a contrast agent no longer used for radiologic
procedures), androgens, estrogens, and α1-antitrypsin deficiency.
Signs and symptoms
Nonspecific symptoms Patients usually present with abdominal pain and
other vague symptoms, including malaise, fever, chills, anorexia, weight loss,
and jaundice.
Physical findings An abdominal mass is noted on physical examination in
one-third of patients. Less common findings include splenomegaly, ascites,
abdominal tenderness, muscle wasting, and spider nevi. Up to 10% of patients
may present with an acute abdomen due to a ruptured tumor.
Screening and diagnosis
Presently, no organization recommends routine screening of average-risk,
asymptomatic adults for liver, gallbladder, and biliary tract cancers.
α-Fetoprotein is produced by 70% of hepatocellular carcinomas. The normal
range for this serum marker is 0-20 ng/mL, and a level > 200 ng/mL is
essentially diagnostic for hepatocellular cancer in the absence of chronic, active
hepatitis B infection. In the presence of active hepatitis B infection, the
diagnostic cutoff is considered to be at least 1,000 ng/mL. In the setting of
hepatitis C infection, the cutoff for diagnosis of hepatocellular carcinoma has
not been well studied, but the specificity of values > 200 ng/mL appears to be
high. False-positive results may be due to acute or chronic hepatitis, germ-cell
tumors, or pregnancy.
Hepatitis B and C Given the association between hepatitis B and C and
hepatocellular cancer, blood should be sent for hepatitis B and C antigen and
antibody determinations.
Imaging The initial diagnostic test in the symptomatic patient may be ultrasonography,
as it is noninvasive and can detect lesions as small as 1 cm.
Ultrasound findings should be followed up with more specific imaging.
Triple-phase, high-resolution CT and contrast-enhanced MRI are the primary
imaging modalities used to diagnose and stage hepatocellular carcinoma. Recent
reports have documented a high number of false-positive results with
CT angioportography (CTAP) and CT hepatic angiography (CTHA). CT scan
predicts resectability in only 40%-50% of cases and does not accurately determine
the functional extent of cirrhosis. Major difficulties arise when the liver
parenchyma is not homogeneous and the lesions are smaller than 1 cm.
Laparoscopy is useful for the evaluation of small tumors, the extent of cirrhosis,
peritoneal seeding, and the volume of noninvolved liver and therefore
may be used prior to open laparotomy for resection. Laparoscopic or intraoperative
ultrasonography should be used to confirm preoperative imaging
tests. The laparoscopic results may change surgical management in up to onethird
of selected patients.
High-risk patients should be screened for hepatocellular carcinoma using
ultrasonography and serum α-fetoprotein levels. At present, however, there
is no standard screening interval, and screening has not been shown to affect
survival. Data suggest that, for screened patients, there is an increase in the
proportion of cancers that are resectable. A study comparing 6-month and
12-month survival intervals in a cohort of HIV-infected patients with hemophilia
showed no substantial benefit to more frequent screening.
Pathology
Three morphologic patterns of hepatocellular carcinoma have been described:
nodular, diffuse, and massive. Diffuse and massive types account for > 90%
of cases. The nodular type usually has multiple lesions in both lobes.
Histologic arrangements Several histologic arrangements have been identified:
trabecular, compact, pseudoglandular or acinar, clear cell, and a
fibrolamellar variant, which is associated with a relatively favorable prognosis.
Staging and prognosis
The staging system for hepatocellular cancer is based on the number and size
of lesions and the presence or absence of vascular invasion (Table 1). The
Okuda staging system accounts for the degree of liver dysfunction and may
better predict prognosis than the TNM stage. However, the Okuda staging
system does not adequately predict resectability. Because of the limited value
of standard staging, the most important factors determining survival are technical
resectability of lesions and degree of dysfunction of the normal liver.
Groups in Italy and China have created prognostic indices that may prove
useful for making treatment decisions.
Of the 5%-30% of patients who can undergo resection, factors associated with
improved survival include curative resection, small tumor size, well-differentiated
tumors, and normal performance status. Cirrhosis, nodal metastases, and
an elevated prothrombin time are indicative of a poor prognosis, as are male
sex, age > 50 years, poor performance status, duration of symptoms
< 3 months, tumor rupture, aneuploidy, high DNA synthesis rate, hypocalcemia,
vascular invasion, and a high serum α-fetoprotein level.
Treatment
SURGERY
Surgery is the form of treatment that offers the greatest potential for cure,
even though only a small minority of patients will actually be cured. Unfortunately,
many patients whose disease is thought to be resectable are clinically
understaged preoperatively.
Only stage I or II tumors have a significant likelihood of being resectable for
cure. However, a large tumor may still be potentially resectable for cure.
Moreover, contiguous involvement of large vessels (including the portal vein
and inferior vena cava) or bile ducts does not automatically mitigate against a
resection, especially in patients with a fibrolamellar histology, although such
resections are considerably more difficult.
Bilobar disease may be addressed with formal resection, tumor ablation techniques
(eg, cryoablation, radiofrequency ablation, and ethanol injection ablation),
or a combination of the two modalities.
Contraindications to resection include imminent clinical hepatic failure
(jaundice in the absence of biliary obstruction), hypoalbuminemia, ascites,
renal insufficiency, hypoglycemia, prolongation of the prothrombin and partial
thromboplastin times, main portal vein involvement, extrahepatic metastatic
disease, or other comorbid diseases that would preclude surgery of any
kind.
Noncirrhotic vs cirrhotic patients Resection should be performed in all
noncirrhotic patients when feasible. Resection of hepatocellular carcinoma
in the presence of cirrhosis is more controversial due to its increased morbidity
in this setting. Cirrhosis has been a major deterrent to resection in western
nations. Resectability rates vary from 0%-43% for cirrhotic patients,
whereas up to 60% of patients without cirrhosis undergo resection. Use of the
modified Child-Pugh classification of liver reserve may guide the surgeon in
preoperative assessment of liver function status and may aid in the selection
of operable patients.
When resection is performed in the presence of cirrhosis, Child class A patients
fare better than Child class B or C patients. Survival rates at 5 years
following resection range from 4%-36%, with noncirrhotic patients living longer
than cirrhotic patients.
Transplantation Owing to the risk of hepatic failure following resection in
cirrhotic patients, transplantation has become an option for patients with hepatocellular
cancer and cirrhosis. In a study of 181 patients with hepatocellular
carcinoma, Starzl and Iwatsuki found similar overall 5-year survival rates in
patients treated with transplantation vs resection (36% vs 33%). Survival rates
were similar in the two groups when tumors were compared for TNM stage.
However, survival was significantly improved in patients with concomitant
cirrhosis if they were treated with transplantation. Tumor recurrence rates for
stages II and III tumors were significantly lower after transplantation than
after resection, but no differences were seen for stage IV tumors.
Patients with cirrhosis and single tumors < 5 cm or multiple tumors (up to 3
with none > 3 cm) can be considered for transplantation. Larger tumors may
be treated with resection when feasible. Chemoembolization followed by transplantation
may be considered in selected patients. The use of transplantation
is significantly limited by the scarcity and lack of immediate availability of
donor organs. Recently, changes in the organ allocation system have decreased
waiting times for patients with documented hepatocellular cancer.
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