Complete and Sustained Off-Therapy Response to Sorafenib in Advanced Hepatocellular Carcinoma.
A 75-year-old Caucasian woman with alcohol-related cirrhosis was admitted to our Unit in October 2012 for the diagnostic evaluation of a focal liver lesion detected by regular surveillance ultrasound. The subsequent dynamic CT and MR led to a diagnosis of infiltrative hepatocellular carcinoma (HCC) of 5 cm in the hepatic segment IV with neoplastic infiltration of the left branch of the portal vein, in absence of extrahepatic metastases. Therapy with sorafenib 400 mg bid was started and the subsequent dynamic CT performed at the 10th month of therapy showed a complete response according to RECIST criteria and mRECIST, while seriated dosages of α-fetoprotein levels showed a progressive reduction up to normalization. After 18 months of therapy, Sorafenib was discontinued due to a grade 3 adverse event. Nonetheless, all subsequent radiological controls, performed over the following two years confirmed a complete off-therapy response despite withdrawal of Sorafenib. After three years the patient is asymptomatic, with a preserved liver function and undetectable solid tumor lesions at dynamic CT. This case represents one of the few examples of complete response to anti-angiogenic drugs and, to our knowledge, the only case of sustained response, even after the discontinuation of Sorafenib, described so far in the literature.


BACKGROUND
hepatocellular carcinoma - HCC - BCLC - sorafenib - complete response.


BACKGROUND
AFP: alpha-fetoprotein; AE: adverse event; BCLC: Barcelona Clinic Liver Cancer; CT: computed tomography; HCC: hepatocellular carcinoma; mRECIST: modified response evaluation criteria in solid tumors; PS: Performance status; RCTs: randomized controlled trials.
Section of Gastroenterology,Villa Sofia-V. Cervello Hospital, Palermo, Italy. marcello.maida@hotmail.it.
Marcello
M
Maida
Section of Internal Medicine, Villa Sofia-V. Cervello Hospital, Palermo, Italy.
Fabio Salvatore
FS
Macaluso
Section of Radiology,Villa Sofia-V. Cervello Hospital, Palermo, Italy.
Franco
F
Valenza
Section of Internal Medicine, Villa Sofia-V. Cervello Hospital, Palermo, Italy.
Roberto
R
Virdone
D000368:Aged; D000970:Antineoplastic Agents / Q000008:administration & dosage*; D006528:Carcinoma, Hepatocellular / Q000097:blood / Q000188:drug therapy* / Q000473:pathology; D004334:Drug Administration Schedule; D005260:Female; D006801:Humans; D008113:Liver Neoplasms / Q000097:blood / Q000188:drug therapy* / Q000473:pathology; D009536:Niacinamide / Q000008:administration & dosage / Q000031:analogs & derivatives*; D010671:Phenylurea Compounds / Q000008:administration & dosage*; D047428:Protein Kinase Inhibitors / Q000008:administration & dosage*; D012074:Remission Induction; D000077157:Sorafenib; D013997:Time Factors; D014057:Tomography, X-Ray Computed; D016896:Treatment Outcome; D047368:Tumor Burden; D000509:alpha-Fetoproteins / Q000378:metabolism
C097353:AFP protein, human; D000970:Antineoplastic Agents; D010671:Phenylurea Compounds; D047428:Protein Kinase Inhibitors; D000509:alpha-Fetoproteins; D009536:Niacinamide; D000077157:Sorafenib