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Deferoxamine Shows Promise for Liver Cancer in People with Advanced Disease

Deferoxamine, a cancer drug that has an anti-proliferative effect on tumor cells, led to improvement of hepatocellular carcinoma (HCC) in 2 out of 10 patients with moderate-to-severe liver disease, according to a letter in the August 11, 2011, New England Journal of Medicine.alt

Takahiro Yamasaki and colleagues from Yamaguchi University Graduate School of Medicine in Ube, Japan, evaluated deferoxamine therapy in patients with advanced HCC and serious liver function impairment.

The researchers previously reported that deferoxamine (an iron chelator) can prevent liver injury and development of precancerous lesions in rats. The anti-proliferative effect of deferoxamine arrests the cell cycle and induces apoptosis, they noted as background. Sorafenib (Nexavar) was recently established as the standard of care for people with advanced HCC and preserved liver function (Child-Pugh class A), but its safety and efficacy for patients with Child-Pugh class B or C (moderate or severe) liver disease is unknown.

The present study included 10 participants (6 men and 4 women) with advanced HCC who did not respond to hepatic arterial infusion chemotherapy using standard anticancer drugs. The average age was 64 years. Of these patients, 7 had hepatitis C virus (HCV) infection, 2 had hepatitis B virus (HBV) infection, and 1 did not have either type of viral hepatitis.

At baseline, 1 patient had HCC tumor stage II, 2 patients had stage IVA, and 7 had stage IVB (according to Liver Cancer Study Group of Japan criteria). Looking at liver function, 3 had Child-Pugh class A (least severe liver disease), 5 had class B (moderate severity), and 2 had class C (most severe).

Participants received arterial infusions of deferoxamine at a dose of 10-80 mg per kilogram of body weight over 24 hours on alternate days, using an injection port. Deferoxamine was administered an average of 27 times (range 9-78 infusions).


  • 2 patients had a partial response, 3 maintained stable disease, and 5 experienced disease progression (according to Eastern Cooperative Oncology Group criteria).
  • The overall response rate, therefore, was 20%.
  • Levels of tumor-related biomarkers -- alpha-fetoprotein, alpha-fetoprotein L3, des-gamma-carboxyprothrombin, or all of these -- decreased in patients with a partial response.
  • In 1 patient, "a massive hepatocellular tumor with lung metastases disappeared with deferoxamine treatment," the researchers wrote.
  • The 1-year cumulative survival rate was 20%.
  • 4 patients developed grade 2 or 3 (moderate or severe) interstitial pneumonia and 1 patient had grade 2 kidney dysfunction.
  • No grade 4 (life-threatening) adverse events were observed.

Based on these findings, the report authors concluded, "Deferoxamine may warrant testing in patients with Child-Pugh class B or C hepatocellular carcinoma."



T Yamasaki, S Terai, and I Sakaida. Deferoxamine for Advanced Hepatocellular Carcinoma (Correspondence). New England Journal of Medicine 365(6): 576-578. August 11, 2011.