The Efficacy and Safety of Peginterferon Alpha-2a (PEGASYS) monotherapy in the Treatment of Chronic Hepatitis C Infected Subjects with Transfusion Dependent Thalassemia

Background and aims: Interferon monotherapy is currently the only approved treatment for chronic hepatitis C (CHC) infection in transfusion dependent thalassemic patients, in whom ribavirin has limited use because of its hematologic complications. Our aim was to evaluate the efficacy and safety of pegylated Interferon monotherapy for the treatment of HCV infection in transfusion dependent thalassemic patients.

Methods: The trial was a multicenteric, open label, single treatment prospective study of Peginterferon alfa-2 a (PEGASYS, 180 micg per week) for a period of 48 weeks. 32 subjects, 18 to 42 years old (mean ± SD: 24.1 ± 9.44 years), whose serum HCV RNA was positive and mean ALT remained greater than 1.5 times upper limit of normal were enrolled. A percutaneous liver biopsy was performed before treatment and all patients underwent monthly assessment of any adverse events and were monitored for serum ALT. Efficacy was assessed by measuring serum HCV RNA following  24 week treatment-free period . One patient missed follow up and another died due to a drug unrelated cause and 30 patients were evaluated.

Results: Liver biopsy showed mild fibrosis in 31.2%, moderate fibrosis in 53.1% and cirrhosis in 15.6% of patients. Siderosis was severe in 16 patients (50%). In 26 out of 30 patients (86.6%) HCV RNA was negative at the end of treatment (ETR response). Data about 24 weeks post treatment was available in 23 patients, which showed a sustained virological response (SVR) of about 14/23 (60.8%). Two patients had an elevated end of treatment serum ALT instead of negative HCV RNA but their ALT returned to normal as soon as the treatment stopped. These 2 patients were considered to have INF toxicity.

Conclusion: Our experience indicates that the cure of HCV-related liver disease in thalassemic patients is not an unrealistic aim and may be reached with Peginterferon alfa-2 a monotherapy in a sizable portion of cases.