BOSTON, MA—Combined ledipasvir/sofosbuvir (LDV/SOF) is associated with 100% sustained virologic response (SVR) in patients with the inherited genetic disease thalassemia major who become chronically infected with some genotypes of hepatitis C virus (HCV), according to researchers at The Liver Meeting® 2016.
Among 100 enrolled white patients infected with HCV genotypes (GT)-1 and -4, LDV/SOF treatment for 12 weeks appeared to be well-tolerated and effective, yielding 100% SVR rates in an interim analysis, reported lead study author Alessandra Mangia, MD, Liver Unit, IRCCS-Ospedale Casa Sollievo della Sofferenza, in San Giovanni Rotondo, Italy.
The combination of interferon (IFN) and ribavirin (RBV) for the treatment of chronic hepatitis C in patients with thalassemia major is poorly tolerated. The study team sought to assess the safety and efficacy of an IFN- and RBV-free LDV/SOF regimen in these patients. Both IFN-naïve and experienced patients with thalassemia major and chronic GT1 and GT4 HCV were included in the study. Exclusion criteria for the study included compensated or uncompensated cirrhosis and prior IFN and RBV treatment failure.
Patients received LDV/SOF (90mg/400mg) FDC once daily for 12 weeks, and at the end of treatment, SVR12 was assessed as undetectable HCV RNA using an assay capable of detecting 12–15 RNA IU/mL.
At baseline, 58% of the patients were men, and the average age was 56 (range 39–74) years; 73% of patients had the IL28B non-CC genotype. Fifteen percent of patients had compensated cirrhosis.
“All but 2 patients achieved undetectable HCV RNA 4 weeks after the start of treatment,” reported Dr. Mangia. “All patients with available data achieved SVR4.”
The number of blood transfusions did not increase during treatment.
Adverse events included fatigue (77%0; headache (69%); nausea (51%) and insomnia (45%). No treatment-related adverse events were reported.
“No drug to drug interactions were registered,” noted Dr Mangia.
Study authors disclosed serving on advisory committees and review panels, and speaking fees for several drug companies, including Gilead Sciences, BMS, Shering-Plough, MSD, Boheringer, and BMS.