Safety and Efficacy of Direct-Acting Antiviral Drugs in Patients with Haemoglobinophaties and Chronic Hepatitis C Infection

Background and Aim: Direct-acting antiviral drugs (DAAs) have a very high efficacy in patients with hepatitis C virus (HCV) infection, but they have not been extensively used in patients with haemoglobinophaties. To evaluate the safety and efficacy of DAA regimens in this subset we used the ITHACA-SITE dataset, which includes patients with haemoglobinophaties and chronic HCV liver disease treated in Italy.

Patients and methods: Between March 2015 and June 2016, 121 patients included in the ITHACA-SITE dataset started DAA regimens. Cirrhosis was defined by FibroScan^®showing≥12 kPa performed within 6 months before the treatment. Regimen choice and use of ribavirin were based on viral genotype and stage of disease, according to guidelines. Negative HCV RNA at week 12 of post-treatment follow up was considered as Sustained Virological Response (SVR).

Results: The mean age of 121 patients was 42 years, 75 (62%) were males, 101 (83.5%) had β-thalassemia major, 9(7.5%) had β-thalassemia intermedia and 10 (8.3%) had sickle cell disease. Sixty-six patients (54%) had the diagnosis of chronic hepatitis and 55 (46%) hadthe diagnosis of cirrhosis. The prevalence of HCV genotypes (G) was: G1a 6 (5%), G1b 70 (57.8%), G2 31 (25.6%), G3 6 (5%), G4 8 (6.6%). Fifty-six patients (46.3%) were Peg-interferon (P) and Ribavirin (R) naïve and 65 (53.7%) were P/R experienced, 20 patients (16.5%) had diabetes and 29 (24%) had heart disease. By June 2016 63 patients (52%) had concluded the treatment and 33 (27.3%) had concluded the post-treatment follow-up. By ITT, the rate of response at the end of treatment (ETR) was 100% (63/63) and 94% (31/33) of patients achieved a SVR. No patients stopped treatment because of adverse events. No interference with chelation therapy was observed.

Conclusions: Use of DAAs regimens in practice is safe and effective in patients with haemoglobinophaties and cirrhosis or chronic hepatitis due to HCV. The therapy is indicated also in patients with co-morbidity. The lifetime utility of HCV eradication in terms of reduction of events and overall survival needs evaluation in long-term observational cohorts.