The study reports safety and efficacy of sofosbuvir-based treatment in non-cirrhotic, treatment-naive β-thalassemia major patients infected with HCV genotype-3. However, further studies with larger patient populations are needed to build up stronger evidence of safety and efficacy of this treatment approach for HCV infection in thalassemic patients.

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Background and aimsIn clinical studies, sofosbuvir–velpatasvir has demonstrated high cure rates and favorable tolerability in patients chronically infected with chronic hepatitis C virus (HCV) of any genotype. We evaluated the effectiveness and safety of sofosbuvir–velpatasvir administered with minimal medical monitoring to patients in India. MethodsAt 16 sites in India, 129 adult patients with chronic HCV infection of any genotype initiated 12 weeks of once-daily sofosbuvir–velpatasvir (400–100 mg). Patients with compensated cirrhosis or prior treatment experience could be included in the study. Study drug was dispensed monthly, but there were no on-treatment study assessments. The primary efficacy endpoint was rate of sustained virologic response (HCV RNA < 15 IU/mL) 12 weeks after treatment (SVR12), which was compared to a pre-specified performance goal of 85%.ResultsThe majority of patients had HCV genotype 3 infection (70%), followed by HCV genotype 1 (22%). The SVR12 rate was 93% (120/129; 95% CI, 87% to 97%) (p = 0.009 compared with the 85% performance goal). Of the nine patients who did not achieve SVR12, 1 experienced virologic failure, 2 relapsed after treatment, 1 withdrew consent after treatment, and 5 were lost to follow-up (1 during and 4 after treatment). Sofosbuvir–velpatasvir was well-tolerated, and no patients discontinued treatment because of an adverse event. The most frequently reported adverse events were headache (3% of patients), upper abdominal pain (2%), and pyrexia (2%).ConclusionsIn this study conducted at multiple sites in India, sofosbuvir–velpatasvir administered without genotype restriction or on-treatment safety assessments was well-tolerated and highly effective.

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