Mehta Rajiv, Kabrawala Mayank, Nandwani Subhash, Desai Pankaj, Bhayani Vishwa, Patel Sanjay, Parekh Viral
Department of Gastroenterology, Surat Institute of Digestive Sciences (SIDS), Surat 395002, India.
J Clin Exp Hepatol. 2018 Mar;8(1):3-6. doi: 10.1016/j.jceh.2017.06.002. Epub 2017 Jun 23.
β-thalassemia major patients are susceptible to Hepatitis C Virus (HCV) infection owing to life-long dependency for blood-transfusion. Moreover, this patient population is at risk of progression of liver fibrosis or development of cirrhosis as a consequence of both iron overload and HCV infection. Hence, this study was carried out to evaluate efficacy and safety of the combination regimen of sofosbuvir and daclatasvir for HCV infection in β-thalassemia major patients.
The present study was a prospective observational study which enrolled multi-transfused β-thalassemia major patients treated with a combination regimen of sofosbuvir (400 mg) and daclatasvir (60 mg) daily for 12 weeks for HCV infection during May 2016 and November 2016 depending upon inclusion and exclusion criteria of the study. Sustained virological response at post-treatment week-12 (SVR-12) was defined as negative HCV-RNA at week-12 after completion of antiviral treatment.
A total of 10 multi-transfused patients with β-thalassemia major were included in the study. Average age of the patient was 13.60 ± 4.38 years. All the included patients were treatment-naïve, non-cirrhotic and infected with HCV genotype-3. All the patients achieved SVR-12. There was significant reduction in aspartate aminotransferase ( = 0.005) and alanine aminotransferase level ( = 0.005) and serum ferritin level ( = 0.028) after completion of the antiviral treatment. The reported adverse events include nausea, vomiting and anorexia which were managed conservatively. None of the patient required dose reduction or termination of antiviral treatment.
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.
重型β地中海贫血患者由于终生依赖输血，易感染丙型肝炎病毒（HCV）。此外，由于铁过载和HCV感染，该患者群体有肝纤维化进展或发展为肝硬化的风险。因此，本研究旨在评估索磷布韦和达卡他韦联合方案治疗重型β地中海贫血患者HCV感染的疗效和安全性。
本研究为前瞻性观察性研究，根据研究的纳入和排除标准，纳入2016年5月至2016年11月期间接受索磷布韦（400mg）和达卡他韦（60mg）联合方案每日治疗12周以治疗HCV感染的多次输血的重型β地中海贫血患者。治疗后第12周的持续病毒学应答（SVR-12）定义为抗病毒治疗完成后第12周HCV-RNA阴性。
本研究共纳入10例多次输血的重型β地中海贫血患者。患者的平均年龄为13.60±4.38岁。所有纳入患者均未接受过治疗、无肝硬化且感染HCV基因3型。所有患者均实现了SVR-12。抗病毒治疗完成后，天冬氨酸转氨酶（P = 0.005）、丙氨酸转氨酶水平（P = 0.005）和血清铁蛋白水平（P = 0.028）均显著降低。报告的不良事件包括恶心、呕吐和厌食，均经保守处理。无一例患者需要减少剂量或终止抗病毒治疗。
本研究报告了基于索磷布韦的治疗方案在未接受过治疗、无肝硬化且感染HCV基因3型的重型β地中海贫血患者中的安全性和疗效。然而，需要对更多患者进行进一步研究，以更有力地证明这种治疗方法对地中海贫血患者HCV感染的安全性和疗效。
