索磷布韦和达卡他韦治疗儿童和青少年地中海贫血合并丙型肝炎病毒感染的疗效和安全性

The Efficacy and Safety of Sofosbuvir and Daclatasvir Treatment in Children and Adolescents With Thalassemia and Hepatitis C Virus Infection.

作者信息

Samadder Riten K, Ray Gautam, Dutta Supradip, Hazra Avijit, Sadhukhan Provash, Chowdhury Abhijit, Ray Raja, Ahammed Sk Mahiuddin

机构信息

Department of Hepatology, School of Digestive and Liver Diseases, Institute of Post Graduate Medical Education and Research, Kolkata, India.

Divisions of Pediatric Gastroenterology, School of Digestive and Liver Diseases, Institute of Post Graduate Medical Education and Research, Kolkata, India.

出版信息

J Clin Exp Hepatol. 2024 May-Jun;14(3):101310. doi: 10.1016/j.jceh.2023.101310. Epub 2023 Dec 1.

DOI:10.1016/j.jceh.2023.101310

PMID:38264577

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10801307/

Abstract

BACKGROUND/AIM: Thalassemia patients are susceptible to hepatitis C virus (HCV) infection due to blood transfusions. Currently, data on treating HCV in thalassemic children with direct-acting antivirals is lacking. This study was performed to determine the efficacy and safety of sofosbuvir-daclatasvir combination therapy in thalassemic children and adolescents.

METHODS

A nonrandomized, open-label, interventional study was carried out in a tertiary care hospital. Consecutive noncirrhotic treatment-naïve thalassemic patients with HCV infection with viremia, within the age group of 6-18 years, were treated with the combination of sofosbuvir-daclatasvir: 200 mg + 30 mg for age 6-11 years (Group A) and 400 mg + 60 mg for age 12-18 years (Group B). The primary endpoint was sustained virological response at 12 weeks (SVR12).

RESULTS

A total of 70 patients (Group A 45, 64% male; Group B 25, 40% male) were recruited. The mean age was 8.5 years and 13.9 years in the two groups. Mean HCV Ribonucleic acid (RNA) levels in Groups A and B were 446906.1 IU/ml and 256187.8 IU/ml, respectively. SVR12 was achieved in 43 of 45 (95.5%) patients on an intention-to-treat basis and 43 of 44 (97.7%) patients on a perprotocol basis in Group A, and all patients in Group B (100%). In both groups, there was a significant improvement in biochemical parameters. Among the two patients who did not achieve SVR12 in Group A, one required termination of therapy due to urticaria.

CONCLUSION

Sofosbuvir-daclatasvir based treatment in noncirrhotic, treatment-naive thalassemic children and adolescents infected with HCV is effective and safe.

摘要

背景/目的：地中海贫血患者因输血易感染丙型肝炎病毒（HCV）。目前，缺乏关于使用直接抗病毒药物治疗地中海贫血儿童HCV感染的数据。本研究旨在确定索磷布韦-达卡他韦联合疗法治疗地中海贫血儿童和青少年的疗效和安全性。

方法

在一家三级护理医院开展了一项非随机、开放标签的干预性研究。对年龄在6至18岁、连续的未经治疗的非肝硬化HCV感染且有病毒血症的地中海贫血患者，采用索磷布韦-达卡他韦联合治疗：6至11岁患者使用200mg + 30mg（A组），12至18岁患者使用400mg + 60mg（B组）。主要终点是12周持续病毒学应答（SVR12）。

结果

共招募了70例患者（A组45例，男性占64%；B组25例，男性占40%）。两组的平均年龄分别为8.5岁和13.9岁。A组和B组的平均HCV核糖核酸（RNA）水平分别为446906.1 IU/ml和256187.8 IU/ml。A组在意向性分析中45例患者中有43例（95.5%）达到SVR12，符合方案分析中44例患者中有43例（97.7%）达到SVR12，B组所有患者（100%）均达到SVR12。两组的生化指标均有显著改善。A组未达到SVR12的2例患者中，1例因荨麻疹需要终止治疗。