Sofosbuvir-velpatasvir in children 3-17 years old with hepatitis C virus infection.

Autor: Jonas MM; Boston Children's Hospital, Boston, Massachusetts, USA., Romero R; Children's Healthcare of Atlanta and Emory University School of Medicine, Atlanta, Georgia, USA., Rosenthal P; Division of Pediatric Gastroenterology, Hepatology and Nutrition, Department of Pediatrics, University of California San Francisco, Benioff Children's Hospital, San Francisco, California, USA., Lin CH; Children's Hospital Los Angeles, Los Angeles, California, USA., Verucchi G; Università di Bologna, Bologna, Italy., Wen J; University of Pennsylvania and The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA., Balistreri WF; Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA., Whitworth S; Cook Children's Health Care System, Fort Worth, Texas, USA., Bansal S; King's College Hospital, London, UK., Leung DH; Baylor College of Medicine, Texas Children's Hospital, Houston, Texas, USA., Narkewicz MR; School of Medicine and Children's Hospital of Colorado, University of Colorado, Aurora, Colorado, USA., Gonzalez-Peralta RP; AdventHealth for Children and AdventHealth Transplant Institute, Orlando, Florida, USA., Mangia A; Ospedale Casa Sollievo della Sofferenza, San Giovanni Rotondo, Italy., Karnsakul W; Johns Hopkins University School of Medicine, Baltimore, Maryland, USA., Rao GS; Riley Hospital for Children, Indiana University School of Medicine, Indiana, Indianapolis, USA., Shao J; Gilead Sciences Inc., Foster City, California, USA., de Jong J; Gilead Sciences Inc., Foster City, California, USA., Parhy B; Gilead Sciences Inc., Foster City, California, USA., Osinusi A; Gilead Sciences Inc., Foster City, California, USA., Kersey K; Gilead Sciences Inc., Foster City, California, USA., Murray KF; Cleveland Clinic Children's Hospital, Cleveland, Ohio, USA., Sokal EM; Cliniques Universitaires Saint-Luc, Service de Gastroentérologie Hépatologie Pédiatrique, Université Catholique de Louvain, Bruxelles, Belgique., Schwarz KB; Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
Jazyk: angličtina
Zdroj: Journal of pediatric gastroenterology and nutrition [J Pediatr Gastroenterol Nutr] 2024 Jun; Vol. 78 (6), pp. 1342-1354. Date of Electronic Publication: 2024 Apr 21.
DOI: 10.1002/jpn3.12045
Abstrakt: Background: The safety and efficacy of sofosbuvir-velpatasvir in children aged 3-17 years with chronic hepatitis C virus (HCV) infection of any genotype were evaluated.
Methods: In this Phase 2, multicenter, open-label study, patients received once daily for 12 weeks either sofosbuvir-velpatasvir 400/100 mg tablet (12-17 years), 200/50 mg low dose tablet or oral granules (3-11 years and ≥17 kg), or 150/37.5 mg oral granules (3-5 years and <17 kg). The efficacy endpoint was sustained virologic response 12 weeks after therapy (SVR12). Dose appropriateness was confirmed by intensive pharmacokinetics in each age group.
Findings: Among 216 patients treated, 76% had HCV genotype 1% and 12% had genotype 3. Rates of SVR12 were 83% (34/41) among 3-5-year-olds, 93% (68/73) among 6-11-year-olds, and 95% (97/102) among 12-17-year-olds. Only two patients experienced virologic failure. The most common adverse events were headache, fatigue, and nausea in 12-17-year-olds; vomiting, cough, and headache in 6-11-year-olds; and vomiting in 3-5-year-olds. Three patients discontinued treatment because of adverse events. Four patients had serious adverse events; all except auditory hallucination (n = 1) were considered unrelated to study drug. Exposures of sofosbuvir, its metabolite GS-331007, and velpatasvir were comparable to those in adults in prior Phase 2/3 studies. Population pharmacokinetic simulations supported weight-based dosing for children in this age range.
Interpretation: The pangenotypic regimen of sofosbuvir-velpatasvir is highly effective and safe in treating children 3-17 years with chronic HCV infection.
(© 2024 European Society for Pediatric Gastroenterology, Hepatology, and Nutrition and North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition.)
Databáze: MEDLINE
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