{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,12]],"date-time":"2026-10-12T03:44:17Z","timestamp":1791776657741,"version":"4.3.4"},"reference-count":25,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2024,4,21]],"date-time":"2024-04-21T00:00:00Z","timestamp":1713657600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J. pediatr. gastroenterol. nutr."],"published-print":{"date-parts":[[2024,6]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>The safety and efficacy of sofosbuvir\u2013velpatasvir in children aged 3\u201317 years with chronic hepatitis C virus (HCV) infection of any genotype were evaluated.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>In this Phase 2, multicenter, open\u2010label study, patients received once daily for 12 weeks either sofosbuvir\u2013velpatasvir 400\/100\u2009mg tablet (12\u201317 years), 200\/50\u2009mg low dose tablet or oral granules (3\u201311 years and \u226517\u2009kg), or 150\/37.5\u2009mg oral granules (3\u20135 years and &lt;17\u2009kg). The efficacy endpoint was sustained virologic response 12 weeks after therapy (SVR12). Dose appropriateness was confirmed by intensive pharmacokinetics in each age group.<\/jats:p><\/jats:sec><jats:sec><jats:title>Findings<\/jats:title><jats:p>Among 216 patients treated, 76% had HCV genotype 1% and 12% had genotype 3. Rates of SVR12 were 83% (34\/41) among 3\u20135\u2010year\u2010olds, 93% (68\/73) among 6\u201311\u2010year\u2010olds, and 95% (97\/102) among 12\u201317\u2010year\u2010olds. Only two patients experienced virologic failure. The most common adverse events were headache, fatigue, and nausea in 12\u201317\u2010year\u2010olds; vomiting, cough, and headache in 6\u201311\u2010year\u2010olds; and vomiting in 3\u20135\u2010year\u2010olds. Three patients discontinued treatment because of adverse events. Four patients had serious adverse events; all except auditory hallucination (<jats:italic>n<\/jats:italic>\u2009=\u20091) were considered unrelated to study drug. Exposures of sofosbuvir, its metabolite GS\u2010331007, and velpatasvir were comparable to those in adults in prior Phase 2\/3 studies. Population pharmacokinetic simulations supported weight\u2010based dosing for children in this age range.<\/jats:p><\/jats:sec><jats:sec><jats:title>Interpretation<\/jats:title><jats:p>The pangenotypic regimen of sofosbuvir\u2013velpatasvir is highly effective and safe in treating children 3\u201317 years with chronic HCV infection.<\/jats:p><\/jats:sec>","DOI":"10.1002\/jpn3.12045","type":"journal-article","created":{"date-parts":[[2024,4,22]],"date-time":"2024-04-22T06:43:20Z","timestamp":1713768200000},"page":"1342-1354","source":"Crossref","is-referenced-by-count":20,"title":["Sofosbuvir\u2013velpatasvir in children 3\u201317 years old with hepatitis C virus infection"],"prefix":"10.1002","volume":"78","author":[{"given":"Maureen M.","family":"Jonas","sequence":"first","affiliation":[{"name":"Boston Children's Hospital Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rene","family":"Romero","sequence":"additional","affiliation":[{"name":"Children's Healthcare of Atlanta and Emory University School of Medicine Atlanta Georgia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Philip","family":"Rosenthal","sequence":"additional","affiliation":[{"name":"Division of Pediatric Gastroenterology, Hepatology and Nutrition, Department of Pediatrics University of California San Francisco, Benioff Children's Hospital San Francisco California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chuan\u2010Hao","family":"Lin","sequence":"additional","affiliation":[{"name":"Children's Hospital Los Angeles Los Angeles California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gabriella","family":"Verucchi","sequence":"additional","affiliation":[{"name":"Universit\u00e0 di Bologna Bologna Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jessica","family":"Wen","sequence":"additional","affiliation":[{"name":"University of Pennsylvania and The Children's Hospital of Philadelphia Philadelphia Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"William F.","family":"Balistreri","sequence":"additional","affiliation":[{"name":"Cincinnati Children's Hospital Medical Center Cincinnati Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Suzanne","family":"Whitworth","sequence":"additional","affiliation":[{"name":"Cook Children's Health Care System Fort Worth Texas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sanjay","family":"Bansal","sequence":"additional","affiliation":[{"name":"King's College Hospital London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniel H.","family":"Leung","sequence":"additional","affiliation":[{"name":"Baylor College of Medicine Texas Children's Hospital Houston Texas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael R.","family":"Narkewicz","sequence":"additional","affiliation":[{"name":"School of Medicine and Children's Hospital of Colorado University of Colorado Aurora Colorado USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Regino P.","family":"Gonzalez\u2010Peralta","sequence":"additional","affiliation":[{"name":"AdventHealth for Children and AdventHealth Transplant Institute Orlando Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alessandra","family":"Mangia","sequence":"additional","affiliation":[{"name":"Ospedale Casa Sollievo della Sofferenza San Giovanni Rotondo Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wikrom","family":"Karnsakul","sequence":"additional","affiliation":[{"name":"Johns Hopkins University School of Medicine Baltimore Maryland USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Girish S.","family":"Rao","sequence":"additional","affiliation":[{"name":"Riley Hospital for Children Indiana University School of Medicine Indiana Indianapolis USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jiang","family":"Shao","sequence":"additional","affiliation":[{"name":"Gilead Sciences Inc. 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