Daniele Serranti1, Gabriella Nebbia2, Mara Cananzi3, Emanuele Nicastro4, Fabiola Di Dato5, Federica Nuti2, Silvia Garazzino6, Erika Silvestro6, Vania Giacomet7, Federica Forlanini7, Michele Pinon8, Pier Luigi Calvo8, Silvia Riva9, Icilio Dodi10, Antonina Marta Cangelosi10, Roberto Antonucci11, Silvia Ricci12, Elisa Bartolini1, Greta Mastrangelo1, Sandra Trapani1, Matteo Lenge13, Paola Gaio3, Pietro Vajro14, Raffaele Iorio5, Lorenzo D'Antiga4, Giuseppe Indolfi15. 1. Paediatric and Liver Unit, Meyer Children's University Hospital of Florence, Firenze. 2. Pediatric Hepatology, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan. 3. Unit of Gastroenterology, Digestive Endoscopy, Hepatology and Care of Children with Liver Transplantation, University Hospital of Padova, Padova. 4. Paediatric Hepatology, Gastroenterology and Transplantation, Hospital Papa Giovanni XXIII, Bergamo. 5. Pediatric Liver Unit, University of Naples Federico II, Napoli. 6. Ospedale Infantile Regina Margherita, Città della Salute e della Scienza della Città di Torino, Infectious Diseases Unit, University of Turin, Torino. 7. Unit of Paediatric Infectious Disease ASST FBF SACCO, University of Milan, Milan. 8. Ospedale Infantile Regina Margherita, Città della Salute e della Scienza della Città di Torino, Pediatric Gastroenterology Unit, Torino. 9. Unità di Epatologia Pediatrica e Trapianto di Fegato ISMETT Istituto Mediterraneo per i Trapianti e Terapie ad Alta Specializzazione, Palermo. 10. U.O.C. Pediatria Generale e d'Urgenza, Ospedale dei Bambini "Pietro Barilla", Azienda Ospedaliero-Universitaria di Parma, Parma. 11. Pediatric Clinic, Department of Medical, Surgical and Experimental Sciences, University of Sassari, Sassari. 12. Immunology Division, Section of Pediatrics, Department of Health Sciences, University of Florence and Meyer Children's University Hospital of Florence. 13. Clinical Trial Office, Meyer Children's University Hospital of Florence, Firenze. 14. Dipartimento di Medicina, Chirurgia e Odontoiatria "Scuola Medica Salernitana", Università di Salerno, Salerno. 15. Department Neurofarba, University of Florence Paediatric and Liver Unit, Meyer Children's University Hospital of Florence, Firenze, Italy.
Abstract
OBJECTIVES: Sofosbuvir/Ledipasvir (SOF/LDV) has been approved by the European Medicine Agency (EMA) for the treatment of children and adolescents (at least 3 years of age) with chronic hepatitis C (CHC) genotype 1, 3, and 4 infection. The aim of this study was to evaluate the efficacy and safety of SOF/LDV in adolescents (12 to <18 years old) with CHC in the real-world setting. METHODS: Prospective, open-label, multicentre study involving 12 Italian centres. Patients received the fixed-dose combination of SOF/LDV (400/90 mg) once daily ± ribavirin as per EMA approval and recommendations. The key efficacy endpoint was sustained virological response 12 weeks after the end of treatment (SVR12) as per intention-to-treat analysis. Safety was assessed by adverse events and clinical/laboratory data. RESULTS: Seventy-eight consecutive adolescents (median age 15.2 years, range 12-17.9; girls 53.8%) were enrolled and treated between June 2018 and December 2019. Genotype distribution was as follows: genotype 1 (82.1%), 3 (2.5%), and 4 (15.4%). Seventy-six (97.4%) patients completed treatment and follow-up. Overall, SVR12 was 98.7%. One patient was lost to follow-up after 4 weeks of treatment; 1 patient completed treatment and missed the follow-up visit. No virological breakthrough or relapse were observed. No patient experienced grade 3 to 4 adverse event or serious adverse event. CONCLUSIONS: The results of this real-world study confirmed the high efficacy and the optimal safety profile of SOF/LDV for treatment of CHC in adolescents.
OBJECTIVES: Sofosbuvir/Ledipasvir (SOF/LDV) has been approved by the European Medicine Agency (EMA) for the treatment of children and adolescents (at least 3 years of age) with chronic hepatitis C (CHC) genotype 1, 3, and 4 infection. The aim of this study was to evaluate the efficacy and safety of SOF/LDV in adolescents (12 to <18 years old) with CHC in the real-world setting. METHODS: Prospective, open-label, multicentre study involving 12 Italian centres. Patients received the fixed-dose combination of SOF/LDV (400/90 mg) once daily ± ribavirin as per EMA approval and recommendations. The key efficacy endpoint was sustained virological response 12 weeks after the end of treatment (SVR12) as per intention-to-treat analysis. Safety was assessed by adverse events and clinical/laboratory data. RESULTS: Seventy-eight consecutive adolescents (median age 15.2 years, range 12-17.9; girls 53.8%) were enrolled and treated between June 2018 and December 2019. Genotype distribution was as follows: genotype 1 (82.1%), 3 (2.5%), and 4 (15.4%). Seventy-six (97.4%) patients completed treatment and follow-up. Overall, SVR12 was 98.7%. One patient was lost to follow-up after 4 weeks of treatment; 1 patient completed treatment and missed the follow-up visit. No virological breakthrough or relapse were observed. No patient experienced grade 3 to 4 adverse event or serious adverse event. CONCLUSIONS: The results of this real-world study confirmed the high efficacy and the optimal safety profile of SOF/LDV for treatment of CHC in adolescents.
Authors: Emanuele Nicastro; Lorenzo Norsa; Angelo Di Giorgio; Giuseppe Indolfi; Lorenzo D'Antiga Journal: World J Gastroenterol Date: 2021-05-28 Impact factor: 5.742