{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,3,27]],"date-time":"2025-03-27T13:55:16Z","timestamp":1743083716638},"reference-count":18,"publisher":"Ovid Technologies (Wolters Kluwer Health)","issue":"1S","license":[{"start":{"date-parts":[[2021,12,1]],"date-time":"2021-12-01T00:00:00Z","timestamp":1638316800000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["lww.com","ovid.com"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2021,12]]},"abstract":"<jats:sec>\n            <jats:title>Background<\/jats:title>\n            <jats:p>Patients with inherited blood disorders (IBLD) have a high risk of hepatitis C virus (HCV) infection. The aim of this work was to assess the efficacy and safety of HCV direct-acting antiviral (DAA)-based treatment in patients with IBLD and chronic HCV infection.<\/jats:p>\n          <\/jats:sec>\n          <jats:sec>\n            <jats:title>Methods<\/jats:title>\n            <jats:p>Twenty-seven patients (25 with sickle cell disease, 1 with \u03b2-thalassemia and 1 with hemoglobin D-Punjab), including 3 with compensated cirrhosis, were included. They were treated with sofosbuvir in combination with ribavirin, daclatasvir, ledipasvir, or velpatasvir or with grazoprevir\/elbasvir for 8 or 12\u2009weeks. In the case of treatment failure, in-vitro assessment of resistance-associated substitutions (RASs) and full-length genome sequence analysis by means of deep sequencing were performed.<\/jats:p>\n          <\/jats:sec>\n          <jats:sec>\n            <jats:title>Results:<\/jats:title>\n            <jats:p>Treatment was safe and well-tolerated and there were no drug discontinuations due to DAA-related adverse events. Twenty-five out of the 27 patients (93%) achieved sustained virological response 12\u2009weeks post-treatment. One patient discontinued after 18\u2009days due to adverse events unrelated to the antiviral treatment. One patient infected with \u2018unusual\u2019 genotype 2 subtype 2m relapsed. Subtype 2m naturally carries the NS5A L31M RAS. In a genotype 2a subgenomic replicon model, L31M increased daclatasvir effective concentration 50% (EC<jats:sub>50<\/jats:sub>) by 97-fold, but velpatasvir EC<jats:sub>50<\/jats:sub> by only 3-fold, without altering the replication capacity. This patient was successfully retreated with sofosbuvir\/velpatasvir for 12\u2009weeks.<\/jats:p>\n          <\/jats:sec>\n          <jats:sec>\n            <jats:title>Conclusion<\/jats:title>\n            <jats:p>DAA-based regimens are well tolerated and highly efficacious in patients with chronic hepatitis C and IBLD in the real-world setting. Thus, DAA-based antiviral treatment should be prioritized in this thus far neglected population of HCV-infected patients.<\/jats:p>\n          <\/jats:sec>","DOI":"10.1097\/meg.0000000000002003","type":"journal-article","created":{"date-parts":[[2020,11,20]],"date-time":"2020-11-20T23:26:14Z","timestamp":1605914774000},"page":"e191-e196","update-policy":"http:\/\/dx.doi.org\/10.1097\/lww.0000000000001000","source":"Crossref","is-referenced-by-count":8,"title":["Real-world efficacy and safety of direct-acting antiviral drugs in patients with chronic hepatitis C and inherited blood disorders"],"prefix":"10.1097","volume":"33","author":[{"given":"Isaac","family":"Ruiz","sequence":"first","affiliation":[{"name":"Department of Hepatology, H\u00f4pital Henri Mondor, Universit\u00e9 Paris-Est"},{"name":"INSERM U955, Team \u201cViruses, Hepatology, Cancer\u201d, Institut Mondor de Recherche Biom\u00e9dicale (IMRB)"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Slim","family":"Fourati","sequence":"additional","affiliation":[{"name":"INSERM U955, Team \u201cViruses, Hepatology, Cancer\u201d, Institut Mondor de Recherche Biom\u00e9dicale (IMRB)"},{"name":"Department of Virology, National Reference Center for Viral Hepatitis B, C and D, H\u00f4pital Henri Mondor, Universit\u00e9 Paris-Est, Cr\u00e9teil, France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Abdelhakim","family":"Ahmed-Belkacem","sequence":"additional","affiliation":[{"name":"INSERM U955, Team \u201cViruses, Hepatology, Cancer\u201d, Institut Mondor de Recherche Biom\u00e9dicale (IMRB)"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Christophe","family":"Rodriguez","sequence":"additional","affiliation":[{"name":"INSERM U955, Team \u201cViruses, Hepatology, Cancer\u201d, Institut Mondor de Recherche Biom\u00e9dicale (IMRB)"},{"name":"Department of Virology, National Reference 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