{"id":4741,"date":"2013-11-05T14:45:19","date_gmt":"2013-11-05T19:45:19","guid":{"rendered":"http:\/\/blogs.nejm.org\/?p=4741"},"modified":"2013-11-05T14:45:19","modified_gmt":"2013-11-05T19:45:19","slug":"sofosbuvir-and-ledipasvir-phase-2-and-when-small-studies-make-a-big-impact","status":"publish","type":"post","link":"https:\/\/blogs.nejm.org\/hiv-id-observations\/index.php\/sofosbuvir-and-ledipasvir-phase-2-and-when-small-studies-make-a-big-impact\/2013\/11\/05\/","title":{"rendered":"Sofosbuvir and Ledipasvir Phase II, and When Small Studies Make a Big Impact"},"content":{"rendered":"

Imagine for a moment the ideal medical future … there’s a vaccine that prevents the common cold … colon cancer screening no longer requires that horrendous “prep” … electronic medical records are easily accessible, intuitive, secure, and all communicate effortlessly with one another … your doctor’s office has an actual person who answers the phone instead of a prerecorded list of “menu options” … and hepatitis C is cured with one pill a day taken for 8 weeks.<\/p>\n

Not sure about the timeline for the first four, but based on this study of sofosbuvir and ledipasvir just published in the Lancet<\/em><\/a>, the hepatitis C outcome is right around the corner:<\/p>\n

In cohort A, SVR12 [no detectable virus 12 weeks after stopping treatment] was achieved by 19 (95%)<\/strong> of 20 patients (95% CI 75\u2013100) in group 1, by 21 (100%)<\/strong> of 21 patients (84\u2013100) in group 2, and by 18 (95%)<\/strong> of 19 patients (74\u2013100) in group 3. In cohort B, SVR12 was achieved by 18 (95%)<\/strong> of 19 patients (74\u2013100) in group 4 and by all 21 (100%)<\/strong> of 21 patients (84\u2013100) in group 5 …\u00a0These findings suggest that the fixed-dose combination of sofosbuvir-ledipasvir alone or with ribavirin has the potential to cure most patients with genotype-1 HCV, irrespective of treatment history or the presence of compensated cirrhosis.<\/p><\/blockquote>\n

You’ll note that the “groups” were pretty small, and that only one of them — group 1 — actually got just the one-pill sofosbuvir\/ledipasvir combination for 8 weeks, but regardless, these are pretty spectacular results. Even the most “complex” treatment group in this study received sofosbuvir\/ledipasvir plus ribavirin for only 12 weeks, which is light years from the complexity of our current interferon-based therapies. Light years better, of course.<\/p>\n

And since sofosbuvir has already been reviewed by the FDA advisory panel, we should be getting at least this drug by December 2013 at the latest; the combination pill with ledipasvir could be available some time next year (according to this article in the New York Times<\/em><\/a>), and several other investigational HCV drugs will likely be approved soon as well (simeprevir this year too).<\/p>\n

But calm down already. Take a deep breath. It’s a small phase II study. Shouldn’t we be more cautious? Shouldn’t we avoid wildly overstating the importance of this particular treatment approach? Of course, that’s the prudent thing to do.<\/p>\n

But can people with HCV and their treaters be ecstatic about these results anyway? You betcha.<\/p>\n","protected":false},"excerpt":{"rendered":"

Imagine for a moment the ideal medical future … there’s a vaccine that prevents the common cold … colon cancer screening no longer requires that horrendous “prep” … electronic medical records are easily accessible, intuitive, secure, and all communicate effortlessly with one another … your doctor’s office has an actual person who answers the phone […]<\/p>\n","protected":false},"author":6,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[3],"tags":[],"class_list":["post-4741","post","type-post","status-publish","format-standard","hentry","category-health-care"],"post_mailing_queue_ids":[],"_links":{"self":[{"href":"https:\/\/blogs.nejm.org\/hiv-id-observations\/index.php\/wp-json\/wp\/v2\/posts\/4741","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/blogs.nejm.org\/hiv-id-observations\/index.php\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/blogs.nejm.org\/hiv-id-observations\/index.php\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/blogs.nejm.org\/hiv-id-observations\/index.php\/wp-json\/wp\/v2\/users\/6"}],"replies":[{"embeddable":true,"href":"https:\/\/blogs.nejm.org\/hiv-id-observations\/index.php\/wp-json\/wp\/v2\/comments?post=4741"}],"version-history":[{"count":0,"href":"https:\/\/blogs.nejm.org\/hiv-id-observations\/index.php\/wp-json\/wp\/v2\/posts\/4741\/revisions"}],"wp:attachment":[{"href":"https:\/\/blogs.nejm.org\/hiv-id-observations\/index.php\/wp-json\/wp\/v2\/media?parent=4741"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/blogs.nejm.org\/hiv-id-observations\/index.php\/wp-json\/wp\/v2\/categories?post=4741"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/blogs.nejm.org\/hiv-id-observations\/index.php\/wp-json\/wp\/v2\/tags?post=4741"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}