{"id":77954,"date":"2022-02-10T16:15:02","date_gmt":"2022-02-10T15:15:02","guid":{"rendered":"https:\/\/cri1149.fr\/actualites\/givosiran-in-acute-intermittent-porphyria-a-personalized-medicine-approach\/"},"modified":"2022-02-10T16:15:02","modified_gmt":"2022-02-10T15:15:02","slug":"givosiran-in-acute-intermittent-porphyria-a-personalized-medicine-approach","status":"publish","type":"post","link":"https:\/\/cri1149.fr\/en\/news-of-the-month\/givosiran-in-acute-intermittent-porphyria-a-personalized-medicine-approach\/","title":{"rendered":"Givosiran in acute intermittent porphyria: A personalized medicine approach"},"content":{"rendered":"<h2 class=\"title\" style=\"text-align: justify;\">Abstract<\/h2>\n<div id=\"enc-abstract\" class=\"abstract-content selected\">\n<p style=\"text-align: justify;\"><strong class=\"sub-title\">Background:\u00a0<\/strong>In patients with acute intermittent porphyria (AIP), induction of delta aminolevulinic acid synthase 1 (ALAS1) leads to haem precursor accumulation that may cause recurring acute attacks. In a recent phase III trial, givosiran significantly reduced the attack rate in severe AIP patients. Frequent adverse events were injection-site reaction, fatigue, nausea, chronic kidney disease and increased alanine aminotransferase.<\/p>\n<p style=\"text-align: justify;\"><strong class=\"sub-title\">Objectives:\u00a0<\/strong>To describe the efficacy and safety of givosiran based on a personalized medical approach.<\/p>\n<p style=\"text-align: justify;\"><strong class=\"sub-title\">Methods:\u00a0<\/strong>We conducted a retrospective patient file study in 25 severe AIP patients treated with givosiran in France. We collected data on clinical and biochemical efficacy along with reports of adverse events.<\/p>\n<p style=\"text-align: justify;\"><strong class=\"sub-title\">Results:\u00a0<\/strong>Givosiran drastically reduced the attack rate in our cohort, as 96% were attack-free at the time of the study. The sustained efficacy of givosiran in most patients allowed us to personalize dosing frequency. In 42%, givosiran was only given when haem precursor levels were increasing. Our data suggest that givosiran is most effective when given early in the disease course. We confirmed a high prevalence of adverse events. One patient discontinued treatment due to acute pancreatitis. All patients had hyperhomocysteinemia, and all patients with initial homocysteine levels available showed an increase under treatment. In this context, one patient was diagnosed with pulmonary embolism.<\/p>\n<p style=\"text-align: justify;\"><strong class=\"sub-title\">Conclusion:\u00a0<\/strong>The sustained effect of givosiran allowed a decrease in dosing frequency without compromising treatment efficacy. The high prevalence of adverse events emphasizes the importance of restricting the treatment to severe AIP and administering the minimum effective dose for each patient.<\/p>\n<\/div>\n<p>&nbsp;<\/p>\n<p><span style=\"text-decoration: underline; font-size: 14pt;\"><strong>Authors :<\/strong><\/span><\/p>\n<p><span class=\"authors-list-item \"><a class=\"full-name\" href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/?term=Poli+A&amp;cauthor_id=35058124\" data-ga-category=\"search\" data-ga-action=\"author_link\" data-ga-label=\"Antoine Poli\">Antoine Poli<\/a><span class=\"comma\">,\u00a0<\/span><\/span><span class=\"authors-list-item \"><a class=\"full-name\" href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/?term=Schmitt+C&amp;cauthor_id=35058124\" data-ga-category=\"search\" data-ga-action=\"author_link\" data-ga-label=\"Caroline Schmitt\">Caroline Schmitt<\/a><span class=\"comma\">,\u00a0<\/span><\/span><span class=\"authors-list-item \"><a class=\"full-name\" href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/?term=Moulouel+B&amp;cauthor_id=35058124\" data-ga-category=\"search\" data-ga-action=\"author_link\" data-ga-label=\"Boualem Moulouel\">Boualem Moulouel<\/a><span class=\"comma\">,\u00a0<\/span><\/span><span class=\"authors-list-item \"><a class=\"full-name\" href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/?term=Mirmiran+A&amp;cauthor_id=35058124\" data-ga-category=\"search\" data-ga-action=\"author_link\" data-ga-label=\"Arienne Mirmiran\">Arienne Mirmiran<\/a><span class=\"comma\">,\u00a0<\/span><\/span><span class=\"authors-list-item \"><a class=\"full-name\" href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/?term=Talbi+N&amp;cauthor_id=35058124\" data-ga-category=\"search\" data-ga-action=\"author_link\" data-ga-label=\"Neila Talbi\">Neila Talbi<\/a><span class=\"comma\">,\u00a0<\/span><\/span><span class=\"authors-list-item \"><a class=\"full-name\" href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/?term=Rivi%C3%A8re+S&amp;cauthor_id=35058124\" data-ga-category=\"search\" data-ga-action=\"author_link\" data-ga-label=\"Sophie Rivi\u00e8re\">Sophie Rivi\u00e8re<\/a><span class=\"comma\">,\u00a0<\/span><\/span><span class=\"authors-list-item \"><a class=\"full-name\" href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/?term=Cerutti+D&amp;cauthor_id=35058124\" data-ga-category=\"search\" data-ga-action=\"author_link\" data-ga-label=\"Diane Cerutti\">Diane Cerutti<\/a><span class=\"comma\">,\u00a0<\/span><\/span><span class=\"authors-list-item \"><a class=\"full-name\" href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/?term=Bouchoule+I&amp;cauthor_id=35058124\" data-ga-category=\"search\" data-ga-action=\"author_link\" data-ga-label=\"Isabelle Bouchoule\">Isabelle Bouchoule<\/a><span class=\"comma\">,\u00a0<\/span><\/span><span class=\"authors-list-item \"><a class=\"full-name\" href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/?term=Faivre+A&amp;cauthor_id=35058124\" data-ga-category=\"search\" data-ga-action=\"author_link\" data-ga-label=\"Anthony Faivre\">Anthony Faivre<\/a><span class=\"comma\">,\u00a0<\/span><\/span><span class=\"authors-list-item \"><a class=\"full-name\" href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/?term=Grobost+V&amp;cauthor_id=35058124\" data-ga-category=\"search\" data-ga-action=\"author_link\" data-ga-label=\"Vincent Grobost\">Vincent Grobost<\/a><span class=\"comma\">,\u00a0<\/span><\/span><span class=\"authors-list-item \"><a class=\"full-name\" href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/?term=Douillard+C&amp;cauthor_id=35058124\" data-ga-category=\"search\" data-ga-action=\"author_link\" data-ga-label=\"Claire Douillard\">Claire Douillard<\/a><span class=\"comma\">,\u00a0<\/span><\/span><span class=\"authors-list-item \"><a class=\"full-name\" href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/?term=Duch%C3%AAne+F&amp;cauthor_id=35058124\" data-ga-category=\"search\" data-ga-action=\"author_link\" data-ga-label=\"Francis Duch\u00eane\">Francis Duch\u00eane<\/a><span class=\"comma\">,\u00a0<\/span><\/span><span class=\"authors-list-item \"><a class=\"full-name\" href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/?term=Fiorentino+V&amp;cauthor_id=35058124\" data-ga-category=\"search\" data-ga-action=\"author_link\" data-ga-label=\"Valeria Fiorentino\">Val\u00e9ria Fiorentino<\/a><span class=\"comma\">,\u00a0<\/span><\/span><span class=\"authors-list-item \"><a class=\"full-name\" href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/?term=Dupr%C3%A9+T&amp;cauthor_id=35058124\" data-ga-category=\"search\" data-ga-action=\"author_link\" data-ga-label=\"Thierry Dupr\u00e9\">Thierry Dupr\u00e9<\/a><span class=\"comma\">,\u00a0<\/span><\/span><span class=\"authors-list-item \"><a class=\"full-name\" href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/?term=Manceau+H&amp;cauthor_id=35058124\" data-ga-category=\"search\" data-ga-action=\"author_link\" data-ga-label=\"Hana Manceau\">Hana Manceau<\/a><span class=\"comma\">,\u00a0<\/span><\/span><span class=\"authors-list-item \"><a class=\"full-name\" href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/?term=Peoc%27h+K&amp;cauthor_id=35058124\" data-ga-category=\"search\" data-ga-action=\"author_link\" data-ga-label=\"Katell Peoc'h\">Katell Peoc\u2019h<\/a><span class=\"comma\">,\u00a0<\/span><\/span><span class=\"authors-list-item \"><a class=\"full-name\" href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/?term=Puy+H&amp;cauthor_id=35058124\" data-ga-category=\"search\" data-ga-action=\"author_link\" data-ga-label=\"Herv\u00e9 Puy\">Herv\u00e9 Puy<\/a><span class=\"comma\">,\u00a0<\/span><\/span><span class=\"authors-list-item \"><a class=\"full-name\" href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/?term=Lefebvre+T&amp;cauthor_id=35058124\" data-ga-category=\"search\" data-ga-action=\"author_link\" data-ga-label=\"Thibaud Lefebvre\">Thibaud Lefebvre<\/a><span class=\"comma\">,\u00a0<\/span><\/span><span class=\"authors-list-item \"><a class=\"full-name\" href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/?term=Gouya+L&amp;cauthor_id=35058124\" data-ga-category=\"search\" data-ga-action=\"author_link\" data-ga-label=\"Laurent Gouya\">Laurent Gouya<\/a><\/span><\/p>\n<p>&nbsp;<\/p>\n<p>Link of publication : <a href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/35058124\/\">https:\/\/pubmed.ncbi.nlm.nih.gov\/35058124\/<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Abstract Background:\u00a0In patients with acute intermittent porphyria (AIP), induction of delta aminolevulinic acid synthase 1 (ALAS1) leads to haem precursor accumulation that may cause recurring acute attacks. In a recent [&hellip;]<\/p>\n","protected":false},"author":9,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_EventAllDay":false,"_EventTimezone":"","_EventStartDate":"","_EventEndDate":"","_EventStartDateUTC":"","_EventEndDateUTC":"","_EventShowMap":false,"_EventShowMapLink":false,"_EventURL":"","_EventCost":"","_EventCostDescription":"","_EventCurrencySymbol":"","_EventCurrencyCode":"","_EventCurrencyPosition":"","_EventDateTimeSeparator":"","_EventTimeRangeSeparator":"","_EventOrganizerID":[],"_EventVenueID":[],"_OrganizerEmail":"","_OrganizerPhone":"","_OrganizerWebsite":"","_VenueAddress":"","_VenueCity":"","_VenueCountry":"","_VenueProvince":"","_VenueState":"","_VenueZip":"","_VenuePhone":"","_VenueURL":"","_VenueStateProvince":"","_VenueLat":"","_VenueLng":"","_VenueShowMap":false,"_VenueShowMapLink":false,"footnotes":""},"categories":[328],"tags":[],"class_list":["post-77954","post","type-post","status-publish","format-standard","hentry","category-news-of-the-month"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.2 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Givosiran in acute intermittent porphyria: A personalized medicine approach - CRI<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/cri1149.fr\/en\/news-of-the-month\/givosiran-in-acute-intermittent-porphyria-a-personalized-medicine-approach\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Givosiran in acute intermittent porphyria: A personalized medicine approach - CRI\" \/>\n<meta property=\"og:description\" content=\"Abstract Background:\u00a0In patients with acute intermittent porphyria (AIP), induction of delta aminolevulinic acid synthase 1 (ALAS1) leads to haem precursor accumulation that may cause recurring acute attacks. 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