{"id":62193,"date":"2022-05-02T15:46:46","date_gmt":"2022-05-02T13:46:46","guid":{"rendered":"https:\/\/cri1149.mlcom-dev.net\/?post_type=publications&#038;p=62193"},"modified":"2025-09-11T16:47:59","modified_gmt":"2025-09-11T14:47:59","slug":"givosiran-in-acute-intermittent-porphyria-a-personalized-medicine-approach","status":"publish","type":"publication","link":"https:\/\/cri1149.mlcom-dev.net\/en\/publication\/givosiran-in-acute-intermittent-porphyria-a-personalized-medicine-approach\/","title":{"rendered":"Givosiran in acute intermittent porphyria: A personalized medicine approach"},"content":{"rendered":"","protected":false},"featured_media":0,"template":"","meta":{"_acf_changed":false},"class_list":["post-62193","publication","type-publication","status-publish","hentry"],"acf":{"numero_de_publication":"Mol Genet Metab.","date_de_publication":"20220301","numero_doi":"","equipe":[124],"auteurs-liste":[{"texte_libre":true,"auteur-lien":null,"auteur-text":"[:fr]Antoine Poli[:]"},{"texte_libre":false,"auteur-lien":13115,"auteur-text":""},{"texte_libre":false,"auteur-lien":13125,"auteur-text":""},{"texte_libre":false,"auteur-lien":13203,"auteur-text":""},{"texte_libre":false,"auteur-lien":14956,"auteur-text":""},{"texte_libre":true,"auteur-lien":null,"auteur-text":"[:fr]Sophie Rivi\u00e8re[:]"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"[:fr]Diane Cerutti[:]"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"[:fr]Isabelle Bouchoule[:]"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"[:fr]Anthony Faivre[:]"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"[:fr]Vincent Grobost[:]"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"[:fr]Claire Douillard[:]"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"[:fr]Francis Duch\u00eane[:]"},{"texte_libre":true,"auteur-lien":null,"auteur-text":"[:fr]Valeria Fiorentino[:]"},{"texte_libre":false,"auteur-lien":14954,"auteur-text":""},{"texte_libre":false,"auteur-lien":13113,"auteur-text":""},{"texte_libre":false,"auteur-lien":13139,"auteur-text":""},{"texte_libre":false,"auteur-lien":13142,"auteur-text":""},{"texte_libre":false,"auteur-lien":13104,"auteur-text":""},{"texte_libre":false,"auteur-lien":13080,"auteur-text":""}],"auteurs-manuel":"","liens_externes":null,"liens":[{"lien":"https:\/\/pubmed.ncbi.nlm.nih.gov\/35058124\/"}],"paragraphe":"[:fr]<h2 class=\"title\" style=\"text-align: justify;\">Abstract<\/h2>\r\n<div id=\"enc-abstract\" class=\"abstract-content selected\">\r\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>\r\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>\r\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>\r\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>\r\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>\r\n<\/div>[:]","paragraphe_en":"[:fr]<h2 class=\"title\" style=\"text-align: justify;\">Abstract<\/h2>\r\n<div id=\"enc-abstract\" class=\"abstract-content selected\">\r\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>\r\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>\r\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>\r\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>\r\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>\r\n<\/div>[:]","documents":null},"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v26.8 - 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=\"noindex, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\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:url\" content=\"https:\/\/cri1149.mlcom-dev.net\/publication\/givosiran-in-acute-intermittent-porphyria-a-personalized-medicine-approach\/\" \/>\n<meta property=\"og:site_name\" content=\"CRI\" \/>\n<meta property=\"article:publisher\" content=\"https:\/\/www.facebook.com\/CRI-Centre-de-recherche-sur-linflammation-100116118884153\" \/>\n<meta property=\"article:modified_time\" content=\"2025-09-11T14:47:59+00:00\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\/\/schema.org\",\"@graph\":[{\"@type\":\"WebPage\",\"@id\":\"https:\/\/cri1149.mlcom-dev.net\/publication\/givosiran-in-acute-intermittent-porphyria-a-personalized-medicine-approach\/\",\"url\":\"https:\/\/cri1149.mlcom-dev.net\/publication\/givosiran-in-acute-intermittent-porphyria-a-personalized-medicine-approach\/\",\"name\":\"Givosiran in acute intermittent porphyria: A personalized medicine approach - 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