{"id":7246,"date":"2025-06-13T10:57:32","date_gmt":"2025-06-13T08:57:32","guid":{"rendered":"https:\/\/rvh-synergie.org\/actualites\/la-therapie-genique-de-lhemophilie-b-demontre-un-succes-a-long-terme\/"},"modified":"2025-06-13T10:57:32","modified_gmt":"2025-06-13T08:57:32","slug":"la-therapie-genique-de-lhemophilie-b-demontre-un-succes-a-long-terme","status":"publish","type":"post","link":"https:\/\/rvh-synergie.org\/actualites\/la-therapie-genique-de-lhemophilie-b-demontre-un-succes-a-long-terme\/","title":{"rendered":"La th\u00e9rapie g\u00e9nique de l’h\u00e9mophilie B d\u00e9montre un succ\u00e8s \u00e0 long terme"},"content":{"rendered":"
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Une approche de transfert de g\u00e8nes pour traiter l’h\u00e9mophilie B du trouble de saignement reste s\u00fbre et efficace \u00e0 long terme, car des scientifiques du St. Jude Children’s Research Hospital and University College de Londres rapportent aujourd’hui treize ans de donn\u00e9es de suivi. L’h\u00e9mophilie B est un trouble g\u00e9n\u00e9tique rare caus\u00e9 par des niveaux insuffisants d’une prot\u00e9ine circulante appel\u00e9e facteur IX, qui favorise la coagulation sanguine.<\/p>\n

Les chercheurs ont utilis\u00e9 une intervention de th\u00e9rapie g\u00e9nique ponctuelle pour traiter le trouble. Publi\u00e9 dans The New England Journal of Medicine<\/i>l’\u00e9tude de suivi de 13 ans est la plus longue signal\u00e9e pour toute th\u00e9rapie g\u00e9nique pour l’h\u00e9mophilie B. Les r\u00e9sultats, y compris une r\u00e9duction presque dix fois du taux de saignement annualis\u00e9, soutiennent la viabilit\u00e9 \u00e0 long terme de la th\u00e9rapie g\u00e9nique pour le traitement de la maladie.<\/p>\n

L’h\u00e9mophilie B est un trouble g\u00e9n\u00e9tique li\u00e9 \u00e0 l’X affectant 1 sur environ 25 000 naissances masculines. Bien que le trouble puisse aller en gravit\u00e9, des saignements spontan\u00e9s fr\u00e9quents et des h\u00e9morragies mortelles se produisent en raison du facteur de coagulation sanguine insuffisante. Le traitement de l’h\u00e9mophilie B est traditionnellement co\u00fbteux pour la suppl\u00e9mentation \u00e0 vie du facteur de coagulation, mais la th\u00e9rapie g\u00e9nique offre un moyen potentiellement transformateur pour aborder le trouble.<\/p>\n

\u00ab\u00a0L’avantage cl\u00e9 est que la th\u00e9rapie g\u00e9nique est une perfusion intraveineuse simple et unique qui est tr\u00e8s simple \u00e0 faire et a potentiellement des effets positifs pour toute une vie\u00a0\u00bb, a d\u00e9clar\u00e9 le co-investigateur de l’\u00e9tude Andrew Davidoff, MD, pr\u00e9sident du d\u00e9partement de chirurgie de St. Jude.<\/p>\n

Innocuit\u00e9, efficacit\u00e9 et viabilit\u00e9 \u00e0 long terme<\/h2>\n

L’\u00e9tude comprenait 10 adultes atteints d’h\u00e9mophilie B s\u00e9v\u00e8re qui a re\u00e7u le m\u00e9dicament de th\u00e9rapie g\u00e9nique entre mars 2010 et novembre 2012, avec des succ\u00e8s initiaux de s\u00e9curit\u00e9 et d’efficacit\u00e9 signal\u00e9s en 2014 The New England Journal of Medicine<\/i>. Maintenant, plus d’une d\u00e9cennie plus tard, le nouveau rapport de suivi de 13 ans renforce ces r\u00e9sultats. Les patients ont \u00e9t\u00e9 suivis sur 10 ann\u00e9es suppl\u00e9mentaires et ont tous maintenu un niveau constant de facteur IX et un excellent avantage en termes de libert\u00e9 de saignement.<\/p>\n

Dans le domaine de la th\u00e9rapie g\u00e9nique, des questions restent concernant la viabilit\u00e9 durable de ces traitements, avec des parties prenantes cl\u00e9s, y compris les patients et les familles, en attendant de voir quels seront les r\u00e9sultats \u00e0 long terme, comme la durabilit\u00e9 et la stabilit\u00e9 de l’expression.<\/p>\n

\u00ab\u00a0Pour ces 10 patients, les niveaux de facteurs sont stables et ont \u00e9t\u00e9 au m\u00eame niveau au cours de ces 13 ann\u00e9es\u00a0\u00bb, a d\u00e9clar\u00e9 le chercheur principal Ulrike Reiss, MD, St. Jude Department of Hematology. \u00ab\u00a0De plus, nous n’avons vu aucun effet secondaire ou \u00e9v\u00e9nements toxiques dans le suivi \u00e0 long terme.\u00a0\u00bb<\/p>\n

Le manque d’\u00e9v\u00e9nements toxiques est remarquable. Lors de l’administration de la th\u00e9rapie g\u00e9nique, plus de 90% se retrouve dans le foie. Les chercheurs ont gard\u00e9 un \u0153il attentif \u00e0 ce sujet pour s’assurer qu’aucun probl\u00e8me ne se pose, et bien qu’un petit degr\u00e9 d’inflammation du foie ait \u00e9t\u00e9 not\u00e9 peu de temps apr\u00e8s l’administration vectorielle, il a \u00e9t\u00e9 frein\u00e9 par l’administration de st\u00e9ro\u00efdes et n’est pas revenu apr\u00e8s la r\u00e9solution initiale.<\/p>\n

Les r\u00e9sultats indiquent une am\u00e9lioration significative de la qualit\u00e9 de vie pour les individus trait\u00e9s. Leur taux de saignement annualis\u00e9 (combien quelqu’un saigne au cours d’une ann\u00e9e) est pass\u00e9 de 14,0 \u00e9pisodes \u00e0 1,5 \u00e9pisodes. De plus, m\u00eame si les patients ne sont pas entr\u00e9s dans la plage normale de l’expression du facteur IX, leur cas a toujours \u00e9t\u00e9 significativement am\u00e9lior\u00e9 avec moins de d\u00e9pendance aux suppl\u00e9ments du facteur IX.<\/p>\n

\u00ab\u00a0Il est incroyablement gratifiant de voir la s\u00e9curit\u00e9 et l’efficacit\u00e9 soutenues, ce qui valide vraiment le potentiel de la th\u00e9rapie g\u00e9nique en tant que traitement unique pour cette condition\u00a0\u00bb, a d\u00e9clar\u00e9 le chercheur en chef de l’\u00e9tude, Amit Nathwani, Ph.D., de l’UCL Cancer Institute et du Royal Free Hospital. \u00ab\u00a0Nos r\u00e9sultats r\u00e9pondent aux questions critiques sur la durabilit\u00e9 \u00e0 long terme de la th\u00e9rapie g\u00e9nique, offrant un espoir profond et une qualit\u00e9 de vie consid\u00e9rablement am\u00e9lior\u00e9e pour les patients.\u00a0\u00bb<\/p>\n

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Une approche de transfert de g\u00e8nes pour traiter l’h\u00e9mophilie B du trouble de saignement reste<\/p>\n","protected":false},"author":1,"featured_media":7247,"comment_status":"closed","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[86],"tags":[725,1064,1687,619,734,1637,1063],"class_list":["post-7246","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-les-maladies","tag-demontre","tag-genique","tag-lhemophilie","tag-long","tag-succes","tag-terme","tag-therapie","generate-columns","tablet-grid-50","mobile-grid-100","grid-parent","grid-50","resize-featured-image"],"yoast_head":"\nLa th\u00e9rapie g\u00e9nique de l'h\u00e9mophilie B d\u00e9montre un succ\u00e8s \u00e0 long terme - RVH-Synergie.org<\/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:\/\/rvh-synergie.org\/actualites\/la-therapie-genique-de-lhemophilie-b-demontre-un-succes-a-long-terme\/\" \/>\n<meta property=\"og:locale\" content=\"fr_FR\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"La th\u00e9rapie g\u00e9nique de l'h\u00e9mophilie B d\u00e9montre un succ\u00e8s \u00e0 long terme - 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