{"id":10881,"date":"2026-07-31T14:19:55","date_gmt":"2026-07-31T12:19:55","guid":{"rendered":"https:\/\/www.ab-science.com\/?page_id=10881"},"modified":"2026-07-31T14:19:55","modified_gmt":"2026-07-31T12:19:55","slug":"etude-clinique-masitinib-drepanocytose","status":"publish","type":"page","link":"https:\/\/www.ab-science.com\/fr\/pipeline\/masitinib-apercu\/etude-clinique-masitinib-drepanocytose\/","title":{"rendered":"Dr\u00e9panocytose"},"content":{"rendered":"

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<\/span>\u00c0 propos de la dr\u00e9panocytose<\/span><\/div><\/div>
<\/div>[vc_column_text]La dr\u00e9panocytose est une maladie g\u00e9n\u00e9tique qui touche des millions de personnes dans le monde. Bien que l’esp\u00e9rance de vie ait augment\u00e9 au cours des 20 derni\u00e8res ann\u00e9es, les complications aigu\u00ebs et chroniques entra\u00eenent toujours des comorbidit\u00e9s, une charge sociale \u00e9lev\u00e9e et un d\u00e9c\u00e8s pr\u00e9matur\u00e9 vers l’\u00e2ge de 40 ans. Environ 1,1 % des couples dans le monde risquent d’avoir un enfant atteint d’un trouble de l’h\u00e9moglobine (dr\u00e9panocytose ou thalass\u00e9mie) et 2,3 conceptions sur 1000 sont affect\u00e9es par la dr\u00e9panocytose. Les estimations sugg\u00e8rent que chaque ann\u00e9e, environ 300 000 enfants naissent avec la dr\u00e9panocytose et que ce nombre pourrait atteindre 400 000 d’ici 2050 . La dr\u00e9panocytose touche plus de 100 000 enfants et adultes aux \u00c9tats-Unis. En France, environ 26 000 patients sont touch\u00e9s (50 % d’enfants, 50 % d’adultes).<\/p>\n

La maladie va bien au-del\u00e0 des crises douloureuses \u00e9pisodiques. L’h\u00e9molyse chronique, la vaso-occlusion et le dysfonctionnement endoth\u00e9lial entra\u00eenent des l\u00e9sions organiques progressives touchant le cerveau (infarctus silencieux, accident vasculaire c\u00e9r\u00e9bral manifeste), les reins, les poumons (hypertension pulmonaire, syndrome thoracique aigu) et la rate. Ces l\u00e9sions cumul\u00e9es r\u00e9duisent l’esp\u00e9rance de vie de 20 \u00e0 30 ans, m\u00eame dans les pays \u00e0 revenu \u00e9lev\u00e9, et les patients d\u00e9veloppent souvent des complications que les traitements actuels ne peuvent pas inverser.[\/vc_column_text]

<\/span>Rationnel pour l\u2019utilisation du masitinib dans la dr\u00e9panocytose<\/span><\/div><\/div>
<\/div>[vc_column_text]Les mastocytes, une cible majeure du masitinib, semblent jouer un r\u00f4le essentiel dans les formes graves de dr\u00e9panocytose et ses complications, telles que les crises vaso-occlusives (CVO), le syndrome thoracique aigu et la douleur . Le masitinib a d\u00e9montr\u00e9 un b\u00e9n\u00e9fice de survie dans un mod\u00e8le murin de dr\u00e9panocytose: toutes les souris de contr\u00f4le atteintes de dr\u00e9panocytose ont eu des crises vaso-occlusives et 83 % d’entre elles sont mortes dans les 3 premi\u00e8res heures, alors que les souris atteintes de dr\u00e9panocytose et trait\u00e9es au pr\u00e9alable avec le masitinib pendant 4 jours n’ont pas eu de crises vaso-occlusives et ne sont pas mortes. En outre, l’histologie pulmonaire et l’immunohistochimie ont montr\u00e9 que le masitinib prot\u00e8ge des l\u00e9sions pulmonaires aigu\u00ebs et de l’infiltration des mastocytes dans un mod\u00e8le murin de dr\u00e9panocytose.[\/vc_column_text]
<\/div>
<\/span>\u00c9tude de phase 1\/2 avec le masitinib dans la dr\u00e9panocytose<\/span><\/div><\/div>
<\/div>[vc_column_text]Le d\u00e9veloppement clinique du masitinib dans la dr\u00e9panocytose est men\u00e9 dans le cadre du programme collaboratif SICKMAST, financ\u00e9 \u00e0 hauteur de 9,2 millions d’euros, qui vise \u00e0 d\u00e9montrer dans un essai clinique de phase 2 l’efficacit\u00e9 du masitinib dans le traitement des complications aigu\u00ebs et chroniques de la dr\u00e9panocytose chez des patients identifi\u00e9s sur la base de biomarqueurs. L\u2019Assistance Publique-H\u00f4pitaux de Paris (AP-HP) est le promoteur de cette \u00e9tude de phase 2, con\u00e7ue en deux \u00e9tapes\u00a0:<\/p>\n
    \n
  • Etape 1 : Identification et validation de biomarqueurs mettant en \u00e9vidence le r\u00f4le des mastocytes et des basophiles dans l’orchestration des complications aigu\u00ebs et chroniques de la dr\u00e9panocytose<\/li>\n
  • Etape 2 : D\u00e9monstration, dans le cadre d’un essai clinique de phase 2, de l’efficacit\u00e9 du masitinib dans le traitement des complications aigu\u00ebs et chroniques de la dr\u00e9panocytose chez des patients identifi\u00e9s sur la base de biomarqueurs<\/li>\n<\/ul>\n

    [\/vc_column_text]

    <\/div>[\/vc_column][\/vc_row]<\/p>\n<\/div>","protected":false},"excerpt":{"rendered":"

    [vc_row][vc_column][vc_column_text]La dr\u00e9panocytose est une maladie g\u00e9n\u00e9tique qui touche des millions de personnes dans le monde. Bien que l’esp\u00e9rance de vie ait augment\u00e9 au cours des 20 derni\u00e8res ann\u00e9es, les complications aigu\u00ebs et chroniques entra\u00eenent toujours des comorbidit\u00e9s, une charge sociale \u00e9lev\u00e9e et un d\u00e9c\u00e8s pr\u00e9matur\u00e9 vers l’\u00e2ge de 40 ans. Environ 1,1 % des couples…<\/p>\n","protected":false},"author":1,"featured_media":0,"parent":3763,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"footnotes":""},"class_list":["post-10881","page","type-page","status-publish","hentry","description-off"],"yoast_head":"\nMasitinib et dr\u00e9panocytose : \u00e9tude clinique | AB Science<\/title>\n<meta name=\"description\" content=\"D\u00e9couvrez l\u2019\u00e9tude clinique du masitinib dans la dr\u00e9panocytose, men\u00e9e dans le cadre de SICKMAST, et son rationnel ciblant les mastocytes.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.ab-science.com\/fr\/pipeline\/masitinib-apercu\/etude-clinique-masitinib-drepanocytose\/\" \/>\n<meta property=\"og:locale\" content=\"fr_FR\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" 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