Son zamanlarda givinostat ile tedavi edilen DMD'li \u00e7ocuklarda\/gen\u00e7 yeti\u015fkinlerde ani \u00f6l\u00fcm vakalar\u0131ndan haberdar olduk.<\/strong><\/p>\n\n\n\n\u00dc\u00e7 vaka, Italfarmaco taraf\u0131ndan d\u00fczenleyici otoritelere, \u00f6zellikle de Birle\u015fik Krall\u0131k'taki MHRA'ya d\u00fczenleyici gereklilikler do\u011frultusunda bildirildi.<\/p>\n\n\n\n
Givinostat \u00fcreten Italfarmaco \u015firketi, givinostat ile tedavi edilen DMD hastalar\u0131nda yak\u0131n zamanda bildirilen \u00f6l\u00fcm vakalar\u0131yla ilgili olarak hasta dan\u0131\u015fma gruplar\u0131na (PAG'ler) a\u015fa\u011f\u0131daki mesaj\u0131 20 Ekim 2025'te yay\u0131nlad\u0131:<\/p>\n\n\n\n
\n\n\n\n\u201cGivinostat'\u0131n g\u00fcvenlik profili, e\u015f zamanl\u0131 steroid tedavisi g\u00f6ren 6 ya\u015f ve \u00fczeri 179 ayaktan DMD hastas\u0131 \u00fczerinde y\u00fcr\u00fct\u00fclen, \u00e7ift k\u00f6r, plasebo kontroll\u00fc, 18 ayl\u0131k bir \u00e7al\u0131\u015fmaya dayanmaktad\u0131r (EPIDYS \u00c7al\u0131\u015fmas\u0131). EPIDYS \u00e7al\u0131\u015fmas\u0131 s\u0131ras\u0131nda ne givinostat kolunda ne de plasebo kolunda \u00f6l\u00fcm bildirilmemi\u015ftir.<\/p>\n\n\n\n
D\u00fcnya \u00e7ap\u0131nda 1700'den fazla hasta klinik \u00e7al\u0131\u015fmalarda, Geni\u015fletilmi\u015f Eri\u015fim Programlar\u0131 s\u0131ras\u0131nda ve ticari ortamda givinostata maruz kalm\u0131\u015ft\u0131r.<\/p>\n\n\n\n
Givinostat'\u0131n ger\u00e7ek d\u00fcnya kullan\u0131m\u0131 ve klinik \u00e7al\u0131\u015fmalardaki fayda-risk profilinin devam eden g\u00f6zetimi kapsam\u0131nda, a\u00e7\u0131k etiketli uzatma (OLE) \u00e7al\u0131\u015fmas\u0131nda 2 ani \u00f6l\u00fcm vakas\u0131 tespit ettik; biri Hollanda'da (Eyl\u00fcl 2025) ve biri ABD'de (Ekim 2024). Ayr\u0131ca, Almanya'daki Givinostat Geni\u015fletilmi\u015f Eri\u015fim Program\u0131'nda (Haziran 2025) 1 ani \u00f6l\u00fcm vakas\u0131 tespit ettik.<\/strong> \u00d6l\u00fcm vakalar\u0131, MHRA dahil olmak \u00fczere yerel y\u00f6nergeler do\u011frultusunda yetkili makamlara bildirildi.<\/p>\n\n\n\nItalfarmaco, bug\u00fcne kadar givinostat ile ili\u015fkili bildirilen hi\u00e7bir \u00f6l\u00fcm olmad\u0131\u011f\u0131n\u0131 de\u011ferlendirmi\u015ftir. Bu nedenle, mevcut verilere dayanarak, givinostat'\u0131n risk-fayda profili de\u011fi\u015fmeden kalmaktad\u0131r.<\/p>\n\n\n\n
Risk profiline ili\u015fkin fayda de\u011fi\u015firse, hekimleri, toplumu uyaraca\u011f\u0131z ve d\u00fczenleyici otoritelerle uygun \u015fekilde ileti\u015fim kuraca\u011f\u0131z.<\/p>\n\n\n\n
Fayda risk profili de\u011fi\u015fmedik\u00e7e s\u00fcrekli g\u00fcncelleme sa\u011flayam\u0131yoruz. Hasta mahremiyeti nedeniyle, bu \u00f6zel vakalarla ilgili ayr\u0131nt\u0131lar\u0131 yaz\u0131l\u0131 olarak payla\u015fam\u0131yoruz.\u201d<\/p>\n\n\n\n
\n\n\n\nBiz klinisyenler olarak, bu vakalar\u0131n ko\u015fullar\u0131n\u0131n yaln\u0131zca bir \u00f6zetini alabildik; bu da kapsaml\u0131 bir risk de\u011ferlendirmesi yapmam\u0131z\u0131 zorla\u015ft\u0131r\u0131yor. Birle\u015fik Krall\u0131k'ta klinik deney, EAP veya rutin NHS bak\u0131m\u0131 kapsam\u0131nda re\u00e7ete edilen herhangi bir ilac\u0131n risk-fayda analizini yapmak d\u00fczenleyici otoritelerin (MHRA) g\u00f6revidir. 22 Ekim'de, MHRA'dan 3 vakayla ilgili bilgileri ald\u0131klar\u0131na dair onay ald\u0131k ve bunlar\u0131 h\u0131zla de\u011ferlendirdikleri konusunda bizi temin ettiler.<\/p>\n\n\n\n
22 Ekim 2025 tarihine kadar mevcut bilgilere dayanarak, bu \u00f6l\u00fcmlerin givinostat veya givinostat'\u0131n bilinen yan etkilerinden biriyle do\u011frudan ili\u015fkili oldu\u011funa dair net bir kan\u0131t bulunmamaktad\u0131r. E\u015flik eden hastal\u0131klar ve birlikte kullan\u0131lan ila\u00e7lar gibi di\u011fer fakt\u00f6rler de bu vakalarda rol oynam\u0131\u015f olabilir. Bu olaylara yan\u0131t olarak ve MHRA'dan daha fazla bilgi beklerken, givinostat tedavisinin risk ve faydalar\u0131n\u0131n ki\u015fiye \u00f6zel de\u011ferlendirilmesini sa\u011flamak i\u00e7in tedaviye ba\u015flayacak ve\/veya halihaz\u0131rda tedavi g\u00f6ren t\u00fcm hastalar\u0131 yak\u0131ndan izlemeye ve de\u011ferlendirmeye devam ediyoruz. Daha fazla bilgi edinildik\u00e7e payla\u015faca\u011f\u0131z.<\/p>\n\n\n\n
Devam\u0131n\u0131 oku<\/strong>:\u00a0Duchenne \u0130\u00e7in Klinik Ara\u015ft\u0131rmalar (T\u00fcm Ara\u015ft\u0131rmalar\u0131n Listesi)<\/a><\/p>","protected":false},"excerpt":{"rendered":"According to an announcement on the Muscular Dystrophy UK website, NorthStar Network stated: ‘As part of ongoing surveillance of the risk profile of Givinostat (Duvyzat) in real-world use and clinical studies, we are aware of three cases of sudden death in the open-label extension (OLE) study.’ Italfarmaco states that there have been no reported deaths […]<\/p>\n","protected":false},"author":1,"featured_media":4869,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[46,47,311,445],"class_list":["post-4868","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-duvyzat","tag-givinostat","tag-italfarmaco","tag-northstar-network"],"subtitle":"Duchenne kas distrofisi olan ve givinostat (Duvyzat) kullanan \u00fc\u00e7 DMD hastas\u0131n\u0131n hayat\u0131n\u0131 kaybetti\u011fi a\u00e7\u0131kland\u0131.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4868","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/comments?post=4868"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4868\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4869"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4868"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4868"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4868"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}
![\"Givinostat](\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/10\/Screenshot-2025-10-22-230132-1024x916.png\" "\\"\\"")
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