{"id":1859,"date":"2023-04-20T17:23:00","date_gmt":"2023-04-20T15:23:00","guid":{"rendered":"https:\/\/niho.sk\/?p=1859"},"modified":"2024-03-30T17:27:06","modified_gmt":"2024-03-30T16:27:06","slug":"29a-liecivo-onasemnogen-abeparvovek-zolgensma-na-liecbu-pacientov-s-5q-spinalnou-muskularnou-atrofiou-sma-s-bialelickou-mutaciou-v-gene-smn1-a-klinicky-diagnostikovanou-sma-1-typu","status":"publish","type":"post","link":"https:\/\/www.niho.sk\/en\/29a-liecivo-onasemnogen-abeparvovek-zolgensma-na-liecbu-pacientov-s-5q-spinalnou-muskularnou-atrofiou-sma-s-bialelickou-mutaciou-v-gene-smn1-a-klinicky-diagnostikovanou-sma-1-typu\/","title":{"rendered":"29A: Lie\u010divo onasemnog\u00e9n abeparvovek (Zolgensma) na lie\u010dbu pacientov s 5q spin\u00e1lnou muskul\u00e1rnou atrofiou (SMA) s bialelickou mut\u00e1ciou v g\u00e9ne SMN1 a klinicky diagnostikovanou SMA 1. typu"},"content":{"rendered":"
    \n
  1. Ak\u00e1 je z\u00e1\u0165a\u017e ochorenia pre pacienta? Ako z\u00e1sadn\u00e9 je ochorenie?<\/strong><\/li>\n<\/ol>\n\n\n\n

    Spin\u00e1lna muskul\u00e1rna atrofia (SMA) je zriedkav\u00e9 genetick\u00e9 ochorenie sp\u00f4sobuj\u00face svalov\u00fa slabos\u0165, postupn\u00fa stratu pohybu a smr\u0165. Naj\u010dastej\u0161ie doch\u00e1dza k degener\u00e1cii motorick\u00fdch neur\u00f3nov miechy, ktor\u00e9 s\u00fa zodpovedn\u00e9 za chodenie, lozenie, pohyb r\u00fak, hlavy a krku, preh\u013atanie a d\u00fdchanie. U pacientov so SMA 1. typu sa ochorenie prejav\u00ed do 6 mesiacov \u017eivota. O\u010dak\u00e1van\u00e1 d\u013a\u017eka \u017eivota bez lie\u010dby je pribli\u017ene 2 roky. Ochorenie m\u00e1 rovnako v\u00fdrazn\u00fd dopad na bl\u00edzkych pacienta, najm\u00e4 rodi\u010dov alebo z\u00e1konn\u00fdch z\u00e1stupcov, nako\u013eko si deti s touto diagn\u00f3zou vy\u017eaduj\u00fa nepretr\u017eit\u00fa starostlivos\u0165, \u010do znemo\u017e\u0148uje minim\u00e1lne jedn\u00e9mu z nich pracova\u0165 na pln\u00fd \u00fav\u00e4zok. \u010eal\u0161\u00edm aspektom je neust\u00e1ly strach o svoje die\u0165a.<\/p>\n\n\n\n

      \n
    1. O ak\u00fd liek ide?<\/strong><\/li>\n<\/ol>\n\n\n\n

      Onasemnog\u00e9n abeparvovek je g\u00e9nov\u00e1 terapia ur\u010den\u00e1 na zavedenie funk\u010dnej k\u00f3pie g\u00e9nu pre\u017e\u00edvania motorick\u00fdch neur\u00f3nov (SMN1) do transdukovan\u00fdch buniek na vyrie\u0161enie monog\u00e9nnej hlavnej pr\u00ed\u010diny ochorenia. O\u010dak\u00e1va sa, \u017ee zabezpe\u010den\u00edm alternat\u00edvneho zdroja produkcie prote\u00ednu SMN v motorick\u00fdch neur\u00f3noch sa podpor\u00ed pre\u017eitie a funkcia transdukovan\u00fdch motorick\u00fdch neur\u00f3nov. Zolgensma sa pod\u00e1va intraven\u00f3zne vo forme suspenzie vektorov\u00fdch gen\u00f3mov, ktor\u00e9 s\u00fa ur\u010den\u00e9 na jednor\u00e1zov\u00e9 podanie.<\/p>\n\n\n\n

      Liek Zolgensma (onasemnog\u00e9n abeparvovek) bol registrovan\u00fd Eur\u00f3pskou liekovou agent\u00farou (EMA) pod \u010d\u00edslom d\u0148a 18.5.2020.<\/p>\n\n\n\n

        \n
      1. Ako sa na axikabtag\u00e9n ciloleucel pozeraj\u00fa odborn\u00edci? <\/strong><\/li>\n<\/ol>\n\n\n\n

        Klinick\u00ed experti participuj\u00faci na hodnoten\u00ed lieku Zolgensma v NICE (anglick\u00fd HTA in\u0161tit\u00fat (National Instute for Health and Care Excellence)) uv\u00e1dzaj\u00fa, \u017ee u niektor\u00fdch pacientov vo veku 7 a\u017e 12 mesiacov by sa o\u010dak\u00e1val podobn\u00fd pr\u00ednos ako u pacientov vo veku do 6 mesiacov. Preto v s\u00falade s NICE navrhuje NIHO zv\u00e1\u017ei\u0165 doplnenie indika\u010dn\u00e9ho obmedzenia pre podanie lieku Zolgensma pre pacientov vo veku 7 a\u017e 12 mesiacov, pokia\u013e sa na tom zhodne t\u00edm \u0161pecialistov v lie\u010dbe predmetn\u00e9ho ochorenia a pacient m\u00e1 aspo\u0148 70 %-n\u00fa \u0161ancu, \u017ee bude schopn\u00fd samostatn\u00e9ho sedu.<\/p>\n\n\n\n

          \n
        1. \u010co hodnotilo NIHO a ak\u00fd je v\u00fdsledok?<\/strong><\/li>\n<\/ol>\n\n\n\n

          Dr\u017eite\u013e registr\u00e1cie (Novartis Europharm Limited (IRL)) podal \u017eiados\u0165 o zaradenie lie\u010diva onasemnog\u00e9n abeparvovek (Zolgensma) na lie\u010dbu pacientov s 5q spin\u00e1lnou muskul\u00e1rnou atrofiou (SMA) s bialelickou mut\u00e1ciou v g\u00e9ne SMN1 a klinicky diagnostikovanou SMA 1. typu.<\/p>\n\n\n\n

          NIHO odpor\u00fa\u010da vyhovie\u0165 \u017eiadosti o kategorizovanie lieku Zolgensma v predmetnej indik\u00e1ci\u00ed, pokia\u013e dr\u017eite\u013e registr\u00e1cie uprav\u00ed po\u017eadovan\u00fa v\u00fd\u0161ku \u00fahrady tak, aby sp\u013a\u0148ala krit\u00e9ri\u00e1 n\u00e1kladovej efekt\u00edvnosti.<\/p>\n\n\n\n

          V\u00fdsledok hodnotenia n\u00e1kladovej efekt\u00edvnosti je aj pri uvedenej \u00fahrade spojen\u00fd s extr\u00e9mne vysokou mierou neistoty, \u017ee v klinickej praxi nebud\u00fa splnen\u00e9 krit\u00e9ri\u00e1 n\u00e1kladovej efekt\u00edvnosti. Preto NIHO odpor\u00fa\u010da po\u017eadova\u0165 od dr\u017eite\u013ea registr\u00e1cie adekv\u00e1tnu dodato\u010dn\u00fa z\u013eavu, ktorou by sa t\u00e1to neistota kompenzovala.<\/p>\n\n\n\n

          Okrem toho NIHO odpor\u00fa\u010da zv\u00e1\u017ei\u0165 doplnenie indika\u010dn\u00e9ho obmedzenia pod\u013ea n\u00e1vrhu NIHO.<\/p>","protected":false},"excerpt":{"rendered":"

          NIHO odpor\u00fa\u010da vyhovie\u0165 \u017eiadosti o kategorizovanie lieku Zolgensma v predmetnej indik\u00e1ci\u00ed, pokia\u013e dr\u017eite\u013e registr\u00e1cie uprav\u00ed po\u017eadovan\u00fa v\u00fd\u0161ku \u00fahrady tak, aby sp\u013a\u0148ala krit\u00e9ri\u00e1 n\u00e1kladovej efekt\u00edvnosti…<\/p>","protected":false},"author":3,"featured_media":1860,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"inline_featured_image":false,"footnotes":""},"categories":[11],"tags":[95,94],"class_list":["post-1859","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-sumar-hodnotenia","tag-onasemnogen-abeparvovek","tag-zolgensma"],"_links":{"self":[{"href":"https:\/\/www.niho.sk\/en\/wp-json\/wp\/v2\/posts\/1859","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.niho.sk\/en\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.niho.sk\/en\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.niho.sk\/en\/wp-json\/wp\/v2\/users\/3"}],"replies":[{"embeddable":true,"href":"https:\/\/www.niho.sk\/en\/wp-json\/wp\/v2\/comments?post=1859"}],"version-history":[{"count":0,"href":"https:\/\/www.niho.sk\/en\/wp-json\/wp\/v2\/posts\/1859\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.niho.sk\/en\/wp-json\/wp\/v2\/media\/1860"}],"wp:attachment":[{"href":"https:\/\/www.niho.sk\/en\/wp-json\/wp\/v2\/media?parent=1859"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.niho.sk\/en\/wp-json\/wp\/v2\/categories?post=1859"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.niho.sk\/en\/wp-json\/wp\/v2\/tags?post=1859"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}