{"id":1902,"date":"2023-07-13T15:38:00","date_gmt":"2023-07-13T13:38:00","guid":{"rendered":"https:\/\/niho.sk\/?p=1902"},"modified":"2024-04-10T15:40:54","modified_gmt":"2024-04-10T13:40:54","slug":"37b-liecivo-kanabidiol-epidyolex-ako-adjuvantna-liecba-zachvatov-spojenych-s-komplexom-tuberoznej-sklerozy-tsc-u-pacientov-vo-veku-od-2-rokov","status":"publish","type":"post","link":"https:\/\/www.niho.sk\/en\/37b-liecivo-kanabidiol-epidyolex-ako-adjuvantna-liecba-zachvatov-spojenych-s-komplexom-tuberoznej-sklerozy-tsc-u-pacientov-vo-veku-od-2-rokov\/","title":{"rendered":"37B: Lie\u010divo kanabidiol (Epidyolex) ako adjuvantn\u00e1 lie\u010dba z\u00e1chvatov spojeny\u0301ch s komplexom tuber\u00f3znej skler\u00f3zy (TSC) u pacientov vo veku od 2 rokov"},"content":{"rendered":"
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  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

    Komplex tuber\u00f3znej skler\u00f3zy (TSC) je zriedkav\u00e9 genetick\u00e9 ochorenie sp\u00f4soben\u00e9 mut\u00e1ciou v g\u00e9noch TSC1 alebo TSC2. T\u00e1to mut\u00e1cia m\u00e1 za n\u00e1sledok tvorbu ben\u00edgnych n\u00e1dorov vo viacer\u00fdch org\u00e1nov\u00fdch syst\u00e9moch vr\u00e1tane ko\u017ee, mozgu, srdca, obli\u010diek o\u010d\u00ed a p\u013e\u00fac. Ochorenie sa m\u00f4\u017ee prejavi\u0165 v ka\u017edom veku, naj\u010dastej\u0161ie v\u0161ak v detstve. Prejavy sa m\u00f4\u017eu u r\u00f4znych \u013eud\u00ed v\u00fdrazne l\u00ed\u0161i\u0165. Okrem fyzick\u00fdch prejavov sa u zna\u010dnej \u010dasti pacientov prejavuj\u00fa aj kognit\u00edvno-behavior\u00e1lne \u0165a\u017ekosti. Pr\u00edtomnos\u0165 ben\u00edgnych \u00fatvarov v mozgu vedie u niektor\u00fdch pacientov k \u0165a\u017ek\u00fdm neurologick\u00fdm po\u0161kodeniam, vr\u00e1tane epilepsie, ktor\u00e1 sa vyskytuje u 80% pacientov. U ur\u010dit\u00e9ho podielu vznik\u00e1 farmakorezistentn\u00e1 forma z\u00e1chvatov. Mnoh\u00ed pacienti s\u00fa odk\u00e1zan\u00ed na nepretr\u017eit\u00fa zdravotn\u00fa starostlivos\u0165, pri\u010dom pr\u00edtomnos\u0165 \u0165a\u017ek\u00fdch a nekontrolovate\u013en\u00fdch z\u00e1chvatov predstavuje dodato\u010dn\u00fa z\u00e1\u0165a\u017e pre o\u0161etrovate\u013eov a aj samotn\u00fdch pacientov. <\/p>\n\n\n\n

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

      Epidyolex obsahuje kanabidiol, liek, ktor\u00fd sa m\u00f4\u017ee pou\u017e\u00edva\u0165 na lie\u010dbu epilepsie, ochorenia, pri ktorom m\u00e1 \u010dlovek z\u00e1chvaty alebo k\u0155\u010de. <\/p>\n\n\n\n

      Epidyolex sa pou\u017e\u00edva v kombin\u00e1cii s klobazamom alebo s klobazamom a in\u00fdmi antiepileptikami na lie\u010dbu z\u00e1chvatov, ktor\u00e9 sa vyskytuj\u00fa pri dvoch zriedkav\u00fdch ochoreniach naz\u00fdvan\u00fdch Dravetovej syndr\u00f3m a Lennox-Gastautov syndr\u00f3m. M\u00f4\u017ee sa pou\u017e\u00edva\u0165 u dospel\u00fdch, dospievaj\u00facich a det\u00ed vo veku najmenej 2 roky. <\/p>\n\n\n\n

      Epidyolex sa tie\u017e pou\u017e\u00edva v kombin\u00e1cii s in\u00fdmi antiepileptikami na lie\u010dbu z\u00e1chvatov, ktor\u00e9 sa vyskytuj\u00fa pri genetickej poruche naz\u00fdvanej komplex tuber\u00f3znej skler\u00f3zy (TSC). M\u00f4\u017ee sa pou\u017e\u00edva\u0165 u dospel\u00fdch, dospievaj\u00facich a det\u00ed vo veku najmenej 2 roky.<\/p>\n\n\n\n

      Epidyolex je v EMA (Eur\u00f3pskej liekovej agent\u00fare) registrovan\u00fd: <\/p>\n\n\n\n

        \n
      • od j\u00fala 2019 \u2013 na adjuvantn\u00fa lie\u010dbu z\u00e1chvatov spojen\u00fdch s DS a LGS v kombin\u00e1cii s klobazamom pre pacientov od 2 rokov\u00a0<\/li>\n\n\n\n
      • od febru\u00e1ru 2021 \u2013 na adjuvantn\u00fa lie\u010dbu z\u00e1chvatov spojen\u00fdch s komplexom tuber\u00f3znej skler\u00f3zy (TSC) pre pacientov od 2 rokov<\/li>\n<\/ul>\n\n\n\n
          \n
        1. Ako sa na hodnoten\u00e9 lie\u010divo pozeraj\u00fa odborn\u00edci?\u00a0<\/strong><\/li>\n<\/ol>\n\n\n\n

          Odborn\u00edci o\u010dak\u00e1vaj\u00fa v\u00fdznamn\u00fd klinick\u00fd pr\u00ednos lieku Epidyolex, nako\u013eko efektivita doteraj\u0161ej lie\u010dby je vo viacer\u00fdch pr\u00edpadoch len \u010diasto\u010dn\u00e1 a doteraz publikovan\u00e9 v\u00fdsledky nazna\u010duj\u00fa posun v lie\u010dbe farmakorezistentnej epilepsie u pacientov s TSC.<\/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 (Jazz Pharmaceuticals Ireland Ltd (IRL)) podal \u017eiados\u0165 o zaradenie lie\u010diva kanabidiol (Epidyolex) ako adjuvantn\u00e1 lie\u010dba z\u00e1chvatov spojen\u00fdch s komplexom tuber\u00f3znej skler\u00f3zy (TSC) u pacientov vo veku od 2 rokov.<\/p>\n\n\n\n

            NIHO odpor\u00fa\u010da vyhovie\u0165 \u017eiadosti o kategorizovanie lieku Epidyolex v predmetnej indik\u00e1ci\u00ed. <\/p>\n\n\n\n

            Z\u00e1rove\u0148, aj pri uvedenej \u00fahrade je pr\u00edtomn\u00e1 vysok\u00e1 neistota, \u017ee krit\u00e9ri\u00e1 n\u00e1kladovej efekt\u00edvnosti nebud\u00fa v praxi splnen\u00e9. S prihliadnut\u00edm na \u00a77 ods. 1 z\u00e1kona 363\/2011 Z.z. preto odpor\u00fa\u010dame po\u017eadova\u0165 od dr\u017eite\u013ea registr\u00e1cie adekv\u00e1tnu z\u013eavu z n\u00e1kladovo efekt\u00edvnej \u00fahrady, ktorou by sa dan\u00e1 neistota kompenzovala. Neistota je spojen\u00e1 najm\u00e4 s odhadom n\u00e1kladov na pridru\u017een\u00fa zdravotn\u00fa starostlivos\u0165, \u010falej s d\u00e1vkovan\u00edm kanabidiolu pou\u017eit\u00fdm v ekonomickej anal\u00fdze, modelovan\u00edm vplyvu na z\u00e1chvaty a pr\u00ednosu v kvalite \u017eivota.<\/p>\n\n\n\n

            NIHO odpor\u00fa\u010da zv\u00e1\u017ei\u0165 doplnenie indika\u010dn\u00e9ho obmedzenia o vetu: \u201e\u00da\u010dinnos\u0165 lie\u010dby sa vyhodnocuje ka\u017ed\u00fdch 6 mesiacov; lie\u010dba je ukon\u010den\u00e1, pokia\u013e z nej pacient na z\u00e1klade vyhodnotenia o\u0161etruj\u00faceho lek\u00e1ra \u010falej neprofituje (napr. v zmysle zn\u00ed\u017eenia frekvencie alebo intenzity z\u00e1chvatov oproti hodnot\u00e1m pred lie\u010dbou).\u201c<\/p>","protected":false},"excerpt":{"rendered":"

            NIHO odpor\u00fa\u010da vyhovie\u0165 \u017eiadosti o kategorizovanie lieku Epidyolex v predmetnej indik\u00e1ci\u00ed…<\/p>","protected":false},"author":3,"featured_media":1900,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"inline_featured_image":false,"footnotes":""},"categories":[11],"tags":[109,110],"class_list":["post-1902","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-sumar-hodnotenia","tag-epidyolex","tag-kanabidiol"],"_links":{"self":[{"href":"https:\/\/www.niho.sk\/en\/wp-json\/wp\/v2\/posts\/1902","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=1902"}],"version-history":[{"count":0,"href":"https:\/\/www.niho.sk\/en\/wp-json\/wp\/v2\/posts\/1902\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.niho.sk\/en\/wp-json\/wp\/v2\/media\/1900"}],"wp:attachment":[{"href":"https:\/\/www.niho.sk\/en\/wp-json\/wp\/v2\/media?parent=1902"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.niho.sk\/en\/wp-json\/wp\/v2\/categories?post=1902"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.niho.sk\/en\/wp-json\/wp\/v2\/tags?post=1902"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}