{"id":15659,"date":"2021-07-22T15:10:12","date_gmt":"2021-07-22T19:10:12","guid":{"rendered":"https:\/\/www.emilysentourage.org\/?p=15659"},"modified":"2021-10-01T09:36:11","modified_gmt":"2021-10-01T13:36:11","slug":"recap-fda-listening-session-for-the-final-10-of-people-with-cystic-fibrosis-hosted-by-emilys-entourage","status":"publish","type":"post","link":"https:\/\/www.emilysentourage.org\/tr\/recap-fda-listening-session-for-the-final-10-of-people-with-cystic-fibrosis-hosted-by-emilys-entourage\/","title":{"rendered":"\u00d6zet: Emily's Entourage'\u0131n Ev Sahipli\u011finde Kistik Fibrozisli Ki\u015filerin Son 10%'si i\u00e7in FDA Dinleme Oturumu"},"content":{"rendered":"<p style=\"text-align: justify;\"><span style=\"font-weight: 400;\">Emily's Entourage (EE), 15 Temmuz 2021 Per\u015fembe g\u00fcn\u00fc, mevcut hedefe y\u00f6nelik tedavilerden yararlanamayan kistik fibrozis (KF) toplulu\u011funun son 10%'si ad\u0131na savunuculuk yapmak \u00fczere ABD G\u0131da ve \u0130la\u00e7 Dairesi (FDA) ile bir \"Hasta Dinleme Oturumu\" d\u00fczenledi. Nihai 10%, uygun olmayan mutasyonlar, yan etkiler veya eri\u015fim eksikli\u011fi nedeniyle \u015fu anda mevcut olan mutasyon hedefli, mod\u00fclat\u00f6r tedavilerden fayda g\u00f6rmeyen ki\u015filerden olu\u015fmaktad\u0131r.\u00a0<\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-weight: 400;\">Dinleme oturumlar\u0131, hasta savunucusu gruplar\u0131n FDA personeli ile bir hastal\u0131kla ya\u015fama ve y\u00f6netme deneyimleri hakk\u0131nda konu\u015ftuklar\u0131, halka kapal\u0131, k\u00fc\u00e7\u00fck, gayri resmi toplant\u0131lard\u0131r. EE'nin dinleme oturumunun hedefleri, KF'li insanlar\u0131n bu kesimi i\u00e7in devam eden acil, kar\u015f\u0131lanmam\u0131\u015f tedavi ihtiya\u00e7lar\u0131n\u0131 payla\u015fmak ve insanile\u015ftirmek; FDA ile g\u00fc\u00e7l\u00fc bir ili\u015fki kurmak ve KF toplulu\u011funun bak\u0131\u015f a\u00e7\u0131lar\u0131n\u0131n ila\u00e7 geli\u015ftirme ve onay s\u00fcre\u00e7lerinin her a\u015famas\u0131na dahil edilmesini sa\u011flamakt\u0131.\u00a0<\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-weight: 400;\">Oturum, bir KF klinisyeni, KF'li \u00fc\u00e7 yeti\u015fkin ve KF'li bireylerin \u00fc\u00e7 aile \u00fcyesi de dahil olmak \u00fczere \u00e7e\u015fitli KF toplulu\u011fu \u00fcyelerini bir araya getirerek hastal\u0131kla ilgili deneyimlerini ve kar\u015f\u0131lanmam\u0131\u015f acil tedavi ihtiya\u00e7lar\u0131n\u0131 payla\u015ft\u0131.<\/span><\/p>\n<div id=\"attachment_15665\" style=\"width: 1395px\" class=\"wp-caption alignnone\"><img fetchpriority=\"high\" decoding=\"async\" aria-describedby=\"caption-attachment-15665\" class=\"wp-image-15665\" src=\"https:\/\/www.emilysentourage.org\/wp-content\/uploads\/FDA-Patient-Listening-Session_website-1-300x147.png\" alt=\"\" width=\"1385\" height=\"680\" srcset=\"https:\/\/www.emilysentourage.org\/wp-content\/uploads\/FDA-Patient-Listening-Session_website-1-300x147.png 300w, https:\/\/www.emilysentourage.org\/wp-content\/uploads\/FDA-Patient-Listening-Session_website-1-1024x503.png 1024w, https:\/\/www.emilysentourage.org\/wp-content\/uploads\/FDA-Patient-Listening-Session_website-1-768x377.png 768w, https:\/\/www.emilysentourage.org\/wp-content\/uploads\/FDA-Patient-Listening-Session_website-1-16x8.png 16w, https:\/\/www.emilysentourage.org\/wp-content\/uploads\/FDA-Patient-Listening-Session_website-1-600x295.png 600w, https:\/\/www.emilysentourage.org\/wp-content\/uploads\/FDA-Patient-Listening-Session_website-1.png 1385w\" sizes=\"(max-width: 1385px) 100vw, 1385px\" \/><p id=\"caption-attachment-15665\" class=\"wp-caption-text\">(Soldan sa\u011fa) Emily Kramer-Golinkoff, KF'li yeti\u015fkin: Dr. Jennifer Taylor-Cousar, KF klinisyeni; Lizi Gilad Silver, KF'li yeti\u015fkin; Abhijit Tirumala, KF'li yeti\u015fkin; Arek Puzia, KF'li bir \u00e7ocu\u011fun babas\u0131; Diane Shader Smith, KF'li bir yeti\u015fkinin annesi; Gabriela Castillo, KF'li yeti\u015fkin; ve Natalie Wasserman Kingston, KF'li yeti\u015fkin (k\u0131z karde\u015fi Mollie Wasserman taraf\u0131ndan temsil edilmektedir).<\/p><\/div>\n<p style=\"text-align: justify;\"><span style=\"font-weight: 400;\">EE Kurucu Orta\u011f\u0131 Emily Kramer-Golinkoff, toplant\u0131ya ev sahipli\u011fi yaparak KF, KF toplulu\u011funun son 10%'si ve Emily's Entourage hakk\u0131nda bilgi verirken kendi hikayesini de anlatt\u0131. Konu\u015fmas\u0131nda, Haziran 2021'de EE taraf\u0131ndan mevcut CFTR mod\u00fclat\u00f6rlerinden yararlanamayan 420'den fazla KF'li ki\u015fiyle yap\u0131lan bir anketten elde edilen bilgilerle hastal\u0131\u011f\u0131n kistik fibrozlu ki\u015filer \u00fczerindeki derin etkisinin alt\u0131n\u0131 \u00e7izdi.\u00a0<\/span><\/p>\n<blockquote><p>Ankete kat\u0131lanlar\u0131n 69%'si COVID-19'dan \u00f6nceki y\u0131l en az bir kez hastanede yatt\u0131\u011f\u0131n\u0131, 14%'si ise o y\u0131l be\u015f veya daha fazla kez hastanede yatt\u0131\u011f\u0131n\u0131 bildirmi\u015ftir.<\/p><\/blockquote>\n<p style=\"text-align: justify;\"><span style=\"font-weight: 400;\">Emily, anket kat\u0131l\u0131mc\u0131lar\u0131n\u0131n 40%'sinin rutin t\u0131bbi tedaviler i\u00e7in g\u00fcnde en az iki saat harcad\u0131\u011f\u0131n\u0131 ve ortalama olarak anket kat\u0131l\u0131mc\u0131lar\u0131n\u0131n \u015fu anda be\u015ften fazla akci\u011ferle ilgili tedavi ve 4,8 akci\u011fer d\u0131\u015f\u0131, KF ile ilgili tedavi uygulad\u0131\u011f\u0131n\u0131 belirterek KF'li ki\u015filerin tedavi y\u00fck\u00fcnden bahsetti.<\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-weight: 400;\">\"Sadece tedavilere ayak uydurmak bile tam zamanl\u0131 bir i\u015f, KF'nin bireyin enerjisi ve sa\u011fl\u0131\u011f\u0131 \u00fczerindeki etkisinden bahsetmeye bile gerek yok,\" diye a\u00e7\u0131kl\u0131yor.\u00a0<\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-weight: 400;\">Hastal\u0131\u011f\u0131n\u0131n y\u0131llar i\u00e7inde nas\u0131l ilerledi\u011fini ve ya\u015fam\u0131 \u00fczerindeki etkisini payla\u015fmaya devam etti.\u00a0<\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-weight: 400;\">\"S\u0131k s\u0131k ya\u015famak ve hayatta kalmak aras\u0131ndaki bu garip arafta s\u0131k\u0131\u015f\u0131p kalm\u0131\u015f gibi hissediyorum. S\u00fcrekli v\u00fccuduma g\u00fcvenmedi\u011fim hissine kap\u0131l\u0131yorum. \u00c7ok \u00e7al\u0131\u015f\u0131yorum, onlarca y\u0131ld\u0131r tedavilerimi aksatmad\u0131m, kestirme yollara ba\u015fvurmuyorum ama yine de hi\u00e7bir \u015fey yeterli olmuyor.\"\u00a0<\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-weight: 400;\">Emily, KF toplulu\u011funun daha iyi bir gelecek ve yeni tedaviler i\u00e7in umudun klinik deneylerde yatt\u0131\u011f\u0131n\u0131n fark\u0131nda oldu\u011funu payla\u015ft\u0131.\u00a0<\/span><\/p>\n<blockquote><p>Anket kat\u0131l\u0131mc\u0131lar\u0131n\u0131n y\u00fczde yetmi\u015f yedisi klinik ara\u015ft\u0131rmalara kat\u0131lmakla ilgilendiklerini belirtirken, 54%'si olas\u0131 faydalar\u0131n risklerden daha a\u011f\u0131r basmas\u0131 halinde potansiyel sa\u011fl\u0131k riskleri kar\u015f\u0131s\u0131nda bile kat\u0131labileceklerini s\u00f6ylemi\u015ftir.<\/p><\/blockquote>\n<p style=\"text-align: justify;\"><span style=\"font-weight: 400;\">Emily'nin a\u00e7\u0131klamalar\u0131n\u0131n ard\u0131ndan Dr. Jennifer L. Taylor-Cousar, MD, MSCS, ATSF, yeti\u015fkin KF program\u0131 e\u015f direkt\u00f6r\u00fc, KF terap\u00f6tik geli\u015ftirme merkezi yeti\u015fkin program\u0131 direkt\u00f6r\u00fc ve National Jewish Health t\u0131p ve pediatri b\u00f6l\u00fcmlerinde profes\u00f6r olarak mevcut KF tedavilerine genel bir bak\u0131\u015f sundu ve KF toplulu\u011funun son 10%'sini kimlerin olu\u015fturdu\u011funu a\u00e7\u0131klad\u0131.\u00a0<\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-weight: 400;\">Dr. Taylor-Cousar, kistik fibrozisli ki\u015filerin 90%'si i\u00e7in kaydedilen ilerlemeyi ve KF toplulu\u011funun 10%'sinin hala di\u011fer pek \u00e7ok ki\u015fiye yeni bir hayat sa\u011flayan d\u00f6n\u00fc\u015f\u00fcmsel ila\u00e7lardan yararlanamad\u0131\u011f\u0131 y\u0131k\u0131c\u0131 ger\u00e7e\u011fini a\u00e7\u0131klad\u0131. <\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-weight: 400;\">Son 10%'ye duyulan acil ihtiyac\u0131 dile getiren bir anekdot payla\u015ft\u0131: <\/span>Akci\u011fer nakli i\u00e7in listeye al\u0131nan be\u015f hastadan \u00fc\u00e7\u00fc CFTR mod\u00fclat\u00f6rleri i\u00e7in uygun hale geldi ve sa\u011fl\u0131k durumlar\u0131 o kadar \u00e7arp\u0131c\u0131 bir \u015fekilde iyile\u015fti ki nakil listesinden \u00e7\u0131kar\u0131ld\u0131lar ve bug\u00fcne kadar nakil listesinde yer almad\u0131lar. Kalan iki hasta mod\u00fclat\u00f6rler i\u00e7in uygun de\u011fildi ve her ikisine de nakil yap\u0131ld\u0131, bu da mod\u00fclat\u00f6rlerden yararlananlar ve yararlanmayanlar aras\u0131ndaki sa\u011fl\u0131k sonu\u00e7lar\u0131ndaki \u00f6nemli farkl\u0131l\u0131\u011f\u0131 vurgulamaktad\u0131r.<\/p>\n<p style=\"text-align: justify;\"><span style=\"font-weight: 400;\">Ayr\u0131ca Dr. Taylor-Cousar, beyaz olmayan n\u00fcfusun mevcut mod\u00fclat\u00f6rlerden yararlanamayan KF toplulu\u011funun son 10%'si aras\u0131nda orant\u0131s\u0131z bir \u015fekilde temsil edildi\u011fini ve bu gruplar\u0131n halihaz\u0131rda kar\u015f\u0131 kar\u015f\u0131ya oldu\u011fu bir\u00e7ok \u00f6nemli sa\u011fl\u0131k e\u015fitsizli\u011fini daha da art\u0131rd\u0131\u011f\u0131n\u0131 payla\u015ft\u0131.\u00a0<\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-weight: 400;\">Ard\u0131ndan, KF toplulu\u011funun alt\u0131 \u00fcyesi hastal\u0131kla ilgili deneyimlerini payla\u015fm\u0131\u015ft\u0131r. Topluluk \u00fcyeleri, KF toplulu\u011fu ve \u00f6zellikle son 10% i\u00e7inde var olan \u00e7e\u015fitlili\u011fi yans\u0131tan \u00e7ok \u00e7e\u015fitli farkl\u0131 hastal\u0131k a\u015famalar\u0131n\u0131, belirtilerini, ya\u015flar\u0131n\u0131 ve etnik k\u00f6kenlerini temsil ediyordu. Konu\u015fmac\u0131lar aras\u0131nda \u015funlar vard\u0131:<\/span><\/p>\n<ul style=\"text-align: justify;\">\n<li style=\"font-weight: 400;\" aria-level=\"1\"><b>Mollie Wasserman<\/b><span style=\"font-weight: 400;\">KF'li bir yeti\u015fkinin k\u0131z karde\u015fi (Natalie Wasserman Kingston ad\u0131na)<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><b>Arek Puzia<\/b><span style=\"font-weight: 400;\">, KF'li bir \u00e7ocu\u011fun babas\u0131<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><b>Gabriela Castillo<\/b><span style=\"font-weight: 400;\">, KF'li yeti\u015fkin<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><b>Abhijit Tirumala<\/b><span style=\"font-weight: 400;\">, KF'li yeti\u015fkin<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><b>Diane Shader Smith<\/b><span style=\"font-weight: 400;\">KF'li bir yeti\u015fkinin annesi<\/span><\/li>\n<li style=\"font-weight: 400;\" aria-level=\"1\"><b>Lizi Gilad Silver<\/b><span style=\"font-weight: 400;\">, KF'li yeti\u015fkin<\/span><\/li>\n<\/ul>\n<p style=\"text-align: justify;\"><span style=\"font-weight: 400;\">Tan\u0131kl\u0131klar\u0131 son derece ki\u015fisel ve zaman zaman da duygusald\u0131. KF'nin g\u00fcnl\u00fck ya\u015famlar\u0131, ili\u015fkileri, ruh sa\u011fl\u0131klar\u0131 ve gelece\u011fe y\u00f6nelik beklentileri \u00fczerindeki etkisinden bahsettiler. Kat\u0131l\u0131mc\u0131lar\u0131n her birine b\u00f6ylesine ham ve g\u00fc\u00e7l\u00fc ki\u015fisel hikayelerini payla\u015fma cesareti g\u00f6sterdikleri i\u00e7in minnettar\u0131z.\u00a0<\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-weight: 400;\">Konu\u015fmalar\u0131n\u0131n ard\u0131ndan Emily, panelistlere FDA'dan gelen sorularla canl\u0131 bir a\u00e7\u0131k soru-cevap oturumu ba\u015flatt\u0131 ve toplant\u0131y\u0131 referanslardan ve anket verilerinden elde edilen temalar\u0131n bir \u00f6zetini yaparak tamamlad\u0131. FDA'y\u0131 \"CF'nin bitmedi\u011fini\" ve son 10%'de olanlar i\u00e7in h\u0131z ve \"zaman kazanman\u0131n\" kritik oldu\u011funu anlamaya zorlayarak, ila\u00e7 incelemelerine \u00f6ncelik vermeye ve beklemenin ya\u015famla \u00f6l\u00fcm aras\u0131ndaki fark olabilece\u011fi insanlar i\u00e7in d\u00fczenleyici esneklik uygulamaya \u00e7a\u011f\u0131rd\u0131. Emily s\u00f6zlerini, KF toplulu\u011fu i\u00e7in yapt\u0131klar\u0131 \u00e7al\u0131\u015fmalardan dolay\u0131 FDA'ya i\u00e7ten te\u015fekk\u00fcrlerini sunarak ve hayat kurtaran tedavileri acilen ihtiya\u00e7 duyan KF'li insanlara h\u0131zl\u0131 bir \u015fekilde ula\u015ft\u0131rma ortak hedefinde onlarla i\u015fbirli\u011fi yapma arzusunu ifade ederek bitirdi.<\/span><\/p>\n<p style=\"text-align: justify;\"><em><span style=\"font-weight: 400;\">Oturumun tam ve ayr\u0131nt\u0131l\u0131 bir \u00f6zetini okuyun <a href=\"https:\/\/www.emilysentourage.org\/wp-content\/uploads\/Emilys-Entourage-Listening-Session-Summary.pdf\" target=\"_blank\" rel=\"noopener\">Burada<\/a>.\u00a0<\/span><\/em><\/p>","protected":false},"excerpt":{"rendered":"<p>Emily's Entourage (EE), 15 Temmuz 2021 Per\u015fembe g\u00fcn\u00fc, mevcut hedefe y\u00f6nelik tedavilerden fayda g\u00f6rmeyen kistik fibrozis (KF) toplulu\u011funun son 10%\u201csi ad\u0131na savunuculuk yapmak \u00fczere ABD G\u0131da ve \u0130la\u00e7 Dairesi (FDA) ile bir \u201dHasta Dinleme Oturumu\" d\u00fczenledi. Nihai 10%, mevcut hedefe y\u00f6nelik tedavilerden fayda g\u00f6rmeyen ki\u015filerden olu\u015fuyor... <\/p>\n<div class=\"clear\"><\/div>\n<p><a href=\"https:\/\/www.emilysentourage.org\/tr\/recap-fda-listening-session-for-the-final-10-of-people-with-cystic-fibrosis-hosted-by-emilys-entourage\/\" class=\"excerpt-read-more\">Daha fazla bilgi edinin<\/a><\/p>","protected":false},"author":1281,"featured_media":15690,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_seopress_titles_title":"","_seopress_titles_desc":"","_seopress_robots_index":"","_seopress_robots_follow":"","_seopress_robots_imageindex":"","_seopress_robots_snippet":"","_seopress_robots_primary_cat":"none","_seopress_robots_breadcrumbs":"","_seopress_robots_freeze_modified_date":"","_seopress_robots_custom_modified_date":"","_seopress_robots_canonical":"","_seopress_social_fb_title":"","_seopress_social_fb_desc":"","_seopress_social_fb_img":"","_seopress_social_fb_img_attachment_id":0,"_seopress_social_fb_img_width":0,"_seopress_social_fb_img_height":0,"_seopress_social_twitter_title":"","_seopress_social_twitter_desc":"","_seopress_social_twitter_img":"","_seopress_social_twitter_img_attachment_id":0,"_seopress_social_twitter_img_width":0,"_seopress_social_twitter_img_height":0,"_seopress_redirections_value":"","_seopress_redirections_enabled":"","_seopress_redirections_enabled_regex":"","_seopress_redirections_logged_status":"","_seopress_redirections_param":"","_seopress_redirections_type":301,"_seopress_analysis_target_kw":"","_seopress_news_disabled":"","_seopress_video_disabled":"","_seopress_video":[],"_seopress_pro_schemas_manual":[{"_seopress_pro_rich_snippets_type":"none"}],"_seopress_pro_rich_snippets_disable_all":"","_seopress_pro_rich_snippets_disable":[],"_seopress_pro_schemas":[],"_monsterinsights_skip_tracking":false,"_monsterinsights_sitenote_active":false,"_monsterinsights_sitenote_note":"","_monsterinsights_sitenote_category":0,"_jetpack_feature_clip_id":0,"_jetpack_memberships_contains_paid_content":false,"footnotes":""},"categories":[1],"tags":[],"class_list":["post-15659","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-uncategorized"],"acf":[],"jetpack_featured_media_url":"https:\/\/www.emilysentourage.org\/wp-content\/uploads\/FDA-Patient-Listening-Session_website-2.png","jetpack_sharing_enabled":true,"post_mailing_queue_ids":[],"_links":{"self":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/15659","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/users\/1281"}],"replies":[{"embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/comments?post=15659"}],"version-history":[{"count":5,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/15659\/revisions"}],"predecessor-version":[{"id":15855,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/15659\/revisions\/15855"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/media\/15690"}],"wp:attachment":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/media?parent=15659"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/categories?post=15659"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/tags?post=15659"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}