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Pwen esansyèl nan 25yèm Reyinyon Anyèl ASGCT
Koreksyon emostatik ki estab ak amelyore kalite lavi ki gen rapò ak emofili: analiz final ki soti nan Faz pivotal 3 HOPE-B esè Etranacogene Dezaparvovec.
Prezante pa: Steven W. Pipe, MD, University of Michigan, Ann Arbor, Michigan, Etazini
Steven W. Pipe1, Frank WG Leebeek2, Michael Recht3, Nigel S. Key4, Giancarlo Castaman5, David Cooper6, Robert Gut6, Ricardo Dolmetsch6, Yanyan Li7, Pòl E. Monahan7, Wolfgang Miesbach8
1University of Michigan, Ann Arbor, MI
2Erasmus MC, University Medical Center, Rotterdam, Netherlands
3Oregon Sante ak Syans Inivèsite, Portland, OSWA
4Inivèsite North Carolina, Chapel Hill, NC
5Sant pou maladi senyen ak koagulasyon, Careggi Inivèsite Lopital, Florence, Itali
6uniQure Inc., Lexington, MA
7CSL Behring, wa Lapris, PA
8Inivèsite lopital Frankfurt, Frankfurt, Almay
Kle Done Pwen
Aktivite FIX nan yon etap pou pasyan modere grav (N = 10) ak grav (N = 44) enfuze ak etranacogene dezaparvovec (AAV5-Padua hFIX) nan yon dòz 2x10.13 gc/kg nan faz 3 HOPE-B esè. Analiz sa a enkli pasyan ki gen ak san antikò netralize AAV5 (NAbs).
Aktivite FIX nan yon eta nan debaz ak nan 18 mwa apre perfusion pou pasyan ki gen ak san AAV5 NAbs pre-egziste. Avèk antikò yo te defini kòm gen yon tit ki pi gran pase limit la nan deteksyon (LOD). San yo pa antikò yo te defini kòm gen yon tit ki mwens pase oswa egal a LOD.
Pwofil sekirite etranacogene dezaparvovec nan 18 mwa te konsistan avèk done yo te prezante anvan. Evènman negatif ki pi souvan ki gen rapò ak tretman (TRAE) ki te rive nan plis pase 10% nan pasyan yo te ogmantasyon ALT (17%), maltèt (15%), maladi ki sanble ak grip (13%), ak reyaksyon ki gen rapò ak perfusion (13%). ).
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