Efikasite ak Sekirite nan Etranacogene Dezaparvovec nan Adilt ki gen grav oswa modere-grav Emofili B: Premye done ki soti nan faz 3 Hope-B Jij Terapi Jijman an
Pwen esansyèl nan 14yèm Kongrè Anyèl EAHAD la

Efikasite ak Sekirite nan Etranacogene Dezaparvovec nan Adilt ki gen grav oswa modere-grav Emofili B: Premye done ki soti nan faz 3 Hope-B Jij Terapi Jijman an

Steven W Pipe, MD1, Michael Recht, MD, PhD2, Nigel S Key, MBChB3, Frank WG Leebeek, MD4, Giancarlo Castaman, MD5, Susan U Lattimore, RN6, Paul van der Valk, MD6, Kathelijne Peerlinck, MD, PhD7, Michiel Coppens, MD, PhD8, Niamh O'Connell, MB PhD9, K. Jan Pasi, MBChB, PhD10, Pyè Kampmann, MD11, Karina Meijer, MD, PhD.12, Annette von Drygalski, MD, PharmD13, Guy Young, MD14, Cedric Hermans, MD15, Jan Astermark, MD, PhD16, Robert Klamroth, MD, PhD17, Richard S Sitwon, MD, PhD18, Natan Visweshwar, MD19, Shelley Crary, MD20, Rashid Kazmi, MBBS21, Emily Symington, MBChB22, Miguel A Escobar, MD23, Esteban Gomez, MD24, Rebecca Kruse-Jarres, MD, MPH25, Adam Kotowski, MD26, Doris Quon, MD, PhD27, Michael Wang, MD28, Allison P Wheeler, MD29, Eileen K Sawyer, PhD30, Stephanie Verweij30, Valerie Colletta, MSc30, Naghmana Bajwa, MD, Robert Gut, MD, PD30, Wolfgang Miesbach, MD, PhD31

1Inivèsite Michigan, Ann Arbor, MI, USA

2Oregon Sante ak Syans Inivèsite, Portland, OSWA, USA

3Inivèsite North Carolina, Chapel Hill, NC, USA

4Erasmus MC, University Medical Center, Rotterdam, Netherlands

5Sant pou maladi senyen ak koagulasyon, Careggi Inivèsite Lopital, Florence, Itali

6Inivèsite Medisch Centrum Utrecht, Netherlands

7Depatman Medsin vaskilè ak Ememostazi ak Emofili Sant, Inivèsite Lopital Louvain, Louvain, Bèljik

8Depatman Medsin vaskilè, Amstèdam Syans kadyo-vaskilè, Amstèdam Inivèsite Sant Medikal, Inivèsite Amstèdam, Amstèdam, Netherlands

9Sant Koagulasyon Nasyonal la, Lopital St James a, Dublin, Iland

10Royal London Emofili Sant, Barts ak lekòl la London nan Medsin ak Dexter, London, UK

11Rigshospitalet, vil Copenhagen, Denmark

12Inivèsite Medikal Sant Groningen, Groningen, Netherlands

13Inivèsite Kalifòni San Diego, La Jolla, CA, USA

14Children's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA, USA

15Cliniques Universitaires Saint ‐ Luc, Université Catholique de Louvain, Brussels, Bèljik

16Lopital Inivèsite Skåne, Malmö

17Vivantes Klinikum im Friedrichshain, Bèlen, Almay

18Inivèsite Utah, Salt Lake City, UT, USA

19Inivèsite nan Sid Florid, Tampa, FL, USA

20University of Arkansas pou Syans Medikal, AR, USA

21Inivèsite Lopital Southampton NHS Fondasyon Trust; Southampton, UK

22Cambridge University - Addenbrooke's Hospital, Cambridge, UK

23Sant Syans Inivèsite Texas nan Houston, Houston, TX, USA

24Lopital Phoenix pou Timoun yo, Phoenix USA

25Bloodworks Nòdwès, Seattle, WA

26WNY BloodCare, NY, USA

27Los Angeles Orthopedic Hospital, Orthopedic Emophilia Treatment Center, Los Angeles, CA, USA

28Inivèsite Kolorado Emofili ak Sant tronbozi, Anschutz Medical Campus, Aurora, CO

29Sant Medikal Inivèsite Vanderbilt, Nashville, TN, USA

30uniQure BV, Amstèdam, Netherlands / uniQure Inc. Lexington, MA, USA

31Inivèsite lopital Frankfurt, Frankfurt, Almay

Kle Done Pwen

FIX Ekspresyon

Figi sa a montre 1 nan 3 pwen final yo ko-prensipal nan etid la, vle di ak medyàn aktivite FIX pou tout patisipan yo nan 26 semèn apre dòz ak 2 × 1013 gc / kg (N = 54). FIX ekspresyon te gaya pa 3 semèn pòs-dòz ak ki estab pa semèn 26, ak yon mwayèn de 37.2%.

Imaj

Paske etid sa a pa t 'eskli patisipan yo ak netralize antikò (NAbs) AAV, li te posib pou evalye rezilta pou korelasyon ant NAbs ak aktivite FIX. Aks vètikal la se semèn obsève 26 FIX aktivite moun ki te resevwa yon dòz konplè vektè. Pasyan ki gen NAbs detektab yo te trase nan 7 sou aks orizontal la (ki te papòt la nan tès la). Aks orizontal la montre tit yo nan lòt patisipan yo sou yon seri jiska 678. Te gen yon patisipan ki pa reponn ki te gen yon tit> 3200, ki pa montre sou echèl grafik sa a. Pa te gen okenn korelasyon obsève nan pre-egziste NAbs ak aktivite FIX jiska yon tit nan 678.

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