Relasyon ant ekspresyon andojèn, transjèn transjèn FVIII ak evènman senyen apre transfè jèn Valoctocogene Roxaparvovec pou emofili grav A: Yon analiz post-hoc nan esè GENER8-1 Faz 3 la
Pwen esansyèl Soti nan 63yèm Reyinyon Anyèl ASH

Relasyon ant ekspresyon andojèn, transjèn transjèn FVIII ak evènman senyen apre transfè jèn Valoctocogene Roxaparvovec pou emofili grav A: Yon analiz post-hoc nan esè GENER8-1 Faz 3 la

Steven W. Pipe, MD1, Margareth C Ozelo, MD, PhD2*, Gili Kenet, MD3, Mark T Reding, MD4, Jane Mason, MBBS Hons, FRACP, FRCPA5,6 *, Andre D Leavitt, MD7, Bella Madan, MD8*, Michael Laffan, DM, PhD9, Doris V. Quon10, Annette von Drygalski, MD, Pharm D11, Sheng-Chieh Chou, MD, PhD12 *, Susan Shapiro, MD, PhD13,14,15 *, Amy L Dunn, MD16 *, Michael Wang, MD17, Nigel S Key18, Radoslaw Kaczmarek, PhD19,20 *, Emily Symington, MD21 *, Adebayo Lawal, MD, MSc, MBA22 *, Reena Mahajan, MD22 *, Konstantia-Maria Chavele, PhD22 *, Divya B Reddy, MD22 *, Hua Yu, PhD22 *, Zèl Yen Wong, MD22 *, Tara M Robinson, MD, PhD22 *, ak Benjamin Kim, MD, MPhil22

1Depatman Pedyatri ak Patoloji, University of Michigan, Ann Arbor, MI
2Hemocentro UNICAMP, Depatman Medsin Entèn, Lekòl Syans Medikal, University of Campinas, Campinas, Brezil
3Sant Nasyonal emofili a, ak Amalia Biron Research Institute of tronboz ak emostaz, Sheba Medical Center, Tel Hashomer, Tel Aviv University, Tel Aviv, Izrayèl.
4Sant pou senyen ak twoub kayo, University of Minnesota Medical Center, Minneapolis, MN
5University of Queensland, Brisbane, QLD, Ostrali
6Queensland Haemophilia Center, Sèvis Swen Kansè, Royal Brisbane ak Lopital Fanm, Brisbane, QLD, Ostrali
7University of California San Fransico, San Francisco, CA
8Guy's & St. Thomas' NHS Foundation Trust, London, Wayòm Ini
9Sant pou ematoloji, Depatman Medsin, Imperial College London, London, Wayòm Ini
10Sant Tretman Òtopedik Emofili, Los Angeles, CA
11Depatman Medsin Molekilè, Scripps Research Institute, La Jolla, CA
12Divizyon ematoloji, Depatman Medsin Entèn, National Taiwan University Hospital, Taipei, Taiwan
13Radcliffe Depatman Medsin, University of Oxford, Oxford, Wayòm Ini
14Oxford University Hospitals National Health Service Foundation Trust, Oxford, Wayòm Ini
15National Institute for Health Research, Oxford Biomedical Research Centre, Oxford, Wayòm Ini
16Sant Tretman Emofili, Lopital pou Timoun nan tout peyi a ak Kolèj Medsin Inivèsite Eta Ohio, Columbus, OH
17Sant emofili ak tronboz, University of Colorado Anschutz Medical Campus, Aurora, CO
18UNC Blood Research Center, University of North Carolina nan Chapel Hill, Chapel Hill, NC
19Depatman Pedyatri, Indiana University School of Medicine, IUPUI-Wells Center for Pediatric Research, Indianapolis, IN
20Laboratwa nan glikobioloji, Hirszfeld Enstiti pou iminoloji ak terapi eksperimantal, Wroclaw, Polòy
21Cambridge Inivèsite Lopital NHS Fondasyon Trust, Cambridge, Wayòm Ini
22BioMarin Pharmaceutical Inc., Novato, CA

Kle Done Pwen

Faz 3 GENER8-1 konsepsyon etid

Rezilta yo nan faz 3 GENER8-1 etid la te analize pou relasyon ki genyen ant aktivite transgene ki sòti FVIII ak senyen. Nan kantite total patisipan yo nan etid la (N = 134), analiz sa a enkli yon ti gwoup pasyan ki te fè eksperyans ≥ 1 senyen trete nan peryòd apre tranzisyon an, ki te kòmanse 1 mwa apre fen peryòd post-profilaktik ki te swiv la. perfusion nan transjèn.

ABR nan patisipan yo ak senyen trete apre tranzisyon (n = 22)

Nan 134 patisipan total nan etid GENER8-1, 22 (16%) te fè eksperyans omwen 1 senyen trete nan peryòd apre tranzisyon an. Yo montre ABR pou patisipan sa yo pou debaz pre-perfusion ak peryòd etid apre perfusion, apre pwofilaktik. Parapò ak debaz, 15 patisipan yo te gen yon diminisyon nan ABR, 2 te gen yon ogmantasyon nan ABR <1 (flèch nwa) ak 5 te gen yon ogmantasyon nan ABR> 1 (flèch zoranj).

RELATED kontni

Webinars entèaktif
Imaj

Please enable the javascript to submit this form

Sipòte pa sibvansyon edikasyon bay Bayer, BioMarin, CSL Behring, Freeline Therapeutics Limited, Pfizer Inc., Spark Therapeutics, ak uniQure, Inc.

Esansyèl SSL