Sorotan Daripada Mesyuarat Tahunan ASH ke-63
Hubungan Antara Ekspresi Endogen, Transgen FVIII dan Peristiwa Pendarahan Berikutan Pemindahan Gen Valoctocogene Roxaparvovec untuk Hemofilia A yang Teruk: Analisis Post-Hoc Percubaan GENER8-1 Fasa 3
Steven W. Pipe, MD1, Margareth C Ozelo, MD, PhD2*, Gili Kenet, MD3, Mark T Reding, MD4, Jane Mason, MBBS Hons, FRACP, FRCPA5,6 *, Andrew 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 *, Wing Yen Wong, MD22 *, Tara M Robinson, MD, PhD22 *, dan Benjamin Kim, MD, MPhil22
1Jabatan Pediatrik dan Patologi, Universiti Michigan, Ann Arbor, MI
2Hemocentro UNICAMP, Jabatan Perubatan Dalaman, Pusat Pengajian Sains Perubatan, University of Campinas, Campinas, Brazil
3Pusat Hemofilia Kebangsaan, dan Institut Penyelidikan Trombosis dan Haemostasis Amalia Biron, Pusat Perubatan Sheba, Tel Hashomer, Universiti Tel Aviv, Tel Aviv, Israel
4Pusat Pendarahan dan Gangguan Pembekuan, Pusat Perubatan Universiti Minnesota, Minneapolis, MN
5Universiti Queensland, Brisbane, QLD, Australia
6Pusat Haemophilia Queensland, Perkhidmatan Penjagaan Kanser, Royal Brisbane dan Hospital Wanita, Brisbane, QLD, Australia
7Universiti California San Fransico, San Francisco, CA
8Guy's & St. Thomas' NHS Foundation Trust, London, United Kingdom
9Pusat Hematologi, Jabatan Perubatan, Imperial College London, London, United Kingdom
10Pusat Rawatan Hemofilia Ortopedik, Los Angeles, CA
11Jabatan Perubatan Molekul, Institut Penyelidikan Scripps, La Jolla, CA
12Bahagian Hematologi, Jabatan Perubatan Dalaman, Hospital Universiti Nasional Taiwan, Taipei, Taiwan
13Jabatan Perubatan Radcliffe, Universiti Oxford, Oxford, United Kingdom
14Amanah Yayasan Perkhidmatan Kesihatan Nasional Hospital Universiti Oxford, Oxford, United Kingdom
15Institut Penyelidikan Kesihatan Kebangsaan, Pusat Penyelidikan Bioperubatan Oxford, Oxford, United Kingdom
16Pusat Rawatan Hemofilia, Hospital Kanak-Kanak Seluruh Negara dan Kolej Perubatan Universiti Ohio State, Columbus, OH
17Pusat Hemofilia dan Trombosis, Kampus Perubatan Universiti Colorado Anschutz, Aurora, CO
18Pusat Penyelidikan Darah UNC, Universiti North Carolina di Chapel Hill, Chapel Hill, NC
19Jabatan Pediatrik, Sekolah Perubatan Universiti Indiana, Pusat Penyelidikan Pediatrik IUPUI-Wells, Indianapolis, IN
20Makmal Glikobiologi, Institut Imunologi dan Terapi Eksperimen Hirszfeld, Wroclaw, Poland
21Amanah Yayasan NHS Hospital Universiti Cambridge, Cambridge, United Kingdom
22BioMarin Pharmaceutical Inc., Novato, CA