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Konferans Briefings

Premye-nan-moun etid katwini swivi sou dirab efikasite ki ka geri ou ak sekirite: AAV jèn Terapi ak Valoctocogene Roxaparvovec pou imilit grav A

Premye-nan-moun etid katwini swivi sou dirab efikasite ki ka geri ou ak sekirite: AAV jèn Terapi ak Valoctocogene Roxaparvovec pou imilit grav A
Pwen esansyèl ki soti nan Summit Virtual WFH 2020

Premye-nan-moun etid katwini swivi sou dirab efikasite ki ka geri ou ak sekirite: AAV jèn Terapi ak Valoctocogene Roxaparvovec pou imilit grav A

K. John Pasi, MB, ChB, PhD, FRCP, FRCPath, FRCPCH1; Savita Rangarajan, MBBS, FRCP, FRCPath2; Nina Mitchell, MB, BChir3; Èske Lester, MB, ChB, PhD, FRCP, FRCPath4; Emily Symington, BSc, MBBS, MRCP, FRCPath5; Bella Madan, MD, FRCP, FRCPath6; Michael Laffan, DM, FRCP, FRCPath7; Chris B. Russell, PhD3; Mingjin Li, MSc3; Benjamin Kim, MD, MPhil3; Glenn F. Pierce, MD, PhD8; Zèl yen wong, MD3

1Barts ak Lekòl la nan Lond Medsin ak Dexter, London, UK

2Inivèsite Lopital Southampton, Southampton, UK

3BioMarin Inc., Novato, CA, USA

4Inivèsite Lopital Birmingham NHS Foundation Trust, Birmingham, UK

5Inivèsite Cambridge Inivèsite Fondasyon NHS Trust, Cambridge, UK

6Guy's & St. Thomas 'Trust Fondasyon NHS, London, UK

7Sant pou ematoloji, Imperial Kolèj London, London, UK.

8Konsiltan, La Jolla, CA, USA

Kle Done Pwen

Dyagram sa a sou bò gòch la rezime kòwòt la dòz pou BMN 270 (valoctocogene roxaparvovec). 2 pasyan yo nan dòz ki pi ba yo (6 x 1012 ak 2 x 1013 vg / kg) pa te montre ase ekspresyon FVIII. Rezilta etid yo revize pou 13 pasyan yo nan 2 koud dòz pi wo yo: 7 nan 6 x 1013 vg / kg (6E13) ak 6 nan 4 x 1013 vg / kg (4E13). Karakteristik debaz pou tout 15 pasyan yo montre sou bò dwat la. Miyò, 14 nan 15 pasyan yo te sou terapi prophylactiques nan debaz, ak yon mwayèn vle to senyen annualisé (ABR) nan 6.5.

Pasyan nan tou de koòdone dòz te montre rediksyon sibstansyèl nan ABR ki te soutni pou jiska 4 ane pòs-perfusion nan kòwòt la 6E13 ak 3 ane nan kòwòt la 4E13. Mwayen ABR kimilatif te redwi pa 95% a 0.8 nan kòwòt 6E13 ak pa 93% a 0.9 nan kòwòt 4E13. Pa te gen okenn emoraji espontane nan 4 ane pou 6 nan 7 patisipan yo nan kòwòt la 6E13 ak nan ane 3 pou 5 nan 6 patisipan yo nan kòwòt la 4E13. Tout patisipan yo rete nan pwofilaktik FVIII.

Pasyan ki nan 2 koort yo dòz (6E13 nan vèt, 4E13 nan jòn) demontre dòz-depandan, soutni ogmante nan nivo aktivite FVIII pou jiska 208 semèn nan kòwòt la 6E13 ak 156 semèn nan kòwòt la 4E13 (ki baze sou tchoul chromogenic a). Nivo vle di FVIII yo te jeneralman pi wo pou kòwòt nan dòz pi wo (n = 7), pik nan 70% -80% sou semèn 16 a 52, ki te swiv pa yon bès ralanti. Nivo vle di FVIII nan kòwòt nan dòz pi ba (n = 6) som nan 20% -30% sou menm peryòd la, tou ki te swiv pa yon bès ralanti.

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

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