Table 1 Key demographic and baseline clinical characteristics of adult male participants with severe hemophilia A who underwent liver biopsy in the phase 1/2 clinical trial of valoctocogene roxaparvovec (AAV5-hFVIII-SQ)

^aParticipant	Dose of AAV5-hFVIII-SQ, vg per kg body weight	^bAge at first enrollment, years	Biopsy time point (after first enrollment), weeks (years)	Biopsy date	Route of biopsy	No. of hepatic lobules in biopsy sample	ALT at time of biopsy, U l⁻¹	^cFVIII activity at time of biopsy, IU dl⁻¹
^aParticipant	Dose of AAV5-hFVIII-SQ, vg per kg body weight	^bAge at first enrollment, years	Biopsy time point (after first enrollment), weeks (years)	Biopsy date	Route of biopsy	No. of hepatic lobules in biopsy sample	ALT at time of biopsy, U l⁻¹	CS result	OS result
1^d	6 × 10¹²	25	201 (3.86)	Aug 2019	Transjugular	12	29	BLD	BLD
11	4 × 10¹³	37	140 (2.69)	Aug 2019	Transjugular	23	11	18.6	28.4
15	4 × 10¹³	37	148 (2.85)	Jan 2020	Percutaneous	15	20	BLD	2.1
3	6 × 10¹³	32	214 (4.12)	Jan 2020	Percutaneous	23	12	8.2	14
4	6 × 10¹³	23	213 (4.10)	Mar 2020	Percutaneous	18	11	13.5	23.9

^aParticipants were numbered according to the order in which they were enrolled and dosed in the clinical trial^4,5.
^bAge at enrollment into the valoctocogene roxaparvovec phase 1/2 clinical trial (NCT02576795).
^cFVIII levels were measured using both a one-stage (OS) activated partial thromboplastin time-based clotting assay and a chromogenic substrate (CS) assay¹⁸.
^dParticipants were admitted to hospital on the morning of the procedure and all except participant 1 were discharged on the following day as planned. Participant 1 experienced abdominal pain during the procedure and had post-procedural bleeding. Computed tomography scan revealed a small hepatic hematoma with no evidence of active bleeding or vascular injury. The participant was treated with FVIII replacement and discharged 2 d after the biopsy procedure due to clinical improvement. The participant was readmitted 1 week after discharge with abdominal pain, vomiting and orthostatic hypotension; repeat computed tomography revealed old blood within the liver capsule and a liver laceration but no active bleeding. Although there were complications involving a fever, the participant was managed successfully with supportive treatment and finally discharged 3 weeks after the biopsy procedure.
BLD, below limit of detection.

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