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. 2026 Jan 19;10(1):103360. doi: 10.1016/j.rpth.2026.103360

Figure 1.

Figure 1

Disposition of participants. aOne participant discontinued the infusion of etranacogene dezaparvovec due to a treatment-emergent adverse event of hypersensitivity after receiving ∼10% of the full dose. This participant was included in the full analysis set and the safety analyses but excluded from the responder analysis set. bThis participant had a non–treatment-related treatment-emergent adverse event of cardiogenic shock that resulted in death at 464 days postdose. Efficacy and safety data from this participant before the event were included in the analyses for the full analysis set, responder analysis set, and safety population set. cThis participant had a baseline adeno-associated viral vector serotype 5 titer of 3212 and did not respond to treatment with etranacogene dezaparvovec. Before the participant withdrew consent, his efficacy and safety data were included in the analyses for the full analysis set and the safety population set but were excluded from the responder analysis set. He continues to be followed for safety through review of medical records. dBefore the liver transplant, efficacy and safety data from this participant were included in the analyses for the full analysis set, responder analysis set, and safety population set. After the liver transplant, hepatocytes from the transplanted liver produced endogenous wild-type factor IX. As such, efficacy data from this participant no longer informed outcomes from gene therapy and were excluded from the analysis. Safety data from this participant were included in the analyses regardless of the liver transplant. DCO, data cutoff; NAb+, with preexisting adeno-associated viral vector serotype 5 neutralizing antibodies.