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Table 4 Disease severity in ENVISION trial participants according to hemin prophylaxis at baseline

From: Disease burden in patients with acute hepatic porphyria: experience from the phase 3 ENVISION study

 

No prior hemin prophylaxis

Prior hemin prophylaxis

Placebo (n = 28)

Givosiran (n = 28)

Placebo (n = 18)

Givosiran (n = 20)

Historical AAR, median (range)

6.0 (0–46)

8.0 (4–34)

9.0 (4–38)

9.0 (4–32)

Chronic symptoms, n (%)

17 (61)

16 (57)

9 (50)

7 (35)

Chronic opioid use, n (%)

7 (25)

6 (21)

6 (33)

8 (40)

EQ VAS, mean (SD)

63.9 (20.7)

58.4 (23.0)

64.8 (18.4)

68.4 (21.3)

SF-12 PCS, mean (SD)

36.5 (10.5)a

39.3 (11.2)

40.5 (8.4)

39.7 (7.8)

  1. AAR annualized attack rate, EQ VAS EuroQol visual analog scale, PCS physical component summary, SF-12 12-Item Short-Form Health Survey, version 2
  2. aData missing for 1 patient