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. 2021 Sep 4;27(6):984–992. doi: 10.1111/hae.14398

TABLE 4.

ABR for PwHA treated with emicizumab by ADA status a

ADA‐negative PwHA (N = 634) ADA‐positive PwHA b (N = 30) ADA‐positive PwHA with decreased exposure  (N = 4)

Duration of efficacy period

Median [IQR], weeks

103

[84‐150]

103

[78‐160]

57 c

[16; 65; 80; 50]

Annualised rate of treated bleeding events

Model‐based d [95% CI]

.9

[.79‐1.11]

.7

[.37‐1.33]

14.1 c

[23.0; 28.0; 0; 5.3]

Proportion of participants with zero treated bleeds, n (%) [95% CI e ]

374 (59.0)

[55.1‐62.9]

16 (53.3)

[34.3‐71.7]

1 (25.0)

[.6‐80.6]

Annualised rate of all bleeding events

Model‐based d [95% CI]

2.3

[1.99‐2.60]

1.2

[.75‐1.91]

15.2 c

[23.0; 28.0; 0; 7.4]

Proportion of participants with zero all bleeds

n (%) [95% CI e ]

224 (35.3)

[31.6‐39.2]

7 (23.3)

[9.9‐42.3]

1 (25.0)

[.6‐80.6]

Abbreviations: ABR, annualised bleed rate; ADA, anti‐drug antibody; CI, confidence interval; IQR, interquartile range; PwHA, persons with haemophilia A.

a

Data includes the up‐titration period.

b

Excluding participants A, B, C, and D with ADA with decreased exposure.

c

Median [individual data for participants A, B, C, and D, respectively] (Figures S1–S4).

d

Model‐based ABR were derived using a negative‐binomial regression model.

e

CIs were derived using the exact method.