IPTACOPAN ASH UPDATE
Iptacopan monotherapy was superior to SoC for transfusion
avoidance
Observed
Population
estimate
Transfusion avoidance
Estimated proportion¹ of
patients, % (95% CI)
100
22843220
60/62
14/35
patients treated patients treated
with iptacopan
with SoC
96.4%
Difference =
70.3%
(95% CI 52.6, 84.9)
P<0.00012
26.1%
Of the patients treated with anti-C5 therapy who
received transfusions, they received on average
more than double the number of transfusions
vs. the few patients on iptacopan
A post hoc sensitivity analysis
using a different approach for handling
missing data confirmed the significance
of the pre-specified analysis:
96.7% (95% CI 91.3, 100.0)
38.9% (95% CI 23.1, 55.8)
Difference = 57.8% (95% CI 39.8, 74.2), P<0.00012
1. Marginal proportions, differences in marginal proportions and 95% CIs were calculated using a logistic regression model that adjusted for baseline covariates and accounted for missing data. Marginal proportions reflect the population
average probability of a patient meeting the endpoint criteria. 2. P values are two-sided and unadjusted
12 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022
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