Table 3.

Parameter Estimates of Final Multivariate Models for Selected Phase 3 Efficacy End Points in Luspatercept‐Treated Patients Who Required Regular Transfusions

Parameter	Estimate	SE	OR	95%CI of OR	P
≥33% Reduction in weeks 13‐24
AUCavg24, μg·day/mL	0.00620	0.00407	1.01	0.998‐1.01	.1275
Age, years	0.0354	0.0154	1.04	1.01‐1.07	.0210
RBCT burden (RBC units/24 weeks)	−0.0985	0.0464	0.906	0.827‐0.993	.0338
≥33% Reduction in weeks 37‐48
AUCavg48, μg·day/mL	0.00571	0.00399	1.01	0.998‐1.01	.1526
Age, years	0.0386	0.0161	1.04	1.01‐1.07	.0168
RBCT burden (RBC units/24 weeks)	−0.109	0.0498	0.896	0.813‐0.988	.0281
Genotype β0/β0	−1.04	0.484	0.354	0.137‐0.913	.0317
≥33% Reduction in any 12 weeks
AUCavg48, μg·day/mL	0.00172	0.00348	1.00	0.995‐1.01	.621
≥33% Reduction in any 24 weeks
AUCavg48, μg·day/mL	0.00203	0.00376	1.00	0.995‐1.01	.58901
Age, years	0.0461	0.0149	1.05	1.02‐1.08	.00194
RBCT burden (RBC units/24 weeks)	−0.111	0.0419	0.895	0.824‐0.971	.00803
Race: Asian (reference = non‐Asian)	−0.832	0.331	0.435	0.228‐0.833	.01193
Mild/moderate renal impairment	−1.11	0.479	0.330	0.129‐0.844	.02070
≥50% Reduction in any 12 weeks
AUCavg48, μg·day/mL	−0.00424	0.00361	0.996	0.989‐1.00	.24111
Age, years	0.0423	0.0143	1.04	1.01‐1.07	.00302
RBCT burden (RBC units/24 weeks)	−0.146	0.0414	0.864	0.797‐0.938	< .001

AUC_avg24, average area under the concentration‐time curve from weeks 1 to 24; AUC_avg48, average area under the concentration‐time curve from weeks 1 to 48; CI, confidence interval; OR, odds ratio; P, tail probability of the normal distribution (Wald test); RBCT, red blood cell transfusion; SE, standard error.

For the 12‐week evaluation period, a reduction of at least 2 units RBCs transfused was required for responders.