Table 2.

Completed clinical trials of luspatercept in MDS

Luspatercept trials			Patient population	End point	No. of erythroid responses		Dosing, mg/kg
Phase	Trial name	NCT no.			Luspatercept	Placebo
1		NCT01432717	Postmenopausal, healthy women (age 45-75 years)	Mean hemoglobin change at day +15	24	8	0.0625-0.25
2	PACE-MDS	NCT01749514	IPSS low or intermediate-1 risk, anemia with or without transfusion dependence	HI-E, RBC-TI ≥ 8 weeks	32 (HI-E, 63%); 16 (RBC-TI, 38%)	—	0.125-1.75
3	MEDALIST	NCT02631070	IPSS-R very low, low, or intermediate risk, ≥15% RS or ≥5% RS with SF3B1 mutation, R/R ESA or serum EPO >200 U/L, transfusion dependence (≥2 units once every 8 weeks)	HI-E, RBC-TI ≥ 8 weeks	81 (HI-E, 53%); 58 (RBC-TI, 38%)	9 (HI-E, 12%); 10 (RBC-TI, 13%)	1.0-1.75

R/R, relapsed or refractory.