Experimental treatments (phase 2/phase 3) of IPSS lower-risk MDS patients with severe thrombocytopenia
| Study . | Dose of drugs . | No. Patients . | Inclusion criteria . | Results (ORR) % . |
|---|---|---|---|---|
| ROM phase 3 randomized vs PBO61,62 | 750 μg/wk × 58 wk SC | 250 | PLT <50 × 109/L | 36.5 (IWG HI-P) |
| EL escalating dose phase 2 vs PBO 2:163 | 50-300 mg os daily | Ongoing | PLT <30 × 109/L | 32 (IWG HI-P) |
| ROM plus azacitidine64 | 500 and 750 μg/wk SC plus azacitidine 75 mg/m2 per d for 7 d on a 28-d cycle | 40 | Stratified PLT <50 and >50 × 109/L (MDS IPSS INT-1 and INT-2) | * |
| ROM plus decitabine randomized vs PBO plus decitabine65 | 750 μg/wk SC plus decitabine 20 mg/m2 per d for 5 d on a 28-d cycle or 15 mg/m2 IV over 3 h repeated every 8 h for 3 d every 6 wk | 29 | Stratified PLT <50 and >50 × 109/L and also higher-risk MDS | 33 |
| ROM plus lenalidomide66 | 500 or 750 μg/wk SC plus lenalidomide 10 mg/d for 21 d on a 28-d cycle | 39 | Del5q and non-del5q | † |
| Study . | Dose of drugs . | No. Patients . | Inclusion criteria . | Results (ORR) % . |
|---|---|---|---|---|
| ROM phase 3 randomized vs PBO61,62 | 750 μg/wk × 58 wk SC | 250 | PLT <50 × 109/L | 36.5 (IWG HI-P) |
| EL escalating dose phase 2 vs PBO 2:163 | 50-300 mg os daily | Ongoing | PLT <30 × 109/L | 32 (IWG HI-P) |
| ROM plus azacitidine64 | 500 and 750 μg/wk SC plus azacitidine 75 mg/m2 per d for 7 d on a 28-d cycle | 40 | Stratified PLT <50 and >50 × 109/L (MDS IPSS INT-1 and INT-2) | * |
| ROM plus decitabine randomized vs PBO plus decitabine65 | 750 μg/wk SC plus decitabine 20 mg/m2 per d for 5 d on a 28-d cycle or 15 mg/m2 IV over 3 h repeated every 8 h for 3 d every 6 wk | 29 | Stratified PLT <50 and >50 × 109/L and also higher-risk MDS | 33 |
| ROM plus lenalidomide66 | 500 or 750 μg/wk SC plus lenalidomide 10 mg/d for 21 d on a 28-d cycle | 39 | Del5q and non-del5q | † |
EL, eltrombopag; IWG HI-P; International Working Group hematological improvement-PLTs; ORR, overall response rate; ROM, romiplostim.
*The incidence of PLT transfusions was 46%, 36%, and 69% in patients receiving ROM 500 μg, ROM 750 μg, or PBO.
†Thrombocytopenia-related adjustments in lenalidomide dose occurred in 6 (50%) patients in the PBO group, 5 (36%) in the ROM 500 μg group, and 2 (15%) in the 750 μg group. No difference in transfusions was noted.