Response rates based on erythroid response criteria in patients with β-thalassemia
| . | 0.6-1.25 mg/kg luspatercept (N = 63)* . | 95% CI . |
|---|---|---|
| Non–transfusion-dependent patients, n/N (%) | n = 31† | |
| Mean hemoglobin increase, g/dL | ||
| ≥1.5 for 14 d‡ | 18/31 (58) | 39.1-75.5 |
| ≥1.0 for 12 wk | 22/31 (71) | 52.0-85.8 |
| ≥1.5 for 12 wk | 14/31 (45) | 27.3-64.0 |
| Decrease in LIC ≥2 mg/g dry weight§ | 5/15 (33) | 11.8-61.6 |
| Transfusion-dependent patients, n/N (%) | n = 32† | |
| Transfusion burden reduction, % | ||
| ≥20‡ | 26/32 (81) | 63.6-92.8 |
| ≥33 | 23/32 (72) | 53.3-86.3 |
| ≥50 | 20/32 (63) | 43.7-78.9 |
| Decrease in LIC ≥2 mg/g dry weight§ | 5/9 (56) | 21.2-86.3 |
| . | 0.6-1.25 mg/kg luspatercept (N = 63)* . | 95% CI . |
|---|---|---|
| Non–transfusion-dependent patients, n/N (%) | n = 31† | |
| Mean hemoglobin increase, g/dL | ||
| ≥1.5 for 14 d‡ | 18/31 (58) | 39.1-75.5 |
| ≥1.0 for 12 wk | 22/31 (71) | 52.0-85.8 |
| ≥1.5 for 12 wk | 14/31 (45) | 27.3-64.0 |
| Decrease in LIC ≥2 mg/g dry weight§ | 5/15 (33) | 11.8-61.6 |
| Transfusion-dependent patients, n/N (%) | n = 32† | |
| Transfusion burden reduction, % | ||
| ≥20‡ | 26/32 (81) | 63.6-92.8 |
| ≥33 | 23/32 (72) | 53.3-86.3 |
| ≥50 | 20/32 (63) | 43.7-78.9 |
| Decrease in LIC ≥2 mg/g dry weight§ | 5/9 (56) | 21.2-86.3 |
Results shown are the numbers of patients achieving any of the specified end points over any unbroken 12-wk period on study compared with baseline; only patients receiving dose levels of 0.6-1.25 mg/kg of luspatercept at any time during the study are shown (63 of 64 total patients; initial and extension stages).
One of the 64 patients initially enrolled is not included because the patient did not receive luspatercept ≥0.6 mg/kg during the study.
One patient classified as non–transfusion dependent during the initial stage of the study was reclassified as transfusion dependent before entering the extension stage and is counted as transfusion dependent in the table.
Primary end point; other end points shown are secondary end points.
Includes patients with baseline LIC ≥3 mg/g dry weight who were treated for ≥4 mo.