Patient demographics and baseline characteristics
| . | Initial luspatercept dose groups, mg/kg . | Total (N = 64) . | |
|---|---|---|---|
| 0.2-0.4 (n = 12) . | 0.6-1.25 (n = 52) . | ||
| Age, median (range), y | 33.5 (22-53) | 41.0 (20-62) | 38.5 (20-62) |
| Female, n (%) | 5 (42) | 26 (50) | 31 (48) |
| Splenectomy, n (%) | 10 (83) | 33 (64) | 43 (67) |
| Non–transfusion-dependent patients, n | 12 | 21 | 33 |
| Hemoglobin, median (range), g/dL | 8.5 (7.0-9.6) | 8.5 (6.5-9.8) | 8.5 (6.5-9.8) |
| LIC, mean (SD), mg/g dry weight | 5.1 (2.9) | 5.6 (4.3) | 5.4 (3.8) |
| Transfusion-dependent patients, n | 0 | 31 | 31 |
| RBC transfusion burden, median (range), units/12 wk* | NA | 8.0 (4-18) | 8.0 (4-18) |
| LIC, mean (SD), mg/g dry weight† | NA | 5.0 (5.3) | 5.0 (5.3) |
| BMD | |||
| Total hip, mean (SD), z score | −1.1 (0.90) | −0.66 (1.27) | −0.74 (1.21) |
| Lumbar spine, mean (SD), z score | −1.62 (0.58) | −1.61 (0.95) | −1.61 (0.88) |
| FACIT-F score, median (range)‡ | NA | 41.5 (25-48) | 41.5 (25-48) |
| . | Initial luspatercept dose groups, mg/kg . | Total (N = 64) . | |
|---|---|---|---|
| 0.2-0.4 (n = 12) . | 0.6-1.25 (n = 52) . | ||
| Age, median (range), y | 33.5 (22-53) | 41.0 (20-62) | 38.5 (20-62) |
| Female, n (%) | 5 (42) | 26 (50) | 31 (48) |
| Splenectomy, n (%) | 10 (83) | 33 (64) | 43 (67) |
| Non–transfusion-dependent patients, n | 12 | 21 | 33 |
| Hemoglobin, median (range), g/dL | 8.5 (7.0-9.6) | 8.5 (6.5-9.8) | 8.5 (6.5-9.8) |
| LIC, mean (SD), mg/g dry weight | 5.1 (2.9) | 5.6 (4.3) | 5.4 (3.8) |
| Transfusion-dependent patients, n | 0 | 31 | 31 |
| RBC transfusion burden, median (range), units/12 wk* | NA | 8.0 (4-18) | 8.0 (4-18) |
| LIC, mean (SD), mg/g dry weight† | NA | 5.0 (5.3) | 5.0 (5.3) |
| BMD | |||
| Total hip, mean (SD), z score | −1.1 (0.90) | −0.66 (1.27) | −0.74 (1.21) |
| Lumbar spine, mean (SD), z score | −1.62 (0.58) | −1.61 (0.95) | −1.61 (0.88) |
| FACIT-F score, median (range)‡ | NA | 41.5 (25-48) | 41.5 (25-48) |
NA, not applicable; SD, standard deviation.
Baseline RBC transfusion burden is defined as the total number of RBC units transfused in the 12 wk before first dose of luspatercept (and confirmed over 6 mo before treatment).
LIC data were not available at baseline for 1 of the 31 transfusion-dependent patients.
FACIT-F score was assessed in 26 of the 64 patients, none of whom received 0.2-0.4 mg/kg of luspatercept. For scoring assessment, a scale of 0 to 52 is used, with 0 indicating worst quality of life and 52, best. A score <44 points is considered to be a FACIT-F deficit.16