Table 2.
Mean Changes in Efficacy Parameters After 6 Months of Somavaratan Treatment
| Parameter | 5.0 mg/kg Monthly (n = 23) | 2.5 mg/kg Twice Monthly (n = 20) | 1.15 mg/kg Weekly (n = 21) |
|---|---|---|---|
| Mean annualized HV, cm/y (SD)a | 7.86 (2.5) | 8.61 (2.7) | 7.58 (2.5) |
| Change in HT-SDS (SD) | 0.28 (0.22) | 0.33 (0.23) | 0.24 (0.22) |
| Mean change in BMI, kg/m2 (SD) | 0.96 (0.72) | 0.84 (0.73) | 1.02 (0.72) |
| Mean change in bone age, years (SD) | 0.40 (0.42) | 0.39 (0.37) | 0.39 (0.23) |
BMI, body mass index; HT-SDS, height standard deviation score; HV, height velocity; SD, standard deviation.
a
Values are for the intention-to-treat population. For the 1 patient in the twice-monthly dose group who did not complete the 6-month study, annualized HV was imputed from the last observation of change in HT-SDS.