Table 1.

Radiographic outcomes in children after 64 weeks in clinical trials of burosumab.

Study	Age at enrollment	N	Dosing	RSS at baseline Mean ± SD (range)	RSS at 64 weeks Mean ± SD	RSS change from baseline LS mean [SE]	Global RGI-C at 64 weeks Mean [SE]	Global RGI-C ≥2 at 64 weeks (%)	Lower limb deformity RGI-C at 64 weeks
Carpenter et al. [35]	5–12 years	26	Burosumab Q2W (initially 0.1 mg/kg, titrated to mean 0.98 mg/kg)	1.9 ± 1.2 (0–4.5)	0.8 ± 0.6	−1.00 [0.11]	+1.56 [0.11]	58%	+0.5 [0.1]a
		26	Burosumab Q4W (initially 0.2 mg/kg, titrated to mean 1.5 mg/kg)	1.7 ± 1.0 (0–3.0)	0.9 ± 0.5	−0.84 [0.10]	+1.58 [0.11]	50%	See abovea
Whyte et al.[36]	1–4 years	13	Burosumab 0.8–1.2 mg/kg Q2W	2.9 ± 1.4 (1.0–6.5)	0.9 ± 0.5b	−2.0 [0.1]	+2.2 [0.1]	100%	+1.6 [0.1]
Imel et al.[22]	1–12 years	29	Burosumab 0.8–1.2 mg/kg Q2W	3.2 ± 1.0	1.0 ± 0.7b	−2.2 [0.1]	+2.1 [0.1]	87%	+1.3 [0.2]
		32	Phosphate and active vitamin D	3.2 ± 1.1	2.2 ± 0.8b	−1.0 [0.2]	+1.0 [0.1]	19%	+0.3 [0.1]

^aThe lower limb deformity score for this trial was only reported for the combined group including those dosed Q2W and Q4W together.

^bThese numbers were not expressly stated in the text, but were estimated for the table from the graphs in the original publications.