Table 1.

Published short-term studies in patients with Noonan syndrome treated with rhGH.

	Macfarlane et al. (49)	Lee et al. (50)	Siklar et al. (48)	Horikawa et al. (51)
Patients, n	23	30	47	51
Publication year	2001	2015	2016	2020
Patient population	United Kingdom	USA	Turkey	Japan
Duration of therapy (years)	3	4	3	4
Age at start, years (range or SD)	9.3 ± 2.6 (4.5 to 14.0)	8.39 ± 3.45 (2.4 to 14.3)	9.8 ± 3.4 (0.08 to 17.8)	6.06 (2.25)
Height at start, SDS (SD)	-2.7 ( ± 0.4)	−2.64 ( ± 0.96)	-3.62 ( ± 1.14)	-3.24 ( ± 0.6) [0.033] -3.25 ( ± 0.71) [0.066]
rhGH doses (mg/kg/day) (SD)	0.047	0.048 ( ± 0.011)	0.035	0.033/0.066
Height at 1 year, SDS (SD)	-2.2 ( ± 0.6)	ND	ND	-2.4 [0.033] -1.78 [0.066]
Height gain at 1 year, SDS (SD)	0.5	0,50 ( ± 0,03)	0.4 ( ± 0.44)	0.84 [0.033] 1.47 [0.066]
Growth Velocity at 1 year (cm/year)	8.4. ± 1.7	ND	ND	ND
Genetic analysis	ND	ND	PTPN11 (n:39)	PTPN11, SOS1, KRAS, RAF1, BRAF, SHOC2, RIT1
Height at therapy end, SDS (SD)	-1.9 ( ± 0.9) (n:23)	-1.48 ( ± 0.09)	-2.85 ( ± 0.96)	-2.39 [0.033] -1.41 [0.066]
Height gain at therapy end, SDS (SD)	0.8	1.14 ( ± 0.13)	0.77 ( ± 0.42)	0.85 [0.033], 1.84 [0.066]

Data are expressed as means [Adapted from Carcavilla et al. (43)].