Short Stature in Noonan Syndrome: Demography and Response to Growth Hormone Treatment in KIGS Barto J Otten KIGS 10 Year Book, 1999
Jan 18, 2018
Short Stature in Noonan Syndrome:Demography and Response to
Growth Hormone Treatment in KIGS
Barto J Otten
KIGS 10 Year Book, 1999
B J Otten - KIGS 10 Year Book, 1999
Auxological characteristics of all patients with Noonan Syndrome at the start of GH treatment
Ta, Tanner et al (1966); Ra, Ranke et al (1988)WHI, weight-for-height index; PAH, projected adult height
Variable Boys GirlsPercentile Percentile
(n) (median) (10th) (90th) (n) (median) (10th) (90th)
Mid-parental height (SDS) 95 -0.4 -2.4 0.9 41 -0.5 -1.7 0.8
Birth weight (SDS) 85 -0.4 -2.1 0.9 41 -0.7 -2.4 1.1Chronological age (yrs) 101 9.7 5.4 15.0 42 9.9 3.5 14.4
Bone age (yrs) 43 7.2 3.5 11.8 13 7.0 2.5 10.0
Height (Ta) (SDS) 101 -3.0 -4.3 -2.2 42 -3.1 -4.4 -2.1Height (Ra) (SDS) 100 -1.3 -2.7 -0.5 41 -1.0 -2.7 -0.1
Weight (SDS) 101 -2.8 -4.3 -1.4 42 -2.5 -4.5 -1.5WHI (%) 101 100 90 115 42 99 86 114Height velocity (cm/yr) 57 4.3 2.9 6.2 23 4.8 2.7 7.8
PAH (Ra) (cm) 100 152.2 141.5 158.1 41 143.9 132.8 150.4GH dose (IU/kg/week) 101 0.64 0.50 0.97 42 0.72 0.45 9.97Injections (n/week) 101 6.0 5.0 7.0 42 6.5 5.0 7.0
B J Otten - KIGS 10 Year Book, 1999
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Height of male patients (n=101) with Noonan syndrome at the start of GH treatment
, prepubertal; , in puberty; , no information on puberty; , median; , + 2 SD; , -4 SD (Noonan-specific standards; Ranke et al 1988)Shaded area, normative standards (Tanner et al 1966)
Chronological age (years)
Hei
ght (
cm)
B J Otten - KIGS 10 Year Book, 1999
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Height of female patients (n=42) with Noonan syndrome at the start of GH treatment
, prepubertal; , in puberty; , no information on puberty; , median; , + 2 SD; , -4 SD (Noonan-specific standards; Ranke et al 1988)Shaded area, normative standards (Tanner et al 1966)
Chronological age (years)
Hei
ght (
cm)
B J Otten - KIGS 10 Year Book, 1999
Growth response of 57 prepubertal patients with Noonan syndrome to 1 year of GH therapy compared with predicted model for first-year growth
response in Turner syndrome
Predicted height velocity (cm/years)
Stud
enti s
e d re
sidu
a ls
Response to GH therapy of patients with Noonan Syndrome(cross-sectional and longitudinal data for boys and girls)
B J Otten - KIGS 10 Year Book, 1999
Variable Cross-sectional data Longitudinal data
Boys Girls Boys Girls
Percentile Percentile (median) (median)
(n) (median) (10th) (90th) (n) (median) (10th) (90th) (n=20) (n=6)
At start
Chronological age (yrs) 101 9.7 5.4 15.0 42 9.9 3.5 14.4 8.8 3.2
Height (Ta) (SDS) 101 -3.0 -4.3 -2.2 42 -3.1 -4.4 -2.1 -2.9 -3.8
Height (Ra) (SDS) 100 -1.3 -2.7 -0.5 41 -1.0 -2.7 -0.1 -1.2 -1.5
Height velocity (cm/yr) 57 4.3 3.9 6.2 23 4.8 2.7 7.8
Projected height (cm) 100 152.2 141.5 158.1 144 143.9 132.8 150.4 152.4 141.2
(1/6)
B J Otten - KIGS 10 Year Book, 1999
Response to GH therapy of patients with Noonan Syndrome(cross-sectional and longitudinal data for boys and girls)
Variable Cross-sectional data Longitudinal data
Boys Girls Boys Girls
Percentile Percentile (median) (median)
(n) (median) (10th) (90th) (n) (median) (10th) (90th) (n=20) (n=6)
1st Year
Height (Ta) (SDS) 76 -2.7 -4.0 -1.9 31 -2.7 -4.5 -1.8 -2.6 -2.9
Height (Ra) (SDS) 76 -0.9 -2.2 0.2 31 -0.5 -2.5 0.4 -0.8 -1.0
Height velocity (cm/yr) 76 7.2 5.5 9.1 31 7.0 4.5 8.8
Projected height (cm) 76 155.5 145.4 162.6 31 147.7 134.3 153.4 155.5 144.1
(2/6)
B J Otten - KIGS 10 Year Book, 1999
Response to GH therapy of patients with Noonan Syndrome(cross-sectional and longitudinal data for boys and girls)
Variable Cross-sectional data Longitudinal data
Boys Girls Boys Girls
Percentile Percentile (median) (median)
(n) (median) (10th) (90th) (n) (median) (10th) (90th) (n=20) (n=6)
2nd Year
Height (Ta) (SDS) 59 -2.5 -3.7 -1.6 27 -2.4 -3.8 -1.7 -2.4 -2.8
Height (Ra) (SDS) 57 -0.7 -1.9 0.8 26 -0.4 -2.2 0.4 -0.7 -1.0
Height velocity (cm/yr) 57 5.7 4.7 8.1 24 6.2 2.3 7.3
Projected height (cm) 57 156.4 147.6 167.5 26 148.3 136.3 153.6 156.4 144.6
(3/6)
B J Otten - KIGS 10 Year Book, 1999
Response to GH therapy of patients with Noonan Syndrome(cross-sectional and longitudinal data for boys and girls)
Variable Cross-sectional data Longitudinal data
Boys Girls Boys Girls
Percentile Percentile (median) (median)
(n) (median) (10th) (90th) (n) (median) (10th) (90th) (n=20) (n=6)
3rd Year
Height (Ta) (SDS) 49 -2.6 -4.3 -1.2 14 -2.5 -3.7 -1.6 -2.5 -2.5
Height (Ra) (SDS) 43 -0.5 -2.2 1.0 14 0.1 -2.1 0.7 -0.7 -0.4
Height velocity (cm/yr) 46 7.2 5.5 9.1 31 7.0 4.5 8.8
Projected height (cm) 43 157.5 145.4 168.5 14 150.5 136.8 156.0 156.6 148.5
(4/6)
B J Otten - KIGS 10 Year Book, 1999
Response to GH therapy of patients with Noonan Syndrome(cross-sectional and longitudinal data for boys and girls)
Variable Cross-sectional data Longitudinal data
Boys Girls Boys Girls
Percentile Percentile (median) (median)
(n) (median) (10th) (90th) (n) (median) (10th) (90th) (n=20) (n=6)
4th Year
Height (Ta) (SDS) 37 -2.3 -3.6 -0.9 12 -2.4 -3.6 -1.3
Height (Ra) (SDS) 30 0.2 -2.3 1.3 11 0.0 -2.0 0.6
Height velocity (cm/yr) 35 5.0 3.8 7.5 12 5.0 1.7 6.8
Projected height (cm) 30 162.8 144.5 171.1 11 151.1 137.5 155.2
(5/6)
B J Otten - KIGS 10 Year Book, 1999
Response to GH therapy of patients with Noonan Syndrome(cross-sectional and longitudinal data for boys and girls)
Variable Cross-sectional data Longitudinal data
Boys Girls Boys Girls
Percentile Percentile (median) (median)
(n) (median) (10th) (90th) (n) (median) (10th) (90th) (n=20) (n=6)
5th Year
Height (Ta) (SDS) 24 -2.0 -3.6 -0.7 8 -2.3 -4.8 0.0
Height (Ra) (SDS) 19 0.5 -2.1 1.9 8 0.0 -2.7 1.9
Height velocity (cm/yr) 23 5.1 3.0 7.5 7 5.6 2.5 9.6
Projected height (cm) 19 165.0 146.1 175.1 8 151.1 132.8 164.0
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Response to 3 years of GH therapy of 26 patients (boys, n = 20; girls, n = 6) with Noonan Syndrome
B J Otten - KIGS 10 Year Book, 1999
Data are shown as box plots, with medians (rules), means (dots), 25th-75th percentiles (shaded boxes) and 10th-90th percentiles (whiskers)
p <0.05
(n = 26) (n = 26) (n = 26) (n = 26)
Start 1 2 3 Years on GH therapy
Pare
ntal
cor
rect
ed h
eigh
t SD
S
B J Otten - KIGS 10 Year Book, 1999
Auxological characteristics of all patients with Noonan Syndrome discontinued from KIGS
Ta, Tanner et al (1966); Ra, Ranke et al (1988)
Variable Boys Girls
Patients Percentile Patients Percentile
(n) (median) (10th) (90th) (n) (median) (10th) (90th)
At start of GH therapy
Chronological age (years) 36 12.0 5.8 15.9 16 12.7 7.5 14.9
Height (Ta) (SDS) 36 -3.4 -4.4 -2.5 16 -3.2 -4.1 -2.8
Height (Ra) (SDS) 36 -1.5 -2.7 -0.7 16 -1.0 -2.1 -0.1
Height velocity (1st year)(cm/yr)
31 6.4 5.5 9.1 13 7.0 3.8 8.0
(1/2)
B J Otten - KIGS 10 Year Book, 1999
Auxological characteristics of all patients with Noonan Syndrome discontinued from KIGS
Ta, Tanner et al (1966); Ra, Ranke et al (1988)
Variable Boys Girls
Patients Percentile Patients Percentile
(n) (median) (10th) (90th) (n) (median) (10th) (90th)
At discontinuation fromKIGSChronological age (years) 36 15.6 9.4 19.1 16 15.8 12.3 17.0
Height (Ta) (SDS) 36 -2.9 -3.8 -1.8 16 -2.4 -3.7 -1.7
Height (Ra) (SDS) 36 -0.8 -2.3 -0.7 16 -0.3 -1.8 0.3
Duration of GH (years) 36 2.7 1.0 6.3 16 2.5 1.0 6.0
Height velocity duringpreceding year (cm/year)
36 4.3 2.7 6.5 16 3.2 0.7 4.7
(2/2)
B J Otten - KIGS 10 Year Book, 1999
Characteristics of Noonan patients on GH therapy close to final height
Ta, Tanner et al (1966); Ra, Ranke et al (1988)
Patients Percentile(n) (median) (10th) (90th)
At start of therapyMid-parental height (SDS) 18 0.1 -1.4 1.3Birth weight (SDS) 16 0.1 -1.9 1.3
Chronological age (years) 18 14.1 12.1 15.9
Height (Ta) (SDS) 18 -3.7 -4.9 -2.8Height (Ra) (SDS) 18 -1.5 -2.7 -0.4
(1/2)
B J Otten - KIGS 10 Year Book, 1999
Characteristics of Noonan patients on GH therapy close to final height
Ta, Tanner et al (1966); Ra, Ranke et al (1988)
Patients Percentile(n) (median) (10th) (90th)
Close to final heightChronological age (years) 18 18.8 17.0 20.9Height (Ta) (SDS) 18 -2.1 -3.0 -0.7Height (Ra) (SDS) 18 0.9 -0.8 3.2Height velocity (cm/year) 18 1.9 0.7 4.5Duration of GH therapy (years) 18 4.7 2.5 7.4Height (Ta) (SDS) 18 1.5 0.2 2.7Height (Ra) (SDS) 18 2.4 0.9 5.0
(2/2)