1. MD.Trần Thị Bích Huyền Children hospital 1 Endocrine department
Efficacy of exogenous Growth hormon (GH) treatment in children with growth hormon deficiency (GHD) in Children Hospital 1
MD.Trần Thị Bích Huyền
Children hospital 1
Endocrine department

2. 1. Introduction
Growth hormon (GH): secreted by anterior pituitary lobe, stimulates cartilage growth.
GH deficiency (GHD) causes growth failure in children.
The etiology of GHD may be congenital or acquired.
Incidence of short stature associated GHD : 1/

3. 1. Introduction
In the world: GH has been available for management of short stature associated GHD from
In1921: initial therapy with bovine GHnot efficiency.
1985: Recombinant DNA-derived human GH (hGH) has been available.

4. 1. Introduction
Vietnam: limitation in diagnosis and treatment GHD (laboratory, drugs)
2010 : diagnostic approach to short stature and started to use exogenous GH for GHD in Children hospital.
We report the efficacy of GH therapy in 7 patients with GHD in my hospital.

5. 2. Background

6. Definition
Growth failure: defined as height velocity less than 2 SD below the mean for age ( corresponds to approximately < 3rd percentile.

7. Aetiology of GHD Congenital forms Acquired forms
Tumours or defects of hypothalamus
Pituitary: tumours, trauma, central nervous system disease, infections, late effects of cancer therapy ...

8. Diagnostic approach to Short stature
Clinical and auxological assessment
Baseline Investigations:
Bone age
Karyotype
Systemic screen
FBC, EUC, TSH, T4, Ca, Phos, urinalysis
IGF-1, GH, LH, FSH, Testosterone/Estrogen
Subsequent Investigations:
GH stimulation tests
Imaging studies as indicated (MRI)

9. Stimulation testing
Clonidine, Arginine, Insulin, Glucagon, Levodopa, GHRH, exercise test.
Diagnosis of GHD: 2 stimulation tests
Children hospital 1: Insulin and exercise test

10. Stimulation testing Peak GH > 20mU/l (10ng/ml): normal
Peak GH: 10-20mU/l (5-10ng/ml): partial GH deficiency
Peak GH< 10mU/l (5ng/ml): GH deficiency

11. Definition of Biochemical GH deficiency
Peak GH cut off for diagnosis of GHD
mU/L
No of provocative tests
Australia
10 2
UK (Kirk 2006)
<20
1 or 2
USA
<30
New Zealand
Germany
2 (1 with low IGF-1 or IGFBP-3)
France
Netherlands
Spain
Sweden
<24
Canada
Israel
Japan(<2005)
Japan(>2005)*
<18
Taiwan

12. Management Indications for GH treatment in CH 1: GHD Turner syndrome
Somatropin (Growth hormon: 0,2-0,3 mg/kg/ week)
Follow up
Side effects
Plotting on growth charts and monitoring growth rate over a 3 to 6 month period
Reassessment : IGF-1, bone age every 6-12 month period

13. GH side effects Uncommon overall
Benign intracranial hypertension (1 in 1000)
Slipped capital femoral epiphyses
Odema
Risk of diabetes
Progression of scoliosis
Cancer

14. 3. Outcome

15. Characteristics of the populationNo
Gender
Age 1 M 10 2
9,5 3 F
4,5 4 5
11,5 6 12 7

16. Characteristics of the populationNo
Gender
Age
Height (cm)
Z-SCORE
TARGET HEIGHT (cm) 1 M 10
114,5
-3,65
164 2
9,5
122
-2,28
161,5 3 F
4,5 78
-6,17
155 4 5
92,5
-3,43
165
11,5
134 -2
170,5 6 12
130
-2,57 7
120
-5,63
150

17. Boys Height

18. Girls height

19. Characteristics of the populationNo
Gender
Age
Weight (Kg)
BMI
Z-SCORE 1 M 10 25
19,1
1,24 2
9,5
25,5
17,1
0,45 3 F
4,5 13
21,4
3,26 4 5
15,2
-0,02
11,5 30
16,7
-0,35 6 12
17,8
0,16 7 26
18,1
-0,55

20. Characteristics of the populationNo
Gender
Age
Bone age
KARYOTYPE 1 M 10
7-8 2
9,5 8 3 F
4,5
1,5
46XX 4 5
2,5
11,5 6 12
9-10 7

21. Characteristics of the populationNo
Gender
Age
IGF1 (ng/ml)
GH (ng/ml) 1 M 10
194
0,12 2
9,5
174
0,04 3 F
4,5
176
0,45 4 5
172
3,03
11,5
214
0,61 6 12
120
0,48 7
182
0,0

22. Characteristics of the populationNo
TSH
(uIU/ml)
FT4
(ng/dl)
Cortisol
(g/dl) 1
1,3
1,68
8,25 2
1,39
1,57
14,7 3
4,65
0,86
15,15 4
3,13
1,72
8,97 5
3,67
1,19
8,87 6
3,52
0,96
8,9 7
4,53
0,83
6,8
Screening tests: FBC, EUC, Ca, Phos, urinalysis: normal

23. Stimulation test No GH (ng/ml) Peak GH (exercise) (test Insulin) 1
0,12
1,41
2,39 2
0,04
1,52
3,49 3
0,45
0,16
0,37 4
3,03
4,05
4,29 5
0,61
1,89
2,89 6
0,48
0,88
0,94 7
0,0
0,06
0,05
Peak GH after 2 stimulation test < 10mU/l (<5 ng/ml)

24. MRI No 2 6 MRI Small pituitary: 4x6x7 mm
Posterior pituitary lobe: absent
Anterior pituitary lobe: normal
The others has normal brain MRI

25. Outcome of therapy STT Dose of GH (mg/Kg/day) Time (month)
Height (before)
(cm)
Height (after)
∆H(cm)
∆H /month 1
0.04 13
114,5
126
11,5
0,88 2
0,03 4
122
125 3
0.75
0,04 14 78
101 23
1.64
3.5
92,5 96
1.00 5
0,035
9.5
134
144 10
1.05 6 24
130
146,5
16.5
0.69 7
120

26. Patient 1
TARGET HEIGHT (164cm)

27. Patient 2
TARGET HEIGHT (161,5cm)

28. Patient 3
TARGET HEIGHT (155cm)

29. Patient 4
TARGET HEIGHT (165cm)

30. Patient 5
TARGET HEIGHT (170,5cm)

31. Patient 6
TARGET HEIGHT (170,5cm)

32. Patient 7
TARGET HEIGHT (150cm)

33. Follow up No IGF1 (ng/ml) IGF1 (ng/ml) (6 months) Bone age BA (1 year)
194
186
7-8 9 2
174 - 8 3
176
146
1,5 4
172
2,5 5
214 6
6-7
120
9-10 11 7
182
Side effects: We did not notice any side effect of GH in all cases

34. Conclusion
All patients were diagnosed and started to treat with GH at mean age 9,5 years.
Height of cases less than 3 SD below the mean for age before treatment .
There was a clear improvement of height in all patients and the mean of height velocity was 1cm/month in the first year .
We did not notice any side effect of GH in all cases.

35. Recommendations
Assessment of growth over time is an essential aspect of child health care
More Scientific studies about GH treatment in Vietnam
Medical insurance

36. Thank you