Paediatric Growth Hormone Deficiency (PGHD)

Genotropin (somatropin, rbe) has proven efficacy across five paediatric indications:1  

1. Genotropin has proven efficacy in growth hormone deficiency (GHD)1 with achievement of genetic height potential2

2. Genotropin achieves catch-up growth and normalises the stature of short ‘small for gestational age (SGA) children’3 who fail to manifest catch-up growth1

3. Genotropin maximises adult height with induction of puberty at an age appropriate time 4,5 in children with Turner Syndrome

4. Genotropin has a proven favourable effect on growth and body composition6 in children with Prader-Willi Syndrome (PWS)1

5. Genotropin induces persistent catch-up growth in chronic renal insufficiency and the majority of patients achieve normal adult height7

Prevalence of PGHD

The prevalence of paediatric growth hormone deficiency is estimated to be between 1 in 3500 and 1 in 4000 children. However, in about 50% of children the cause of growth hormone deficiency is unknown (idiopathic growth hormone deficiency).

Genotropin is effective in treating idiopathic PGHD2

Achievement of genetic height potential2

Genetic height potential normalised by growth hormone (GH) treatment (0.65 IU/kg body weight/week)2

Genetic height potential graph

Patients achieved their genetic height potential following treatment with growth hormone (0.65 IU/kg body weight/week for a median duration of 9.4 years) 2

Study methods
Analysis of final height in idiopathic GHD patients enrolled in KIGS database. Of 10,657 idiopathic GHD patients enrolled from 43 participating countries, 369 fulfilled the necessary final height criteria for inclusion in the analysis and were treated on an average dose of 0.49 IU/kg body weight/week (0.16 mg/kg body weight/week) growth hormone (GH) for a median of 8.1 years. A Swedish subgroup of patients (n=69) received conventional therapy (0.65 IU/kg body weight/week) for a mean of 9.4 years. The criteria used to define final height were height velocity less than 2 cm/year, calculated over a minimum of 9 months; chronological age more 17 years or bone age of more than 16 years in boys; chronological age more than 15 years or bone age more than 14 years in girls. To be included in the analysis the children had to have more than 2 years of GH treatment prior to puberty, and more than 5 years of GH treatment in total.
Adapted from Cutfield et al, 1999 2

Genotropin Dosage in Paediatric Patients1

Dosage recommendations in Paediatric Patients1

Indication

mg/kg body weight

dose per day

mg/m2 body surface area

dose per day

Growth hormone deficiency in children

0.025 - 0.035

0.7 - 1.0

Prader-Willi syndrome in children

0.035

1.0

Turner syndrome

0.045 - 0.050

1.4

Chronic renal insufficiency

0.045 - 0.050

1.4

Children born small for gestational age

0.035

1.0

 

Where childhood onset GHD persists into adolescence, treatment should be continued to achieve full somatic development (e.g. body composition, bone mass).1

For guidance on dosing in transition patients, please see the full Genotropin Summary of Product Characteristics.

Adverse Events in Children treated for Growth Disturbance with Genotropin (somatropin). 1

 

Long-term Treatment of Children with Growth Disturbance due to insufficient secretion of growth hormone1

System Organ Class

Very Common

≥ 1/10

Common

≥ 1/100 to <1/10

Uncommon

≥ 1/1,000 to <1/100

Rare

≥ 1/10,000 to <1/1,000

Very Rare

<1/10,000

Not Known (cannot be estimated from available data)

Neoplasms Benign, Malignant and Unspecified (including cysts and polyps)

  

  

Leukaemia†

  

  

  

Metabolism and Nutrition Disorders

 

 

 

 

 

Type 2 diabetes mellitus

Nervous System Disorders

 

 

 

 

 

Paraesthesia*

Benign intracranial hypertension

Musculoskeletal, Connective Tissue and Bone Disorders

 

 

Arthralgia*

 

 

Myalgia*

Musculoskeletal stiffness*

General Disorders and Administration Site Conditions

Injection site reaction$

 

 

 

 

Oedema peripheral*

Investigations

 

 

 

 

 

Blood cortisol decreased

 

*In general, these adverse effects are mild to moderate, arise within the first months of treatment, and subside spontaneously or with dose-reduction. The incidence of these adverse effects is related to the administered dose, the age of the patients, and possibly inversely related to the age of the patients at the onset of growth hormone deficiency.

$ Transient injection site reactions in children have been reported.

‡ Clinical significance is unknown

† Reported in growth hormone deficient children treated with somatropin, but the incidence appears to be similar to that in children without growth hormone deficiency. 

References

  1. Genotropin Summary of Product Characteristics.  http://www.medicines.org.uk/emc/search/?q=Genotropin Accessed March 2018  
  2. Cutfield W, et al. Final Height in Idiopathic Growth Hormone Deficiency: the KIGS Experience. Acta Paediatr 1999; 428(Suppl): 72–75. 
  3. De Zegher F, et al. Growth Hormone Treatment of Children Born Small for Gestational Age: Growth Responses with Continuous and Discontinuous Regimens over 6 Years. J Clin Endocrinol Metab 2000; 85(8): 2816–2821.
  4. Ranke MB, Price DA, Reiter EO (eds): Growth Hormone Therapy in Pediatrics –  20 Years of KIGS. Turner Syndrome within KIGS Including an Analysis of 1146 Patients Grown to Near Adult Height. Basel, Karger, 2007, pp 332–339.
  5. Ranke MB, Price DA, Reiter EO (eds): Growth Hormone Therapy in Pediatrics – 20 Years of KIGS. Turner Syndrome–Growth Hormone Treatment. Basel, Karger, 2007, pp 326–331.
  6. Lindgren AC, Ritzén EM. Five Years of Growth Hormone Treatment in Children with Prader-Willi Syndrome. Acta Paediatr 1999; 433(Suppl): 109–111.
  7. Haffner D. Effect of growth hormone treatment on the adult height of children with chronic renal failure. NEJM 2000; 343: 923–930.
     

Abbreviations: GHD, growth hormone deficiency; GH, growth hormone.

 

PP-GEN-GBR-0469. July 2018