Appeal type: Medical necessity
Decision: Upheld

Diagnosis: Growth Hormone Deficiency
Treatment: Growth Hormone Therapy (Genotropin 12 milligrams)
The insurer denied: Growth Hormone Therapy (Genotropin 12 milligrams)
The denial is upheld

The patient is an adolescent male with history of small for gestational age (SGA) and fetal alcohol syndrome. His growth chart shows that his height used to track along the tenth percentile. However, starting around five years old, the patient's height has been tracking close to the fifth percentile. Recently the patient's insulin-like growth hormone one (IGF-1) was normal at 117 while insulin-like growth factor binding protein three (IGFBP-3) was 3531. The patient underwent growth hormone (GH) stimulation testing using Arginine and Clonidine, which showed a peak growth hormone (GH) of 7.3. 

The proposed treatment is not medically necessary.

Per the Consensus Guidelines for the Diagnosis and Treatment of Growth Hormone Deficiency (GHD) in Childhood and Adolescence by the GH Research Society (reference #1): Diagnosing growth hormone deficiency requires a multifaceted process including comprehensive clinical and auxological assessment, combined with biochemical tests of the growth hormone-insulin-like growth factor (IGF) axis and sometimes radiological evaluation which include:
1) Severe short stature, defined as a height more than 3.0 standard deviations below the mean. 
2) Height more than 1.5 standard deviations below the mid-parental height. 
3) Height more than 2.0 standard deviations below the mean and a height velocity over one year more than 1.0 standard deviations below the mean for chronological age, or a decrease in height standard deviations of more than 0.5 over one year in children over two years of age. 
4) In the absence of short stature, a height velocity more than 2.0 standard deviations below the mean over one year or more than 1.5 standard deviations sustained over two years; this may occur in growth hormone deficiency, presenting in infancy, or in organic acquired growth hormone deficiency. 
5) Signs indicative of an intracranial lesion. 
6) Signs of multiple pituitary hormone deficiency (MPHD); and 
7) Neonatal symptoms and signs of growth hormone deficiency.

Biochemical Assessment of growth hormone deficiency: 
1) A combination of two provocative growth hormone (GH) testing can be used to test for growth hormone deficiency and failing both tests has traditionally been used to support the diagnosis. 
Based on the clinical data submitted, the patient did not meet many of the criteria for the diagnosis of an isolated growth hormone deficiency.

This patient has no significant short stature and there is no clear demonstration of persistent linear growth deceleration to suspect growth hormone deficiency. Pretreatment height was close to the fifth percentile and growth velocity was 4.6 centimeters per year (-1.0 standard deviations for age and gender), both are within acceptable range. Serum insulin-like growth factor one (IGF-1) level (a good surrogate marker of growth hormone status) was noted to be normal despite an attenuated peak growth hormone response to provocative tests. Bone age was normal, while there was no mention of pituitary abnormality on brain magnetic resonance image (MRI). These findings are not consistent with growth hormone deficiency per the above Consensus Guidelines for the Diagnosis and Treatment of Growth Hormone (GH) Deficiency in Childhood and Adolescence by The GH Research Society and the referenced medical literature.
This patient also does not meet criteria for growth hormone treatment under the Food and Drug Administration (FDA) indication for small for gestational age (SGA) with failure to catch up as the patient's pre-treatment height is not greater than 2.0 standard deviations below mean for age and gender.
Therefore, the drug is not being requested for a medically accepted indication as supported by current medical literature and any applicable diagnostic/treatment guidelines.