Appeal type: Medical necessity
Decision: Overturned

Diagnosis: ISS (idiopathic short stature).
Treatment: Zomacton Injection 10MG.

The insurer denied the Zomacton Injection 10MG. The denial is overturned. 

The patient is a female diagnosed with ISS (idiopathic short stature). This request is for Zomacton. 

The patient's menarche was earlier this year. Her IGF1 (insulin-like growth factor 1) was 359; IGFBP3 (insulin-like growth factor binding protein 3) was 4837. 

The patient's mother's height is 157.48 cm (centimeters), and her father's height is 165.1 cm. Her so mid-parental height is 154.79 cm (-1.32 SDS [standard deviation score]).

The patient's height was 142.5 cm (-2.32 SDS). 
Her predicted adult height is to be 148 cm, -2.36 SD (standard deviations).

Yes, the treatment with Zomacton is medically necessary.

The peer-reviewed literature, including consensus clinical guidelines and randomized controlled trials, are summarized below:

Classic GH (growth hormone) deficiency
Classic GH deficiency criteria are outlined in the consensus guidelines for the diagnosis and treatment of GH deficiency published by the GH research society (1), the Lawson Wilkins Pediatric Endocrinology Society (2, 3), and the medical guidelines for GH use provided by the American Association of Clinical Endocrinologists (4). The above-referenced guidelines are the latest available guidelines from these respective societies. The diagnosis is based on auxological, biochemical and sometimes radiological findings. 

The auxological criteria based on consensus guidelines upon which a biochemical evaluation is to be initiated are 
1)	Severe short stature, defined as a height of more than 3 SD below the mean; 
2)	Height of more than 1.5 SD below the mid-parental height; 
3)	Height of more than 2 SD below the mean, and a height velocity over one year of more than 1 SD below the mean for chronological age, or a decrease in height SD 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 of more than 2 SD below the mean over one year, or more than 1.5 SD sustained over two years. 

The biochemical criteria include low IGF1 (insulin-like growth factor 1) or IGFBP3 (insulin-like growth factor binding protein 3) or GH stimulation testing, with a peak GH of less than 10 ng/ml (nanograms per milliliter).		

The patient did not meet biochemical criteria. Growth hormone stimulation testing is not reported. IGF1 was normal.

Idiopathic Short stature
Growth hormone in the treatment of idiopathic short stature (ISS) is supported by randomized controlled trials and consensus guidelines (4-9). FDA (Food and Drug Administration) approval for this indication is for "non-growth hormone-deficient short stature, defined by height SDS less than or equal to -2.25, and associated with growth rates unlikely to permit attainment of adult height in the normal range, in pediatric patients whose epiphyses are not closed and for whom diagnostic evaluation excludes other causes associated with short stature that should be observed or treated by other means."

The patient had a pretreatment height more than 2.25 SD below the mean. The patient's height prediction is below the normal range. The request meets accepted criteria for the diagnosis of idiopathic short stature. The request is consistent with generally accepted standards of medical practice and is therefore medically necessary.