Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Idiopathic short stature 
Treatment: Zomacton
The Zomacton is medically necessary, as described below.

The patient is a female diagnosed with ISS (idiopathic short stature). This request is for Zomacton. Her GH (growth hormone) stimulation test with arginine and clonidine peaked at 15.60. Her bone age on was eight years four months at CA (chronological age) ten years four months. At one point, it was six years ten months. 

The patient's height velocity is reported to be 3.42 cm/year (between 5.83 years old and 6.83 years old). Her height at 9.33 years old was 118.1 cm (-2.71 SDS), and at 10.75 years old was 123 cm (-2.82 SDS), with height velocity over one year four months of 3.5 cm/year. Her mother's height is 161.29 cm, and father's height is 160.02 cm, giving a mid-parental height of 154.155 cm (-1.41 SDS). Her IGF1 (insulin-like growth factor one) was 174 (49-451).

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

Classic GH 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 midparental 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 or IGFBP3 or GH stimulation testing, with a peak GH of less than 10ng/ml. 
		
The patient did not meet biochemical criteria. Growth hormone stimulation testing peaked at greater than ten. 

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 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 has a pretreatment height less than or equal to -2.25 SD. The height velocity is extremely poor. The patient therefore met idiopathic short stature criteria. 

The request meets nationally accepted criteria for use of growth hormone. The request is therefore medically necessary.