Appeal type: Medical necessity
Decision: Upheld

Diagnosis: Short Stature.
Treatment: Zomacton, Pre-service.
The insurer denied the Zomacton, Pre-service.
The denial is upheld. 

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

The patient's growth hormone (GH) stimulation test with arginine and clonidine peaked at 11. His insulin-like growth factor one (IGF1) was 166 (108-293) and insulin-like growth factor binding protein three (GFBP3) was 2.90. The patient's testes were four cubic centimeters bilaterally and pubic hair was at tanner-one. 

The patient's height was 128.80 centimeters (-1.97 standard deviations [SD]) and 10 months later was 131.80 centimeters (-2.07 SD), with height velocity over ten months of 3.6 centimeters per year. 

The patient's mother's height is 152.4 centimeters and father's height is 172.72 centimeters and mid parental height is 174.14 centimeters (-0.38 SD).

No, the proposed Zomacton is not medically necessary.

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

Classic Growth Hormone (GH) Deficiency

Classic growth hormone deficiency criteria are outlined in the consensus guidelines for the diagnosis and treatment of growth hormone deficiency published by the GH Research Society [1], the Lawson Wilkins Pediatric Endocrinology Society [2, 3], and the medical guidelines for growth hormone 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 height, height velocity, biochemical and sometimes radiological findings. The height and height velocity criteria based on consensus guidelines upon which a biochemical evaluation is to be initiated are 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 standard deviation; 3) height more than 2.0 standard deviations below the mean and a height velocity over one year more than 1.0 standard deviation below the mean for chronological age, or a decrease in height standard deviation 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. The biochemical criteria include low insulin-like growth factor one (IGF1) or insulin-like growth factor binding protein three (IGFBP3) or growth hormone stimulation testing with a peak growth hormone less than ten nanograms per milliliter.

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, 5, 6, 7, 8, 9].  Food and Drug Administration (FDA) approval for this indication is for "non-growth hormone-deficient short stature, defined by height SDS (standard deviation score) 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 does not have a pretreatment height less than or equal to -2.25 SDS. The patient therefore does not meet idiopathic short stature criteria. 

The request does not meet any nationally accepted criteria for use of growth hormone. The request is therefore not medically necessary.