Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Short Stature.
Treatment: Zomacton 10mg vial.

The insurer denied the Zomacton 10mg vial.  The denial is overturned.

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

The patient's GH (growth hormone) stimulation test peaked at 12.7.  Her breasts and pubic hair were at tanner 1. Her IGFBP3 (insulin-like growth factor binding protein 3) was 4300 (2456-6992). Her IGF1 (insulin-like growth factor 1) was 137 (76-549). Her Z-score was -0.9. 

The patient's height was 115.8 cm (centimeters) (-3.82 SDS [standard deviation score]). Almost a year later, it was 118.2 cm (centimeters) (-3.97 SDS [standard deviation score]). This was a height velocity over 11 months of 2.5 cm/yr (centimeters per year).

The patient's mother's height is 149.86 cm (centimeters). The father's height is 167.64 cm (centimeters). This provides for a mid-parental height of 152.25 cm (centimeters) (-1.71 SDS [standard deviation score]).

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 criteria are outlined in the consensus guidelines for the diagnosis and treatment of GH (growth hormone) deficiency published by the GH (growth hormone) research society (1), the Lawson Wilkins Pediatric Endocrinology Society (2, 3), and the medical guidelines for GH (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 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 (standard deviation) below the mean, 2) Height of more than 1.5 SD (standard deviation) below the midparental height, 3) Height of more than 2 SD (standard deviation) below the mean, and a height velocity over one year of more than 1 SD (standard deviation) below the mean for chronological age, or a decrease in height SD (standard deviation) of more than 0.5 over one year in children over two years of age, or 4) In the absence of short stature, a height velocity of more than 2 SD (standard deviation) below the mean over one year, or more than 1.5 SD (standard deviation) 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 (growth hormone) stimulation testing, with a peak GH (growth hormone) of less than 10 ng/ml (nanograms per milliliter).

The patient did not meet biochemical criteria. Growth hormone stimulation testing peaked at greater than 10.

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 (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." (10)

The patient had a pretreatment height less than or equal to -2.25 SD (standard deviations). The patient's height velocity and bone age would not allow the patient to reach a normal adult height. The request therefore meets idiopathic short stature criteria.

Therefore, given the above, the request is consistent with generally accepted standards of medical practice. The request is likely to be health beneficial for the patient. The request is therefore medically necessary.