Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Idiopathic short stature
Treatment: Humatrope
The insurer denied the Humatrope.
The denial is overturned.

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

The patient was treated on GH (growth hormone). He completed the course of Testosterone a few years ago. His GH (growth hormone) stimulation test with arginine and clonidine peaked at 17.8. His GH (growth hormone) stimulation test with arginine and clonidine peaked at 11.06.

The patient's IGF1 (insulin-like growth factor-1) was 298; Z-score was -0.73 SD (standard deviations). His testes are 12 ml (milliliters) bilaterally and pubic hair is at tanner 4. His height velocity on therapy is reported to be 11.12 cm/yr (centimeters per year). A year later his testes are 6 ml (milliliters) at right and 8 ml (milliliters) at left, and pubic hair is at tanner 3.

The patient's mother's height is 167.64 cm (centimeters). His father's height is 175.26 cm (centimeter). His mid-parental height is 177.95 cm (centimeters) (+0.15 SDS [standard deviation score]).

The patient's height on therapy was 158.8 cm (centimeters) (-2.01 SDS).

The patient's pretreatment height was 139.5 cm (centimeters) (-2.86 SDS [standard deviation score]) and a little over a year later was 145.01 cm (centimeters) (-2.89 SDS [standard deviation score]) with height velocity over one year of 5.3 cm/yr (centimeters per year).

Yes, the treatment with Humatrope 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 (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;
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.

Idiopathic Short stature

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 (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 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. The patient is a continuing therapy patient who has benefited from growth hormone. The request is consistent with generally accepted standards of medical practice and is medically necessary.