Appeal type: Medical necessity
Decision: Upheld

Diagnosis: Endocrine/Metabolic/Nutritional
Treatment: Pharmacy/Prescription Drugs
The insurer denied: growth hormone (Humatrope)
The denial is: Upheld in whole.

The patient is a male diagnosed with idiopathic short stature (ISS). This request is for Humatrope. He has Hirschsprung's disease. He started growth hormone (GH) therapy. His GH stimulation test with Levo (L)-dopa and Glucagon peaked at 12 and 13 respectively. His pretreatment height velocity is reported to be 4.7 centimeters per year (cm/yr.) The Insulin-like Growth Factor 1 (IGF1) was 115 (normal); Insulin-like growth factor binding protein 3 (IGFBP3) was 3.74 (normal). 
His mother's height is 162.56 centimeter (cm) and father's height is 182.88 cm and so mid parental height is 179.22 cm (0.33 standard deviation score [SDS]). His pretreatment height was 105.51 cm (-2.19 SDS) with height velocity over 11 months of 6.2 centimeters per year (cm/yr.)

The health plan's denial is upheld in whole.

The peer reviewed literature including consensus clinical guidelines, 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 more than 3 SD below the mean; 2) height more than 1.5 standard deviation (SD) below the mid parental height; 3) height more than 2 SD below the mean and a height velocity over 1 yr. more than 1 SD below the mean for chronological age, or a decrease in height SD of more than 0.5 over 1 year in children over 2 years of age; 4) in the absence of short stature, a height velocity more than 2 SD below the mean over 1 year or more than 1.5 SD sustained over 2 years. The biochemical criteria include low IGF1 or IGFBP3 or GH stimulation testing with a peak GH less than 10 nanograms/milliliter (ng/ml).
The patient did not meet biochemical criteria. Growth hormone stimulation testing peaked at greater than 10.
Idiopathic Short stature: Growth hormone in the treatment of idiopathic short stature (ISS) is supported by randomized controlled trials and consensus guidelines (4-9). Food and Drug Administration (FDA) approval for this indication is for "non-growth hormone-deficient short stature, defined by height standard deviation score (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 does not have a pretreatment height less than or equal to -2.25 SD. 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.