Appeal type: Medical necessity
Decision: Upheld

Diagnosis: Idiopathic short stature.
Treatment: Nutropin.
The insurer denied Nutropin.
The determination is upheld.

This is a patient with idiopathic short stature. The letter of medical necessity reports that the patient had an extensive evaluation including magnetic resonance imaging (MRI) of the pituitary, growth hormone stimulation testing, all of which were unrevealing. He had normal genetic testing for short stature homeobox-containing gene (SHOX), karyotype and other genetic testing as well as a MicroArray. His height standard deviation (SD) was -1.3 and his height was at -1.23 SD. His bone age was reported as 13  with a height prediction of 69.6 inches. He had some growth deceleration when he was started on stimulant medication. His height was at the 10th percentile at 155 centimeters (cm). The insulin growth factor-1 (IGF-1) was 218 (range 64-508). Growth at most in relation to testing with arginine and clonidine peaked at 14.6. MRI of the brain was unremarkable. At issue is the medical necessity of Nutropin.

No. The Nutropin is not medically necessary.
The peer reviewed literature including consensus clinical guidelines, randomized controlled trials are summarized below:
Classic growth hormone (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 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 standard deviations (SD) below the mean; 2) height more than 1.5 SD below the mid parental height SD; 3) height more than 2 SD below the mean and a height velocity over 1 year (yr) more than 1 SD below the mean for chronological age, or a decrease in height SD of more than 0.5 over 1 yr in children over 2 yr of age; 4) in the absence of short stature, a height velocity more than 2 SD below the mean over 1 yr or more than 1.5 SD sustained over 2 yr. The biochemical criteria include low IGF-1 or Insulin-like growth factor binding protein 3 (IGFBP3) or GH stimulation testing with a peak GH less than 10 nanograms per 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, [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 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.