Appeal type: Medical necessity
Decision: Upheld

Endocrine/Metabolic/Nutritional
Pharmacy/Prescription Drug
Diagnosis: Short Stature
Treatment:  Humatrope 36 Unit Cartridge

The patient was diagnosed with skeletal dysplasia. This request is for Humatrope. The patient had genetic testing that was positive for skeletal dysplasia. Her height velocity is reported to be 4.52 centimeters per year (cm/yr). Her Insulin-like Growth Factor 1 (IGF1) was 206 (range 121-541). Additional heights were 114.7 cm (-3.92 standard deviation score (SDS)) and  116 cm (-3.79 SDS) with height velocity over 3 months of 5.6 cm/yr. Her father's height was 155.2 cm. At issue is the medical necessity of Humatrope.
It is not medically necessary. 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 growth hormone (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 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 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 yr or more than 1.5 SD sustained over 2 years. The biochemical criteria include low IGF-1 or Insulin-like growth factor binding protein-3 (IGFBP-3) or GH stimulation testing with a peak GH less than 10ng/ml. The patient did not meet biochemical criteria. Growth hormone stimulation testing is not reported and the IGF-1 is normal.
Idiopathic Short stature
Growth hormone in the treatment of idiopathic short stature (ISS) is supported by randomized controlled trials and consensus guidelines [4-9]. The Food and Drug Administration (FDA) approval for this indication is for "non-growth hormone-deficient short stature, defined by height 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 had a pretreatment height less than or equal to -2.25 SD. She has skeletal dysplasia. Idiopathic short stature requires that other causes have been ruled out. The patient therefore does not meet idiopathic short stature criteria. There are no studies showing safety or efficacy of growth hormone in patients with skeletal dysplasia.
The request does not meet any nationally accepted criteria for the use of growth hormone.