Appeal type: Medical necessity
Decision: Upheld

Diagnosis: Endocrine/Metabolic/Nutritional/ Idiopathic Short Stature. 
Treatment: Pharmacy/Prescription Drugs/Genotropin. 

The health plan denied the requested prescription medication, Genotropin as not medically necessary. 
The health plan's determination is upheld. 

This is an adolescent male diagnosed with short stature. This request is for Genotropin 1.9 milligrams (mg) daily. As reported, he is on growth hormone (GH) therapy; he is taking Genotropin 1.7 mg daily (for 6 months as per visit note.) His GH stimulation test with Arginine and Clonidine peaked at 20.1. His magnetic resonance imaging (MRI) was normal. His height velocity is 4.4 centimeters/year (cm/year). His testes were 8-10 milliliters (ml) on the left and 6 ml on the right and pubic hair was at tanner 1. His pretreatment Insulin-like Growth Factor 1 (IGF1) was 185. His testes were 5-6 milliters (mls). 

 His pretreatment height was 142.24 centimeters (cm) (-0.96 standard deviation score (SDS)) and was 144.4 cm (-1.68 SDS) with height velocity over 1.19 year of 1.8 cm. His height on therapy was 162.56 cm (-0.48 SDS). His mid parental height is 176.022 cm (-0.12 SDS). 

The health plan's determination of medical necessity is upheld, in whole. 

No, the requested health service/treatment of Genotropin is not medically necessary for this patient. 

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 deviation (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 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 Insulin-like growth factor binding protein 3 (IGFBP3) or GH stimulation testing with a peak GH less than 10 nanograms/milliliter (ng/ml). 

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 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."[10-13] 

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 patient appears to have prepubertal slowing associated with constitutional delay of growth and puberty. Observation is appropriate for this patient. 
The request does not meet any nationally accepted criteria for use of growth hormone. The request is therefore not medically necessary.