Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Idiopathic short stature 
Treatment: Genotropin
The insurer denied Genotropin.
The determination is overturned.

This is a female patient diagnosed with idiopathic short stature (ISS). The request is for Genotropin. Her growth hormone (GH) stimulation test on  with glucagon and L-dopa peaked at 26 and 12 respectively. She had short stature homeobox (SHOX) gene testing and it was normal. Her Insulin-like Growth Factor 1 (IGF1) was 48-450. Her weight on was 5.876 kilograms (kg).
Her height on was 102.387 centimeters (cm) (-2.73 standard deviation score (SDS)) and 104.90 cm (-2.72 SDS) with a height velocity over 5 months of 5.7 centimeters per year (cm/yr)
Her mother's height is 157.48 cm and father's height is 175.26 cm and mid parental height is 159.87 cm (-0.54 SDS). At issue is the medical necessity of Genotropin

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 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 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 (yr) in children over 2 yrs 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 Insulin-like Growth Factor 1 (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).
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."[10-13]
The patient had a pretreatment height less than or equal to -2.25 SD. 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 request is consistent with generally accepted standards of medical practice and is therefore medically necessary.