Appeal type: Medical necessity
Decision: Overturned

Diagnosis:  Short Stature.
Treatment:  Nutropin AQ NuSpin (Pre-Service).

The insurer denied the Nutropin AQ NuSpin (Pre-Service).  The denial is overturned. 

The patient is a male diagnosed with idiopathic short stature (ISS).  This request is for Nutropin.  He was born appropriate for gestational age (AGA).  His growth hormone (GH) stimulation test with glucagon and L-dopa peaked at 17 and 16, respectively.  His height velocity is reported to be three to four centimeters per year (cm/yr).  His insulin like growth factor one (IGF1) was 88 (60-280); insulin like growth factor binding protein three (IGFBP3) was 2399.  The patient's testes were two cubic centimeter (cc) and pubic hair was at tanner 1.  

The patient's mother's height is 151 centimeters (cm) and father's height is 173 centimeters (cm) and mid-parental height is 168.5 centimeters (cm) (-1.16 standard deviations [SDS]).  

The patient's height the previous year was 103.99 centimeters (cm) (-2.29 standard deviations [SDS]) and earlier this year was 107.90 centimeters (cm) (-2.37 standard deviations [SDS]) with height velocity over 0.83 years of 4.7 centimeters per year (cm/yr), -1.5 standard deviations (SD). 

Yes, the proposed treatment with Nutropin AQ NuSpin is medically necessary.

The peer reviewed literature including consensus clinical guidelines, randomized controlled trials are summarized below:

Classic growth hormone (GH) deficiency:
Classic growth hormone (GH) deficiency criteria are outlined in the consensus guidelines for the diagnosis and treatment of growth hormone (GH) deficiency published by the Growth Hormone (GH) Research Society [1], the Lawson Wilkins Pediatric Endocrinology Society [2, 3], and the medical guidelines for growth hormone (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 3.0 standard deviations (SD) below the mean; 2) height more than 1.5 standard deviations (SD) below the mid parental height standard deviation (SD); 3) height more than 2.0 standard deviations (SD) below the mean and a height velocity over one year more than 1.0 standard deviations (SD) below the mean for chronological age, or a decrease in height standard deviations (SD) of more than 0.5 over one year in children over two years of age; or 4) in the absence of short stature, a height velocity more than 2.0 standard deviations (SD) below the mean over one year or more than 1.5 SD sustained over two years.  The biochemical criteria include low insulin-like growth factor one (IGF1) or insulin-like growth factor binding protein three (IGFBP3) or growth hormone (GH) stimulation testing with a peak growth hormone (GH) less than ten nanograms per milliliter (ng/mL).

The patient did not meet biochemical criteria.  Growth hormone stimulation testing peaked at greater than ten. 

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 deviations 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 had a pretreatment height less than or equal to -2.25 standard deviation (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 likely to be beneficial for the patient's health.  The request is therefore medically necessary.