Appeal type: Medical necessity
Decision: Upheld

Diagnosis:  Idiopathic Short Stature.
Treatment:  Humatrope.

The insurer denied the Humatrope.  The denial is upheld. 

The patient is a male diagnosed with idiopathic short stature (ISS).  This request is for Humatrope. 

The patient started growth hormone (GH) therapy.  His growth hormone (GH) stimulation test peaked at 10.1.  His growth hormone (GH) stimulation test with clonidine peaked at 17.1.  His magnetic resonance image (MRI) was normal.  Approximately two years prior to treatment, the patient's testes were 2.5 cubic centimeters (cc) bilaterally, and pubic hair was at tanner 2.  Approximately one year prior to treatment, his testes were three to four cc bilaterally with tanner 1-2, and pubic hair was at tanner 2.  Approximately two months after the start of treatment his testes were 8 cc bilaterally with tanner 3, as well as pubic hair.  He has normal insulin-like growth factor one (IGF1) and insulin-like growth factor binding protein three (IGFBP3) as reported. 

The patient's pretreatment height was 129.1 centimeters (cm) (-1.98 standard deviations [SDS]) and was 134.4 cm (-1.69 standard deviations [SDS]) with height velocity over 9 months of 6.9 centimeters per year. 

The patient's height on therapy was 140.792 cm (-1.10 standard deviations [SDS]).  
His mother's height is 157.48 cm and father's height is 175.26 cm; therefore, mid-parental height is 172.87 cm (-0.56 standard deviations [SDS]). 

No, the proposed treatment with Humatrope is not 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 deviation (SD) below the mid-parental height standard deviation (SD); 3)
height more than 2.0 standard deviation (SD) below the mean and a height velocity
over one year more than 1.0 standard deviation (SD) below the mean for
chronological age, or a decrease in height standard deviation (SD) of more than 0.5
over one year in children over two years of age; 4) in the absence of short stature, a
height velocity more than 2.0 standard deviation (SD) below the mean over one
year or more than 1.5 standard deviation (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 10 nanograms per milliliter
(ng/ml).

This patient did not meet biochemical criteria.  His growth hormone stimulation
testing peaked at greater than ten.

Idiopathic short stature:
Growth hormone (GH) 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 (SD) 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."

This patient does not have a pretreatment height less than or equal to -2.25
standard deviation (SD).  Therefore, the patient does not meet idiopathic short
stature criteria.  Furthermore, the patient has a normal height prediction.  The request does not meet any nationally accepted criteria for use of growth
hormone.  Therefore, the request is not medically necessary.