Appeal type: Medical necessity
Decision: Upheld

Diagnosis: Idiopathic short stature.
Treatment: Omnitrope (pre-service).
The insurer denied the Omnitrope (pre-service).
The denial is upheld. 

This is a male diagnosed with idiopathic short stature (ISS). This request is for Omnitrope. As reported; the patient is on growth hormone (GH) therapy with a good result and Omnitrope is covering the cost. His GH (growth hormone) stimulation test peaked at 12.20. His pretreatment height velocity is reported to four centimeters per year (cm/yr). His insulin-like growth factor one (IGF1) was 200 (23-386); Z-score was +0.6 standard deviations (SD); insulin-like growth factor binding protein three (IGFBP3) was 5390 (1932-5858). The patient's testes were 2.5 cubic centimeters (cc) bilaterally with tanner-1. His pretreatment height z-score is reported to be -2.02 SD. 

The patient's mother's height is 158.75 centimeters and father's height is 180.34 centimeters and mid parental height is 176.045 centimeters (-0.11 standard deviations [SDS]). 

The patient's height on therapy was 136.4 centimeters (cm) (-1.11 SDS).  His pretreatment height was 110 centimeters (+0.18 SDS) and was 115 cm (-1.03 SDS) with height velocity over 1.75 years of 2.9 centimeters per year.

No, the proposed treatment with Omnitrope is not medically necessary..

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

Classic GH (growth hormone) deficiency:
Classic GH (growth hormone) deficiency criteria are outlined in the consensus guidelines for the diagnosis and treatment of GH (growth hormone) deficiency published by the GH (growth hormone) Research Society [1], the Lawson Wilkins Pediatric Endocrinology Society [2], [3], and the medical guidelines for GH (growth hormone) 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 SD (standard deviation) below the mid parental height SD (standard deviation); 3) height more than 2.0 SD (standard deviation) below the mean and a height velocity over one year more than 1.0 SD (standard deviation) below the mean for chronological age, or a decrease in height SD (standard deviation) 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 SD (standard deviation) below the mean over one year or more than 1.5 SD (standard deviation) 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 GH (growth hormone) stimulation testing with a peak GH (growth hormone) less than ten nanograms per milliliter (ng/mL).

In this case, 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 SDS (standard deviation score) 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."

In this case, the patient does not have a pretreatment height less than or equal to -2.25 SD (standard deviation). The patient therefore does not meet idiopathic short stature criteria. 

The request does not meet any nationally accepted criteria for use of growth hormone. The request is therefore not medically necessary.