Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Idiopathic short stature
Treatment: Humatrope 72-unit cartridge, quantity of two per 28 days
The insurer denied the Humatrope 72-unit cartridge, quantity of two per 28 days. 
The denial is overturned. 

This is an male diagnosed with idiopathic short stature (ISS). This request is for Humatrope. As reported, the patient was started on Omnitrope; which he is currently receiving through manufacturer assistance; he is responding very well; his height is improved from -2.74 to -2.24 standard deviations (SDS). He started growth hormone (GH) therapy; according to the visit note.  His GH stimulation test peaked at 12.3. His insulin-like growth factor one (IGF1) was 141. 

Yes, the proposed treatment is medically necessary, as described below.

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 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 3.0 SD below the mean; 2) height more than 1.5 SD below the mid parental height SD; 3) height more than 2.0 SD below the mean and a height velocity over one year more than 1.0 SD below the mean for chronological age, or a decrease in height 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 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 GH stimulation testing with a peak 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]. 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."

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.