Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Idiopathic Short Stature.
Treatment: Nutropin AQ Nuspin.
The insurer denied the Nutropin AQ Nuspin.
The determination is overturned.

The patient was diagnosed with idiopathic short stature (ISS). He had started growth hormone (GH) therapy and has showed excellent response to GH. He is on Nutropin and doing well. His GH stimulation test with clonidine and L-dopa peaked at 19.1. His height velocity is reported to be improved from less than (<) 3% to 9.4 centimeters per year [cm/yr]. His testes were 8-10 cubic centimeters (cc). His Insulin-like growth factor binding protein 3 (IGFBP3) was 3360 (range 1828-6592) ; insulin like growth factor 1 (IGF1) was 114.
His height on therapy was 144 cm (-1.51 standard deviation score (SDS))
His mother's height is 149.86 cm, his father's height is 170.18 cm and mid parental height is 166.52 cm (-1.44 SDS) His pretreatment height  was 124 cm (-2.15 SDS) and was 127 cm (-2.3 SDS) with height velocity over one year of 3 cm/year.
At issue is the medical necessity of Nutropin AQ Nuspin.
The Nutropin AQ Nuspine is medically necessary.
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 standard deviation(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 year (yr) more than 1 SD below the mean for chronological age, or a decrease in height SD of more than 0.5 over 1 yr in children over 2 yr 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 IGF1 or 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], [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 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 pretreatment height velocity and bone age would not allow the patient to reach a normal adult height. The patient met accepted criteria for idiopathic short stature. The patient was started on growth hormone for idiopathic short stature. The has benefited from growth hormone with an improvement in height velocity. Continuation of growth hormone is necessary is medically necessary. The request is consistent with generally accepted standards of medical practice and is therefore medically necessary.