Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Idiopathic Short Stature
Treatment: Genotropin

The insurer denied the Genotropin.
The determination is overturned.

The patient was diagnosed with idiopathic short stature (ISS). This request is for Genotropin.
His Insulin-like Growth Factor 1 (IGF1) was 462. His height velocity is reported to be 4.91 centimeters per year (cm/yr). His testes were 6 cubic centimeters (cc) bilaterally and pubic hair was at Tanner 2. His IGF1 was 159; z-score was -1.6; Insulin-like growth factor binding protein 3 (IGFBP3) was 5.
His height 139.5 centimeter (cm) (-2.17 standard deviation score (SDS)) and then 147.3 cm (-2.49 SDS) with height velocity over 1y7m of 4.9 centimeters per year (cm/yr). His mid-parental height is 172 cm (-0.68 SDS). At issue is the medical necessity of Genotropin.

Yes. The Genotropin is medically necessary. The peer-reviewed literature including consensus clinical guidelines and randomized controlled trials is 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 growth hormone (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, and 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 deviations (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 is not reported and IGF-I is normal.

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 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.