Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Endocrine/Metabolic/Nutritional - Short Stature
Treatment: Pharmacy/Prescription Drugs - Nutropin
The health plan denied the prescription drug Nutropin as not medically necessary for this patient. 
The reviewer has overturned in whole the health plan's determination.

This is a pediatric patient diagnosed with idiopathic short stature (ISS). This request is for Nutropin 1.2 milligrams (mg) daily. 

Classic growth hormone (GH) deficiency criteria are outlined in the consensus guidelines for the diagnosis and treatment of GH deficiency published by the GH research society, the Lawson Wilkins Pediatric Endocrinology Society, and the medical guidelines for GH use provided by the American Association of Clinical Endocrinologists. 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 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 more than 1 SD below the mean for chronological age, or a decrease in height SD of more than 0.5 over 1 year in children over 2 years of age; 4) in the absence of short stature, a height velocity more than 2 SD below the mean over 1 year or more than 1.5 SD sustained over 2 years. The biochemical criteria include low insulin-like growth factor 1 (IGF1) or insulin-like growth factor binding protein 3 (IGFBP3) or GH stimulation testing with a peak GH less than 10ng/ml (nanograms per milliliter).

The patient did not meet biochemical criteria. Growth hormone stimulation testing peaked at greater than 10.

GH is indicated for the treatment of GH deficiency in certain patients who do not fail GH stimulation tests based on the Lawson Wilkins Pediatric Endocrine Society alternate criteria, which state, "No gold standard exists for the diagnosis of growth hormone deficiency [GHD]. Although children severely affected by GHD fail GH stimulation tests, there is no doubt that some children with GHD achieve stimulated GH concentrations above the arbitrary cutoffs that have been applied. A trial of GH therapy should be approved for children with otherwise unexplained short stature who pass GH stimulation tests, but who meet most of the following criteria: (1) height greater than 2.25 SD below the mean for age or greater than 2 SD below the mid parental height percentile; (2) growth velocity less than 25th percentile for bone age;(3) bone age greater than 2 SD below the mean for age; (4) low serum insulin-like growth factor 1 (IGF-I) and/or insulin-like growth factor binding protein 3 (IGFBP3); and/or (5) other clinical features suggestive of GHD."

This patient met criterion 1 as pretreatment height was - 2.28 SD. IGF1 was low. Height velocity is less than the 25th percentile. The patient has a small pituitary on magnetic resonance imaging (MRI). The patient therefore met 4 of the above criteria. The patient therefore meets accepted criteria for the diagnosis of growth hormone deficiency.

Idiopathic Short stature: Growth hormone in the treatment of idiopathic short stature (ISS) is supported by randomized controlled trials and consensus guidelines. United States 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 does not meet idiopathic short stature criteria because the patient has growth hormone deficiency as described above.

The request is consistent with generally accepted standards of medical practice and is therefore medically necessary.