Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Idiopathic Short Stature.
Treatment: Nutropin AQ Nuspin 10 Injector.
The insurer denied the Nutropin AQ Nuspin 10 Injector.
The denial is overturned.

The patient is a male diagnosed with short stature and growth failure. This request is for Nutropin.

The patient's GH (growth hormone) stimulation test with arginine and clonidine peaked at 15.60. His IGF1 (insulin-like growth factor 1) was 147 (168-576); Z-score was - (minus) 2.1 SD (standard deviation). His height velocity is reported to be 3.2 centimeters per year.

His weight was 35.5 kg (kilograms).

The patient's height was 142.8 centimeters (- [minus] 2.16 SDS [standard deviation score]) was 144.4 centimeters (- [minus] 2.42 SDS [standard deviation score]) with height velocity over 7 months of 2.6 centimeters per year.

Yes, the requested Nutropin is medically necessary.
The peer-reviewed literature, including consensus clinical guidelines and randomized controlled trials, are summarized below:

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 auxological, biochemical and sometimes radiological findings.

The auxological criteria based on consensus guidelines upon which a biochemical evaluation is to be initiated are
1)	Severe short stature, defined as a height of more than 3 SD (standard deviation) below the mean;
2)	Height of more than 1.5 SD (standard deviation) below the midparental height;
3)	Height of more than 2 SD (standard deviation) below the mean, and a height velocity over one year of more than 1 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 of more than 2 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 IGF1 (insulin-like growth factor 1) or IGFBP3 (insulin-like growth factor binding protein 3) or GH (growth hormone) stimulation testing, with a peak GH (growth hormone) of less than 10 ng/ml (nanograms per milliliter).

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

In addition, GH (growth hormone) is indicated for the treatment of GH (growth hormone) deficiency in certain patients who do not fail GH (growth hormone) stimulation tests, based on the Lawson Wilkins Pediatric Endocrine Society (LWPES) alternate criteria (2), which state, "No gold standard exists for the diagnosis of GH [growth hormone deficiency]. Although children severely affected by GH [growth hormone deficiency] fail GH [growth hormone] stimulation tests, there is no doubt that some children with GH [growth hormone deficiency] achieve stimulated GH [growth hormone] concentrations above the arbitrary cutoffs that have been applied. A trial of GH [growth hormone] therapy should be approved for children with otherwise unexplained short stature who pass GH [growth hormone] stimulation tests, but who meet most of the following criteria:
1) Height greater than 2.25 SD [standard deviation] below the mean for age or greater than 2 SD (standard deviation) below the midparental height percentile;
2) Growth velocity less than 25th percentile for bone age;
3) Bone age greater than 2 SD [standard deviation] 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 GH (growth hormone deficiency)."

This patient met criterion 1, as pretreatment height was - [minus] 2.42 SD (standard deviation). IGF1 (insulin-like growth factor 1) was low. Bone age was delayed. Height velocity is less than the 25th percentile. The patient therefore met four of the above criteria. The patient meets criteria for the diagnosis of growth hormone deficiency. Growth hormone in the treatment of growth hormone deficiency is expected to be health beneficial and is medically necessary.

Idiopathic Short stature
Growth hormone in the treatment of idiopathic short stature (ISS) is supported by randomized controlled trials and consensus guidelines (4-9). FDA (Food and Drug Administration) approval for this indication is for "non-growth hormone-deficient short stature, defined by height SDS (standard deviation score) less than or equal to - [minus] 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 - [minus] 2.25 SD (standard deviations). The patient's height velocity and bone age would not allow the patient to reach a normal adult height. The patient has growth hormone deficiency as described above. Short stature is a diagnosis of exclusion. The patient does not meet idiopathic short stature criteria because the patient has a diagnosis.

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

No, the health plan did not act reasonably, with sound medical judgment, and in the best interest of the patient.