Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Short Stature.
Treatment: Nutropin AQ Inj.
The insurer denied the Nutropin AQ Inj.
The denial is overturned.

The patient is a female diagnosed with short stature. This request is for Nutropin. As reported; she has a medical history significant for Juvenile Idiopathic arthritis (JIA); JIA (Juvenile Idiopathic arthritis) symptoms were initially noted when she was a toddler. She failed a trial off of Humira approximately four years ago and has been treated with Humira since.

The patient's GH (growth hormone) stimulation test peaked at 19 and 14 respectively. She was started on treatment with Nutropin.  She is on Nutropin 1.1 mg (milligrams) daily (0.28 mg (milligrams) x kg (kilograms) x week). Her height velocity on therapy is reported to be 9.7 centimeters per year.

The patient's weight was 27.4 kg (kilograms). Her IGF1 (insulin-like growth factor 1) was 32. Two months later her IGF1 (insulin-like growth factor 1) was 24; Z-score was - (minus) 2.33 SD (standard deviation). A little over a year later, her IGF1 (insulin-like growth factor 1) was 56 (80-307); IGFBP3 (insulin-like growth factor binding protein 3) was 3.4 (1.6-6.5).

The patient's pretreatment height was 113 cm (centimeters) (-2.84 SDS [standard deviation score]) and was 115 cm (centimeters) (-3.10 SDS [standard deviation score]) with height velocity over 0.8 years of 2.5 centimeters per year. Her height on therapy was 132.5 cm (centimeters) (-1.43 SDS [standard deviation score]).

The patient's mother's height is 162.56 cm (centimeters), and father's height is 162.56 cm (centimeters). The mid-parental height is 156.06 cm (centimeters) (-1.12 SDS [standard deviation score]).

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
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.

Alternate GH (growth hormone) deficiency (GHD) criteria
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 -3.1. 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 is growth hormone deficient. Growth hormone in the treatment of growth hormone deficiency 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, or in the best interest of the patient, as the request is medically necessary as described above.