Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Short Stature
Treatment: Norditropin
The health plan denied: Norditropin
The determination is: Overturned

The patient is a female diagnosed with idiopathic short stature (ISS). As reported; she started growth hormone (GH) therapy and stopped within 3 months. She has anorexia nervosa and has lost 4.6 pounds, but her most recent weights are normal. She is on Norditropin 0.8 milligrams (mg) daily. Her GH stimulation test with arginine and clonidine peaked at 12.5. Her height velocity is reported to be 4.8 centimeters per year (cm/yr). Her breasts were at Tanner 1-2 on the right and Tanner 1 on the left; the pubic hair was scored at Tanner 2. 

The patient's magnetic resonance imaging (MRI) was normal. Her weight was 39.10 kilograms (kg). Her height off therapy was 141.3 centimeters (cm) (-2.84 standard deviation score [SDS]). Her mid parental height is 166.37 cm (+0.47 SDS).

At issue is the medical necessity of Norditropin 1.7 mg once daily.

The health plan's determination of medical necessity is overturned in whole.

The requested health service/treatment of Norditropin is medically necessary for this patient.

The peer reviewed literature including consensus clinical guidelines and randomized controlled trials are summarized below.
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:

 	Severe short stature, defined as a height more than 3 standard deviations (SD) below the mean;

 	Height more than 1.5 SD below the mid parental height SD;

 	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;

 	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 10.

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

Growth hormone in the treatment of idiopathic short stature (ISS) is supported by randomized controlled trials and consensus guidelines [4-9]. 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." [10-13]

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.