Appeal type: Medical necessity
Decision: Overturned

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

The patient was diagnosed with idiopathic short stature (ISS). This request is for Nutropin. As reported, she started having breast development. She reached menarche. Her growth chart shows a growth spurt  Her height velocity is reported to be 5.9 centimeters per year (cm/yr). Her breasts and pubic hair were at Tanner 3. 
Her luteinizing hormone (LH) was 3.4; estradiol was 28.1. The follicle-stimulating hormone (FSH) was 4.78; LH was 6.68.
Her height was 135 centimeters (cm) (+0.72 standard deviation score (SDS)) and 138.7 cm (+0.88 SDS) with a height velocity over 0.57 years of 6.5 cm/yr.
Her mother's height is 158.75 cm and father's height is 168.91 cm and mid parental height 157.33 cm (-0.93 SDS).
At issue is the medical necessity of Nutropin AQ Nuspin 10.
The Nutropin AQ Nuspin 10 is medically necessary.
The peer reviewed literature including consensus clinical guidelines, randomized controlled trials are 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 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 1) severe short stature, defined as a height more than 3 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 Insulin-like Growth Factor 1 (IGF1) or Insulin-like growth factor binding protein 3 (IGFBP3) or growth hormone (GH) stimulation testing with a peak growth hormone (GH) less than 10 nanograms per milliliter (ng/ml).
The patient did not meet biochemical criteria. Growth hormone stimulation testing is not reported.
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 does not have a pretreatment height less than or equal to -2.25 SD. The patient therefore does not meet idiopathic short stature criteria.
However, there are studies in patients with precocious puberty and a poor height prediction, such as this patient, showing safety and efficacy of growth hormone and improved height prediction (10-12). The requested treatment is expected to be effective for this patient based on these studies. It is off label but has support in the literature cited. Therefore, the request is medically necessary.