Appeal type: Medical necessity
Decision: Upheld

Diagnosis: Idiopathic short stature
Treatment: Nutropin AQ Nuspin 10

The insurer denied coverage for Nutropin AQ Nuspin 10.
The denial is upheld. 

This patient is a male child diagnosed with idiopathic short stature. This request is for Nutropin. The patient's growth hormone (GH) stimulation test with clonidine and arginine peaked at 20.30 and 17.40, respectively. His insulin-like growth factor binding protein 3 (IGFBP-3) was 6720; insulin-like growth factor 1 (IGF-1) was Z-score +1.8 standard deviation (SD). The patient's bone age was below his chronological age. The patient's height velocity is reported to be 6.9 centimeters (cm) per year. The patient's predicted adult height based on the latest bone age was 174.4 cm, -0.34 SD. The patient's mother's height is 166.37 cm and father's height is 182.88 cm and mid parental height is 181.125 cm (+0.60 SDS). 

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 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 standard deviation (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 a certain 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 10 nanograms per milliliter (ng/mL). In this case, the patient did not meet the biochemical criteria for GH deficiency. 

Growth hormone in the treatment of idiopathic short stature is supported by randomized controlled trials and consensus guidelines. Food and Drug Administration (FDA) approval for this indication is for "non-growth hormone-deficient short stature, defined by height [standard deviation score (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." In this case, the patient has a pretreatment height less than or equal to -2.25 standard deviation (SD). The patient's height velocity and bone age predicted a normal adult height. The patient therefore, does not meet the idiopathic short stature criteria. 

Based on the review of the medical record and literature, the request does not meet any nationally accepted criteria for use of growth hormone. The coverage for Nutropin AQ, growth hormone therapy is therefore, not medically necessary for this patient.

The carrier's denial of coverage for Nutropin AQ Nuspin 10 for idiopathic short stature (ISS) should be upheld. The medical necessity is not substantiated.