Appeal type: Medical necessity
Decision: Upheld

Diagnosis: Growth hormone deficiency

Treatment: Nutropin AQ Nuspin 20

The insurer denied coverage for growth hormone therapy with Nutropin AQ Nuspin 20

The denial is upheld. 

This patient is a male child with a diagnosis of growth hormone deficiency (GHD). This request is for Nutropin. The patient's GH stimulation test with clonidine and arginine peaked at 10.10. The patient's growth hormone (GH) stimulation test with clonidine and arginine peaked at 7.19 and 0.51, respectively. The patient's bone age was 10-years old at a chronological age (CA) of 10-years old. The patient's bone age was 6-years old at CA of 6-years and 4-months old. The patient had magnetic resonance imaging (MRI) done that showed a tiny cyst measuring 8x3x5 millimeters. The patient's insulin-like growth factor 1 (IGF1) was 71.  5 months prior to that, his IGF1 was 129. Prior to that, his IGF1 was 96.6 and his insulin-like growth factor-binding protein (IGFBP3) was 3. The patient's testes were noted to be 3 cubic centimeters (cc) with Tanner 1. The patient's height at 8.16-years old was 123.30 centimeters (cm), which is -0.92 standard deviations (SD), and at 10.16-years old, was 131.50 cm (-1.18 SD) with height velocity over 2 years of 4.1 cm per year. The patent's mother's height was 161.29 cm and his father's height was 177.8 cm, which gave this patient's mid-parental height of 176.045 cm (-0.11 SD). The patient's height velocity over 1 year was 4.9 cm per year (-0.5 SD).

Classic 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 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 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 year of 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 yr. The biochemical criteria include low IGF1 or IGFBP3 or GH stimulation testing with a peak GH less than 10 nanograms per milliliter (ng/mL). 

This patient did not meet height and height velocity criteria as pretreatment height and height velocity are normal. Height was 1.1 SD below the mean. Height velocity was normal at -0.5 SD. GH stimulation testing peaked at less than 10 ng/mL. The pediatric endocrine society recommends against making a diagnosis of GH deficiency based solely on failed GH stimulation testing.

GH in the treatment of idiopathic short stature (ISS) is supported by randomized controlled trials and consensus guidelines. The 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."

This patient does not have a pretreatment height less than or equal to -2.25 SD; therefore, does not meet idiopathic short stature criteria. 

The request does not meet any nationally accepted criteria for use of growth hormone. Thus, the request for GH therapy is not medically necessary.

The health plan acted reasonably with sound medical judgment, and in the best interest of the patient.

Based on the above, the medical necessity for Nutropin AQ Nuspin 20 is not substantiated.  The insurer's denial should be upheld.