Appeal type: Medical necessity
Decision: Upheld

Diagnosis: Idiopathic short stature (ISS).
Treatment: Nutropin AQ Nuspin.

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

This patient is a male child diagnosed with idiopathic short stature (ISS).  The patient's growth hormone (GH) stimulation test with glucagon and L-dopa peaked at 11 and 6.2 respectively.  The patient's bone age was noted to be different from a specified older chronological age (CA).  The patient's insulin-like growth factor (IGF)-1 was 359; and his IGF-binding protein-3 was 4374. The patient's mother's height was 160.02 cm and his father's height was 170.18 cm; so, his mid-parental height was 171.6 cm (-0.73 SDS).  The patient's height at a specified CA was 139 cm (-1.82 standard deviations (SDS)) and a specified age was 148.79 cm (-2.16 SDS) with height velocity over 2 years of 4.9 cm/year. 

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 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 more than 3 SDS below the mean, 2) height more than 1.5 SD below the mid-parental height, 3) height more than 2 SDS below the mean and a height velocity over 1 year more than 1 SDS below the mean for chronological age, or a decrease in height SDS of more than 0.5 over 1 year in children over 2 years of age, or 4) in the absence of short stature, a height velocity more than 2 SDS below the mean over 1 year or more than 1.5 SDS sustained over 2 years.  The biochemical criteria include low IGF1 or IGFBP3 or GH stimulation testing with a peak GH less than 10ng/ml.  In this case, the patient did not meet the biochemical criteria for growth hormone deficiency.  This patient's 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).  FDA approval for this indication is for "non-growth hormone-deficient short stature, defined by height SDSS 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 did not have a pre-treatment height less than or equal to -2.25 SDS.  Thus, the patient did not meet the clinical criteria for idiopathic short stature diagnosis. 

Based on the review of the medical record, this patient's clinical presentation failed to meet the criteria for either growth hormone deficiency or idiopathic short status diagnosis.  Thus, the treatment with growth hormone therapy is not medically necessary for this patient. 

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

The carrier's denial of coverage for the medication Nutropin AQ Nuspin for idiopathic short stature should be upheld.  The medical necessity is not substantiated.