Appeal type: Medical necessity
Decision: Upheld

Diagnosis: Idiopathic Short Stature
Treatment: Nutropin AQ Nuspin 20
The insurer denied Nutropin AQ Nuspin 20.
The denial was upheld.  

This is a male patient with idiopathic short stature (ISS).  This request is for Nutropin.  The patient has started on a trial of growth hormone (GH) in early 2020 with an excellent response in his growth velocity from 3.3 cm/year to 8.07 cm/year on four months of growth hormone (GH) therapy.  The patient's GH stimulation test in mid-2019 with glucagon and L-dopa peaked at 20 and 17 respectively.  The patient's bone age around that time was at chronological.  The patient's IGFBP3 was 3806 and IGF1 was 169.  In mid-2020, the patient's testes were descended 3 cc bilaterally and his pubic hair was at tanner 3.  The patient's height on therapy was 139.09 cm (-1.20 SDS [standard deviations]). The patient's pretreatment height was 128.80 cm (-1.32 SDS) and several months prior to that it was 136.398 cm (-1.34 SDS) with height velocity over 1 year 9months of 4.3 cm/year.  

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 [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 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; 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 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 GH deficiency, and his GH 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, [5, [6, [7, [8, [9].  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." In this case, the patient does not have a pretreatment height less than or equal to -2.25 SD.  The patient therefore, does not meet the idiopathic short stature criteria. 

Based on the review of the medical record and literature, this patient did not meet any nationally accepted criteria for use of growth hormone. The patient's condition neither met growth hormone (GH) deficiency nod idiopathic short stature (ISS). Thus, Nutropin AQ Nuspin 20 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 Nutropin AQ Nuspin 20 should be upheld.  The medical necessity is not substantiated.