Diagnosis: Idiopathic Short Stature (ISS)

Treatment: Nutropin AQ Nuspin 20

The insurer denied coverage for Nutropin AQ Nuspin 20. The denial was upheld.

This patient is a male child diagnosed with Idiopathic Short Stature (ISS). This request is for Nutropin. He started growth hormone (GH) therapy several years ago. His mother's height is 154.94 cm and father's height is 163.83 cm and so mid-parental height is 165.885 cm (-1.53 SDS). His height on therapy in mid-2019 was 151.2 cm (-2.06 SDS). In mid-2019 his testes are 20 cc bilaterally and pubic hair is at tanner 3. His bone age in mid-2019 was at chronological age (CA) 13 years, 6 months; in early-2017 was at CA 12 years, 7 months; in late-2014 was at CA 10 years 2 months. In late-2014 his IGF1 was 150 (100-449) and IGFBP3 was 3.8 (2.1-7.7). His pretreatment height in late-2014 was 125 cm (-2.15 SDS) and in early-2015 was 126.8 cm (-2.07 SDS) with height velocity over 4 months is 3.6 cm/year. In early-2015 his testes are 2 cc bilaterally and pubic hair is at tanner 1.

The plan acted reasonably as growth hormone is not medically necessary as described below.

The peer reviewed literature including consensus clinical guidelines, and randomized controlled trials are summarized below:
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 standard deviations (sd) below the mean; 2) height more than 1.5 sd below the mid-parental height; 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.
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.(4-9) 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."

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. The request does not meet any nationally accepted criteria for use of growth hormone. The request is therefore not medically necessary.

The carrier's denial of coverage for Nutropin AQ Nuspin 20 is upheld. The medical necessity is not substantiated.