Appeal type: Medical necessity
Decision: Upheld

Diagnosis: Idiopathic Short Stature 

Treatment: Nutropin

The insurer denied the Nutropin. The denial was upheld. 

This is a young male who was diagnosed with idiopathic short stature. He has a history of inguinal hernia and orchidopexy. He has been under the care of a pediatric endocrinologist for the past three years. He had a magnetic resonance imaging of the brain which revealed a hypoenhancing nodule in the left pituitary gland. In the beginning of 2019 the patient had an elevated cortisol level (24) and prolactin level (21), and normal thyroid levels, IGF1, and IGFB3. 

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 yr more than 1 sd below the mean for chronological age, or a decrease in height sd of more than 0.5 over 1 yr in children over 2 yr of age; 4) in the absence of short stature, a height velocity more than 2 sd below the mean over 1 yr 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 10ng/ml.
		
The patient did not meet biochemical criteria. 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 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."

His GH stimulation test in 2017 with arginine and clonidine peaked at 14. His height on therapy in 2019 was 145.5 cm (-2.23 SDS). His height in early 2018 was 137.3 cm (-2.05 SDS) and late 2018 was 143.8 cm (-2.04 SDS) with height velocity over 0.9 years of 7.1 cm/yr. His mother's height is 160.02 cm and father's height is 177.8 cm and so mid- parental height is 175.41 cm (-0.20 SDS). In 2019 his ACTH was 48; IGF1 was 179; IGFBP3 was 4.51. In 2017 his IGF1 was 123. In 2016 his IGF1 was 134 and IGFBP3 was 2.7. 

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. 

Based on the above, the medical necessity for Nutropin is not substantiated. The insurer's denial is upheld.