Appeal type: Medical necessity
Decision: Upheld

Diagnosis: Small for Gestational Age (SGA) plus Short Stature/Growth Failure
 
Treatment: Medication-Humatrope 24 mg cartridge, 1.2 mg subcutaneously once daily
 

The insurer denied the Humatrope 24 mg cartridge. The denial was upheld. 

This patient is a young male diagnosed with small for gestational age (SGA) and idiopathic short stature (ISS). This request is for Humatrope. He was born at 38.6 gestational weeks; birth length was 46.99 cm and birth weight was 6 lbs 2.5 oz. The cutoff for SGA is 5.14 lbs and 46.1 cm. The patient was not SGA. His growth hormone (GH) stimulation test in late 2016 with glucagon and Arginine by report was not deficient. He had another GH stimulation test in early 2018 with glucagon and L-dopa peaking at 13. In early 2018, his insulin-like growth factor 1 (IGF1) was 136 and his insulin-like growth factor binding protein 3 (IGFBP3) was 3.10. His mother's height is 157.48 cm and his father's height is 169.926 cm; therefore, the mid parental height is 170.20 cm (-0.93 SDS). His testes are 2 cc and pubic hair is at Tanner 1. 

Classic GH deficiency
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 deviation [SD] below the mean; 2) height more than 1.5 SD below the midparental 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 peaked at greater than 10.

Idiopathic Short stature
Growth hormone in the treatment of idiopathic short stature (ISS) is supported by randomized controlled trials and consensus guidelines [4-9]. Food and Drug Administration [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. 

Based on the above, the medical necessity for the Humatrope 24 mg cartridge, 1.2 mg subcutaneously once daily is not substantiated. The insurer's denial is upheld.