Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Idiopathic short stature (ISS)

Treatment: Zomacton 10 mg vial

The insurer denied coverage for Zomacton 10 mg vial. The denial was reversed.

This patient is a male child with idiopathic short stature (ISS). This request is for Zomacton. His growth hormone (GH) stimulation test with arginine and clonidine peaked at 10.5. His height velocity is reported to be 3.1 cm/year. His mother's height is 157.48 cm and his father's height is 177.8 cm; therefore, the mid parental height is 174.14 cm (-0.38 standard deviation score [SDS]). His testis was 2.9 cm at the right and 3 cm at the left and pubic hair was at Tanner 2 his insulin-like growth factor 1 (IGF1) was 322 (49-487) and the insulin-like growth factor-binding protein 3 (IGFBP3) was 5.26. His bone age was 11 years 6 months at an older chronological age. The predicted adult height is more than 2 standard deviation (SD) below the mean. 

The peer reviewed literature including consensus clinical guidelines, randomized controlled trials are summarized below:

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 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 has a pretreatment height less than or equal to -2.25 SD. The height velocity is slow given that he is in puberty. The height prediction is more than 2SD below the mean. Other causes of short stature were ruled out, including familial short stature. The patient therefore met idiopathic short stature criteria. 

The request meets nationally accepted criteria for use of growth hormone. The request is therefore medically necessary. The health plan did not act reasonably or with sound medical judgment or in the best interest of the patient as the request is medically necessary. 

Based on the above, the medical necessity for Zomacton 10 mg vial is substantiated.  The insurer's denial is reversed.