Appeal type: Medical necessity
Decision: Upheld

Diagnosis: Idiopathic short stature (ISS)
Treatment: Genotropin for idiopathic short stature (ISS)

The insurer denied coverage for Genotropin for idiopathic short stature (ISS).
The denial is upheld. 

This patient is a male child diagnosed with idiopathic short stature (ISS). This request is for Genotropin. The patient's height was 126.88 centimeters (cm) (-1.57 SDS (standard deviation score)) and at an older age was 138 cm (-0.12 SDS). The patient's mid-parental height is reported to be 169 cm (-1.10 SDS). 

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, the Lawson Wilkins Pediatric Endocrinology Society, and the medical guidelines for GH use provided by the American Association of Clinical Endocrinologists. 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 standard deviation (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 above a certain 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 insulin-like growth factor-1 (IGF1) or insulin-like growth factor binding protein-3 (IGFBP3) or GH stimulation testing with a peak GH less than 10 nanograms per milliliter. In this case, the patient did not meet biochemical criteria. Growth hormone stimulation testing is not reported.

Growth hormone in the treatment of idiopathic short stature (ISS) is supported by randomized controlled trials and consensus guidelines. Food and Drug Administration (FDA) approval for this indication is for "non-growth hormone-deficient short stature, defined by height standard deviation score (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." Idiopathic short stature is determined based on the current height. In this case, the patient does not have a pretreatment height less than or equal to -2.25 SDS. The patient therefore, does not meet idiopathic short stature criteria. 

Based on the review of the medical record and literature, the request does not meet any nationally accepted criteria for use of GH. Thus, the request for Genotropin treatment is not considered medically necessary for this patient.

The health plan acted reasonably with sound medical judgment, and in the best interest of the patient.

Based on the above, the medical necessity for Genotropin for idiopathic short stature (ISS) is not substantiated. The insurer's denial should be upheld.