Appeal type: Medical necessity
Decision: Upheld

Diagnosis: Idiopathic short stature.

Treatment: Growth hormone to treat idiopathic short stature.

The insurer denied coverage for growth hormone to treat idiopathic short stature.

The denial is upheld.

This patient is a female child diagnosed with idiopathic short stature (ISS). The patient has also been diagnosed with type 1 diabetes mellitus (DM). She reports continued pubertal progression with axillary hair; increased pubic hair and increased breast development. Her Growth Hormone (GH) stimulation test with clonidine and arginine peaked at 31.90. Her height velocity is reported to be 6.019 centimeters (cm)/year. Her breasts and genitourinary (GU) were at tanner 4. Her mother's height is 165.10 cm and father's height is 162.56 cm and mid-parental height is 157.33 cm (-0.93 SDS [standard deviation score]). This request is for Zomacton.

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 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 SD [standard deviations] 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 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 insulin-like growth factor-1 (IGF-1) or insulin-like growth factor-binding protein 3 (IGFBP3) or GH stimulation testing with a peak GH less than 10 nanograms per milliliter (ng/mL). The patient did not meet biochemical criteria. Growth hormone stimulation testing peaked at greater than 10. 

Growth hormone in the treatment of ISS is supported by randomized controlled trials and consensus guidelines [4, [5, [6, [7, [8, [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. 

The request does not meet any nationally accepted criteria for use of growth hormone. 

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

The insurer's denial of coverage for growth hormone to treat idiopathic short stature is upheld. Medical necessity is not substantiated.