Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Idiopathic Short Stature

Treatment: Humatrope 12 milligrams (mg) Cartridge (Procedure code j2941)

The insurer denied coverage for Humatrope 12 milligrams (mg) Cartridge (Procedure code j2941)

The denial is overturned.

This is a female child diagnosed with idiopathic short stature (ISS). This request is for Humatrope. The patient's growth hormone (GH) stimulation test with glucagon and L-dopa peaked at 14. The patient's bone age was 3 years (y) 6 months (m) at calculated age (CA) 5y5m.  The patient has normal SHOX gene. The patient's height velocity is reported to be 4.318 centimeters (cm)/y. The patient's insulin-like growth factor-1 (IGF1) was 98; insulin-like growth factor-binding protein-3 (IGFBP3) was 2941.

The patient's most recent weight was 17.463 kilograms (kg). The patient's height at 6y6m was 106.095 cm (-2.57 standard deviations [SDS]) and at 7y4m was 110.11 cm (-2.70 SDS) with height velocity over 9 months of 4.9 cm/y. The patient's mother's height is 154.94 cm and father's height is 167.64 and mid parental height is 162.41 cm (-0.14 SDS).

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 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 deviations (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 y more than 1 SD below the mean for chronological age, or a decrease in height SD of more than 0.5 over 1 y in children over 2 y of age; 4) in the absence of short stature, a height velocity more than 2 SD below the mean over 1 y 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 10.
		
The patient does not meet growth hormone deficiency criteria.

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" (10-13).

The patient had a pretreatment height less than or equal to -2.25 SD. The patient's height at 6y6m was 106.095 cm (-2.57 SDS) and 7y4m was 110.11 cm (-2.70 SDS) with height velocity over 9 months of 4.9 cm/y. The patient's height velocity and bone age would not allow the patient to reach a normal adult height.

The request therefore meets idiopathic short stature criteria.

Based on the above, the insurer's denial must be reversed. The health care plan did not act reasonably and with sound medical judgment and in the best interest of the patient. 

The medical necessity for Humatrope 12milligrams (mg) Cartridge (Procedure code j2941) services is substantiated.