Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Growth hormone deficiency

Treatment: Humatrope (J2941) 24mg cartridge 1.7mg by daily subcutaneous injection 

The insurer denied coverage Humatrope (J2941) 24mg cartridge 1.7mg by daily subcutaneous injection. The denial was reversed.

This patient is a female child diagnosed with growth hormone deficiency (GHD). This request is for Humatrope. Her insulin-like growth factor-1 (IGF-1) was 114. Her MRI showed an empty sella which is frequently associated with GHD. Her mother's height is 164.465 cm and her father's height is 179.07 cm; therefore, her mid parental height is 165.26 cm (0.30 standard deviation score [SDS]). Her height in late-2017 was 132.1 cm (-1.23 SDS) and137.1 cm (-1.68 SDS) in early-2019 with a height velocity over 1.33 years of 3.8 cm/year, (-2.5 standard deviation [SD]).  In late-2017, her breasts are at Tanner 2 and pubic hair is at Tanner 1. Her bone age in 2017 was 8 years 10 months; in mid-2018 bone at was 10 years. In late-2017 her insulin-like growth factor binding protein3 (IGFBP3) was 3.5 (2.1-7.7); her IGFBP3 was 114 (125-541). In mid-2018 her IGF1 was 152 (152-593). Her height velocity is reported to be 7.2cm/year. Her MRI was normal.

Classic GH deficiency published by the GH research society(1), the Lawson Wilkins Pediatric Endocrinology Society (LWPES) (2, 3), and the medical guidelines for GH use provided by the American Association of Clinical Endocrinologists(4). The above 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, severe short stature, defined as a height more than 3 standard deviation (SD) below the mean, height more than 1.5 SD below the midparental height, 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, and 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 was not performed.

Alternate GH deficiency criteria: LWPES alternate criteria (2) states no gold standard exists for the diagnosis of GHD. Although children severely affected by GHD fail GH stimulation tests, there is no doubt that some children with GHD achieve stimulated GH concentrations above the arbitrary cutoffs that have been applied. A trial of GH therapy should approved for children with otherwise unexplained short stature who pass GH stimulation tests, but who meet most of the following criteria,  height greater than 2.25 SD below the mean for age or greater than 2 SD below the midparental height percentile, growth velocity less than 25th percentile for bone age, bone age greater than 2 SD below the mean for age, low serum insulin-like growth factor 1 (IGF-I) and/or insulin-like growth factor binding protein 3 (IGFBP3), and/or other clinical features suggestive of GHD.

The IGF1 was low, bone age was delayed, and height velocity is -2.5 SD. The patient had a partially empty sella. The patient therefore met 4 of the above criteria. The patient is therefore growth hormone deficient. Treatment with growth hormone is therefore consistent with generally accepted standards of medical practice and 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.

The medical necessity for Humatrope (J2941) 24mg cartridge 1.7mg by daily subcutaneous injection is substantiated.  The insurer's denial is reversed.