Appeal type: Medical Necessity
Decision: Overturned

Nature of Statutory Criteria/ Case Summary:  The enrollees parent is requesting authorization and coverage for the medication, Norditropin Flexpro. The enrollee has severe short stature, poor growth velocity and a three-year delay in bone maturation. The pediatric endocrinology office note indicates the enrollees magnetic resonance imaging (MRI) revealed a seven millimeter anterior intrasellar lesion. Pituitary function testing was performed twice and was normal. Neurosurgery recommended a follow-up MRI in six to eight weeks. The plan was to start growth hormone (GH) if the lesion was stable. The repeat MRI showed no change. The enrollees height was documented as 112.5 centimeters; less than the first percentile, and her weight was 23.8 kg, placing her in the 13th percentile. Her bone age was five years, nine months at a chronological age of eight years, seven months. The enrollees bone age is greater than two standard deviations (SDs) behind the expected mean for her chronological age. The endocrinologist is requesting authorization for GH therapy. The provider notes GH therapy for children with ISS is approved by the Food and Drug Administration for those whose height falls below -2.25 SDs of the mean, and with a predicted adult height below the normal range of 59 inches (150 cm) for females. The enrollee is severely growth-restricted, with a growth velocity of 0.8 centimeters over four months. Her mothers height is 165.1 centimeters and her fathers height is 180.34 centimeters. The mid-parental height is 166.22 centimeters (0.45 SDS). Classic GH deficiency criteria are outlined in the consensus guidelines for the diagnosis and treatment of GH deficiency, and the medical guidelines for GH use. The diagnosis of GH deficiency 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 three SDS below the mean; a height more than 1.5 SDS below the mid-parental height; a height more than two SDS below the mean and a height velocity over one year of more than one SDS below the mean for chronological age, or a decrease in height SDS of more than 0.5 over one year in children over two years of age; and, in the absence of short stature, a height velocity more than two SDS below the mean over one year or more than 1.5 SDS sustained over two 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 of less than 10ng/ml. Growth hormone in the treatment of idiopathic short stature (ISS) is supported by randomized controlled trials and consensus guidelines. Approval for this indication is for non-growth hormone-deficient short stature. This is defined by a height SDS of 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 individuals 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 enrollee has a pretreatment height less than or equal to -2.25 SD. Her height velocity is poor and would not allow her to reach a height in the normal range. The enrollee therefore meets idiopathic short stature criteria and  nationally accepted criteria for the use of growth hormone.Final Result: The reviewer determined that the requested service is medically necessary for the treatment of the enrollees medical condition. Credentials/Qualifications: The physician reviewer is board certified in pediatric endocrinology, diabetes & metabolism and is actively practicing. The reviewer is an expert in the treatment of the enrollees medical condition and knowledgeable about the proposed treatment through recent or current actual clinical experience treating those with the same or similar medical condition.