Economic Burden of Growth Hormone Deficiency in a US Pediatric Population

Abstract Pediatric GHD is a rare disorder of short stature that is currently treated with daily injections of GH. In addition to short stature, GHD is associated with other comorbidities such as impaired musculoskeletal development, cardiovascular disease, and decreased quality of life. The objective of this study was to analyze GH utilization, adherence, and healthcare costs among children with GHD who had either Medicaid or commercial health insurance. Children (age <18 years) with a diagnosis of GHD between January 1, 2007, and December 31, 2017 were identified in the IBM Marketscan Commercial and Medicaid Databases. Children with GHD were direct matched (1:3) to controls without GHD (or other short stature-related disorder) on age, gender, plan type, region, and race (Medicaid only). The index date was set as the date of the first GHD diagnosis during the selection window for GHD patients and using random assignment for controls. Patients were followed for the 12 months prior to the index date until the end of continuous database enrollment or December 31, 2018. Baseline comorbidities and medications were measured during the 12 months pre-index. Treatment patterns and all-cause and GHD-related healthcare costs were measured during the variable follow-up period. Multivariable modeling was used to compare costs between GHD patients and controls and between GH treated and untreated GHD patients while adjusting for baseline characteristics. There were 6,820 Medicaid and 14,070 commercial patients with GHD who met the study inclusion criteria. Mean (SD) age at index was 9.5 (4.5) years for Medicaid patients and 11.1 (3.7) years for commercial patients. A majority of patients were male (>65%) and followed for at least 3.7 years. Overall, 63.2% of Medicaid and 68.4% of commercial patients were treated with GH during follow-up. Among Medicaid patients, the treatment rate was highest among white males and lowest among black females. Adherence, as measured by proportion of days covered, was low, with 18.4% of Medicaid patients and 32.3% of commercial patients considered “adherent” (PDC ≥ 0.8). Nearly half (49.1%) of treated Medicaid patients and 24.3% of commercial patients discontinued GH therapy before age 13. After adjusting for baseline characteristics, all-cause non-GH costs were 5.7times higher (Δ$19,309) for Medicaid patients and 5.5 times higher (Δ$12,305) for commercial patients than matched non-GHD controls. Adjusted all-cause non-GH costs were 0.6 times lower (Δ$14,416) for treated Medicaid patients and 0.7 times lower (Δ$7,650) for treated commercial patients than for untreated patients. Pediatric GHD presents a significant healthcare burden, and many patients remain untreated or undertreated. Untreated GHD was associated with higher non-GH healthcare costs than treated GHD. Strategies to improve adherence may reduce the healthcare burden faced by these patients.