Presentation (CTI)

OBJECTIVES: Growth hormone deficiency (GHD) is the most common pediatric endocrine disorder. Neonatal GHD most frequently presents as persistent severe hypoglycemia, which can lead to seizures and brain injury. Growth hormone replacement therapy results in recovery from hypoglycemia while enhancing growth and producing significant metabolic effects in children with GHD. However, treatment with somatropin, a recombinant human growth hormone (rhGH), is costly and has not been included in the benefit package in Thailand yet. This study aimed to evaluate the cost-utility of somatropin replacement compared to no treatment for neonates and young children with GHD-associated hypoglycemia.
METHODS: A Markov model was developed to assess lifetime costs and health outcomes of growth hormone replacement compared with no treatment. Cost data were obtained from published literature. Health outcomes were measured in life years, quality-adjusted life-years (QALY), and incremental cost-effectiveness ratios (ICER), and future costs and outcomes were discounted at 3% per annum. One-way and probabilistic sensitivity analyses were conducted to investigate the uncertainties of all parameters.
RESULTS: For GHD-associated hypoglycemia, somatropin treatment resulted in 3.04 additional QALYs at an incremental cost of 966,822 baht, compared with no treatment. The ICER of somatropin replacement compared to no treatment was 317,568 baht per quality-adjusted life year (QALY) gained. At the Thai societal willingness-to-pay threshold of 160,000 baht per QALY gained, somatropin replacement would be cost-effective if the cost per milliliter would be reduced by 52%. One-way sensitivity analysis results revealed that the model was the most sensitive to the utility scores for no treatment and for somatropin replacement and cost of somatropin, respectively.
CONCLUSIONS: At a societal willingness to pay of 160,000 THB per QALY gained, no treatment was cost-effective for children with GHD-associated hypoglycemia. Although somatropin improved growth and metabolic outcomes, it incurred the highest cost. However, it could become cost-effective if its price were reduced.