TransCon human growth hormone for children with growth hormone deficiency: a technology evaluation

摘要

Introduction Standard-of-care treatment for growth hormone deficiency consists of daily subcutaneous injections of recombinant human growth hormone, also known as somatropin. Although somatropin treatment is well established, the burden of daily injections can lead to poor adherence and quality of life. In this regard, a TransCon human growth hormone (lonapegsomatropin-tcgd) technology was developed to optimize the therapeutic effect of daily somatropin for the treatment of children with growth hormone deficiency. Areas covered The authors reviewed the effects of lonapegsomatropin-tcgd in children with growth hormone deficiency. Expert opinion Lonapegsomatropin-tcgd was found to be non-inferior to and superior to daily somatropin for annualized height velocity. In addition, the safety was comparable between them. As a result, the convenient dosing of lonapegsomatropin-tcgd has the potential to improve patient adherence, leading to increased efficacy and quality of life. Medication adherence, quality of life, long-term safety, and cost-effectiveness studies comparing lonapegsomatropin-tcgd and daily somatropin are required to confirm these possible benefits.