Advances in growth hormone therapy: a new registry tool

Growth hormone (GH) deficiency is a rare condition, and physicians may lack clinical experience of its treatment. Despite evidence for the efficacy and tolerability of GH therapy, a substantial proportion of patients remain untreated. Registries and other large-scale databases are an important tool for expanding the evidence base for GH treatment. Since the mid-1980s, registries have provided data on important aspects of the safety and efficacy of GH treatment. Registries have also allowed pooling of data from patients with rare conditions that could otherwise not be recruited in sufficient numbers for clinical trials. Importantly for patients and their relatives, use of registry data has allowed the development of prediction models that indicate the likely outcomes of treatment. MEGHA (Metabolic Endocrinology and Growth Hormone Assessment) is a recently developed, observational database with a number of features not found in existing registries, including its use as a day-to-day clinical management tool, the ability to create individual sub-studies, direct comparison of personal data against the full database, and a particular focus on the transition from childhood to adulthood. This creative registry is a promising instrument for further research into GH-related disorders that will improve GH therapy and thus provide benefits for patients.