ESPE Abstracts

Background: Daily recombinant human growth hormone (rhGH) has been utilized since 1985 and has been proven to increase height velocity and improve body composition in growth hormone deficiency, various genetic syndromes and chronic kidney disease. Safety and efficacy are well established. Long-acting growth hormone (LAGH) analogs have been developed to improve compliance and patient experience. There are several LAGH preparations in development or early commercial use. A recent meta-analysis which included seven studies comparing LAGH and daily rhGH, found no significant difference in height velocity and height standard deviation scores or a significant difference in incidence of adverse effects.

Methods: This survey was developed on behalf of the ESPE Bone & Growth Plate working group to evaluate current knowledge, perceptions and reservations of paediatricians regarding LAGH analog use. Clinicians were invited by direct email request, society updates and social media post to complete a questionnaire on current practices and future intentions regarding growth hormone prescription.

Results: The majority of the 30 respondents were paediatric endocrinologists (83%). Replies came from Europe (80%), Latin America (10%), Asia (7%) and Middle East (3%). Current practice regarding initiation of daily rhGH treatment, monitoring of efficacy and adverse effects were based on recognized guidelines.

The majority of respondents (90%) have been provided with information on LAGH analogs, usually from conference symposia. Only one responder is currently prescribing LAGH as part of a clinical trial. Based on current knowledge, one third would prescribe LAGH rather than daily treatment but would welcome further information, 43% are undecided while 23% of respondents would not at this time. Reasons to move from daily to long-acting GH analogs include patient compliance (95%), frequency of dosing (83%), pain or distress of child (70%) and safety data (40%). The majority would use the same clinical indications, efficacy and safety parameters that exist for daily treatment. Reservations are based on current knowledge (46%) and pharmacokinetic profile (53%) compared to physiological GH secretion. Cost of LAGH analogs would impact use in 80% of respondents. The majority would prefer increased monitoring (70%) and expect pharmaceutical companies to provide clear dosing guidelines (87%), updated reference ranges for IGF-1/IGF-BP3 (75%) and provision of a surveillance registry (83%)

Conclusion: Introduction of LAGH analogs will require increased information on dosing, comparable cost and provision of surveillance registries to encourage clinician use over daily rhGH therapy.