ESPE Yearbook of Paediatric Endocrinology

Most papers selected for this chapter tackle issues with implications for clinical practice. Phase 2 and 3 long-acting GH clinical trials in GHD, SGA and even ISS children have been published in the last year. Use of the IGF-1/IGFBP-3 molar ratio has been re-proposed, with more convincing evidence, for the diagnosis of GH deficiency. An umpteenth reassuring pharma sponsored observational study on rhGH safety with short patient follow-up has been published. The safety and efficacy of long-term rhGH therapy in large cohorts of short children born SGA followed up to the achievement of adult height and even at 12 years after the completion of treatment have been extensively investigated in different articles. The importance of a comprehensive genetic approach to better characterize short SGA children and possibly predict the responders to rhGH therapy has been highlighted in an analytic observational study. The close link between early postnatal growth and adult height has been confirmed in a large retrospective longitudinal study. Two new classes of factors, pappalysins and stanniocalcins, have been identified as modulators of IGF bioavailability at different stages of development. Unexpectedly, GH has been reported to exert a direct effect on neuroinflammation in an animal model. Finally, the yet unanswered old question if faster height growth rate may be associated and even contribute to the development of type 1 diabetes in childhood has probably obtained an answer eventually.