ESPE Yearbook of Paediatric Endocrinology

PrefaceThe selection of papers in this chapter focused on clinical research, as major advancements in the pathophysiology, diagnostic work-up, management and treatment of growth disorders have been reported in 2020–21. The new cut-off values of blood GH concentrations for diagnosis of GH deficiency in the newborn, the insights into the phenotype and therapeutic response to rhGH in patients with NPR2 mutations, the individualisation of the o...

Address correspondence to: Stefano Cianfarani Bambino Gesù Children’s Hospital, Dipartimento Pediatrico Universitario Ospedaliero, IRCCS “Bambino Gesù” Children’s Hospital, Rome, Italy. P.zza S. Onofrio, 4 00165, Roma, Italy Tel. +39 06 6859 3074 (Hosp.) / +39 06 72596178 (Lab.) Fax. +39 06 6859 2508 (Hosp.)/ +39 06 72596172 (Lab.) E-mail: stefano.cianfarani@uniroma2.it<p class="abs...

To read the full abstract: PLoS One. 2018;13:e0201566.NAFLD is a multifactorial disease characterized by an abnormal accumulation of fat in the liver without a history of significant alcohol intake. NAFLD is the most common form of chronic liver disease, affecting 30% of adults and 10% of children in the USA [1]. Alarmingly, its prevalence is increasing worldwide paralleling to the incre...

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 effic...