PMU142 ASSESSING THE HUMAN AND ECONOMIC BURDEN OF SHORT STATURE: A SYSTEMATIC LITERATURE REVIEW

Abstract

We sought to better understand the human and economic burden of short stature (SS) using a systematic literature review. Studies of the human and economic burden of SS in adults and children, due to any cause, were searched using Embase, MEDLINE and Cochrane databases in December 2018, capturing publications from 2008 onwards. Case series and populations with adult-onset growth hormone deficiency (GHD) were excluded. Of 1239 publications identified, 26 studies (17 in children, 9 in adults) met inclusion criteria, with sample sizes of the SS populations ranging from 15 to 285. Thirteen studies were in mixed populations, four in GHD only, four in chronic kidney disease (CKD), and one each in Turner syndrome, Prader-Willi syndrome, skeletal dysplasia, congenital adrenal hyperplasia, and childhood-onset multiple pituitary hormone deficiency. No economic studies were identified. Twelve studies compared quality of life (QoL) in children with SS and those with normal height; 10 found that QoL was significantly poorer in children with SS and two (both in CKD populations) found no association. Of five studies that investigated height as a predictor of QoL among children with SS, all found greater height was associated with better QoL. One study highlighted negative social experiences in children with SS. Two studies evaluated cognitive function, finding cognitive scores were significantly poorer in children with SS than in those with normal height. In adults, six studies demonstrated impaired QoL with SS versus normal height, while two found no association and one reported mixed results. The literature suggests that compared with those with normal height, children and adults with SS of any cause experience poorer QoL and in children it may negatively impact social and cognitive functioning. Further research could extend our understanding of the human and economic burden in this field.