Linglart Agnès, Merzoug Valérie, Lambert Anne-Sophie, Adamsbaum Catherine
Service d'endocrinologie et diabétologie pour enfants, centre de référence des maladies rares du métabolisme du calcium et du phosphate, plateforme d'expertise Paris Sud des maladies rares, filière OSCAR, hôpital Bicêtre Paris Sud, AP-HP, 94270 Le Kremlin-Bicêtre, France.
Service de radiologie pédiatrique, plateforme d'expertise Paris Sud des maladies rares, filière OSCAR, Medical School, hôpital Bicêtre Paris Sud, Paris-Sud University, AP-HP, 94270 Le Kremlin-Bicêtre, France.
Ann Endocrinol (Paris). 2017 Jun;78(2):114-122. doi: 10.1016/j.ando.2017.04.011. Epub 2017 May 8.
Bone dysplasia is a large group that encompasses 436 rare diseases. Many of them are characterized by short stature or decreased growth velocity during puberty. The diagnosis of short stature due to skeletal dysplasia relies on (i) physical features such as disproportionate trunk/limbs, short limbs or extremities and/or stocky build, (ii) radiographic features to analyze mineralization, maturation and bone morphology, and (iii) whenever possible, the genetic characterization. Bone dysplasia mostly affect many organs, and therefore require multidisciplinary follow-up and care. The role of the pediatric endocrinologist is to assess the growth potential of these patients in coordination with the other caregivers, offer the best management of the growth to limit the psychosocial consequences of the extreme short stature and bone deformities.
骨发育异常是一个包含436种罕见疾病的大类。其中许多疾病的特征是身材矮小或青春期生长速度减慢。骨骼发育异常所致身材矮小的诊断依赖于:(i)身体特征,如躯干/四肢比例失调、肢体短小或粗壮体型;(ii)用于分析矿化、成熟度和骨形态的影像学特征;以及(iii)尽可能进行基因特征分析。骨发育异常大多会影响多个器官,因此需要多学科随访和护理。儿科内分泌学家的作用是与其他护理人员协作评估这些患者的生长潜力,提供最佳的生长管理,以限制极端身材矮小和骨骼畸形带来的心理社会后果。