Department of Endocrinology and Metabolism, Amsterdam UMC, University of Amsterdam, Meibergdreef 9, 1105 AZ, Amsterdam, the Netherlands.
Rev Endocr Metab Disord. 2018 Mar;19(1):81-92. doi: 10.1007/s11154-018-9460-5.
Inborn errors of metabolism encompass a wide spectrum of disorders, frequently affecting bone. The most important metabolic disorders that primarily influence calcium or phosphate balance, resulting in skeletal pathology, are hypophosphatemic rickets and hypophosphatasia. Conditions involving bone marrow or affecting skeletal growth and development are mainly the lysosomal storage disorders, in particular the mucopolysaccharidoses. In these disorders skeletal abnormalities are often the presenting symptom and early recognition and intervention improves outcome in many of these diseases. Many disorders of intermediary metabolism may impact bone health as well, resulting in higher frequencies of osteopenia and osteoporosis. In these conditions factors contributing to the reduced bone mineralization can be the disorder itself, the strict dietary treatment, reduced physical activity or sunlight exposure and/or early ovarian failure. Awareness of these primary or secondary bone problems amongst physicians treating patients with inborn errors of metabolism is of importance for optimization bone health and recognition of skeletal complications.
先天性代谢缺陷涵盖了广泛的疾病谱,常累及骨骼。最重要的代谢性疾病主要影响钙或磷平衡,导致骨骼病理,即低磷性佝偻病和低磷酸酯酶症。涉及骨髓或影响骨骼生长发育的主要是溶酶体贮积症,特别是黏多糖贮积症。在这些疾病中,骨骼异常往往是首发症状,早期识别和干预可改善许多此类疾病的预后。许多中间代谢紊乱也可能影响骨骼健康,导致更高的骨质疏松症和骨量减少症发病率。在这些情况下,导致骨矿物质化减少的因素可能是疾病本身、严格的饮食治疗、体力活动减少或阳光暴露减少,以及/或卵巢早衰。治疗先天性代谢缺陷患者的医生了解这些原发性或继发性骨骼问题对于优化骨骼健康和识别骨骼并发症非常重要。