PATHOGENESIS OF OSTEOPOROSIS IN YOUNG CHILDREN WITH CYSTIC FIBROSIS

本子项目是利用由NIH/NCRR资助的中心赠款提供的资源的众多研究子项目之一。子项目和研究者（PI）可能已经从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。列出的机构是中心的，不一定是研究者的机构。骨质疏松、骨骼疼痛和骨折是囊性纤维化（CF）的常见后遗症。CF患者骨病的发病机制是复杂的，既有减少骨形成的过程，也有增加骨吸收的过程。CF患者的骨转换已被证明是异常的，骨吸收增加，但骨形成正常。没有一项研究全面评估CF患儿的维生素D代谢。这项研究将CF患儿与其未受影响的兄弟姐妹进行比较。选择兄弟姐妹作为对照，以便为骨转换测量和密度测量提供比较组，并探索杂合CF跨膜受体突变是否对骨矿物质积累有影响。对患有CF的青春期前儿童的骨量和骨代谢进行评估，将有助于确定这些因素在这些个体骨质疏松症发展中的作用。这将有助于制定合理的管理和预防性干预措施和疗法。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Osteoporosis, skeletal pain, and fractures are frequent sequelae of cystic fibrosis (CF). The pathogenesis of bone disease in individuals with CF is complex, resulting both from processes that decrease bone formation and that increase bone resorption. Bone turnover in adults with CF has been shown to be abnormal with increased bone resorption yet normal bone formation. NO study as comprehensively assessed vitamin D metabolism in young children with CF. This study will compare children with CF to their unaffected siblings. Siblings were chosen as the controls in order to provide a comparison group for bone turnover measures and densitometric measurements and also to explore if there are effects of heterozygous CF transmembrane receptor mutations on bone mineral accrual. Assessment of bone mass and bone metabolism in prepubertal children with CF will allow determination of the role of these factors in the development of osteoporosis in these individuals. This will enable development of rational management and prophylactic interventions and therapies.