BONE MINERAL DENSITY IN CHILDREN AFTER HEMATOPOIETIC STEM CELL TRANSPLANTATION

该子项目是利用NIH/NCRR资助的中心赠款提供的资源的许多研究子项目之一。子项目和研究者（PI）可能从另一个NIH来源获得主要资金，因此可以在其他CRISP条目中表示。所列机构为中心，不一定是研究者所在机构。随着更有效的抗癌治疗和造血干细胞移植（HSCT）的使用，更多的儿童在原发性恶性肿瘤中存活或治愈原发性热或代谢疾病。由于生存率的提高，HCST后患者的人数正在增加，治疗的继发性影响成为一个重要问题。HSCT的代谢并发症之一是骨质疏松症。充足的骨矿化对儿科人群尤为重要，因为45%的峰值骨矿物质增加发生在青春期最活跃的生长期。在此期间未达到最佳骨矿物质密度（BMD）的患者可能会终身衰弱。 对儿童HSCT后骨丢失的危险因素、发病率、时间、机制和程度的了解非常有限。目前，尚无关于HSCT后儿童BMD的前瞻性研究报告。本初步研究的目的是收集HSCT后儿童BMD的初步前瞻性数据。这项试点研究的目的是收集骨密度和骨转换标志物的初步前瞻性数据，这些数据将构成申请外部赠款支持的基础。 每名患者将有4个预约，这将是干细胞移植前后常规护理的一部分，即移植后100天，6个月和12个月。每次访视将包括体格检查、血液和尿液检查，以评估骨骼和与骨骼生长相关的各种内分泌腺的状况。此外，在4次就诊中的3次，患者将进行骨骼DEXA扫描，这是一种无创，无痛，安全，快速的方法来确定骨骼的矿物质含量和骨质疏松症的风险。在首次访视和1年随访访视期间，将拍摄左手腕的X光片以确定骨骼的年龄，这对于解释DEXA扫描的结果很重要。

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. With the use of more effective anti-cancer therapy and hematopoietic stem cell transplantation (HSCT), more children survive their primary malignancies or are cured of the primary heatologic or metabolic disorders. As a consequence of improved survival, the population of post-HCST patients is increasing and secondary effects of treatment become an important issue. One of the metabolic complications of HSCT is osteoporosis. Adequate bome mineralization is of particular importance in the pediatric population because 45% of peak bone mineral accretion occurs during the most active growth during puberty. Patients who do not reach the optimal bone mineral density (BMD) during this period may suffer from a lifelong debilitating condition. The knowledge of risk factors, incidence, timing, the mechanisms and the magnitude of bone loss following HSCT in children is very limited. At present, no prospective studies have been reported on BMD in children after HSCT. The goal of this pilot study is to collect preliminary prospective data on BMD in children after HSCT. The goal of this pilot study is to collect preliminary prospective data on BMD and markers of bone turnover that would constitute the basis for an application for the external grant support. Each patient will have 4 appointments which will be part of a routine care before and after stem cell transplant, that is 100 days, 6 months, and 12 months after transplant. Each visit will involve a physical examination, blood and urine tests to assess the condition of the bones and various endocrine glands related to the bone growth. In addition, during 3 out of 4 visits, the patients will have a DEXA scan of the bones, which is a non-invasive, painless, safe, and quick method to determine the mineral content of the bones and the risk of osteoporosis. During the initial visit, and at a 1-year follow up visit, the x-ray of the left wrist will be taken to determine the age of the bones, which will be important to interpret the results of the DEXA scan.