造血干细胞移植在骨硬化症患者血液系统以外的作用  

Extra-hematologic actions of hemopoietic stem cell transplantation on osteopetrosis patients

作　　者：李想 王梓媛 任娜 章振林[1] 汪纯[1] LI Xiang;WANG Zi-yuan;REN Na;ZHANG Zhen-lin;WANG Chun(Department of Osteoporosis and Bone Disease,the Sixth People's Hospital Affiliated to Shanghai Jiaotong University,Shanghai Bone Disease Clinical Research Center,Shanghai 200233,China)

机构地区：[1]上海交通大学附属第六人民医院骨质疏松和骨病专科,上海市骨疾病临床研究中心,上海200233

出　　处：《中华骨质疏松和骨矿盐疾病杂志》2022年第5期545-555,共11页Chinese Journal Of Osteoporosis And Bone Mineral Research

基　　金：国家重点研发计划(2018YFA0800801);国家自然科学基金(82070903);上海申康医院发展中心临床科技创新项目(SHDC12018120)。

摘　　要：骨硬化症(osteopetrosis,OPT)是一类以骨密度异常增高、多发脆性骨折、视力及听力障碍等为主要临床表现的遗传性疾病,目前造血干细胞移植(hemopoietic stem cell transplantation,HSCT)是治愈该疾病的唯一手段。重症OPT患者常存在造血衰竭,多在幼年时期死亡,而HSCT能显著延长患者的生存期、恢复骨髓造血,但HSCT是否能为患者带来造血系统以外的获益目前仍无一致结论。本文旨在综述HSCT在骨重塑、视力、听力、生命质量等方面为OPT患者带来的潜在获益。Osteopetrosis(OPT)is a hereditary disease,characterized by abnormal dense bone,multiple fragility fractures,visual and hearing impairment.At present,hemopoietic stem cell transplantation(HSCT)is the only cure.Severe cases suffer from hematopoietic failure and die at infancy.And HSCT promotes their survival as well as restores hematopoiesis,but whether it brings extra-hematologic benefits remains controversial.This paper aims to explore the potential benefits of HSCT in bone remodeling,vision,hearing,and quality of life of OPT patients.

关 键 词：骨硬化症 造血干细胞移植 视力 听力 

分 类 号：R681[医药卫生—骨科学]