Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management

被引:733
作者
Birnkrant, David J. [1 ]
Bushby, Katharine [2 ]
Bann, Carla M. [3 ]
Alman, Benjamin A. [4 ]
Apkon, Susan D. [5 ]
Blackwell, Angela [3 ]
Case, Laura E. [7 ]
Cripe, Linda [8 ]
Hadjiyannakis, Stasia [9 ,10 ]
Olson, Aaron K. [6 ]
Sheehan, Daniel W. [11 ]
Bolen, Julie [12 ]
Weber, David R. [13 ]
Ward, Leanne M. [9 ,10 ]
机构
[1] Case Western Reserve Univ, MetroHlth Med Ctr, Dept Pediat, Cleveland, OH 44109 USA
[2] Newcastle Univ, Inst Genet Med, John Walton Muscular Dystrophy Res Ctr, Newcastle Upon Tyne, Tyne & Wear, England
[3] RTI Int, Res Triangle Pk, NC USA
[4] Duke Univ, Sch Med & Hlth Syst, Dept Orthopaed Surg, Durham, NC USA
[5] Seattle Childrens Hosp, Dept Rehabil Med, Seattle, WA USA
[6] Seattle Childrens Hosp, Dept Pediat, Seattle, WA USA
[7] Duke Univ, Sch Med, Dept Orthopaed, Phys Therapy Div, Durham, NC USA
[8] Ohio State Univ, Nationwide Childrens Hosp, Dept Pediat, Columbus, OH 43210 USA
[9] Childrens Hosp Eastern Ontario, Div Endocrinol & Metab, Ottawa, ON, Canada
[10] Univ Ottawa, Ottawa, ON, Canada
[11] SUNY Buffalo, John R Oishei Childrens Hosp, Buffalo, NY USA
[12] Ctr Dis Control & Prevent, Disorders & Hlth Outcomes Team, Natl Ctr Birth & Dev Disabil, Atlanta, GA USA
[13] Univ Rochester, Med Ctr, Golisano Childrens Hosp, Div Endocrinol & Diabet, Rochester, NY 14642 USA
关键词
INCIDENT VERTEBRAL FRACTURES; LUNG-VOLUME RECRUITMENT; ASSOCIATION TASK-FORCE; FAT-EMBOLISM SYNDROME; INTRAVENOUS BISPHOSPHONATE THERAPY; VENTRICULAR ASSIST DEVICE; NONINVASIVE VENTILATION; OSTEOGENESIS IMPERFECTA; AMERICAN-COLLEGE; VITAL CAPACITY;
D O I
10.1016/S1474-4422(18)30025-5
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
A coordinated, multidisciplinary approach to care is essential for optimum management of the primary manifestations and secondary complications of Duchenne muscular dystrophy (DMD). Contemporary care has been shaped by the availability of more sensitive diagnostic techniques and the earlier use of therapeutic interventions, which have the potential to improve patients' duration and quality of life. In part 2 of this update of the DMD care considerations, we present the latest recommendations for respiratory, cardiac, bone health and osteoporosis, and orthopaedic and surgical management for boys and men with DMD. Additionally, we provide guidance on cardiac management for female carriers of a disease-causing mutation. The new care considerations acknowledge the effects of long-term glucocorticoid use on the natural history of DMD, and the need for care guidance across the lifespan as patients live longer. The management of DMD looks set to change substantially as new genetic and molecular therapies become available.
引用
收藏
页码:347 / 361
页数:15
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