Developing standardized corticosteroid treatment for Duchenne muscular dystrophy

被引：56

作者：

Guglieri, Michela ^{[1
]}

Bushby, Kate ^{[1
]}

McDermott, Michael P. ^{[2
]}

Hart, Kimberly A. ^{[2
]}

Tawil, Rabi ^{[2
]}

Martens, William B. ^{[2
]}

Herr, Barbara E. ^{[2
]}

McColl, Elaine ^{[3
]}

Wilkinson, Jennifer ^{[3
]}

Kirschner, Janbernd ^{[4
]}

King, Wendy M. ^{[2
]}

Eagle, Michele ^{[1
]}

Brown, Mary W. ^{[2
]}

Willis, Tracey ^{[5
]}

Hirtz, Deborah ^{[6
]}

Shieh, Perry B. ^{[7
]}

Straub, Volker ^{[1
]}

Childs, Anne-Marie ^{[8
]}

Ciafaloni, Emma ^{[2
]}

Butterfield, Russell J. ^{[9
]}

Horrocks, Iain ^{[10
]}

Spinty, Stefan ^{[11
]}

Flanigan, Kevin M. ^{[12
]}

Kuntz, Nancy L. ^{[13
]}

Baranello, Giovanni ^{[14
]}

Roper, Helen ^{[15
]}

Morrison, Leslie ^{[16
]}

Mah, Jean K. ^{[17
]}

Manzur, Adnan Y. ^{[18
]}

McDonald, Craig M. ^{[19
]}

Schara, Ulrike ^{[20
]}

von der Hagen, Maja ^{[21
]}

Barohn, Richard J. ^{[22
]}

Campbell, Craig ^{[23
]}

Darras, Basil T. ^{[24
]}

Finkel, Richard S. ^{[25
]}

Vita, Giuseppe ^{[26
]}

Hughes, Imelda ^{[27
]}

Mongini, Tiziana ^{[28
]}

Pegoraro, Elena ^{[29
]}

Wicklund, Matthew ^{[30
]}

Wilichowski, Ekkehard ^{[31
]}

Bumette, W. Bryan ^{[32
]}

Howard, James F. ^{[33
]}

McMillan, Hugh J. ^{[34
]}

Thangarajh, Mathula ^{[35
]}

Griggs, Robert C. ^{[2
]}

机构：

[1] Newcastle Univ, John Walton Muscular Dystrophy Res Ctr, Newcastle Upon Tyne, Tyne & Wear, England

[2] Univ Rochester, Med Ctr, Rochester, NY 14642 USA

[3] Newcastle Univ, Newcastle Upon Tyne, Tyne & Wear, England

[4] Univ Med Ctr, Freiburg, Germany

[5] NHS Fdn Trust, Robert Jones & Agnes Hunt Orthopaed Hosp, Oswestry, Shrops, England

[6] NIH, Bldg 10, Bethesda, MD 20892 USA

[7] UCLA, Los Angeles, CA USA

[8] Leeds Teaching Hosp, Leeds, W Yorkshire, England

[9] Univ Utah, Salt Lake City, UT 84112 USA

[10] Greater Glasgow & Clyde NHS Yorkhill Hosp, Glasgow, Lanark, Scotland

[11] Alder Hey Childrens Hosp, Liverpool, Merseyside, England

[12] Nationwide Childrens Hosp, Columbus, OH USA

[13] Ann & Robert H Lurie Childrens Hosp, Chicago, IL USA

[14] Neurol Inst Carlo Sesta, Tubingen, Germany

[15] Birmingham Heartlands Hosp, Birmingham, W Midlands, England

[16] Univ Mexico, Mexico City, DF, Mexico

[17] Univ Calgary, Calgary, AB, Canada

[18] UCL, GOSH, London, England

[19] UC Davis Med Ctr, Sacramento, CA USA

[20] Univ Essen Gesamthsch, Essen, Germany

[21] Tech Univ Dresden, Childrens Hosp, Dresden, Germany

[22] Univ Kansas, Med Ctr, Lawrence, KS 66045 USA

[23] Childrens Hosp London, Hlth Sci Ctr, London, ON, Canada

[24] Boston Childrens Hosp, Boston, MA USA

[25] Nemours Childrens Hosp, Orlando, FL USA

[26] Univ Messina, AOU Policlin Gaetano Martino, Messina, Italy

[27] Royal Manchester Childrens Hosp, Manchester, Lancs, England

[28] Univ Torino, Turin, Italy

[29] Univ Padua, Padua, Italy

[30] Penn State Coll Med, Hershey, PA USA

[31] Childrens Univ Hosp, Gottingen, Germany

[32] Vanderbilt Childrens Hosp, Nashville, TN USA

[33] Univ N Carolina, Sch Med, Chapel Hill, NC USA

[34] Childrens Hosp Eastern Ontario, Ottawa, ON, Canada

[35] Childrens Natl Med Ctr, Washington, DC 20010 USA

来源：

CONTEMPORARY CLINICAL TRIALS | 2017年 / 58卷

基金：

美国国家卫生研究院;

关键词：

Duchenne muscular dystrophy; Standards of care; Prednisolone; Deflazacort; Randomized; GENERIC CORE SCALES; PREDNISONE THERAPY; LOW-DOSAGE; DEFLAZACORT; TRIAL; TIME;

D O I：

10.1016/j.cct.2017.04.008

中图分类号：

R-3 [医学研究方法]; R3 [基础医学];

学科分类号：

1001 ;

摘要：

Despite corticosteroids being the only treatment documented to improve strength and function in boys with Duchenne muscular dystrophy (DMD) corticosteroid prescription is inconsistent and in some countries, corticosteroids are not prescribed. We are conducting a clinical trial that (1) compares the 3 most frequently prescribed corticosteroid regimes; (2) standardizes treatment of DMD complications; and (3) standardizes prevention of corticosteroid side effects. Investigators at 38 sites in 5 countries plan to recruit 300 boys aged 4-7 who are randomly assigned to one of three regimens: daily prednisone; daily deflazacort; or intermittent prednisone (10 days on/10 days off). Boys are followed for a minimum of 3 years to assess the relative effectiveness and adverse event profiles of the different regimens. The primary outcome is a 3-dimensional variable consisting of log-transformed time to rise from the floor, forced vital capacity, and subject/parent satisfaction with treatment, each averaged over all post-baseline visits. The study protocol includes evidence- and consensus-based treatment of DMD complications and of corticosteroid side effects. This study seeks to establish a standard corticosteroid regimen for DMD. Since all new interventions for DMD are being developed as add-an therapies to corticosteroids, defining the optimum regimen is of importance for all new treatments.

引用

页码：34 / 39

页数：6

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