Revised upper limb module for spinal muscular atrophy: 12 month changes

被引:88
作者
Pera, Maria Carmela [1 ,2 ,3 ]
Coratti, Giorgia [1 ,2 ,3 ]
Mazzone, Elena S. [1 ,2 ,3 ]
Montes, Jacqueline [4 ,5 ,6 ]
Scoto, Mariacristina [7 ,8 ,9 ]
De Sanctis, Roberto [1 ,2 ,3 ]
Main, Marion [7 ,8 ,9 ]
Mayhew, Anna [10 ]
Lofra, Robert Muni [10 ]
Young, Sally Dunaway [4 ,5 ,6 ]
Glanzman, Allan M. [11 ]
Duong, Tina [12 ]
Pasternak, Amy [13 ,14 ]
Ramsey, Danielle [7 ,8 ,9 ]
Darras, Basil [13 ]
Day, John W. [12 ]
Finkel, Richard S. [15 ]
De Vivo, Darryl C. [6 ]
Sormani, Maria Pia [16 ]
Bovis, Francesca [16 ]
Straub, Volker [10 ]
Muntoni, Francesco [7 ,8 ,9 ]
Pane, Marika [1 ,2 ,3 ]
Mercuri, Eugenio [1 ,2 ,3 ]
Bettolo, Chiara Marini
Civitiello, Matthew
Mirek, Elizabeth
Salazar, Rachel
Forcina, Nicola
Norcia, Giulia
Carnicella, Sara
Antonaci, Laura
机构
[1] Catholic Univ, Paediat Neurol, Largo Gemelli 8, I-00168 Rome, Italy
[2] Catholic Univ, Ctr Clin Nemo, Rome, Italy
[3] Fdn Policlin Univ Agostino Gemelli IRCSS, Policlin Gemelli, Rome, Italy
[4] Columbia Univ, Med Ctr, Dept Rehabil Med, New York, NY USA
[5] Columbia Univ, Med Ctr, Dept Regenerat Med, New York, NY USA
[6] Columbia Univ, Dept Neurol, Med Ctr, New York, NY USA
[7] Dubowitz Neuromuscular Ctr, London, England
[8] Inst Child Hlth, NIHR Great Ormond St Hosp Biomed Res Ctr, London, England
[9] Great Ormond St Hosp Sick Children, London, England
[10] Newcastle Univ, John Walton Muscular Dystrophy Res Ctr, Newcastle Upon Tyne, Tyne & Wear, England
[11] Childrens Hosp Philadelphia, Dept Phys Therapy, Philadelphia, PA 19104 USA
[12] Stanford Univ, Dept Neurol, Stanford, CA 94305 USA
[13] Harvard Med Sch, Dept Neurol, Boston Childrens Hosp, Boston, MA 02115 USA
[14] Boston Childrens Hosp, Dept Phys Therapy & Occupat Therapy, Boston, MA USA
[15] Univ Cent Florida, Nemours Childrens Hosp, Coll Med, Orlando, FL 32816 USA
[16] Univ Genoa, Sect Biostat, Dept Hlth Sci DISSAL, Genoa, Italy
关键词
Spinal Muscular Atrophy; Outcome measures; Neuromuscular disorders; Upper limb; Functional assessment; Longitudinal Data; CLINICAL-TRIAL; TYPE-2;
D O I
10.1002/mus.26419
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
Introduction: The aim of the study was to assess 12 month changes in upper limb function in patients affected by spinal muscular atrophy type 2 and 3. Methods: Longitudinal 12 month data was collected in 114 patients, 60 type 2 and 54 type 3, using the Revised Upper Limb Module. Results: The 12 month changes ranged between -7 and 9 (mean: -0.41; SD: 2.93). The mean changes were not significantly different between the three spinal muscular atrophy groups (-0.45 in type 2, -0.23 in non-ambulant type 3 and -0.34 in ambulant type 3, p = 0.96) and the relationship between 12 month change and age classes was not significantly different among the three types of SMA patients. Discussion: Our results confirm that the Module explores a wide range of functional abilities and can be used in ambulant and non-ambulant patients of different ages in conjunction with other functional scales. Muscle Nerve 59:426-430, 2019
引用
收藏
页码:426 / 430
页数:5
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