Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy: results from the Muscular Dystrophy Association DMD Clinical Research Network

被引：83

作者：

Connolly, Anne M. ^{[1
,2
]}

Florence, Julaine M. ^{[1
]}

Cradock, Mary M. ^{[2
]}

Malkus, Elizabeth C. ^{[1
]}

Schierbecker, Jeanine R. ^{[1
]}

Siener, Catherine A. ^{[1
]}

Wulf, Charlie O. ^{[1
]}

Anand, Pallavi ^{[1
]}

Golumbek, Paul T. ^{[1
,2
]}

Zaidman, Craig M. ^{[1
,2
]}

Miller, J. Philip ^{[3
]}

Lowes, Linda P. ^{[4
]}

Alfano, Lindsay N. ^{[4
]}

Viollet-Callendret, Laurence ^{[4
]}

Flanigan, Kevin M. ^{[4
]}

Mendell, Jerry R. ^{[4
]}

McDonald, Craig M. ^{[5
]}

Goude, Erica ^{[5
]}

Johnson, Linda ^{[5
]}

Nicorici, Alina ^{[5
]}

Karachunski, Peter I. ^{[6
]}

Day, John W. ^{[6
]}

Dalton, Joline C. ^{[6
]}

Farber, Janey M. ^{[6
]}

Buser, Karen K. ^{[6
]}

Darras, Basil T. ^{[7
]}

Kang, Peter B. ^{[7
]}

Riley, Susan O. ^{[7
]}

Shriber, Elizabeth ^{[7
]}

Parad, Rebecca ^{[7
]}

Bushby, Kate ^{[8
,9
]}

Eagle, Michelle ^{[8
,9
]}

机构：

[1] Washington Univ, Sch Med, Dept Neurol, St Louis, MO 63110 USA

[2] Washington Univ, Sch Med, Dept Pediat, St Louis, MO 63110 USA

[3] Washington Univ, Sch Med, Div Biostat, St Louis, MO 63110 USA

[4] Ohio State Univ, Dept Pediat, Nationwide Childrens Hosp, Columbus, OH 43210 USA

[5] Univ Calif Davis, Davis Med Ctr, Dept Phys Med & Rehabil, Sacramento, CA 95817 USA

[6] Univ Minnesota, Dept Neurol, Minneapolis, MN 55455 USA

[7] Harvard Univ, Boston Childrens Hosp, Dept Neurol, Boston, MA 02115 USA

[8] Dept Neurol, Newcastle Upon Tyne, Tyne & Wear, England

[9] Inst Med Genet, Newcastle Upon Tyne, Tyne & Wear, England

来源：

NEUROMUSCULAR DISORDERS | 2013年 / 23卷 / 07期

基金：

美国国家卫生研究院;

关键词：

Duchenne Muscular Dystrophy; Bayley III Scales of Infant and Toddler Development; Hammersmith Functional Motor Scale Extended; North Star Ambulatory Assessment; Infant; 6-MINUTE WALK TEST; STAR AMBULATORY ASSESSMENT; NATURAL-HISTORY; RELIABILITY; SCALE; CHILDREN; ATROPHY; TRIALS; STRENGTH; PREDNISONE;

D O I：

10.1016/j.nmd.2013.04.005

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

100204 [神经病学];

摘要：

Therapeutic trials in Duchenne Muscular Dystrophy (DMD) exclude young boys because traditional outcome measures rely on cooperation. The Bayley III Scales of Infant and Toddler Development (Bayley III) have been validated in developing children and those with developmental disorders but have not been studied in DMD. Expanded Hammersmith Functional Motor Scale (HFMSE) and North Star Ambulatory Assessment (NSAA) may also be useful in this young DMD population. Clinical evaluators from the MDA-DMD Clinical Research Network were trained in these assessment tools. Infants and boys with DMD (n = 24; 1.9 +/- 0.7 years) were assessed. The mean Bayley III motor composite score was low (82.8 +/- 8; p <= .0001) (normal = 100 +/- 15). Mean gross motor and fine motor function scaled scores were low (both p <= .0001). The mean cognitive comprehensive (p = .0002), receptive language (p <= .0001), and expressive language (p = .0001) were also low compared to normal children. Age was negatively associated with Bayley III gross motor (r = -0.44; p = .02) but not with fine motor, cognitive, or language scores. HFMSE (n = 23) showed a mean score of 31 +/- 13. NSAA (n = 18 boys; 2.2 +/- 0.4 years) showed a mean score of 12 +/- 5. Outcome assessments of young boys with DMD are feasible and in this multicenter study were best demonstrated using the Bayley III. (C) 2013 Elsevier B.V. All rights reserved.

引用

页码：529 / 539

页数：11

共 59 条

[1]

The effects of iron deficiency on infants' developmental test performance [J].

Akman, M ;

Cebeci, D ;

Okur, V ;

Angin, H ;

Abali, O ;

Akman, AC .

ACTA PAEDIATRICA, 2004, 93 (10) :1391-1396

[2]

Almeida KM, 2008, J PEDIAT-BRAZIL, V84, P442, DOI [10.2223/JPED.1836, 10.1590/S0021-75572008000600011]

[3]

[Anonymous], 2005, BAYLEY SCALES INFANT

[4]

Baillieu Nicole, 2008, J Neurol Phys Ther, V32, P118, DOI 10.1097/NPT.0b013e3181846232

[5]

A COMPARATIVE-STUDY OF PROFOUNDLY MULTIPLY HANDICAPPED CHILDRENS SCORES ON THE BAYLEY AND THE GRIFFITHS DEVELOPMENTAL SCALES [J].

BEAIL, N .

CHILD CARE HEALTH AND DEVELOPMENT, 1985, 11 (01) :31-36

[6]

DEVELOPMENT OF A QUALITY-OF-MOVEMENT MEASURE FOR CHILDREN WITH CEREBRAL-PALSY [J].

BOYCE, WF ;

GOWLAND, C ;

HARDY, S ;

ROSENBAUM, PL ;

LANE, M ;

PLEWS, N ;

GOLDSMITH, C ;

RUSSELL, DJ .

PHYSICAL THERAPY, 1991, 71 (11) :820-832

[7]

CLINICAL INVESTIGATION OF DUCHENNE MUSCULAR-DYSTROPHY - INTERESTING RESULTS IN A TRIAL OF PREDNISONE [J].

BROOKE, MH ;

FENICHEL, GM ;

GRIGGS, RC ;

MENDELL, JR ;

MOXLEY, RT ;

MILLER, JP ;

KAISER, KK ;

FLORENCE, JM ;

PANDYA, S ;

SIGNORE, L ;

KING, W ;

ROBISON, J ;

HEAD, RA ;

PROVINCE, MA ;

SEYFRIED, W ;

MANDEL, S .

ARCHIVES OF NEUROLOGY, 1987, 44 (08) :812-817

[8]

HYPOXANTHINE AND MCARDLE DISEASE - A CLUE TO METABOLIC STRESS IN THE WORKING FOREARM [J].

BROOKE, MH ;

PATTERSON, VH ;

KAISER, KK .

MUSCLE & NERVE, 1983, 6 (03) :204-206

[9]

DUCHENNE MUSCULAR-DYSTROPHY - PATTERNS OF CLINICAL PROGRESSION AND EFFECTS OF SUPPORTIVE THERAPY [J].

BROOKE, MH ;

FENICHEL, GM ;

GRIGGS, RC ;

MENDELL, JR ;

MOXLEY, R ;

FLORENCE, J ;

KING, WM ;

PANDYA, S ;

ROBISON, J ;

SCHIERBECKER, J ;

SIGNORE, L ;

MILLER, JP ;

GILDER, BF ;

KAISER, KK ;

MANDEL, S ;

ARFKEN, C .

NEUROLOGY, 1989, 39 (04) :475-481

[10]

BROOKE MH, 1981, T AM NEUROL ASSOC, V106, P195

← 1 2 3 4 5 6 →