24 Month Longitudinal Data in Ambulant Boys with Duchenne Muscular Dystrophy

被引：96

作者：

Mazzone, Elena Stacy ^{[1
]}

Pane, Marika ^{[1
]}

Sormani, Maria Pia ^{[2
]}

Scalise, Roberta ^{[1
]}

Berardinelli, Angela ^{[3
]}

Messina, Sonia ^{[4
]}

Torrente, Yvan ^{[5
]}

D'Amico, Adele ^{[6
]}

Doglio, Luca ^{[7
]}

Viggiano, Emanuela ^{[8
]}

D'Ambrosio, Paola ^{[8
]}

Cavallaro, Filippo ^{[4
]}

Frosini, Silvia ^{[9
]}

Bello, Luca ^{[10
]}

Bonfiglio, Serena ^{[11
]}

De Sanctis, Roberto ^{[1
]}

Rolle, Enrica ^{[12
]}

Bianco, Flaviana ^{[1
]}

Magri, Francesca ^{[5
]}

Rossi, Francesca ^{[13
]}

Vasco, Gessica ^{[1
]}

Vita, GianLuca ^{[4
]}

Motta, Maria Chiara ^{[3
]}

Donati, Maria Alice ^{[13
]}

Sacchini, Michele ^{[13
]}

Mongini, Tiziana ^{[12
]}

Pini, Antonella ^{[11
]}

Battini, Roberta ^{[9
]}

Pegoraro, Elena ^{[10
]}

Previtali, Stefano ^{[14
]}

Napolitano, Sara ^{[14
]}

Bruno, Claudio ^{[7
]}

Politano, Luisa ^{[8
]}

Comi, Giacomo Pietro ^{[5
]}

Bertini, Enrico ^{[6
]}

Mercuri, Eugenio ^{[1
]}

机构：

[1] Univ Cattolica Sacro Cuore, Dept Paediat Neurol, Rome, Italy

[2] Univ Genoa, Dept Hlth Sci, Biostat Unit, Genoa, Italy

[3] C Mondino Fdn, Child Neurol & Psychiat Unit, Pavia, Italy

[4] Univ Messina, Dept Neurosci Psychiat & Anaesthesiol, Messina, Italy

[5] Univ Milan, Dept Pathophysiol & Transplantat DEPT, Ca Granda Osped Maggiore Policlin, Dino Ferrari Ctr,Neurosci Sect,Neurol Unit, Milan, Italy

[6] Bambino Gesu Pediat Hosp, Mol Med Unit, Dept Lab Med, Rome, Italy

[7] G Gaslini Inst Children, Neuromuscular Dis Unit, Genoa, Italy

[8] Univ Naples 2, Dipartimento Med Sperimentale, Naples, Italy

[9] Univ Pisa, Stella Maris Inst, Dept Dev Neurosci, Pisa, Italy

[10] Univ Padua, Dept Neurosci, Padua, Italy

[11] Maggiore Hosp, Child Neurol & Psychiat Unit, Bologna, Italy

[12] Univ Turin, Neuromuscular Ctr, SG Battista Hosp, Turin, Italy

[13] Meyer Hosp, Metab & Neuromuscular Unit, Florence, Italy

[14] Ist Sci San Raffaele, Dept Neurol, I-20132 Milan, Italy

来源：

PLOS ONE | 2013年 / 8卷 / 01期

关键词：

6-MINUTE WALK TEST;

D O I：

10.1371/journal.pone.0052512

中图分类号：

O [数理科学和化学]; P [天文学、地球科学]; Q [生物科学]; N [自然科学总论];

学科分类号：

070301 [无机化学]; 070403 [天体物理学]; 070507 [自然资源与国土空间规划学]; 090105 [作物生产系统与生态工程];

摘要：

Objectives: The aim of the study was i) to assess the spectrum of changes over 24 months in ambulant boys affected by Duchenne muscular dystrophy, ii) to establish the difference between the first and the second year results and iii) to identify possible early markers of loss of ambulation. Methods: One hundred and thirteen patients (age range 4.1-17, mean 8.2) fulfilled the inclusion criteria, 67 of the 113 were on daily and 40 on intermittent steroids, while 6 were not on steroids. All were assessed using the 6 Minute Walk Test (6MWT), the North Star Ambulatory Assessment (NSAA) and timed test. Results: On the 6MWT there was an average overall decline of -22.7 (SD 81.0) in the first year and of -64.7 (SD 123.1) in the second year. On the NSAA the average overall decline was of -1.86 (SD 4.21) in the first year and of -2.98 (SD 5.19) in the second year. Fourteen children lost ambulation, one in the first year and the other 13 in the second year of the study. A distance of at least 330 meters on the 6MWT, or a NSAA score of 18 at baseline reduced significantly the risk of losing ambulation within 2 years. Conclusions: These results can be of help at the time of using inclusion criteria for a study in ambulant patients in order to minimize the risk of patients who may lose ambulation within the time of the trial.

引用

页数：6

共 10 条

[1]

Bushby Kate, 2011, Clin Investig (Lond), V1, P1217

[2]

ATS statement: Guidelines for the six-minute walk test [J].

Crapo, RO ;

Casaburi, R ;

Coates, AL ;

Enright, PL ;

MacIntyre, NR ;

McKay, RT ;

Johnson, D ;

Wanger, JS ;

Zeballos, RJ ;

Bittner, V ;

Mottram, C .

AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE, 2002, 166 (01) :111-117

[3]

Henricson E, PLOS CURR, V4

[4]

Moving towards meaningful measurement: Rasch analysis of the North Star Ambulatory Assessment in Duchenne muscular dystrophy [J].

Mayhew, Anna ;

Cano, Stefan ;

Scott, Elaine ;

Eagle, Michelle ;

Bushby, Kate ;

Muntoni, Francesco .

DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY, 2011, 53 (06) :535-542

[5]

Functional changes in Duchenne muscular dystrophy A 12-month longitudinal cohort study [J].

Mazzone, E. ;

Vasco, G. ;

Sormani, M. P. ;

Torrente, Y. ;

Berardinelli, A. ;

Messina, S. ;

D'Amico, A. ;

Doglio, L. ;

Politano, L. ;

Cavallaro, F. ;

Frosini, S. ;

Bello, L. ;

Bonfiglio, S. ;

Zucchini, E. ;

De Sanctis, R. ;

Scutifero, M. ;

Bianco, F. ;

Rossi, F. ;

Motta, M. C. ;

Sacco, A. ;

Donati, M. A. ;

Mongini, T. ;

Pini, A. ;

Battini, R. ;

Pegoraro, E. ;

Pane, M. ;

Gasperini, S. ;

Previtali, S. ;

Napolitano, S. ;

Martinelli, D. ;

Bruno, C. ;

Vita, G. ;

Comi, G. ;

Bertini, E. ;

Mercuri, E. .

NEUROLOGY, 2011, 77 (03) :250-256

[6]

Reliability of the North Star Ambulatory Assessment in a multicentric setting [J].

Mazzone, E. S. ;

Messina, S. ;

Vasco, G. ;

Main, M. ;

Eagle, M. ;

D'Amico, A. ;

Doglio, L. ;

Politano, L. ;

Cavallaro, F. ;

Frosini, S. ;

Bello, L. ;

Magri, F. ;

Corlatti, A. ;

Zucchini, E. ;

Brancalion, B. ;

Rossi, F. ;

Ferretti, M. ;

Motta, M. G. ;

Cecio, M. R. ;

Berardinelli, A. ;

Alfieri, P. ;

Mongini, T. ;

Pini, A. ;

Astrea, G. ;

Battini, R. ;

Comi, G. ;

Pegoraro, E. ;

Morandi, L. ;

Pane, M. ;

Angelini, C. ;

Bruno, C. ;

Villanova, M. ;

Vita, G. ;

Donati, M. A. ;

Bertini, E. ;

Mercuri, E. .

NEUROMUSCULAR DISORDERS, 2009, 19 (07) :458-461

[7]

North Star Ambulatory Assessment, 6-minute walk test and timed items in ambulant boys with Duchenne muscular dystrophy [J].

Mazzone, Elena ;

Martinelli, Diego ;

Berardinelli, Angela ;

Messina, Sonia ;

D'Amico, Adele ;

Vasco, Gessica ;

Main, Marion ;

Doglio, Luca ;

Politano, Luisa ;

Cavallaro, Filippo ;

Frosini, Silvia ;

Bello, Luca ;

Carlesi, Adelina ;

Bonetti, Anna Maria ;

Zucchini, Elisabetta ;

De Sanctis, Roberto ;

Scutifero, Marianna ;

Bianco, Flaviana ;

Rossi, Francesca ;

Motta, Maria Chiara ;

Sacco, Annalisa ;

Donati, Maria Alice ;

Mongini, Tiziana ;

Pini, Antonella ;

Battini, Roberta ;

Pegoraro, Elena ;

Pane, Marika ;

Pasquini, Elisabetta ;

Bruno, Claudio ;

Vita, Giuseppe ;

de Waure, Chiara ;

Bertini, Enrico ;

Mercuri, Eugenio .

NEUROMUSCULAR DISORDERS, 2010, 20 (11) :712-716

[8]

MCDONALD CM, 1995, AM J PHYS MED REHAB, V74, pS70, DOI 10.1097/00002060-199509001-00003

[9]

THE 6-MINUTE WALK TEST AS A NEW OUTCOME MEASURE IN DUCHENNE MUSCULAR DYSTROPHY [J].

McDonald, Craig M. ;

Henricson, Erik K. ;

Han, Jay J. ;

Abresch, R. Ted ;

Nicorici, Alina ;

Elfring, Gary L. ;

Atkinson, Leone ;

Reha, Allen ;

Hirawat, Samit ;

Miller, Langdon L. .

MUSCLE & NERVE, 2010, 41 (04) :500-510

[10]

Scott E, 2011, PHYSIOTHER RES INT

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