24 month longitudinal data in ambulant boys with Duchenne muscular dystrophy. 2013

Elena Stacy Mazzone, and Marika Pane, and Maria Pia Sormani, and Roberta Scalise, and Angela Berardinelli, and Sonia Messina, and Yvan Torrente, and Adele D'Amico, and Luca Doglio, and Emanuela Viggiano, and Paola D'Ambrosio, and Filippo Cavallaro, and Silvia Frosini, and Luca Bello, and Serena Bonfiglio, and Roberto De Sanctis, and Enrica Rolle, and Flaviana Bianco, and Francesca Magri, and Francesca Rossi, and Gessica Vasco, and Gianluca Vita, and Maria Chiara Motta, and Maria Alice Donati, and Michele Sacchini, and Tiziana Mongini, and Antonella Pini, and Roberta Battini, and Elena Pegoraro, and Stefano Previtali, and Sara Napolitano, and Claudio Bruno, and Luisa Politano, and Giacomo Pietro Comi, and Enrico Bertini, and Eugenio Mercuri
Department of Paediatric Neurology, Catholic University, Rome, Italy.

OBJECTIVE 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.

UI MeSH Term Description Entries
D008137 Longitudinal Studies Studies in which variables relating to an individual or group of individuals are assessed over a period of time. Bogalusa Heart Study,California Teachers Study,Framingham Heart Study,Jackson Heart Study,Longitudinal Survey,Tuskegee Syphilis Study,Bogalusa Heart Studies,California Teachers Studies,Framingham Heart Studies,Heart Studies, Bogalusa,Heart Studies, Framingham,Heart Studies, Jackson,Heart Study, Bogalusa,Heart Study, Framingham,Heart Study, Jackson,Jackson Heart Studies,Longitudinal Study,Longitudinal Surveys,Studies, Bogalusa Heart,Studies, California Teachers,Studies, Jackson Heart,Studies, Longitudinal,Study, Bogalusa Heart,Study, California Teachers,Study, Longitudinal,Survey, Longitudinal,Surveys, Longitudinal,Syphilis Studies, Tuskegee,Syphilis Study, Tuskegee,Teachers Studies, California,Teachers Study, California,Tuskegee Syphilis Studies
D008297 Male Males
D011241 Prednisone A synthetic anti-inflammatory glucocorticoid derived from CORTISONE. It is biologically inert and converted to PREDNISOLONE in the liver. Dehydrocortisone,delta-Cortisone,Apo-Prednisone,Cortan,Cortancyl,Cutason,Dacortin,Decortin,Decortisyl,Deltasone,Encorton,Encortone,Enkortolon,Kortancyl,Liquid Pred,Meticorten,Orasone,Panafcort,Panasol,Predni Tablinen,Prednidib,Predniment,Prednison Acsis,Prednison Galen,Prednison Hexal,Pronisone,Rectodelt,Sone,Sterapred,Ultracorten,Winpred,Acsis, Prednison
D011282 Pregnenediones Unsaturated pregnane derivatives containing two keto groups on side chains or ring structures. Diketopregnenes,Dioxopregnenes
D002648 Child A person 6 to 12 years of age. An individual 2 to 5 years old is CHILD, PRESCHOOL. Children
D002675 Child, Preschool A child between the ages of 2 and 5. Children, Preschool,Preschool Child,Preschool Children
D005080 Exercise Test Controlled physical activity which is performed in order to allow assessment of physiological functions, particularly cardiovascular and pulmonary, but also aerobic capacity. Maximal (most intense) exercise is usually required but submaximal exercise is also used. Arm Ergometry Test,Bicycle Ergometry Test,Cardiopulmonary Exercise Testing,Exercise Testing,Step Test,Stress Test,Treadmill Test,Cardiopulmonary Exercise Test,EuroFit Tests,Eurofit Test Battery,European Fitness Testing Battery,Fitness Testing,Physical Fitness Testing,Arm Ergometry Tests,Bicycle Ergometry Tests,Cardiopulmonary Exercise Tests,Ergometry Test, Arm,Ergometry Test, Bicycle,Ergometry Tests, Arm,Ergometry Tests, Bicycle,EuroFit Test,Eurofit Test Batteries,Exercise Test, Cardiopulmonary,Exercise Testing, Cardiopulmonary,Exercise Tests,Exercise Tests, Cardiopulmonary,Fitness Testing, Physical,Fitness Testings,Step Tests,Stress Tests,Test Battery, Eurofit,Test, Arm Ergometry,Test, Bicycle Ergometry,Test, Cardiopulmonary Exercise,Test, EuroFit,Test, Exercise,Test, Step,Test, Stress,Test, Treadmill,Testing, Cardiopulmonary Exercise,Testing, Exercise,Testing, Fitness,Testing, Physical Fitness,Tests, Arm Ergometry,Tests, Bicycle Ergometry,Tests, Cardiopulmonary Exercise,Tests, EuroFit,Tests, Exercise,Tests, Step,Tests, Stress,Tests, Treadmill,Treadmill Tests
D005938 Glucocorticoids A group of CORTICOSTEROIDS that affect carbohydrate metabolism (GLUCONEOGENESIS, liver glycogen deposition, elevation of BLOOD SUGAR), inhibit ADRENOCORTICOTROPIC HORMONE secretion, and possess pronounced anti-inflammatory activity. They also play a role in fat and protein metabolism, maintenance of arterial blood pressure, alteration of the connective tissue response to injury, reduction in the number of circulating lymphocytes, and functioning of the central nervous system. Glucocorticoid,Glucocorticoid Effect,Glucorticoid Effects,Effect, Glucocorticoid,Effects, Glucorticoid
D006801 Humans Members of the species Homo sapiens. Homo sapiens,Man (Taxonomy),Human,Man, Modern,Modern Man
D000293 Adolescent A person 13 to 18 years of age. Adolescence,Youth,Adolescents,Adolescents, Female,Adolescents, Male,Teenagers,Teens,Adolescent, Female,Adolescent, Male,Female Adolescent,Female Adolescents,Male Adolescent,Male Adolescents,Teen,Teenager,Youths

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