Timed rise from floor as a predictor of disease progression in Duchenne muscular dystrophy: An observational study

E. S. Mazzone, Giorgia Coratti, M. P. Sormani, S. Messina, M. Pane, A. D'Amico, G. Colia, L. Fanelli, Angela Lucia Berardinelli, A. Gardani, V. Lanzillotta, P. D'Ambrosio, Roberta Petillo, F. Cavallaro, S. Frosini, L. Bello, S. Bonfiglio, R. De Sanctis, Enrica Rolle, Nicola ForcinaF. Magri, G. Vita, Concetta Palermo, M.A. Donati, Elena Procopio, M.T. Arnoldi, G. Baranello, T. Mongini, A. Pini, R. Battini, E. Pegoraro, Y. Torrente, S.C. Previtali, C. Bruno, L. Politano, G.P. Comi, M.G. D'Angelo, E. Bertini, E. Mercuri

Research output: Contribution to journal › Article

Abstract

Background: The role of timed items, and more specifically, of the time to rise from the floor, has been reported as an early prognostic factor for disease progression and loss of ambulation. The aim of our study was to investigate the possible effect of the time to rise from the floor test on the changes observed on the 6MWT over 12 months in a cohort of ambulant Duchenne boys. Subjects and methods: A total of 487 12-month data points were collected from 215 ambulant Duchenne boys. The age ranged between 5.0 and 20.0 years (mean 8.48 ±2.48 DS). Results: The results of the time to rise from the floor at baseline ranged from 1.2 to 29.4 seconds in the boys who could perform the test. 49 patients were unable to perform the test at baseline and 87 at 12 month The 6MWT values ranged from 82 to 567 meters at baseline. 3 patients lost the ability to perform the 6mwt at 12 months. The correlation between time to rise from the floor and 6MWT at baseline was high (r = 0.6, p
Original languageEnglish
JournalPLoS One
Volume11
Issue number3
DOIs
Publication statusPublished - 2016

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muscular dystrophy
Duchenne Muscular Dystrophy
observational studies
disease course
Observational Studies
Disease Progression
testing
Walking
methodology

Keywords

  • adolescent
  • adult
  • Article
  • building
  • child
  • cohort analysis
  • disease course
  • Duchenne muscular dystrophy
  • exercise test
  • human
  • Italy
  • major clinical study
  • male
  • observational study
  • prediction
  • prospective study
  • six minute walk test
  • time
  • walking difficulty

Cite this

Timed rise from floor as a predictor of disease progression in Duchenne muscular dystrophy: An observational study. / Mazzone, E. S.; Coratti, Giorgia; Sormani, M. P.; Messina, S.; Pane, M.; D'Amico, A.; Colia, G.; Fanelli, L.; Berardinelli, Angela Lucia; Gardani, A.; Lanzillotta, V.; D'Ambrosio, P.; Petillo, Roberta; Cavallaro, F.; Frosini, S.; Bello, L.; Bonfiglio, S.; De Sanctis, R.; Rolle, Enrica; Forcina, Nicola; Magri, F.; Vita, G.; Palermo, Concetta; Donati, M.A.; Procopio, Elena; Arnoldi, M.T.; Baranello, G.; Mongini, T.; Pini, A.; Battini, R.; Pegoraro, E.; Torrente, Y.; Previtali, S.C.; Bruno, C.; Politano, L.; Comi, G.P.; D'Angelo, M.G.; Bertini, E.; Mercuri, E.

In: PLoS One, Vol. 11, No. 3, 2016.

Research output: Contribution to journal › Article

Mazzone, ES, Coratti, G, Sormani, MP, Messina, S, Pane, M, D'Amico, A, Colia, G, Fanelli, L, Berardinelli, AL, Gardani, A, Lanzillotta, V, D'Ambrosio, P, Petillo, R, Cavallaro, F, Frosini, S, Bello, L, Bonfiglio, S, De Sanctis, R, Rolle, E, Forcina, N, Magri, F, Vita, G, Palermo, C, Donati, MA, Procopio, E, Arnoldi, MT, Baranello, G, Mongini, T, Pini, A, Battini, R, Pegoraro, E, Torrente, Y, Previtali, SC, Bruno, C, Politano, L, Comi, GP, D'Angelo, MG, Bertini, E & Mercuri, E 2016, 'Timed rise from floor as a predictor of disease progression in Duchenne muscular dystrophy: An observational study', PLoS One, vol. 11, no. 3. https://doi.org/10.1371/journal.pone.0151445
Mazzone ES, Coratti G, Sormani MP, Messina S, Pane M, D'Amico A et al. Timed rise from floor as a predictor of disease progression in Duchenne muscular dystrophy: An observational study. PLoS One. 2016;11(3). https://doi.org/10.1371/journal.pone.0151445
Mazzone, E. S. ; Coratti, Giorgia ; Sormani, M. P. ; Messina, S. ; Pane, M. ; D'Amico, A. ; Colia, G. ; Fanelli, L. ; Berardinelli, Angela Lucia ; Gardani, A. ; Lanzillotta, V. ; D'Ambrosio, P. ; Petillo, Roberta ; Cavallaro, F. ; Frosini, S. ; Bello, L. ; Bonfiglio, S. ; De Sanctis, R. ; Rolle, Enrica ; Forcina, Nicola ; Magri, F. ; Vita, G. ; Palermo, Concetta ; Donati, M.A. ; Procopio, Elena ; Arnoldi, M.T. ; Baranello, G. ; Mongini, T. ; Pini, A. ; Battini, R. ; Pegoraro, E. ; Torrente, Y. ; Previtali, S.C. ; Bruno, C. ; Politano, L. ; Comi, G.P. ; D'Angelo, M.G. ; Bertini, E. ; Mercuri, E. / Timed rise from floor as a predictor of disease progression in Duchenne muscular dystrophy: An observational study. In: PLoS One. 2016 ; Vol. 11, No. 3.
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title = "Timed rise from floor as a predictor of disease progression in Duchenne muscular dystrophy: An observational study",
abstract = "Background: The role of timed items, and more specifically, of the time to rise from the floor, has been reported as an early prognostic factor for disease progression and loss of ambulation. The aim of our study was to investigate the possible effect of the time to rise from the floor test on the changes observed on the 6MWT over 12 months in a cohort of ambulant Duchenne boys. Subjects and methods: A total of 487 12-month data points were collected from 215 ambulant Duchenne boys. The age ranged between 5.0 and 20.0 years (mean 8.48 ±2.48 DS). Results: The results of the time to rise from the floor at baseline ranged from 1.2 to 29.4 seconds in the boys who could perform the test. 49 patients were unable to perform the test at baseline and 87 at 12 month The 6MWT values ranged from 82 to 567 meters at baseline. 3 patients lost the ability to perform the 6mwt at 12 months. The correlation between time to rise from the floor and 6MWT at baseline was high (r = 0.6, p",
keywords = "adolescent, adult, Article, building, child, cohort analysis, disease course, Duchenne muscular dystrophy, exercise test, human, Italy, major clinical study, male, observational study, prediction, prospective study, six minute walk test, time, walking difficulty",
author = "Mazzone, {E. S.} and Giorgia Coratti and Sormani, {M. P.} and S. Messina and M. Pane and A. D'Amico and G. Colia and L. Fanelli and Berardinelli, {Angela Lucia} and A. Gardani and V. Lanzillotta and P. D'Ambrosio and Roberta Petillo and F. Cavallaro and S. Frosini and L. Bello and S. Bonfiglio and {De Sanctis}, R. and Enrica Rolle and Nicola Forcina and F. Magri and G. Vita and Concetta Palermo and M.A. Donati and Elena Procopio and M.T. Arnoldi and G. Baranello and T. Mongini and A. Pini and R. Battini and E. Pegoraro and Y. Torrente and S.C. Previtali and C. Bruno and L. Politano and G.P. Comi and M.G. D'Angelo and E. Bertini and E. Mercuri",
note = "Cited By :1 Export Date: 27 March 2017 CODEN: POLNC References: McDonald, C.M., Henricson, E.K., Abresch, R.T., Florence, J.M., Eagle, M., Gappmaier, E., THE 6-minute walk test and other endpoints in Duchenne muscular dystrophy: Longitudinal natural history observations over 48 weeks from a multicenter study (2013) Muscle Nerve, 48, pp. 343-356. , PMID: 23681930; McDonald, C.M., Henricson, E.K., Han, J.J., Abresch, R.T., Nicorici, A., Atkinson, L., The 6-minute walk test in Duchenne/Becker muscular dystrophy: Longitudinal observations (2010) Muscle Nerve, 42, pp. 966-974. , PMID: 21038378; Mazzone, E., Vasco, G., Sormani, M.P., Torrente, Y., Berardinelli, A., Messina, S., Functional changes in Duchenne muscular dystrophy: A 12-month longitudinal cohort study (2011) Neurology, 77, pp. 250-256. , PMID: 21734183; Bushby, K., Finkel, R., Wong, B., Barohn, R., Campbell, C., Comi, G.P., Ataluren treatment of patients with nonsense mutation dystrophinopathy (2014) Muscle Nerve, 50, pp. 477-487. , PMID: 25042182; Goemans, N., Klingels, K., Van Den Hauwe, M., Van Orshoven, A., Vanpraet, S., Feys, H., Test-retest reliability and developmental evolution of the 6-min walk test in Caucasian boys aged 5-12 years (2013) Neuromuscul Disord, 23, pp. 19-24. , PMID: 23137525; Goemans, N., Van Den Hauwe, M., Wilson, R., Van Impe, A., Klingels, K., Buyse, G., Ambulatory capacity and disease progression as measured by the 6-minute-walk-distance in Duchenne muscular dystrophy subjects on daily corticosteroids (2013) Neuromuscul Disord, 23, pp. 618-623. , PMID: 23770101; Mazzone, E.S., Pane, M., Sormani, M.P., Scalise, R., Berardinelli, A., Messina, S., 24 Month longitudinal data in ambulant boys with Duchenne muscular dystrophy (2013) PLoS One, 8. , PMID: 23326337; Pane, M., Mazzone, E.S., Sivo, S., Sormani, M.P., Messina, S., D'Amico, A., Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-Month changes (2014) PLoS One, 9. , PMID: 25271887; Mazzone, E., Martinelli, D., Berardinelli, A., Messina, S., D'Amico, A., Vasco, G., North Star Ambulatory Assessment, 6-minute walk test and timed items in ambulant boys with Duchenne muscular dystrophy (2010) Neuromuscul Disord, 20, pp. 712-716. , PMID: 20634072; McDonald, C.M., Henricson, E.K., Han, J.J., Abresch, R.T., Nicorici, A., Elfring, G.L., The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy (2010) Muscle Nerve, 41, pp. 500-510. , PMID: 19941337; McDonald, C.M., Abresch, R.T., Carter, G.T., Fowler, W.M., Jr., Johnson, E.R., Kilmer, D.D., Profiles of neuromuscular diseases. Duchenne muscular dystrophy (1995) Am J Phys Med Rehabil, 74, pp. S70-S92. , PMID: 7576424; McDonald, C.M., Henricson, E.K., Abresch, R.T., Florence, J., Eagle, M., Gappmaier, E., The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: Reliability, concurrent validity, and minimal clinically important differences from a multicenter study (2013) Muscle Nerve, 48, pp. 343-356. , PMID: 23681930",
year = "2016",
doi = "10.1371/journal.pone.0151445",
language = "English",
volume = "11",
journal = "PLoS One",
issn = "1932-6203",
publisher = "Public Library of Science",
number = "3",

}

TY - JOUR

T1 - Timed rise from floor as a predictor of disease progression in Duchenne muscular dystrophy: An observational study

AU - Mazzone, E. S.

AU - Coratti, Giorgia

AU - Sormani, M. P.

AU - Messina, S.

AU - Pane, M.

AU - D'Amico, A.

AU - Colia, G.

AU - Fanelli, L.

AU - Berardinelli, Angela Lucia

AU - Gardani, A.

AU - Lanzillotta, V.

AU - D'Ambrosio, P.

AU - Petillo, Roberta

AU - Cavallaro, F.

AU - Frosini, S.

AU - Bello, L.

AU - Bonfiglio, S.

AU - De Sanctis, R.

AU - Rolle, Enrica

AU - Forcina, Nicola

AU - Magri, F.

AU - Vita, G.

AU - Palermo, Concetta

AU - Donati, M.A.

AU - Procopio, Elena

AU - Arnoldi, M.T.

AU - Baranello, G.

AU - Mongini, T.

AU - Pini, A.

AU - Battini, R.

AU - Pegoraro, E.

AU - Torrente, Y.

AU - Previtali, S.C.

AU - Bruno, C.

AU - Politano, L.

AU - Comi, G.P.

AU - D'Angelo, M.G.

AU - Bertini, E.

AU - Mercuri, E.

N1 - Cited By :1 Export Date: 27 March 2017 CODEN: POLNC References: McDonald, C.M., Henricson, E.K., Abresch, R.T., Florence, J.M., Eagle, M., Gappmaier, E., THE 6-minute walk test and other endpoints in Duchenne muscular dystrophy: Longitudinal natural history observations over 48 weeks from a multicenter study (2013) Muscle Nerve, 48, pp. 343-356. , PMID: 23681930; McDonald, C.M., Henricson, E.K., Han, J.J., Abresch, R.T., Nicorici, A., Atkinson, L., The 6-minute walk test in Duchenne/Becker muscular dystrophy: Longitudinal observations (2010) Muscle Nerve, 42, pp. 966-974. , PMID: 21038378; Mazzone, E., Vasco, G., Sormani, M.P., Torrente, Y., Berardinelli, A., Messina, S., Functional changes in Duchenne muscular dystrophy: A 12-month longitudinal cohort study (2011) Neurology, 77, pp. 250-256. , PMID: 21734183; Bushby, K., Finkel, R., Wong, B., Barohn, R., Campbell, C., Comi, G.P., Ataluren treatment of patients with nonsense mutation dystrophinopathy (2014) Muscle Nerve, 50, pp. 477-487. , PMID: 25042182; Goemans, N., Klingels, K., Van Den Hauwe, M., Van Orshoven, A., Vanpraet, S., Feys, H., Test-retest reliability and developmental evolution of the 6-min walk test in Caucasian boys aged 5-12 years (2013) Neuromuscul Disord, 23, pp. 19-24. , PMID: 23137525; Goemans, N., Van Den Hauwe, M., Wilson, R., Van Impe, A., Klingels, K., Buyse, G., Ambulatory capacity and disease progression as measured by the 6-minute-walk-distance in Duchenne muscular dystrophy subjects on daily corticosteroids (2013) Neuromuscul Disord, 23, pp. 618-623. , PMID: 23770101; Mazzone, E.S., Pane, M., Sormani, M.P., Scalise, R., Berardinelli, A., Messina, S., 24 Month longitudinal data in ambulant boys with Duchenne muscular dystrophy (2013) PLoS One, 8. , PMID: 23326337; Pane, M., Mazzone, E.S., Sivo, S., Sormani, M.P., Messina, S., D'Amico, A., Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-Month changes (2014) PLoS One, 9. , PMID: 25271887; Mazzone, E., Martinelli, D., Berardinelli, A., Messina, S., D'Amico, A., Vasco, G., North Star Ambulatory Assessment, 6-minute walk test and timed items in ambulant boys with Duchenne muscular dystrophy (2010) Neuromuscul Disord, 20, pp. 712-716. , PMID: 20634072; McDonald, C.M., Henricson, E.K., Han, J.J., Abresch, R.T., Nicorici, A., Elfring, G.L., The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy (2010) Muscle Nerve, 41, pp. 500-510. , PMID: 19941337; McDonald, C.M., Abresch, R.T., Carter, G.T., Fowler, W.M., Jr., Johnson, E.R., Kilmer, D.D., Profiles of neuromuscular diseases. Duchenne muscular dystrophy (1995) Am J Phys Med Rehabil, 74, pp. S70-S92. , PMID: 7576424; McDonald, C.M., Henricson, E.K., Abresch, R.T., Florence, J., Eagle, M., Gappmaier, E., The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: Reliability, concurrent validity, and minimal clinically important differences from a multicenter study (2013) Muscle Nerve, 48, pp. 343-356. , PMID: 23681930

PY - 2016

Y1 - 2016

N2 - Background: The role of timed items, and more specifically, of the time to rise from the floor, has been reported as an early prognostic factor for disease progression and loss of ambulation. The aim of our study was to investigate the possible effect of the time to rise from the floor test on the changes observed on the 6MWT over 12 months in a cohort of ambulant Duchenne boys. Subjects and methods: A total of 487 12-month data points were collected from 215 ambulant Duchenne boys. The age ranged between 5.0 and 20.0 years (mean 8.48 ±2.48 DS). Results: The results of the time to rise from the floor at baseline ranged from 1.2 to 29.4 seconds in the boys who could perform the test. 49 patients were unable to perform the test at baseline and 87 at 12 month The 6MWT values ranged from 82 to 567 meters at baseline. 3 patients lost the ability to perform the 6mwt at 12 months. The correlation between time to rise from the floor and 6MWT at baseline was high (r = 0.6, p

AB - Background: The role of timed items, and more specifically, of the time to rise from the floor, has been reported as an early prognostic factor for disease progression and loss of ambulation. The aim of our study was to investigate the possible effect of the time to rise from the floor test on the changes observed on the 6MWT over 12 months in a cohort of ambulant Duchenne boys. Subjects and methods: A total of 487 12-month data points were collected from 215 ambulant Duchenne boys. The age ranged between 5.0 and 20.0 years (mean 8.48 ±2.48 DS). Results: The results of the time to rise from the floor at baseline ranged from 1.2 to 29.4 seconds in the boys who could perform the test. 49 patients were unable to perform the test at baseline and 87 at 12 month The 6MWT values ranged from 82 to 567 meters at baseline. 3 patients lost the ability to perform the 6mwt at 12 months. The correlation between time to rise from the floor and 6MWT at baseline was high (r = 0.6, p

KW - adolescent

KW - adult

KW - Article

KW - building

KW - child

KW - cohort analysis

KW - disease course

KW - Duchenne muscular dystrophy

KW - exercise test

KW - human

KW - Italy

KW - major clinical study

KW - male

KW - observational study

KW - prediction

KW - prospective study

KW - six minute walk test

KW - time

KW - walking difficulty

U2 - 10.1371/journal.pone.0151445

DO - 10.1371/journal.pone.0151445

M3 - Article

VL - 11

JO - PLoS One

JF - PLoS One

SN - 1932-6203

IS - 3

ER -

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