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Multicenter Study
. 2014 Oct 1;9(10):e108205.
doi: 10.1371/journal.pone.0108205. eCollection 2014.

Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes

Marika Pane  1 , Elena Stacy Mazzone  1 , Serena Sivo  1 , Maria Pia Sormani  2 , Sonia Messina  3 , Adele D'Amico  4 , Adelina Carlesi  4 , Gianluca Vita  3 , Lavinia Fanelli  1 , Angela Berardinelli  5 , Yvan Torrente  6 , Valentina Lanzillotta  7 , Emanuela Viggiano  8 , Paola D Ambrosio  8 , Filippo Cavallaro  3 , Silvia Frosini  9 , Andrea Barp  10 , Serena Bonfiglio  11 , Roberta Scalise  1 , Roberto De Sanctis  1 , Enrica Rolle  12 , Alessandra Graziano  1 , Francesca Magri  6 , Concetta Palermo  1 , Francesca Rossi  13 , Maria Alice Donati  14 , Michele Sacchini  14 , Maria Teresa Arnoldi  13 , Giovanni Baranello  13 , Tiziana Mongini  12 , Antonella Pini  15 , Roberta Battini  9 , Elena Pegoraro  10 , Stefano Previtali  16 , Claudio Bruno  7 , Luisa Politano  8 , Giacomo P Comi  6 , Enrico Bertini  4 , Eugenio Mercuri  1
Affiliations
Multicenter Study

Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes

Marika Pane et al. PLoS One. .

Erratum in

Abstract

The 6 minute walk test has been recently chosen as the primary outcome measure in international multicenter clinical trials in Duchenne muscular dystrophy ambulant patients. The aim of the study was to assess the spectrum of changes at 3 years in the individual measures, their correlation with steroid treatment, age and 6 minute walk test values at baseline. Ninety-six patients from 11 centers were assessed at baseline and 12, 24 and 36 months after baseline using the 6 minute walk test and the North Star Ambulatory Assessment. Three boys (3%) lost the ability to perform the 6 minute walk test within 12 months, another 13 between 12 and 24 months (14%) and 11 between 24 and 36 months (12%). The 6 minute walk test showed an average overall decline of -15.8 (SD 77.3) m at 12 months, of -58.9 (SD 125.7) m at 24 months and -104.22 (SD 146.2) m at 36 months. The changes were significantly different in the two baseline age groups and according to the baseline 6 minute walk test values (below and above 350 m) (p<0.001). The changes were also significantly different according to steroid treatment (p = 0.01). Similar findings were found for the North Star Ambulatory Assessment. These are the first 36 month longitudinal data using the 6 minute walk test and North Star Ambulatory Assessment in Duchenne muscular dystrophy. Our findings will help not only to have a better idea of the progression of the disorder but also provide reference data that can be used to compare with the results of the long term extension studies that are becoming available.

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Conflict of interest statement

Competing Interests: Dr. Pane, Dr. Sivo and E. Mazzone report no disclosures. Dr. Sormani: serves on a scientific advisory board for Biogen Idec.; has received funding for travel or speaker honoraria from Merck Serono; serves as consultant for Merck Serono, Actelion Pharmaceuticals Lts. Biogen Idec, and Synthon; and serves on the speakers' bureaus of Teva Pharmaceutical Industries Ltd., Merck Serono, and Biogen Idec. Dr. D'Amico and Dr. Messina report no disclosures. Dr. Vita serves as an Associate Editor for Neurological Sciences. L. Fanelli, Dr. Berardinelli, Dr. Torrente, V. Lanzillotta, Dr. Viggiano, Dr. D'Ambrosio, F. Cavallaro, S. Frosini, Dr. Barp, Dr. Bonfiglio, Dr. Scalise, R. De Sanctis, E. Rolle, Dr. Graziano, Dr. Magri, Dr. Palermo, F. Rossi, Dr. Donati, Dr. Sacchini, Dr. Arnoldi, Dr. Baranello report no disclosures. Dr. Mongini has served on a scientific advisory board for Telethon Italy; has received funding for travel from Genzyme Corporation; and has received research support from AIFA (Italian Government Drug Agency) and Telethon Italy. Dr. Pini, Dr. Battini report no disclosures. Dr. Pegoraro has served on a scientific advisory board for BioMarin Pharmaceutical Inc.; has received funding for travel from Genzyme Corporation; and has received speaker honoraria from MedaPharma; and receives research support from Wellstone and Telethon Italy. Dr. Previtali, Dr. Bruno, Dr. Politano report no disclosures. Dr. Comi: is site PI for the PTC extension study of Ataluren in DMD, for the GSK study on exon skipping; receives research support from Telethon Italy and SMA Europe. Dr. Bertini is site PI for the PTC extension study of Ataluren in DMD, for the GSK study on exon skipping. He also receives funds from the Italian Telethon, the Italian Ministry of Health and SMA Europe for observational studies on outcome measures. Dr. Mercuri is site PI for the PTC extension study of Ataluren in DMD, for the GSK Prosensa and Sarepta studies on exon skipping. He also receives funds from the Italian Telethon and SMA Europe. He has acted as advisory board for Acceleron Pharma, Shire and PTC Therapeutics, Inc, Prosensa. The disclosures shown in the paper do not alter the authors' adherence to PLOS ONE policies on sharing data and materials.

Figures

Figure 1
Figure 1. 6MWT (left pannel) and NSAA (right pannel) at baseline, 1, 2 and 3 years in DMD boys, below (red) and above (blu) the age of 7 years.
Figure 2
Figure 2. 6MWT (left pannel) and NSAA (right pannel) at baseline, 1, 2 and 3 years in DMD boys, below (green) and above (orange) 350 m of 6MWD.
Figure 3
Figure 3. 6MWT (left pannel) and NSAA (right pannel) at baseline, 1, 2 and 3 years in DMD boys, subdivided according to a combination of age (below and above 7 years) and distance (below and above 350 m of 6MWD).
See this image and copyright information in PMC

References

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