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Exploring physical activity levels and sleep efficiency relationships among boys with Duchenne muscular dystrophy (DMD)
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Abstracts 2017 / Neuromuscular Disorders 27 (2017) S96–S249
were computed. The walking, running and stairs climbing or descending episodes were detected with an estimated accuracy greater than 99.7%. These results show that activity patterns can be recognized and classified in a controlled environment with high accuracy. Validation in non-controlled environment is ongoing. This information could be useful for regulators and funders in drug approval and refunding process in order to provide an objective and clinically meaningful measurement of daily life related activities. http://dx.doi.org/10.1016/j.nmd.2017.06.498
P.459 Hand function in boys and men with Duchenne muscular dystrophy (DMD) M. Hunnekens 1, J. Huijben 2, I. de Groot 2 1 Rehabilitation centre Klimmendaal, Arnhem, Netherlands; 2 Radboudumc, Nijmegen, Netherlands Life expectancy is increasing for persons with DMD. It is of importance to preserve hand function for activities of daily living as long as possible. Not much is known concerning the natural disease course of the hands, therefore developing interventions is as yet difficult. Explorative study, cross-sectional design, to examine the natural disease course of the hands in patients with DMD and to explore relations between the level of body functions/structures and the level of activities, according to the ICF model. Patients: 51 boys/men (age range 5.2–40.0) with established diagnosis of DMD. Strength measures: MyoPinch and MyoGrip. Range of motion by goniometry. Activities: several instruments were used e.g. Brooke, PUL, MFM, 9-HPT, Timed-TIHM and all participants completed a questionnaire containing ABILHAND-plus and CUE. The most obvious decrease of ROM with increasing age was seen in wrist supination, both right and left. Strength increased with age until the age of 10. A decrease was seen thereafter with a more rapid decline around 20 years. On the level of activities great variability was seen on the different tests; some boys around the age of 10 already experienced difficulties. Subtests of the Timed-TIHM could also be performed at older age. Correlations were found i.a. between thumb mobility and activities. Large variation in hand function was seen. In some younger patients disabilities were already present. The use of splints or specific hand therapy is still uncommon, but should be considered. The results of this study give basis for possible interventions. This study was performed within clinical practice; thus some data are missing. Clinical message: It is important to pay already early attention to hand function in DMD. http://dx.doi.org/10.1016/j.nmd.2017.06.499
P.460 Unraveling upper extremity performance in DMD: a biophysical model M. Janssen 1, J. Harlaar 2, H. Koopman 3, I. de Groot 1 1 Radboudumc, Nijmegen, Netherlands; 2 Free University medical centre, Amsterdam, Netherlands; 3 University of Twente, Enschede, Netherlands Upper extremity (UE) activity scales are currently used as the golden standard for defining UE function in non-ambulant Duchenne muscular dystrophy (DMD) patients. These scales, however, are not able to explain the biophysical working mechanism behind changes in UE function. Therefore, we aimed to identify critical physiological outcome variables underlying reduced UE task performance in DMD. These critical variables were used to construct an explanatory biophysical model of the UE working mechanism in DMD. The most critical physiologic outcome variables related to UE task performance (Brooke scale and Performance of Upper Limb (PUL) scale) were maximal active joint angle, followed by maximal muscle torque, maximal muscle activity, echogenicity (fatty infiltration) and maximal passive range of motion. These variables explained more than 90% of the variability in UE task performance. Based on these critical variables a biophysical model was constructed. In this model we considered UE task performance to be mainly dependent on the ability to move your arms actively, which is influenced by both
passive range of motion and the available joint torque. The available joint torque is dependent on muscle capacity, and should be sufficient to lift the weight of the arm against gravity and to overcome passive joint torque (stiffness). Muscle capacity is influenced by both the ability to activate the muscle and the muscle force that is generated at the joint level. This model allows us to understand the underlying cause of UE limitations. Based on this knowledge one can select appropriate interventions to improve UE function, such as arm supports to improve the reachable workspace, contracture prevention, or medication to improve muscle strength. In addition, the model could form the basis for the development of new composite outcome measures for clinical trials. http://dx.doi.org/10.1016/j.nmd.2017.06.500
P.461 Performance of the upper limb module for Chinese patients with Duchenne muscular dystrophy: a new useful clinical tool to monitor the disease progress and as an outcome measure for therapeutic drug trial Y. Chiu 1, W. Choi 1, C. Hui 1, S. Li 1, S. Lee 1, S. Chan 2 1 The Duchess of Kent Children’s Hospital, Hospital Authority, Hong Kong, Hong Kong SAR; 2 LKS Faculty of Medicine, The University of Hong Kong, Hong Kong, Hong Kong SAR The recent development of therapeutic treatment to Duchenne muscular dystrophy (DMD), which is a common childhood neuromuscular disorder has highlighted the need to identify a valid and reliable outcome measures for planned clinical trials. This disease has a progressive deterioration from both lower limbs to trunk and upper limbs involvement from teenage onwards. Thus, outcome measures should be able to cover the disease spectrum from ambulatory to non-ambulatory stage. Majority of the existing clinical assessment tools are targeted at walking and lower limb gross motor skills and are inadequate to serve the above purposes. Performance of the upper limb module (PUL) for Duchenne muscular dystrophy is a new tool which was found to be reliably used in both ambulant and non-ambulant patients with DMD (Marika 2014). This study aims to establish the reliability of its use in our Chinese patients with DMD. A study was carried out in 2016 & 2017 to establish the reliability of PUL to be used in Chinese population of DMD with ethical approval from HA HKWC Institution review board. Twenty-three DMD subjects aged 7 to 32, under the care of Paediatric Neuromuscular Disorder Program and Pulmonary Rehabilitation Program, were recruited. Each participant has at least one session of PUL data collection with video recording. The recorded first PUL performance was rated by three individual physiotherapists immediately and also individually at one month interval. There is a good intra-rater and inter-rater reliability results with ICC range from 0.85 to 0.95. The PUL scoring can also satisfactorily reflect the functional decline in patients of both ambulatory and non-ambulatory stage. The pattern of decline in upper limbs corresponds well to clinical course of disease and can sensitively reflect functional difference upon drug treatment including chronic steroid use. This new upper limb module for DMD is a reliable and valid tool for clinical use and research purpose in Chinese patients of DMD for both disease and therapy monitoring. http://dx.doi.org/10.1016/j.nmd.2017.06.501
P.462 Exploring physical activity levels and sleep efficiency relationships among boys with Duchenne muscular dystrophy (DMD) R. Bendixen 1, J. Anning 2, A. Kelleher 1, M. Yuhas 1, M. Feltman 1, D. Lorenzin 3, H. Morizono 4, E. Hoffman 5 1 University of Pittsburgh, Pittsburgh, USA; 2 Slippery Rock University, Slippery Rock, USA; 3 Venetasoft Corporation, Florence, Italy; 4 Children’s National Medical Center, Washington, DC, USA; 5 Binghampton University, Binghampton, USA The purpose of this pilot study was to explore relationships between daily physical activity levels and sleep efficiency in ambulatory boys with DMD. We used the Microsoft Band (MSBand) coupled to a Wi-Fi phone with
Abstracts 2017 / Neuromuscular Disorders 27 (2017) S96–S249 control software to collect raw data. Fifteen ambulatory boys with DMD (7.5 ± 1.5 y) wore the MSBand and used the mobile app for two weeks. The MSBand/mobile app collected step counts to establish daily physical activity and sleep patterns to determine nightly sleep efficiency percentages. Pearson correlation demonstrated a positive relationship between daily step counts and sleep efficiency (r = 0.669; p < 0.000). On average, the boys with DMD achieved 6,170 ± 3,016 daily steps while experiencing 78 ± 14% sleep efficiency. The coinciding elevations and declines in activity levels and sleep efficiency showed large variability. Interestingly, fluctuating activity and sleep may demonstrate an innate self-regulatory ability. For example, for many of our boys, as daily activity increased, sleep efficiency increased; when activity reached a higher “pivotal” level, sleep efficiency began to decline followed by a decline in activity. These declines in daily activity may be required for rest and fortification (self-regulation). Following these rest periods, daily activity began to increase as did sleep efficiency. Importantly, it is unclear whether optimal amounts of daily activity assist to increase sleep efficiency or if higher percentages of sleep efficiency assist to increase daily activity levels. Further study is needed to examine if self-regulation capabilities exist among ambulatory boys with DMD, and if so, what would be an optimal step count range prescription to improve sleep efficiency (or vice versa). Such a discovery would offer the potential of using the MSBand as a resource for creating daily activity/sleep schedules with optimal recovery to prevent a sedentary lifestyle while avoiding fatigue in ambulatory boys with DMD. http://dx.doi.org/10.1016/j.nmd.2017.06.502
P.463 Longitudinal home-monitoring data in non-ambulant patients with Duchenne muscular atrophy A. Seferian 1, G. Quicke 1, E. Gargaun 1, A. Moraux 1, S. Gillabert 1, C. Lilien 1, E. Gasnier 1, V. Che 1, T. Gidaro 1, M. Annoussamy 1, D. Vissiere 2, L. Servais 1 1 Institute of myology, Paris, France; 2 SYSNAV, Vernon, France Measuring disease progression and response to treatment in Duchenne muscular dystrophy (DMD) is a challenge for all clinical development plans, especially in non-ambulant patients. Motion analysis measured with magnetoinertial sensors represents an objective evaluation of the movement in laboratory environment and in everyday life. Worn at the wrist, ActiMyo® is able to capture all movements through the sensor measurements. Dedicated algorithms allow precise qualification and quantification of patient’s upper limb activity in non-controlled environment. To evaluate non-ambulant DMD patients, four variables have been considered for each individual patient: mean rotation rate of the wrist, average acceleration, vertical acceleration and power. Eleven DMD patients with a mean age of 14 ± 2.6 (range 9 to 18) years worn the device continuously at home. The mean number of hours per day when ActiMyo® was worn was 12. Patients were assessed with standard tests at baseline, at 6 (n = 9) and at 12 months (n = 5). Their median Brooke score was 3 (range 2 to 5). Their maximal pinch strength was 1.34 ± 3.00 kg. The total MFM score (n = 8) was 34.23 ± 14.76. We demonstrated that 180 hours of ActiMyo® recording (approximately two weeks) induces a very low intrapatient variability (<=5%) for all the ActiMyo® variables. Month 1, month 6 and month 12 were evaluated for patients having more than 50 hours of ActiMyo® data. ActiMyo® variables estimated on 180h at baseline showed a good significant correlation with pinch strength (rho>0.6), Brooke score and MFM scores (rho>0.8). Data showed a 12-month decrease of 18.8 ± 21.7% for mean rotation rate of the wrist, 20.5 ± 14.9% for average acceleration, 20.6 ± 14.3% for vertical acceleration and 21.8 ± 19.4% for power, which is a very favourable effect size. ActiMyo® allows a continuous measure of nonambulant DMD patients even in advanced stages of loss of function and allows the detection of negative change on a 12 months period. http://dx.doi.org/10.1016/j.nmd.2017.06.503
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P.464 The 100-meter timed test: ability to detect change over time in Duchenne muscular dystrophy N. Miller, L. Alfano, K. Flanigan, S. Al-Zaidy, C. Tsao, J. Mendell, L. Lowes Nationwide Children’s Hospital, Columbus, USA The 100-meter timed test (100m) has been proposed as an outcome measure for use in Duchenne muscular dystrophy (DMD) that has the potential to expand the recruitment pool to include both younger and more able boys as it is a concrete concept of running 2 laps around a set of cones. Many of the current assessments used in DMD have a ceiling effect as the most able boys can achieve a perfect score. The 100m eliminates the ceiling effect by allowing the boys to run if able. Although the most able boys may walk as fast as age matched peers, their running speeds are significantly slower. This study documents the sensitivity of the 100m to measure decline in boys with DMD over time. To this end, 40 boys with DMD (age range: 4–13 years) performed the 100m at baseline and then repeated testing at subsequent regularly scheduled clinic visits with time points at 6 months (N = 30), 12 months (N = 17), 18 months (N = 10), and 24 months (N = 2). Longitudinal change in time to complete the 100m was consistent with natural history studies in other functional measures as it improved until the age of 7 and then declined over time. Spearman’s rho showed excellent correlations between the 100m and other functional tests performed at concurrent visits including the Six Minute Walk Test (n = 51; r=-0.744, p < 0.001), North Star Ambulatory Assessment (n = 88; r=-0.877, p < 0.001), Time to Rise (n = 82; r = 0.768, p < 0.001), and Ten Meter Walk/Run Test (n = 97; r = 0.926, p < 0.001). Change in percent predicted 100m time over a 6 month (mean = -2.9 ± 7.8) and 12 month (mean = -6.0 ± 9.5) time period suggest it would be an optimal outcome measure in upcoming clinical trials. Sample size calculations using the change in 6 months and 12 months will be presented to demonstrate the utility of the 100m for future clinical trial planning as it could expand the recruitment pool and shorten trial duration. http://dx.doi.org/10.1016/j.nmd.2017.06.504
P.465 The reliability and validity of Turkish version of pedsQL multidimensional fatigue scale in Duchenne muscular dystrophy I. Alemdarog˘lu, N. Bulut, S. Bozgeyik, A. Karaduman, H. Topalog˘lu, O. Yılmaz Hacettepe University, Ankara, Turkey Fatigue is one of the most common-difficult to evaluate-symptom of Duchenne muscular dystrophy (DMD). However, there is not enough valid and reliable instrument to assess fatigue in neuromuscular disorders in literature. Our aim was to investigate the reliability and validity of pedsQL multidimensional fatigue scale (MFS) Turkish version in DMD. Seventy one DMD children whose ages were between 5–13 years were participated in this study. For the reliability, Turkish PedsQL MFS was applied children and their parents with two weeks interval by same physiotherapist. For the validity assessment, pediatrics quality of life 3.0 Neuromuscular module (PedsQL 3.0NMM) was used as gold standart. The intraclass correlation coefficent (ICC) and Spearman’s correlation coefficients (rho) were used to analyze the reliability and validity, respectively. The mean age of children was 8.57 ± 1.93 years. The ICC values of the scale for children and parents were found 0.87 and 0.91. A positive, moderate, statistically significant correlation was found between PedsQL 3.0-NMM and PedsQL-MFS in children (r:0.62) while positive, strong correlation in parents (r:0.79) (p < 0.05). The Turkish version of PedsQL-MFS is found to be a reliable and valid instrument to measure fatigue in children with DMD. The scale may be used as an outcome measure in clinical practice and trials, as well as a guide to set physiotherapy and home programs. http://dx.doi.org/10.1016/j.nmd.2017.06.505
Abstracts 2017 / Neuromuscular Disorders 27 (2017) S96–S249
were computed. The walking, running and stairs climbing or descending episodes were detected with an estimated accuracy greater than 99.7%. These results show that activity patterns can be recognized and classified in a controlled environment with high accuracy. Validation in non-controlled environment is ongoing. This information could be useful for regulators and funders in drug approval and refunding process in order to provide an objective and clinically meaningful measurement of daily life related activities. http://dx.doi.org/10.1016/j.nmd.2017.06.498
P.459 Hand function in boys and men with Duchenne muscular dystrophy (DMD) M. Hunnekens 1, J. Huijben 2, I. de Groot 2 1 Rehabilitation centre Klimmendaal, Arnhem, Netherlands; 2 Radboudumc, Nijmegen, Netherlands Life expectancy is increasing for persons with DMD. It is of importance to preserve hand function for activities of daily living as long as possible. Not much is known concerning the natural disease course of the hands, therefore developing interventions is as yet difficult. Explorative study, cross-sectional design, to examine the natural disease course of the hands in patients with DMD and to explore relations between the level of body functions/structures and the level of activities, according to the ICF model. Patients: 51 boys/men (age range 5.2–40.0) with established diagnosis of DMD. Strength measures: MyoPinch and MyoGrip. Range of motion by goniometry. Activities: several instruments were used e.g. Brooke, PUL, MFM, 9-HPT, Timed-TIHM and all participants completed a questionnaire containing ABILHAND-plus and CUE. The most obvious decrease of ROM with increasing age was seen in wrist supination, both right and left. Strength increased with age until the age of 10. A decrease was seen thereafter with a more rapid decline around 20 years. On the level of activities great variability was seen on the different tests; some boys around the age of 10 already experienced difficulties. Subtests of the Timed-TIHM could also be performed at older age. Correlations were found i.a. between thumb mobility and activities. Large variation in hand function was seen. In some younger patients disabilities were already present. The use of splints or specific hand therapy is still uncommon, but should be considered. The results of this study give basis for possible interventions. This study was performed within clinical practice; thus some data are missing. Clinical message: It is important to pay already early attention to hand function in DMD. http://dx.doi.org/10.1016/j.nmd.2017.06.499
P.460 Unraveling upper extremity performance in DMD: a biophysical model M. Janssen 1, J. Harlaar 2, H. Koopman 3, I. de Groot 1 1 Radboudumc, Nijmegen, Netherlands; 2 Free University medical centre, Amsterdam, Netherlands; 3 University of Twente, Enschede, Netherlands Upper extremity (UE) activity scales are currently used as the golden standard for defining UE function in non-ambulant Duchenne muscular dystrophy (DMD) patients. These scales, however, are not able to explain the biophysical working mechanism behind changes in UE function. Therefore, we aimed to identify critical physiological outcome variables underlying reduced UE task performance in DMD. These critical variables were used to construct an explanatory biophysical model of the UE working mechanism in DMD. The most critical physiologic outcome variables related to UE task performance (Brooke scale and Performance of Upper Limb (PUL) scale) were maximal active joint angle, followed by maximal muscle torque, maximal muscle activity, echogenicity (fatty infiltration) and maximal passive range of motion. These variables explained more than 90% of the variability in UE task performance. Based on these critical variables a biophysical model was constructed. In this model we considered UE task performance to be mainly dependent on the ability to move your arms actively, which is influenced by both
passive range of motion and the available joint torque. The available joint torque is dependent on muscle capacity, and should be sufficient to lift the weight of the arm against gravity and to overcome passive joint torque (stiffness). Muscle capacity is influenced by both the ability to activate the muscle and the muscle force that is generated at the joint level. This model allows us to understand the underlying cause of UE limitations. Based on this knowledge one can select appropriate interventions to improve UE function, such as arm supports to improve the reachable workspace, contracture prevention, or medication to improve muscle strength. In addition, the model could form the basis for the development of new composite outcome measures for clinical trials. http://dx.doi.org/10.1016/j.nmd.2017.06.500
P.461 Performance of the upper limb module for Chinese patients with Duchenne muscular dystrophy: a new useful clinical tool to monitor the disease progress and as an outcome measure for therapeutic drug trial Y. Chiu 1, W. Choi 1, C. Hui 1, S. Li 1, S. Lee 1, S. Chan 2 1 The Duchess of Kent Children’s Hospital, Hospital Authority, Hong Kong, Hong Kong SAR; 2 LKS Faculty of Medicine, The University of Hong Kong, Hong Kong, Hong Kong SAR The recent development of therapeutic treatment to Duchenne muscular dystrophy (DMD), which is a common childhood neuromuscular disorder has highlighted the need to identify a valid and reliable outcome measures for planned clinical trials. This disease has a progressive deterioration from both lower limbs to trunk and upper limbs involvement from teenage onwards. Thus, outcome measures should be able to cover the disease spectrum from ambulatory to non-ambulatory stage. Majority of the existing clinical assessment tools are targeted at walking and lower limb gross motor skills and are inadequate to serve the above purposes. Performance of the upper limb module (PUL) for Duchenne muscular dystrophy is a new tool which was found to be reliably used in both ambulant and non-ambulant patients with DMD (Marika 2014). This study aims to establish the reliability of its use in our Chinese patients with DMD. A study was carried out in 2016 & 2017 to establish the reliability of PUL to be used in Chinese population of DMD with ethical approval from HA HKWC Institution review board. Twenty-three DMD subjects aged 7 to 32, under the care of Paediatric Neuromuscular Disorder Program and Pulmonary Rehabilitation Program, were recruited. Each participant has at least one session of PUL data collection with video recording. The recorded first PUL performance was rated by three individual physiotherapists immediately and also individually at one month interval. There is a good intra-rater and inter-rater reliability results with ICC range from 0.85 to 0.95. The PUL scoring can also satisfactorily reflect the functional decline in patients of both ambulatory and non-ambulatory stage. The pattern of decline in upper limbs corresponds well to clinical course of disease and can sensitively reflect functional difference upon drug treatment including chronic steroid use. This new upper limb module for DMD is a reliable and valid tool for clinical use and research purpose in Chinese patients of DMD for both disease and therapy monitoring. http://dx.doi.org/10.1016/j.nmd.2017.06.501
P.462 Exploring physical activity levels and sleep efficiency relationships among boys with Duchenne muscular dystrophy (DMD) R. Bendixen 1, J. Anning 2, A. Kelleher 1, M. Yuhas 1, M. Feltman 1, D. Lorenzin 3, H. Morizono 4, E. Hoffman 5 1 University of Pittsburgh, Pittsburgh, USA; 2 Slippery Rock University, Slippery Rock, USA; 3 Venetasoft Corporation, Florence, Italy; 4 Children’s National Medical Center, Washington, DC, USA; 5 Binghampton University, Binghampton, USA The purpose of this pilot study was to explore relationships between daily physical activity levels and sleep efficiency in ambulatory boys with DMD. We used the Microsoft Band (MSBand) coupled to a Wi-Fi phone with
Abstracts 2017 / Neuromuscular Disorders 27 (2017) S96–S249 control software to collect raw data. Fifteen ambulatory boys with DMD (7.5 ± 1.5 y) wore the MSBand and used the mobile app for two weeks. The MSBand/mobile app collected step counts to establish daily physical activity and sleep patterns to determine nightly sleep efficiency percentages. Pearson correlation demonstrated a positive relationship between daily step counts and sleep efficiency (r = 0.669; p < 0.000). On average, the boys with DMD achieved 6,170 ± 3,016 daily steps while experiencing 78 ± 14% sleep efficiency. The coinciding elevations and declines in activity levels and sleep efficiency showed large variability. Interestingly, fluctuating activity and sleep may demonstrate an innate self-regulatory ability. For example, for many of our boys, as daily activity increased, sleep efficiency increased; when activity reached a higher “pivotal” level, sleep efficiency began to decline followed by a decline in activity. These declines in daily activity may be required for rest and fortification (self-regulation). Following these rest periods, daily activity began to increase as did sleep efficiency. Importantly, it is unclear whether optimal amounts of daily activity assist to increase sleep efficiency or if higher percentages of sleep efficiency assist to increase daily activity levels. Further study is needed to examine if self-regulation capabilities exist among ambulatory boys with DMD, and if so, what would be an optimal step count range prescription to improve sleep efficiency (or vice versa). Such a discovery would offer the potential of using the MSBand as a resource for creating daily activity/sleep schedules with optimal recovery to prevent a sedentary lifestyle while avoiding fatigue in ambulatory boys with DMD. http://dx.doi.org/10.1016/j.nmd.2017.06.502
P.463 Longitudinal home-monitoring data in non-ambulant patients with Duchenne muscular atrophy A. Seferian 1, G. Quicke 1, E. Gargaun 1, A. Moraux 1, S. Gillabert 1, C. Lilien 1, E. Gasnier 1, V. Che 1, T. Gidaro 1, M. Annoussamy 1, D. Vissiere 2, L. Servais 1 1 Institute of myology, Paris, France; 2 SYSNAV, Vernon, France Measuring disease progression and response to treatment in Duchenne muscular dystrophy (DMD) is a challenge for all clinical development plans, especially in non-ambulant patients. Motion analysis measured with magnetoinertial sensors represents an objective evaluation of the movement in laboratory environment and in everyday life. Worn at the wrist, ActiMyo® is able to capture all movements through the sensor measurements. Dedicated algorithms allow precise qualification and quantification of patient’s upper limb activity in non-controlled environment. To evaluate non-ambulant DMD patients, four variables have been considered for each individual patient: mean rotation rate of the wrist, average acceleration, vertical acceleration and power. Eleven DMD patients with a mean age of 14 ± 2.6 (range 9 to 18) years worn the device continuously at home. The mean number of hours per day when ActiMyo® was worn was 12. Patients were assessed with standard tests at baseline, at 6 (n = 9) and at 12 months (n = 5). Their median Brooke score was 3 (range 2 to 5). Their maximal pinch strength was 1.34 ± 3.00 kg. The total MFM score (n = 8) was 34.23 ± 14.76. We demonstrated that 180 hours of ActiMyo® recording (approximately two weeks) induces a very low intrapatient variability (<=5%) for all the ActiMyo® variables. Month 1, month 6 and month 12 were evaluated for patients having more than 50 hours of ActiMyo® data. ActiMyo® variables estimated on 180h at baseline showed a good significant correlation with pinch strength (rho>0.6), Brooke score and MFM scores (rho>0.8). Data showed a 12-month decrease of 18.8 ± 21.7% for mean rotation rate of the wrist, 20.5 ± 14.9% for average acceleration, 20.6 ± 14.3% for vertical acceleration and 21.8 ± 19.4% for power, which is a very favourable effect size. ActiMyo® allows a continuous measure of nonambulant DMD patients even in advanced stages of loss of function and allows the detection of negative change on a 12 months period. http://dx.doi.org/10.1016/j.nmd.2017.06.503
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P.464 The 100-meter timed test: ability to detect change over time in Duchenne muscular dystrophy N. Miller, L. Alfano, K. Flanigan, S. Al-Zaidy, C. Tsao, J. Mendell, L. Lowes Nationwide Children’s Hospital, Columbus, USA The 100-meter timed test (100m) has been proposed as an outcome measure for use in Duchenne muscular dystrophy (DMD) that has the potential to expand the recruitment pool to include both younger and more able boys as it is a concrete concept of running 2 laps around a set of cones. Many of the current assessments used in DMD have a ceiling effect as the most able boys can achieve a perfect score. The 100m eliminates the ceiling effect by allowing the boys to run if able. Although the most able boys may walk as fast as age matched peers, their running speeds are significantly slower. This study documents the sensitivity of the 100m to measure decline in boys with DMD over time. To this end, 40 boys with DMD (age range: 4–13 years) performed the 100m at baseline and then repeated testing at subsequent regularly scheduled clinic visits with time points at 6 months (N = 30), 12 months (N = 17), 18 months (N = 10), and 24 months (N = 2). Longitudinal change in time to complete the 100m was consistent with natural history studies in other functional measures as it improved until the age of 7 and then declined over time. Spearman’s rho showed excellent correlations between the 100m and other functional tests performed at concurrent visits including the Six Minute Walk Test (n = 51; r=-0.744, p < 0.001), North Star Ambulatory Assessment (n = 88; r=-0.877, p < 0.001), Time to Rise (n = 82; r = 0.768, p < 0.001), and Ten Meter Walk/Run Test (n = 97; r = 0.926, p < 0.001). Change in percent predicted 100m time over a 6 month (mean = -2.9 ± 7.8) and 12 month (mean = -6.0 ± 9.5) time period suggest it would be an optimal outcome measure in upcoming clinical trials. Sample size calculations using the change in 6 months and 12 months will be presented to demonstrate the utility of the 100m for future clinical trial planning as it could expand the recruitment pool and shorten trial duration. http://dx.doi.org/10.1016/j.nmd.2017.06.504
P.465 The reliability and validity of Turkish version of pedsQL multidimensional fatigue scale in Duchenne muscular dystrophy I. Alemdarog˘lu, N. Bulut, S. Bozgeyik, A. Karaduman, H. Topalog˘lu, O. Yılmaz Hacettepe University, Ankara, Turkey Fatigue is one of the most common-difficult to evaluate-symptom of Duchenne muscular dystrophy (DMD). However, there is not enough valid and reliable instrument to assess fatigue in neuromuscular disorders in literature. Our aim was to investigate the reliability and validity of pedsQL multidimensional fatigue scale (MFS) Turkish version in DMD. Seventy one DMD children whose ages were between 5–13 years were participated in this study. For the reliability, Turkish PedsQL MFS was applied children and their parents with two weeks interval by same physiotherapist. For the validity assessment, pediatrics quality of life 3.0 Neuromuscular module (PedsQL 3.0NMM) was used as gold standart. The intraclass correlation coefficent (ICC) and Spearman’s correlation coefficients (rho) were used to analyze the reliability and validity, respectively. The mean age of children was 8.57 ± 1.93 years. The ICC values of the scale for children and parents were found 0.87 and 0.91. A positive, moderate, statistically significant correlation was found between PedsQL 3.0-NMM and PedsQL-MFS in children (r:0.62) while positive, strong correlation in parents (r:0.79) (p < 0.05). The Turkish version of PedsQL-MFS is found to be a reliable and valid instrument to measure fatigue in children with DMD. The scale may be used as an outcome measure in clinical practice and trials, as well as a guide to set physiotherapy and home programs. http://dx.doi.org/10.1016/j.nmd.2017.06.505