Defining ambulation status in patients with Duchenne muscular dystrophy using the 10-metre walk test and the motor function measure scale

Petian-Alonso, Danila Cristina; Castro, Ani Caroline de; Davoli, Gabriela Barroso de Queiroz; Martinez, Edson Zangiacomi; Mattiello-Sverzut, Ana Cláudia

doi:10.1080/09638288.2022.2112098

Cited by 6 publications

(7 citation statements)

References 22 publications

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“…of the MFM results is able to distinguish between ambulatory and nonambulatory patients. 21 In addition, D1 of the MFM is valid, reliable, and provides a unidimensional measure of motor function in ambulatory DMD patients. Nevertheless, D2 and D3 of the MFM must be used with caution with this group of patients.…”

Section: Discussionmentioning

confidence: 98%

“…6 This may be explained by the fact that the 6 MWT and muscle strength of the knee extensor have a negative and significant correlation with muscle fat. 21,26 Thus, as the disease progresses, the amount of fat in the muscle tissue increases, emphasizing knee extensor weakness and, consequently, worsening of the patient's performance on the 6 MWT.…”

Section: Discussionmentioning

confidence: 99%

“…The timed tests and MFM scale assess disease severity and are correlated with muscle degeneration as measured by MRI 20 . The association between the 10 MWT and D1 of the MFM results is able to distinguish between ambulatory and nonambulatory patients 21 . In addition, D1 of the MFM is valid, reliable, and provides a unidimensional measure of motor function in ambulatory DMD patients.…”

Section: Discussionmentioning

confidence: 99%

“…The decline in walking ability measured by the 6 MWT occurs despite small changes in strength values of the knee extensor muscles across 1 year of longitudinal evaluation 6 . This may be explained by the fact that the 6 MWT and muscle strength of the knee extensor have a negative and significant correlation with muscle fat 21,26 . Thus, as the disease progresses, the amount of fat in the muscle tissue increases, emphasizing knee extensor weakness and, consequently, worsening of the patient's performance on the 6 MWT.…”

Section: Discussionmentioning

confidence: 99%

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Generation of percentile curves for strength and functional abilities for boys with Duchenne muscular dystrophy

et al. 2023

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Introduction/Aims Considering the heterogeneity of the clinical manifestations of Duchenne muscular dystrophy (DMD), it is important to describe their various clinical profiles. Thus, in this study we aimed to develop percentile curves for DMD using a battery of measures to define the patterns of functional abilities, timed tests, muscle strength, and range of motion (ROM). Methods This retrospective data analysis was based on the records of patients with DMD using the Motor Function Measure (MFM) scale, isometric muscle strength (IS), dorsiflexion ROM, 10‐meter walk test (10 MWT), and 6‐minute walk test (6 MWT). Percentile curves (25th, 50th, and 75th percentiles) with MFM, IS, ROM, 10 MWT, and 6 MWT on the y axis and patient age on the x axis were constructed using the generalized additive model for location, scale, and shape, with Box‐Cox power exponential distribution. Results There were records of 329 assessments of patients between 4 and 18 years of age. The MFM percentiles showed a gradual reduction in all dimensions. Muscle strength and ROM percentiles showed that the knee extensors were the most affected from 4 years of age, and dorsiflexion ROM negative values were noted from the age of 8 years. The 10 MWT showed a gradual increase in performance time with age. For the 6 MWT, the distance curve remained stable until 8 years, with a subsequent progressive decline. Discussion In this study we generated percentile curves that can help health professionals and caregivers follow the trajectory of disease progression in DMD patients.

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Section: Discussionmentioning

confidence: 98%

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

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Generation of percentile curves for strength and functional abilities for boys with Duchenne muscular dystrophy

et al. 2023

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“…In addition to their ease, the 10-meter run/walk test and rise-from-floor test have utility in the assessment of motor performance in DMD (23) and have been employed in clinical trials of exon-skipping therapy to evaluate its efficacy (8). Therefore, it is expected that the natural history of the 10-meter run/walk test and rise-from-floor test will be more widely used in clinical practice as a method for motor function evaluation in DMD in the future.…”

Section: Discussionmentioning

confidence: 99%

Natural history of serum creatine kinase levels and motor, pulmonary, and cardiac functions in 337 patients with Duchenne muscular dystrophy: a retrospective study at a single referral center in Japan

Awano

Nambu

Ito

et al. 2022

Preprint

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IntroductionDuchenne muscular dystrophy (DMD) is characterized by fatal progressive muscular atrophy resulting from dystrophin deficiency due toDMDgene mutations. DMD develops with symptoms of skeletal muscle weakness in early childhood; as a patient ages, muscle weakness progressively worsens and is further complicated by the involvement of the respiratory or cardiac muscles. Nonetheless, only few large-scale studies have investigated the secular changes in these symptoms from infancy to adolescence. Over recent years, with the development of DMD treatments, the need for a natural history of DMD to serve as a control for determining treatment efficacy in clinical trials has dramatically increased. The present study aimed to determine the natural history of patients with DMD and to examine the natural history of individual patients with gene mutations eligible for exon-skipping therapy by conducting a retrospective analysis of medical records of patients with DMD at a single institution in Japan.MethodsMedical records of 337 patients with DMD who visited Kobe University Hospital in western Japan over a period of 30 years from their first visit until 20 years of age were examined. Data on serum creatine kinase (CK) levels, 10-meter run/walk test results and rise-from-floor test results for motor function evaluation, % forced vital capacity (%FVC) and forced expiratory volume in 1 second (%FEV1) values for respiratory function evaluation, and left ventricular ejection fraction (LVEF) and left ventricular end-diastolic diameter (LVDd) values for cardiac function evaluation were extracted. Changes in these values over time were examined.ResultsSerum CK levels showed a stair-step pattern of decline, with extremely high values until 6 years of age, a rapid decline from 7 to 12 years of age, and a plateau thereafter. Both the median 10-meter run/walk velocity and rise-from-floor velocity peaked at the age of 4 years and declined with age. Both the median %FVC and %FEV1 declined from the age of 11 years. The median LVEF was >60% until the age of 12 years and rapidly declined from 13 to 15 years of age. Cardiac dysfunction (LVEF <53%) occurred in approximately 80% of patients at the age of 20 years. Furthermore, examination of the relationship between gene mutations eligible for exon-skipping therapy and natural history revealed no characteristic findings.ConclusionWe analyzed the natural history of patients with DMD in Japan during childhood and adolescence and showed that CK levels and motor, respiratory, and cardiac functions each exhibited unique changes over time. These findings will be useful in developing and designing new therapeutic agents for DMD and in determining their efficacy in clinical trials.

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Longitudinal data of serum creatine kinase levels and motor, pulmonary, and cardiac functions in 337 patients with Duchenne muscular dystrophy

Awano,

Nambu,

Itoh

et al. 2024

Muscle and Nerve

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Introduction/AimsDuchenne muscular dystrophy (DMD) presents with skeletal muscle weakness, followed by cardiorespiratory involvement. The need for longitudinal data regarding DMD that could serve as a control for determining treatment efficacy in clinical trials has increased notably. The present study examined the longitudinal data of Japanese DMD patients collectively and assessed individual patients with pathogenic variants eligible for exon‐skipping therapy.MethodsPatients with DMD who visited Kobe University Hospital between March 1991 and March 2019 were enrolled. Data between the patients' first visit until age 20 years were examined.ResultsThree hundred thirty‐seven patients were included. Serum creatine kinase levels showed extremely high values until the age of 6 years and a rapid decline from ages 7–12 years. Both the median 10‐m run/walk velocity and rise‐from‐floor velocity peaked at the age of 4 years and declined with age. The values for respiratory function declined from the age of 11 years. The median left ventricular ejection fraction was >60% until the age of 12 years and rapidly declined from ages 13–15 years. Examination of the relationship between pathogenic variants eligible for exon‐skipping therapy and longitudinal data revealed no characteristic findings.DiscussionWe found that creatine kinase levels and motor, respiratory, and cardiac functions each exhibited various changes over time. These findings provide useful information about the longitudinal data of several outcome measures for patients with DMD not receiving corticosteroids. These data may serve as historical controls in comparing the natural history of DMD patients not on regular steroid use in appropriate clinical trials.

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Defining ambulation status in patients with Duchenne muscular dystrophy using the 10-metre walk test and the motor function measure scale

Cited by 6 publications

References 22 publications

Generation of percentile curves for strength and functional abilities for boys with Duchenne muscular dystrophy

Generation of percentile curves for strength and functional abilities for boys with Duchenne muscular dystrophy

Natural history of serum creatine kinase levels and motor, pulmonary, and cardiac functions in 337 patients with Duchenne muscular dystrophy: a retrospective study at a single referral center in Japan

Longitudinal data of serum creatine kinase levels and motor, pulmonary, and cardiac functions in 337 patients with Duchenne muscular dystrophy

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