Hip kinetics during gait are clinically meaningful outcomes in young boys with Duchenne muscular dystrophy

Heberer, Kent; Fowler, Eileen; Staudt, Loretta A.; Sienko, Susan; Buckon, Cathleen E.; Bagley, Anita; Sison-Williamson, Mitell; McDonald, Craig M.; Sussman, Michael D.

doi:10.1016/j.gaitpost.2016.05.013

Cited by 26 publications

(28 citation statements)

References 27 publications

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“…51,74 Hip kinematics during gait are clinically meaningful outcome measures at 4–8 years. 75 Other measures assessing antigravity function, considered exploratory in DMD, include the Alberta Infant Motor Scale, Hammersmith Functional Motor Scale Expanded, and the Gross Motor Function Measure. 49,61,76 Assessment of, and intervention for, learning, attention, and sensory processing should begin at young ages.…”

Section: Rehabilitation Managementmentioning

confidence: 99%

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management

Birnkrant

Bushby

Bann

et al. 2018

The Lancet Neurology

927

1,080

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Since the publication of the Duchenne muscular dystrophy (DMD) care considerations in 2010, multidisciplinary care of this severe, progressive neuromuscular disease has evolved. In conjunction with improved patient survival, a shift to more anticipatory diagnostic and therapeutic strategies has occurred, with a renewed focus on patient quality of life. In 2014, a steering committee of experts from a wide range of disciplines was established to update the 2010 DMD care considerations, with the goal of improving patient care. The new care considerations aim to address the needs of patients with prolonged survival, to provide guidance on advances in assessments and interventions, and to consider the implications of emerging genetic and molecular therapies for DMD. The committee identified 11 topics to be included in the update, eight of which were addressed in the original care considerations. The three new topics are primary care and emergency management, endocrine management, and transitions of care across the lifespan. In part 1 of this three-part update, we present care considerations for diagnosis of DMD and neuromuscular, rehabilitation, endocrine (growth, puberty, and adrenal insufficiency), and gastrointestinal (including nutrition and dysphagia) management.

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Section: Rehabilitation Managementmentioning

confidence: 99%

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management

Birnkrant

Bushby

Bann

et al. 2018

The Lancet Neurology

927

1,080

View full text Add to dashboard Cite

show abstract

“…Studies on DMD and SMA patients were conducted from the perspective of improving the problem that the currently used outcome measures lack sensitivity and specificity to detect significant improvements within the first 6–12 months of intervention. Heberer et al collected spatial–temporal data and quantified kinematics and kinetics at the hip, knee, and ankle of 21 DMD boys between 4 and 8 years old using three-dimensional gait analysis over one year [ 17 ]. Between the baseline and post-treatment visits, 12 boys began a corticosteroid regimen (mean duration 10.8 +/− 2.4 months) while 9 boys remained steroid-naïve.…”

Section: Resultsmentioning

confidence: 99%

Digital Biomarkers for Neuromuscular Disorders: A Systematic Scoping Review

Youn

Moon

et al. 2021

Diagnostics

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Biomarkers play a vital role in clinical care. They enable early diagnosis and treatment by identifying a patient’s condition and disease course and act as an outcome measure that accurately evaluates the efficacy of a new treatment or drug. Due to the rapid development of digital technologies, digital biomarkers are expected to grow tremendously. In the era of change, this scoping review was conducted to see which digital biomarkers are progressing in neuromuscular disorders, a diverse and broad-range disease group among the neurological diseases, to discover available evidence for their feasibility and reliability. Thus, a total of 10 studies were examined: 9 observational studies and 1 animal study. Of the observational studies, studies were conducted with amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy (DMD), and spinal muscular atrophy (SMA) patients. Non-peer reviewed poster presentations were not considered, as the articles may lead to erroneous results. The only animal study included in the present review investigated the mice model of ALS for detecting rest disturbances using a non-invasive digital biomarker.

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“…A recent long-term natural history study of laboratory-based gait kinematic parameters in DMD demonstrated that stride length declines more than cadence (steps per minute) with disease progression -as does self-selected walking speed (the product of step length and cadence) [18]. Furthermore, steroids produce an increase in stride length in the 1-year period typically employed in clinical trials, which independently confirms the need to precisely measure step length and stride velocity in DMD [17].…”

Section: What Is Sv95c?mentioning

confidence: 88%

“…accelerometers -have been studied in DMD; DMD patients differ from typically developing youth in total steps and step frequency at the highest step rates or speeds [16]. The digital parameters of total steps per day and steps at the highest rate decline with disease progression and are correlated with traditional endpoints, such as 10-m velocity in meters per second [17,18]. However, these monitors only provide accurate step counts and do not measure the instantaneous stride length of each stride.…”

Section: What Is Sv95c?mentioning

confidence: 99%

First Regulatory Qualification of a Novel Digital Endpoint in Duchenne Muscular Dystrophy: A Multi-Stakeholder Perspective on the Impact for Patients and for Drug Development in Neuromuscular Diseases

et al. 2021

Self Cite

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Background: Functional outcome measures used to assess efficacy in clinical trials of investigational treatments for rare neuromuscular diseases like Duchenne muscular dystrophy (DMD) are performance-based tasks completed by the patient during hospital visits. These are prone to bias and may not reflect motor abilities in real-world settings. Digital tools, such as wearable devices and other remote sensors, provide the opportunity for continuous, objective, and sensitive measurements of functional ability during daily life. Maintaining ambulation is of key importance to individuals with DMD. Stride velocity 95th centile (SV95C) is the first wearable acquired digital endpoint to receive qualification from the European Medicines Agency (EMA) to quantify the ambulation ability of ambulant DMD patients aged ≥5 years in drug therapeutic studies; it is also currently under review for the US Food and Drug Administration (FDA) qualification. Summary: Focusing on SV95C as a key example, we describe perspectives of multiple stakeholders on the promise of novel digital endpoints in neuromuscular disease drug development.

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Hip kinetics during gait are clinically meaningful outcomes in young boys with Duchenne muscular dystrophy

Cited by 26 publications

References 27 publications

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management

Digital Biomarkers for Neuromuscular Disorders: A Systematic Scoping Review

First Regulatory Qualification of a Novel Digital Endpoint in Duchenne Muscular Dystrophy: A Multi-Stakeholder Perspective on the Impact for Patients and for Drug Development in Neuromuscular Diseases

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