Health related quality of life in young, steroid-naïve boys with Duchenne muscular dystrophy

Campbell, Craig; McColl, Elaine; McDermott, Michael P.; Martens, William; Guglieri, Michela; Griggs, Robert C.; Straub, Volker; Childs, Anne‐Marie; Ciafaloni, Emma; Shieh, Perry B.; Spinty, Stefan; Butterfield, Russell J.; Horrocks, Iain; Roper, Helen; Maggi, Lorenzo; Baranello, Giovanni; Flanigan, Kevin M.; Kuntz, Nancy L.; Darras, Basil T.; Kang, Peter B.; Mah, Jean K.; Mongini, Tiziana; Ricci, Federica; Morrison, Leslie; Krzesniak‐Swinarska, Monika; Hagen, Maja von der; Finkel, Richard S.; Kumar, Ashutosh; Wicklund, Matthew; McDonald, Craig; Henricson, E.; Schara‐Schmidt, Ulrike; Wilichowski, Ekkehard; Barohn, Richard J.; Statland, Jeffrey; Kirschner, Janbernd; Vita, Giuseppe; Vita, Gian Luca; Howard, James F.; Hughes, Imelda; McMillan, Hugh J.; Pegoraro, Elena; Bello, Luca; Burnette, W. Bryan; Thangarajh, Mathula; Chang, Taeun

doi:10.1016/j.nmd.2021.06.001

Cited by 5 publications

(2 citation statements)

References 20 publications

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“…As DMD progresses, there is an increase in upper extremity weakness, pain, and restricted movement (Janssen et al., 2014). Participation in physical activities declines with age (Bendixen et al., 2012; Campbell et al., 2021; Heutinck et al., 2015), and activities of daily living can cause discomfort or pain (Zebracki & Drotar, 2008). The DVA aims to evaluate the level of compensated movement for each movement task, with the highest severity score being the inability to perform the task independently.…”

Section: Introductionmentioning

confidence: 99%

Home‐based video assessment of ease of movement for patients with Duchenne: Interviews with physical therapists to select movement tasks

Contesse

Hodges

Staunton

et al. 2023

Physiotherapy Res Intl

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Background and Purpose: Patients with Duchenne muscular dystrophy (DMD) change their movement patterns to compensate for muscle weakness. The Duchenne Video Assessment (DVA) measures ease of movement through evaluation of compensatory movements. The DVA directs caregivers to video record patients performing home-based movement tasks using a mobile application, and DVA-certified physical therapists evaluate the videos using scorecards with prespecified compensatory movement criteria. Two qualitative interview studies were conducted to select movement tasks for the DVA that are relevant to patients with DMD and able to reflect changes in function.Methods: Qualitative interviews with eligible physical therapists were conducted remotely. Physical therapists were asked to spontaneously suggest movement tasks prior to evaluating specific movement tasks selected a priori. Analysts conducted a content analysis to investigate whether movement tasks selected a priori were confirmed as relevant to the population of interest and able to show changes in function. Results:The studies included five physical therapists to select tasks for ambulatory patients with DMD and six for non-ambulatory patients. For an ambulatory population, all five experts confirmed Climb Five Stairs, Run, Stand Up from Sitting, Sit Up from Supine, and Jump Forward, and four (80%) confirmed Walk as relevant and able to show functional changes. For a non-ambulatory population, all six experts confirmed Eat 10 Bites, Roll Over in Bed, Shift Weight in Bed, Take T-Shirt Off, Put T-Shirt On, Put Arms on Armrest, and Reach Across Table to Grab Cell Phone, and five (83%) confirmed Raise Hands Above Head as relevant and able to show functional changes. Discussion: Physical therapists confirmed the DVA movement tasks as relevant to patients with DMD and able to reflect changes in function. The use of the DVA in clinical trials provides an opportunity to collect data not seen in clinic and reduce travel burden for families.

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Section: Introductionmentioning

confidence: 99%

Home‐based video assessment of ease of movement for patients with Duchenne: Interviews with physical therapists to select movement tasks

Contesse

Hodges

Staunton

et al. 2023

Physiotherapy Res Intl

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“…People living with chronic medical conditions—as most people with a neuromuscular disease—are at higher risk of developing mental disorders like anxiety and depression. To investigate the effect of the burden of the disease on mental health, patient reported outcome measures (PROM) and quality of life (QOL) instruments are available, both general and disease-specific ones, e.g., for Spinal Muscular Atrophy (SMA) and Duchenne Muscular Dystrophy (DMD) [ 12 , 13 , 14 , 15 ]. For the pediatric population, questionnaires for parents are available to report information about their child, and age-specific questionnaires using appropriate language for that child’s development have been created and validated [ 16 , 17 ].…”

Section: Introductionmentioning

confidence: 99%

Psychopharmacological Treatments for Mental Disorders in Patients with Neuromuscular Diseases: A Scoping Review

et al. 2022

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Mental disorders are observed in neuromuscular diseases, especially now that patients are living longer. Psychiatric symptoms may be severe and psychopharmacological treatments may be required. However, very little is known about pharmacotherapy in these conditions. We aimed to summarize the current knowledge on the use of psychopharmacological treatments for mental disorders in patients living with a neuromuscular disease. A scoping review was performed using the methodology of the Joanna Briggs Institute. Four databases were searched from January 2000 to July 2021. Articles were screened based on titles and abstracts. Full-text papers published in peer-reviewed journals in English were selected. Twenty-six articles met eligibility criteria, all being case reports/series focusing on the psychopharmacological control of psychiatric symptoms for the following conditions: myasthenia gravis (n = 11), Duchenne (n = 5) and Becker (n = 3) muscular dystrophy, mitochondrial disorders (n = 3), glycogen storage disease (n = 1), myotonic dystrophy (n = 1), hyperkalemic periodic paralysis (n = 1), and congenital myasthenic syndrome (n = 1). None of the articles provided details on the decision-making process to choose a specific drug/regimen or on follow-up strategies to monitor safety and efficacy. Larger studies showing real-world data would be required to guide consensus-based recommendations, thus improving current standards of care and, ultimately, the quality of life of patients and their families.

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Current Challenges of Using Patient-Level Claims and Electronic Health Record Data for the Longitudinal Evaluation of Duchenne Muscular Dystrophy Outcomes

Gooch,

Audhya,

Ricchetti-Masterson

et al. 2024

Adv Ther

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Introduction:Insurance claims data and electronic health records (EHRs) have been used to characterize Duchenne muscular dystrophy (DMD) in real-world populations. The ability to assess patient-level DMD disease progression within insurance claims or EHR data infrastructures is unknown. Insurance claims and EHR data were comprehensively examined for availability and reliability of DMD outcomes that describe functional status and disease progression at the individual patient level over time. Methods: MarketScan Commercial and Medicaid claims, and EHR-linked Clarivate open claims datasets were examined for data measuring 54 previously identified DMD-relevant outcomes in patients with DMD. Each outcome was assigned to one of five categories: functional

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Health related quality of life in young, steroid-naïve boys with Duchenne muscular dystrophy

Cited by 5 publications

References 20 publications

Home‐based video assessment of ease of movement for patients with Duchenne: Interviews with physical therapists to select movement tasks

Home‐based video assessment of ease of movement for patients with Duchenne: Interviews with physical therapists to select movement tasks

Psychopharmacological Treatments for Mental Disorders in Patients with Neuromuscular Diseases: A Scoping Review

Current Challenges of Using Patient-Level Claims and Electronic Health Record Data for the Longitudinal Evaluation of Duchenne Muscular Dystrophy Outcomes

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