Evaluation of a Program for Long-Term Treatment of Duchenne Muscular Dystrophy. Experience at the University Hospitals of Cleveland*

Vignos, Paul J.; Wagner, Marilyn B.; Karlinchak, Brenda; Katirji, Bashar

doi:10.2106/00004623-199612000-00007

Cited by 87 publications

(37 citation statements)

References 20 publications

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“…Again, a multidisciplinary approach is essential involving physiotherapists, rehabilitation specialists and orthopaedic surgeons (19). Orthosis can be beneficial in helping to prevent the progression of lower limb contractures (20), but surgical management to release contractures may be necessary.…”

Section: Interventions Before Surgerymentioning

confidence: 99%

Duchenne muscular dystrophy: the management of scoliosis

et al. 2016

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This study summaries the current management of scoliosis in patients with Duchenne Muscular Dystrophy.A literature review of Medline was performed and the collected articles critically appraised. This literature is discussed to give an overview of the current management of scoliosis within Duchenne Muscular Dystrophy.Importantly, improvements in respiratory care, the use of steroids and improving surgical techniques have allowed patients to maintain quality of life and improved life expectancy in this patient group.

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Section: Interventions Before Surgerymentioning

confidence: 99%

Duchenne muscular dystrophy: the management of scoliosis

et al. 2016

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“…The prevention of joint contractures is a multidisciplinary effort, involving specialist hubs and community spokes. 32 Regional consultant neurologists and specialist neuromuscular physiotherapists review disease progress and treatment regimens at clinic visits, typically twice a year, and offer management suggestions to community physiotherapists. 28 While a boy is still able to walk, a community paediatric physiotherapist will monitor him for hip and ankle contractures until he becomes wheelchair dependent.…”

Section: Physiotherapymentioning

confidence: 99%

Aquatic therapy for children with Duchenne muscular dystrophy: a pilot feasibility randomised controlled trial and mixed-methods process evaluation

Hind

Parkin

Whitworth

et al. 2017

Health Technol Assess

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BackgroundDuchenne muscular dystrophy (DMD) is a rare disease that causes the progressive loss of motor abilities such as walking. Standard treatment includes physiotherapy. No trial has evaluated whether or not adding aquatic therapy (AT) to land-based therapy (LBT) exercises helps to keep muscles strong and children independent.ObjectivesTo assess the feasibility of recruiting boys with DMD to a randomised trial evaluating AT (primary objective) and to collect data from them; to assess how, and how well, the intervention and trial procedures work.DesignParallel-group, single-blind, randomised pilot trial with nested qualitative research.SettingSix paediatric neuromuscular units.ParticipantsChildren with DMD aged 7–16 years, established on corticosteroids, with a North Star Ambulatory Assessment (NSAA) score of 8–34 and able to complete a 10-m walk without aids/assistance. Exclusions: > 20% variation between baseline screens 4 weeks apart and contraindications.InterventionsParticipants were allocated on a 1 : 1 ratio to (1) optimised, manualised LBT (prescribed by specialist neuromuscular physiotherapists) or (2) the same plus manualised AT (30 minutes, twice weekly for 6 months: active assisted and/or passive stretching regime; simulated or real functional activities; submaximal exercise). Semistructured interviews with participants, parents (n = 8) and professionals (n = 8) were analysed using Framework analysis. An independent rater reviewed patient records to determine the extent to which treatment was optimised. A cost-impact analysis was performed. Quantitative and qualitative data were mixed using a triangulation exercise.Main outcome measuresFeasibility of recruiting 40 participants in 6 months, participant and therapist views on the acceptability of the intervention and research protocols, clinical outcomes including NSAA, independent assessment of treatment optimisation and intervention costs.ResultsOver 6 months, 348 children were screened – most lived too far from centres or were enrolled in other trials. Twelve (30% of target) were randomised to AT (n = 8) or control (n = 4). People in the AT (n = 8) and control (n = 2: attrition because of parental report) arms contributed outcome data. The mean change in NSAA score at 6 months was –5.5 [standard deviation (SD) 7.8] for LBT and –2.8 (SD 4.1) in the AT arm. One boy suffered pain and fatigue after AT, which resolved the same day. Physiotherapists and parents valued AT and believed that it should be delivered in community settings. The independent rater considered AT optimised for three out of eight children, with other children given programmes that were too extensive and insufficiently focused. The estimated NHS costs of 6-month service were between £1970 and £2734 per patient.LimitationsThe focus on delivery in hospitals limits generalisability.ConclusionsNeither a full-scale frequentist randomised controlled trial (RCT) recruiting in the UK alone nor a twice-weekly open-ended AT course delivered at tertiary centres is feasible. Further intervention development research is needed to identify how community-based pools can be accessed, and how families can link with each other and community physiotherapists to access tailored AT programmes guided by highly specialised physiotherapists. Bayesian RCTs may be feasible; otherwise, time series designs are recommended.Trial registrationCurrent Controlled Trials ISRCTN41002956.FundingThis project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full inHealth Technology Assessment; Vol. 21, No. 27. See the NIHR Journals Library website for further project information.

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“…Bu kısalıklar DMD ve CMT'de kas zayıflığı ve denge bozukluğunun karakteristik paternlerine bağlı olarak vücut ağırlık merkezindeki değişimlerin ve süregelen postüral kompansasyonların sonucu olarak meydana gelmektedir (5,6). Bu durum hastaların mobilite ve dengelerini limitlerken, sağlıkla ilgili yaşam kalitelerinde de azalmaya sebep olur (3,7,8).…”

Section: Introductionunclassified

Farkli Nöromusküler Hastaliklarda Ayak Bi̇leği̇ Li̇mi̇tasyonunun Yürüme Beceri̇si̇ Üzeri̇ne Etki̇si̇

Kaya¹,

Alemdaroğlu²,

Yılmaz³

et al. 2014

Turk J Physiother Rehabil

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Amaç: Çalışmanın amacı farklı bölgesel kas zayıflığına sahip iki grupta (proksimal ve distal) ayak bileği limitasyonunun, yürüme becerisi üzerine olan etkisini araştırmak ve karşılaştırmak idi.Yöntem: Çalışmaya Brooke Alt Ekstremite Fonksiyonel Sınıflandırması'na göre devre 1-3, primer olarak proksimal kas tutulumu olan 20 çocuk (yaş ortalaması:9.05±3.1 yıl), ve distal kas tutulumu olan (yaş ortalaması:12.95±3.3 yıl) diğer 20 çocuk olmak üzere, toplam 40 çocuk dahil edildi. Çocukların demografik bilgileri kaydedildi. Ayak bileği eklem limitasyonu gonyometre ile ölçüldü. Yürüme kapasiteleri, 6 dakika yürüme testi (6DYT) ile değerlendirildi. 6 dakikadaki yürüme mesafesi, attıkları tur, adım sayısı, adım uzunlukları ve kadansları kaydedildi. Sonuçlar:Çalışmada ayak bileği dorsifleksiyon limitasyonu ve 6 dakikada yürüdükleri toplam mesafe açısından gruplar arasında fark bulunmadı (p>0.05). 6DYT'nin diğer performans parametreleri açısından, test sırasındaki adım uzunluklarında distal kas tutulumlu grup lehine istatistiksel olarak anlamlı fark bulundu (p<0.01). Proksimal tutulumlu grupta 6 dakika süre-since yürünen toplam mesafe ve sağ ve sol ayak bileği limitasyon sonuçları arasında; sağ (r= -0.579, p= 0.01) ve sol (r=-0.445, p= 0.05) negatif yönde, orta kuvvette, istatistiksel olarak anlamlı ilişki bulundu (p<0.05).Tartışma: Çalışmanın sonuçları NMH'ye sahip bireylerde, özellikle de proksimal kas zayıflığı-nın hakim olduğu musküler distrofili çocuklarda hastalıklarının erken dönemlerinden itibaren ayak bileği eklem hareket açıklığının yürüme performansı üzerindeki önemini ortaya koymuş-tur.Anahtar Kelimeler: Nöromusküler hastalıklar; ayak eklemleri; yürüme RESEARCH ARTICLE EFFECT OF ANKLE JOINT LIMITATION ON WALKING ABILITY IN DIFFERENT NEUROMUSCULAR DISEASES ABSTRACTPurpose: In this study, we aimed to investigate the impact of the ankle limitation on the walking ability among two groups that have different spatial distribution of the weakness. Methods:In total, 40 children at the levels 1-3 of the Brooke Lower Extremity Scale are included in this study. 20 of them have been diagnosed distal muscle weakness (mean ages: 12.9±3.3 year), and the other 20 have been proximal muscle weakness (mean ages: 9±3.1). The demographic information were recorded. Ankle joint limitation were measured with goniometer. Their ambulatory capacities were evaluated with the 6 minutes walk test (6MWT). Total walking distances in 6 minutes, strolls that taken, step counts, stride lengths, and cadences were saved.Results: There was not any significant difference between the groups with respect to the limitations in ankle joints' movement and 6 minute total walking distance (p > 0.05). For the other 6MWT walking parametres, there was a statistically significant difference on the stride lengths in the test in favour of (to the good) the group with distal muscle weakness (p< 0.01). There was a negative correlation between the right (r=-0.579, p=0.01) and left (r=-0.445, p= 0.05) ankle dorsiflexion limitations and 6MWT results in the group with ...

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Evaluation of a Program for Long-Term Treatment of Duchenne Muscular Dystrophy. Experience at the University Hospitals of Cleveland*

Cited by 87 publications

References 20 publications

Duchenne muscular dystrophy: the management of scoliosis

Duchenne muscular dystrophy: the management of scoliosis

Aquatic therapy for children with Duchenne muscular dystrophy: a pilot feasibility randomised controlled trial and mixed-methods process evaluation

Farkli Nöromusküler Hastaliklarda Ayak Bi̇leği̇ Li̇mi̇tasyonunun Yürüme Beceri̇si̇ Üzeri̇ne Etki̇si̇

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