Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade

Biggar, W. Douglas; Harris, Valerie; Eliasoph, Laila; Alman, Benjamin A.

doi:10.1016/j.nmd.2006.01.010

Cited by 316 publications

(298 citation statements)

References 38 publications

Supporting

Mentioning

273

Contrasting

Unclassified

Order By: Relevance

“…from deflazacort compared to prednisone, including reduced bone mineral loss, less weight gain, and good evidence of a therapeutic benefit [31,32]. Interestingly, we found that those reporting usage of deflazacort instead of prednisone had a significant delay in age at loss of ambulation.…”

Section: Cc-by-ndmentioning

confidence: 52%

Online Self-Report Data for Duchenne Muscular Dystrophy confirms natural history and can be used to assess for therapeutic benefits

Wang

Fadlon

Ulm

et al. 2014

Preprint

View full text Add to dashboard Cite

To assess the utility of online patient self-report outcomes in a rare disease, we attempted to observe the effects of corticosteroids in delaying age at fulltime wheelchair use in Duchenne muscular dystrophy (DMD) using data from 1,057 males from DuchenneConnect, an online registry. Data collected were compared to prior natural history data in regard to age at diagnosis, mutation spectrum, and age at loss of ambulation. Because registrants reported differences in steroid and other medication usage, as well as age and ambulation status, we could explore these data for correlations with age at loss of ambulation. Using multivariate analysis, current steroid usage was the most significant and largest independent predictor of improved wheelchair-free survival. Thus, these online self-report data were sufficient to retrospectively observe that current steroid use by patients with DMD is associated with a delay in loss of ambulation. Comparing commonly used steroid drugs, deflazacort prolonged ambulation longer than prednisone (median 14 years and 13 years, respectively). Further, use of Vitamin D and Coenzyme Q10, insurance status, and age at diagnosis after 4 years were also significant, but smaller, independent predictors of longer wheelchair-free survival. Nine other common supplements were also individually tested but had lower study power. This study demonstrates the utility of DuchenneConnect data to observe therapeutic differences, and highlights needs for improvement in quality and quantity of patient-report data, which may allow exploration of drug/therapeutic practice combinations impractical to study in clinical trial settings.

show abstract

Section: Cc-by-ndmentioning

confidence: 52%

Online Self-Report Data for Duchenne Muscular Dystrophy confirms natural history and can be used to assess for therapeutic benefits

Wang

Fadlon

Ulm

et al. 2014

Preprint

View full text Add to dashboard Cite

show abstract

“…GC-induced growth retardation was first described 60 years ago after an equivalent cortisone dose of only 1.5 mg/kg/day (Blodgett et al 1956) and can be considerable; by 15 years of age, boys with DMD who are treated with deflazacort are 21 cm shorter on average than untreated boys (Biggar et al 2006). GC-induced growth retardation can also occur following GC exposure by several alternative routes including inhaled GC in asthma (Allen et al 1994) and intraarticular GC injections in juvenile arthritis (Umlawska & Prusek-Dudkiewicz 2010).…”

Section: Introductionmentioning

confidence: 99%

Animal models to explore the effects of glucocorticoids on skeletal growth and structure

Wood

Souček

Wong

et al. 2018

Journal of Endocrinology

View full text Add to dashboard Cite

Glucocorticoids (GCs) are effective for the treatment of many chronic conditions, but their use is associated with frequent and wide-ranging adverse effects including osteoporosis and growth retardation. The mechanisms that underlie the undesirable effects of GCs on skeletal development are unclear, and there is no proven effective treatment to combat them. An in vivo model that investigates the development and progression of GC-induced changes in bone is, therefore, important and a wellcharacterized pre-clinical model is vital for the evaluation of new interventions. Currently, there is no established animal model to investigate GC effects on skeletal development and there are pros and cons to consider with the different protocols used to induce osteoporosis and growth retardation. This review will summarize the literature and highlight the models and techniques employed in experimental studies to date.

show abstract

“…Pulmoner fonksiyonlardaki kayıp-lar ortalama 9-11 yaşlarında başlamaktadır. Zorlu vital kapasitenin (ZVK) yılda ortalama % 5-10 arasında düşmeye başlamasıyla beraber öksürme kuvveti yetersizdir ve zayıf öksürüğe bağlı havayollarından sekresyonun uzaklaştırılmasındaki bozukluk ilerleyen dönemde sık görülen bulgular arasındadır (12)(13)(14)(15). ZVK'deki progresif düşüş, gece hiperkapnisinin gündüzleri de görülmesi şeklinde ilerleyerek ciddi solunum yetmezliğinin gelişmesine zemin hazırlamaktadır (16).…”

Section: Introductionunclassified

“…Gençlik dönemlerinin orta ve son kısmı arasında geceleri ventilasyon yardımcılarına ihtiyaç duyulmaktadır. Gelişmiş pulmoner bakım ve akciğer enfeksiyonlarının tedavisi ile yaşam süresi uzatılabilmektedir (12,13).…”

Section: Introductionunclassified

Duchenne musküler distrofi’de üst ekstremite dinamik egzersizinin solunum fonksiyonu ve yaşam kalitesi üzerine etkisi

Alemdaroğlu

Karaduman

Yılmaz

et al. 2014

Turk J Physiother Rehabil

View full text Add to dashboard Cite

Amaç: Çalışmamızın amacı; Duchenne Musküler Distrofi'li (DMD) çocuklarda üst ekstremite dinamik egzersizinin solunum fonksiyonu ile yaşam kalitesi üzerindeki etkilerini incelemek idi.Yöntemler: Bu çalışmada DMD tanısı almış, Brooke Üst Ekstremite Fonksiyonel Sınıflandır-ması (BÜEFS)'na göre Devre 1 veya 2'de yer alan çocuklar yazı-tura yöntemi ile randomize olarak çalışma ve kontrol gruplarına ayrıldı. Demografik bilgileri kaydedildi. Süreli performans testleri (sırtüstü yatıştan ayağa kalkma, T-shirt giyme/çıkarma), elektronik bir solunum kas kuvveti ölçüm aleti, PedsQL-3.0 Nöromusküler Modül çocuk/ebeveyn anketi sırasıyla üst ekstremite performansları, tepe ekspiratuar akım (TEA) ve sağlıkla ilişkili yaşam kalitelerini değerlendirmek için kullanıldı. Çalışma grubu elektronik kol ergometresinde 8 hafta, haftada 3 gün, ortalama 40 dakika çalıştırıldı. Kontrol grubuna ev programı olarak normal eklem hareketlerini içeren üst ekstremite egzersizleri uygulandı. 8 hafta sonunda değerlendirmeler tekrarlandı. Sonuçlar:Çalışmaya yaş ortalamaları sırasıyla 9.33±1.37 ve 9.50±1.38 olan 12'si kontrol ve 12'si çalışma grubuna toplam 24 DMD'li çocuk dahil edildi. Egzersiz programı sonrası sırtüstü yatıştan ayağa kalkma ve T-shirt giyme süresinde çalışma grubu lehine istatistiksel olarak anlamlı fark bulundu (p<0.05). Çalışma grubunda egzersiz sonrası TEA litre değerinde anlamlı artış bulundu (p<0.01). PedsQL-çocuk skorları ile TEA arasında, pozitif yönde, orta kuvvette (r=0.590; p<0.05); TEA ile T-Shirt giyme süresi arasında negatif yönde, kuvvetli (r=-0.629; p<0.05) ilişki bulundu.Tartışma: Çalışmamızda, DMD'li çocuklarda kol ergometresi ile gerçekleştirilen üst ekstremite dinamik egzersizinin, çocukların solunum performansını arttırdığı ve yaşam kaliteleri üzerinde olumlu etkiye sahip olduğu belirlenmiştir.Anahtar Kelimeler: Duchenne kas distrofisi; ekshalasyon; aerobik egzersiz; üst ekstremite RESEARCH ARTICLE EFFECTS OF UPPER EXTREMITY DYNAMIC EXERCISE ON RESPIRATORY FUNCTION AND QUALITY OF LIFE IN DUCHENNE MUSCULAR DYSTROPHY ABSTRACTPurpose: The aim of this study was to investigate the effects of upper extremity dynamic exercise on respiratory function and quality of life in children with Duchenne Muscular Dystrophy (DMD). Methods:In this study, children who diagnosed DMD scored as Level 1 or 2 according to the Brooke Upper Extremity Functional Classification (BUEFC) were divided into study and control groups with coin toss method randomly. Demographic datas were recorded. Timed performance tests (standing from supine, T-Shirt wearing/removing), electronic respiratory muscle strength device and Pediatric Quality of Life-3.0 (PedsQL-3.0) Neuromuscular Module child/parent questionnaire were used for evaluation of upper extremity performances, peak expiratory flow (PEF) and health-related quality of life, respectively. Study group had trained in arm ergometer, 8 weeks, 3 days per week, mean 40 minutes. Control group was applied normal range of motion upper extremity exercises as home programme. Assessments were repeated aft...

show abstract

Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade

Cited by 316 publications

References 38 publications

Online Self-Report Data for Duchenne Muscular Dystrophy confirms natural history and can be used to assess for therapeutic benefits

Online Self-Report Data for Duchenne Muscular Dystrophy confirms natural history and can be used to assess for therapeutic benefits

Animal models to explore the effects of glucocorticoids on skeletal growth and structure

Duchenne musküler distrofi’de üst ekstremite dinamik egzersizinin solunum fonksiyonu ve yaşam kalitesi üzerine etkisi

Contact Info

Product

Resources

About