Ventilatory assistance in patients with Duchenne muscular dystrophy

Lofaso, Frédéric; David, Olivier; Jc, Raphael

doi:10.1183/09031936.06.00059906

Cited by 24 publications

(25 citation statements)

References 10 publications

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“…Compared to tracheostomy ventilation, NIV greatly simplifies administration of care, is more comfortable for patients and reduces costs [49][50][51]. Night-time NIV means that patients are not restricted in the daytime and enables them to be cared for at home, whereas with long-term tracheostomy ventilation patients may need admission to healthcare services or highly trained and well-motivated home professional or familiar caregivers.…”

Section: Niv Versus Tracheostomymentioning

confidence: 99%

Chronic respiratory care for neuromuscular diseases in adults

Ambrosino¹,

Carpene²,

Gherardi³

2009

Eur Respir J

162

121

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Neuromuscular diseases (NMD) may affect respiratory muscles, leading to respiratory failure. Studies show that long-term noninvasive mechanical ventilation (NIV) improves symptoms, gas exchange, quality of life and survival. NIV improved these parameters in muscular dystrophies and also in patients with amyotrophic lateral sclerosis without severe bulbar dysfunction. NIV should be started at the onset of nocturnal hypoventilation. In selected cases, NIV may be simpler, better accepted by patients and cheaper than invasive mechanical ventilation, but it cannot be used as an alternative. Tracheostomy may be preferred by patients unable to protect their airways and wishing to survive as long as possible, or by ventilatordependent patients.Glossopharyngeal breathing consists of taking air and propelling it into the lungs. Chest percussions and vibrations can help to mobilise airway secretions but they cannot substitute coughing. Manually assisted coughing requires substantial lung inflation through air stacking or deep lung insufflation, followed by an abdominal thrust with open glottis. The combination of mechanical in-exsufflation with an abdominal thrust is a mechanically assisted cough.In conclusion, recent advances in respiratory care of NMD have improved prognosis and many caregivers have changed from a traditional noninterventional to a more aggressive, supportive approach.

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Section: Niv Versus Tracheostomymentioning

confidence: 99%

Chronic respiratory care for neuromuscular diseases in adults

Ambrosino¹,

Carpene²,

Gherardi³

2009

Eur Respir J

162

121

View full text Add to dashboard Cite

show abstract

“…However, the commentary by LOFASO et al [3] on this outstanding work was uninformed. The commentators failed to present the outcomes of continuous NIV from other centres [4][5][6] and they have, apparently, no experience in continuous long-term NIV.…”

mentioning

confidence: 97%

“…Together, we currently have more than 1,000 continuous NIV users in our centres, some aged .50 yrs and most using a simple mouthpiece during daytime hours. LOFASO et al [3] are correct to state that there are ''no controlled studies that demonstrate that NIV prolongs life.'' The evidence to date suggests that there is no clinical equipoise regarding the use of NIV.…”

mentioning

confidence: 99%

“…As such, withholding such support would be unethical. LOFASO et al [3] quote a conservative consensus statement which suggested that when NIV is inadequate ''tracheostomy may be considered'', but they have never reported using continuous NIV for their own patients. The recent American Thoracic Society consensus panel endorsed NIV as the therapy of choice in supporting breathing in DMD, while allowing for the possibility of tracheostomy in cases in which bulbar weakness precluded its use or expert support for NIV was unavailable locally [8].…”

mentioning

confidence: 99%

“…The recent American Thoracic Society consensus panel endorsed NIV as the therapy of choice in supporting breathing in DMD, while allowing for the possibility of tracheostomy in cases in which bulbar weakness precluded its use or expert support for NIV was unavailable locally [8]. LOFASO et al [3] also seem to be unaware that there were signicantly fewer hospitalisations and pulmonary complications in the NIV group as compared with the tracheostomy group [9].…”

mentioning

confidence: 99%

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Tracheostomy tubes are not needed for Duchenne muscular dystrophy

Bach¹,

Bíanchi²,

Finder³

et al. 2007

Eur Respir J

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Population longitudinal analysis of Gait Profile Score and North Star Ambulatory Assessment in children with Duchenne muscular dystrophy

Deng,

Liu,

Feng

et al. 2024

CPT Pharmacom & Syst Pharma

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Duchenne muscular dystrophy (DMD) is a rare X‐linked recessive disorder characterized by loss‐of‐function mutations in the gene encoding dystrophin. These mutations lead to progressive functional deterioration including muscle weakness, respiratory insufficiency, and musculoskeletal deformities. Three‐dimensional gait analysis (3DGA) has been used as a tool to analyze gait pathology through the quantification of altered joint kinematics, kinetics, and muscle activity patterns. Among 3DGA indices, the Gait Profile Score (GPS), has been used as a sensitive overall measure to detect clinically relevant changes in gait patterns in children with DMD. To enhance our understanding of the clinical translation of 3DGA, we report here the development of a population nonlinear mixed‐effect model that jointly describes the disease progression of the 3DGA index, GPS, and the functional endpoint, North Star Ambulatory Assessment (NSAA). The final model consists of a quadratic structure for GPS progression and a linear structure for GPS‐NSAA correlation. Our model was able to capture the improvement in function in GPS and NSAA in younger subjects, as well as the decline of function in older subjects. Furthermore, the model predicted NSAA (CFB) at 1 year reasonably well for DMD subjects ≤7 years old at baseline. The model tended to slightly underpredict the decline in NSAA after 1 year for those >7 years old at baseline, but the prediction summary statistics were well maintained within the standard deviation of observed data. Quantitative models such as this may help answer clinically relevant questions to facilitate the development of novel therapies in DMD.

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Ventilatory assistance in patients with Duchenne muscular dystrophy

Cited by 24 publications

References 10 publications

Chronic respiratory care for neuromuscular diseases in adults

Chronic respiratory care for neuromuscular diseases in adults

Tracheostomy tubes are not needed for Duchenne muscular dystrophy

Population longitudinal analysis of Gait Profile Score and North Star Ambulatory Assessment in children with Duchenne muscular dystrophy

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