Neuromuscular disorders : NMD
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Neuromuscul. Disord. · Jan 2011
Duchenne muscular dystrophy: survival by cardio-respiratory interventions.
We describe survival in Duchenne dystrophy by invasive and noninvasive ventilation vs. untreated. Patients were untreated prior to 1984 (Group 1), underwent tracheotomy from 1984 until 1991 (Group 2), and were managed by noninvasive mechanical ventilation and cardioprotective medications subsequently (Group 3). Symptoms, vital capacity, and blood gases were monitored for all and spirometry, cough peak flows, carbon dioxide tension, and oximetry for Group 3. ⋯ Survival was compared by Kaplan-Meier analysis. The 56 of Group 1 died at 18.6±2.9, the 21 Group 2 at 28.1±8.3 years of age with three still alive, and the 88 using noninvasive ventilation had 50% survival to 39.6 years, p<0.001, respectively. We conclude that noninvasive mechanical ventilation and assisted coughing provided by specifically trained physicians and therapists, and cardioprotective medication can result in more favorable outcomes and better survival by comparison with invasive treatment.
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Neuromuscul. Disord. · Aug 2010
Clinical TrialImpact of tracheostomy on swallowing performance in Duchenne muscular dystrophy.
Mechanical ventilation has improved survival in patients with Duchenne muscular dystrophy (DMD). Over time, these patients experience upper airway dysfunction, swallowing impairments, and dependency on the ventilator that may require invasive mechanical ventilation via a tracheostomy. Tracheostomy is traditionally believed to further impair swallowing. ⋯ Half the swallows were followed by inspiration before tracheostomy. Total bolus swallowing time was significantly shorter (P=0.009), and the number of swallows per bolus significantly smaller (P=0.01), after than before tracheostomy. Invasive ventilation via a tracheostomy may improve swallowing.
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Neuromuscul. Disord. · Jun 2010
Randomized Controlled Trial Multicenter Study Comparative StudyOral dexamethasone pulse therapy versus daily prednisolone in sub-acute onset myositis, a randomised clinical trial.
To determine if high-dose pulsed dexamethasone is more effective and safer than daily high-dose prednisolone in treatment-naive adult patients with inflammatory myopathies (sporadic inclusion body myositis excluded) we performed a multicenter, double-blind randomised controlled clinical trial with 18 months follow-up. Sixty-two patients were randomised into 28-day cycles of oral high-dose dexamethasone or daily high-dose prednisolone. Primary outcome measures included (1) seven point composite score of six clinically relevant outcomes and (2) (time-to) remission and (time-to) relapse. ⋯ Side-effects occurred significantly less frequently in the dexamethasone group. Median time to relapse was 60 (2.9) weeks in the prednisolone and 44 (4.7) weeks in the dexamethasone group (log-rank test p=0.03). In conclusion, pulsed high-dose oral dexamethasone is not superior to daily prednisolone as first-line treatment of idiopathic inflammatory myopathies, but is a good alternative by causing substantially fewer side-effects.