McDonald, CM, Meier T, Voit T, et al. “Idebenone Reduces Respiratory Complications In Patients with Duchenne Muscular Dystrophy.” Neuromuscular Disorders May 2016
Submitted by Leigh Maria K. Ramos Platt, MD, News Science Editorial Board
Idebenone is a short chain benzoquinone with well-known antioxidant properties. This article reports the data from a multi-center Phase III randomized controlled trial to study the effects of Idebenone in patients with Duchenne Muscular Dystrophy (DMD Long-term Idebenone Study, DELOS). Patients enrolled had to be either steroid naïve or off-steroid for at least 12 months. The DELOS trial mainly measured the incidence of bronchopulmonary adverse events (BAEs) and the need for systemic antibiotics in the duration of the study (52 weeks). BAEs consisted of upper respiratory tract infections, bronchitis, pneumonia, cough, Influenza with respiratory symptoms, viral infection with respiratory symptoms, acute respiratory failure, dyspnea, laryngitis, and respiratory failure. 64 boys with DMD between the ages of 10 and 18 years old who were largely in the non-ambulatory phase of their disease were randomized into a placebo group (33 boys) and a 900mg/day Idebenone group (31 boys) with the placebo group being slightly older (15 years vs 13.5 years). 6 patients had 7 BAEs in the Idebenone group as compared with 17 patients with 28 BAEs in the placebo group. 7/31 boys had 8 events requiring the need for systemic antibiotics in the Idebenone group in comparison to 13/33 boys with 17 events in the placebo group. Furthermore, patients in the placebo group used systemic antibiotics for longer (105 days) compared to patients in the idebenone group (65 days). Therefore, it was concluded that the use of Idebenone appears to decrease the incidence of both BAEs and need for systemic antibiotics in patients not treated with steroids between the ages of 10-18 years.
Progressive loss of respiratory function leading to restricted pulmonary disease is a significant cause of morbidity and mortality in DMD patients. This DELOS trial showed that Idebenone at 900 mg/day oral dosing provides statistically significant benefits to protect the respiratory function and slow the progression of pulmonary disease compared with placebo in steroid naïve or steroid-off DMD patients over a 1-year study period. It broadens our therapeutic approaches to this devastating disease. Data from studies in patients who are taking Idebenone and steroids concomitantly would be very helpful.
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