Abstract
In Duchenne muscular dystrophy (DMD), progressive loss of respiratory function leads to restrictive pulmonary disease and places patients at significant risk for severe respiratory complications. Of particular concerns are ineffective cough, secretion retention and recurrent respiratory tract infections. In a Phase 3 randomized controlled study (DMD Long-term Idebenone Study, DELOS) in DMD patients 10-18 years of age and not taking concomitant glucocorticoid steroids, idebenone (900 mg/day) reduced significantly the loss of respiratory function over a 1-year study period. In a post-hoc analysis of DELOS we found that more patients in the placebo group compared to the idebenone group experienced bronchopulmonary adverse events (BAEs): placebo: 17 of 33 patients, 28 events; idebenone: 6 of 31 patients, 7 events. The hazard ratios (HR) calculated "by patient" (HR 0.33, p = 0.0187) and for "all BAEs" (HR 0.28, p = 0.0026) indicated a clear idebenone treatment effect. The overall duration of BAEs was 222 days (placebo) vs. 82 days (idebenone). In addition, there was also a difference in the use of systemic antibiotics utilized for the treatment of BAEs. In the placebo group, 13 patients (39.4%) reported 17 episodes of antibiotic use compared to 7 patients (22.6%) reporting 8 episodes of antibiotic use in the idebenone group. Furthermore, patients in the placebo group used systemic antibiotics for longer (105 days) compared to patients in the idebenone group (65 days). This post-hoc analysis of DELOS indicates that the protective effect of idebenone on respiratory function is associated with a reduced risk of bronchopulmonary complications and a reduced need for systemic antibiotics.
| Original language | English (US) |
|---|---|
| Journal | Neuromuscular Disorders |
| DOIs | |
| State | Accepted/In press - Apr 5 2016 |
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Keywords
- Airway infection
- Duchenne muscular dystrophy
- Idebenone
- Respiratory function
ASJC Scopus subject areas
- Pediatrics, Perinatology, and Child Health
- Medicine(all)
- Neurology
- Clinical Neurology
- Genetics(clinical)
Cite this
Idebenone reduces respiratory complications in patients with Duchenne muscular dystrophy. / McDonald, Craig M.; Meier, Thomas; Voit, Thomas; Schara, Ulrike; Straathof, Chiara S M; D'Angelo, M. Grazia; Bernert, Günther; Cuisset, Jean Marie; Finkel, Richard S.; Goemans, Nathalie; Rummey, Christian; Leinonen, Mika; Spagnolo, Paolo; McDonald, Craig M.
In: Neuromuscular Disorders, 05.04.2016.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Idebenone reduces respiratory complications in patients with Duchenne muscular dystrophy
AU - McDonald, Craig M.
AU - Meier, Thomas
AU - Voit, Thomas
AU - Schara, Ulrike
AU - Straathof, Chiara S M
AU - D'Angelo, M. Grazia
AU - Bernert, Günther
AU - Cuisset, Jean Marie
AU - Finkel, Richard S.
AU - Goemans, Nathalie
AU - Rummey, Christian
AU - Leinonen, Mika
AU - Spagnolo, Paolo
AU - McDonald, Craig M
PY - 2016/4/5
Y1 - 2016/4/5
N2 - In Duchenne muscular dystrophy (DMD), progressive loss of respiratory function leads to restrictive pulmonary disease and places patients at significant risk for severe respiratory complications. Of particular concerns are ineffective cough, secretion retention and recurrent respiratory tract infections. In a Phase 3 randomized controlled study (DMD Long-term Idebenone Study, DELOS) in DMD patients 10-18 years of age and not taking concomitant glucocorticoid steroids, idebenone (900 mg/day) reduced significantly the loss of respiratory function over a 1-year study period. In a post-hoc analysis of DELOS we found that more patients in the placebo group compared to the idebenone group experienced bronchopulmonary adverse events (BAEs): placebo: 17 of 33 patients, 28 events; idebenone: 6 of 31 patients, 7 events. The hazard ratios (HR) calculated "by patient" (HR 0.33, p = 0.0187) and for "all BAEs" (HR 0.28, p = 0.0026) indicated a clear idebenone treatment effect. The overall duration of BAEs was 222 days (placebo) vs. 82 days (idebenone). In addition, there was also a difference in the use of systemic antibiotics utilized for the treatment of BAEs. In the placebo group, 13 patients (39.4%) reported 17 episodes of antibiotic use compared to 7 patients (22.6%) reporting 8 episodes of antibiotic use in the idebenone group. Furthermore, patients in the placebo group used systemic antibiotics for longer (105 days) compared to patients in the idebenone group (65 days). This post-hoc analysis of DELOS indicates that the protective effect of idebenone on respiratory function is associated with a reduced risk of bronchopulmonary complications and a reduced need for systemic antibiotics.
AB - In Duchenne muscular dystrophy (DMD), progressive loss of respiratory function leads to restrictive pulmonary disease and places patients at significant risk for severe respiratory complications. Of particular concerns are ineffective cough, secretion retention and recurrent respiratory tract infections. In a Phase 3 randomized controlled study (DMD Long-term Idebenone Study, DELOS) in DMD patients 10-18 years of age and not taking concomitant glucocorticoid steroids, idebenone (900 mg/day) reduced significantly the loss of respiratory function over a 1-year study period. In a post-hoc analysis of DELOS we found that more patients in the placebo group compared to the idebenone group experienced bronchopulmonary adverse events (BAEs): placebo: 17 of 33 patients, 28 events; idebenone: 6 of 31 patients, 7 events. The hazard ratios (HR) calculated "by patient" (HR 0.33, p = 0.0187) and for "all BAEs" (HR 0.28, p = 0.0026) indicated a clear idebenone treatment effect. The overall duration of BAEs was 222 days (placebo) vs. 82 days (idebenone). In addition, there was also a difference in the use of systemic antibiotics utilized for the treatment of BAEs. In the placebo group, 13 patients (39.4%) reported 17 episodes of antibiotic use compared to 7 patients (22.6%) reporting 8 episodes of antibiotic use in the idebenone group. Furthermore, patients in the placebo group used systemic antibiotics for longer (105 days) compared to patients in the idebenone group (65 days). This post-hoc analysis of DELOS indicates that the protective effect of idebenone on respiratory function is associated with a reduced risk of bronchopulmonary complications and a reduced need for systemic antibiotics.
KW - Airway infection
KW - Duchenne muscular dystrophy
KW - Idebenone
KW - Respiratory function
UR - http://www.scopus.com/inward/record.url?scp=84969972170&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84969972170&partnerID=8YFLogxK
U2 - 10.1016/j.nmd.2016.05.008
DO - 10.1016/j.nmd.2016.05.008
M3 - Article
C2 - 27238057
AN - SCOPUS:84969972170
JO - Neuromuscular Disorders
JF - Neuromuscular Disorders
SN - 0960-8966
ER -