Treatment effect of idebenone on inspiratory function in patients with Duchenne muscular dystrophy

Autor: Buyse, G.M., Voit, Thomas, Schara, Ulrike, Straathof, C.S.M., D’Angelo, M.G., Bernert, G., Cuisset, J.-M., Finkel, Richard S., Goemans, N., Rummey, Christian, Leinonen, Mika, Mayer, Oscar H., Spagnolo, Paolo, Meier, Thomas, McDonald, Craig M., Knipp, F., Van den Hauwe, M., Doppler, V., Gidaro, T., Coopman, S., Lutz, S., Kirschner, J., Borell, S., Will, M., Brighina, E., Gandossini, S., Gorni, K., Falcier, E., Politano, L., D’Ambrosio, P., Taglia, A., Verschuuren, J.J.G.M., Vílchez Padilla, J.J., Muelas Gómez, N., Sejersen, T., Hovmöller, M., Jeannet, P.-Y., Bloetzer, C., Iannaccone, S., Castro, D., Tennekoon, G., Bönnemann, C., Henricson, E., Joyce, N., Apkon, S., Richardson, R.C.
Přispěvatelé: Van den Hauwe, M
Jazyk: angličtina
Rok vydání: 2016
Předmět:
0301 basic medicine
Male
Ubiquinone
Duchenne muscular dystrophy
Medizin
Pediatrics
Antioxidants
Pulmonary function testing
law.invention
0302 clinical medicine
Randomized controlled trial
law
respiratory function
Idebenone
Respiratory function
Lung volumes
Muscular Dystrophy
Child
Genetics (clinical)
Respiration
Perinatology and Child Health
Inspiratory flow
Pediatrics
Perinatology and Child Health
Pulmonary and Respiratory Medicine
3. Good health
Respiratory Function Tests
Treatment Outcome
Neurology
Cardiology
Inspiratory Reserve Volume
inspiratory flow
Female
Original Article
medicine.drug
medicine.medical_specialty
Adolescent
Outcomes
Placebo
03 medical and health sciences
FEV1/FVC ratio
Internal medicine
medicine
Humans
Treatment effect
In patient
business.industry
Original Articles
idebenone
Muscular Dystrophy
Duchenne
Duchenne
medicine.disease
Surgery
030104 developmental biology
Neurology (clinical)
business
030217 neurology & neurosurgery
Zdroj: Pediatric Pulmonology
Neuromuscular Disorders, 26, S156-S157
r-IIS La Fe. Repositorio Institucional de Producción Científica del Instituto de Investigación Sanitaria La Fe
instname
ISSN: 8755-6863
Popis: Assessment of dynamic inspiratory function may provide valuable information about the degree and progression of pulmonary involvement in patients with Duchenne muscular dystrophy (DMD). The aims of this study were to characterize inspiratory function and to assess the efficacy of idebenone on this pulmonary function outcome in a large and well-characterized cohort of 10-18 year-old DMD patients not taking glucocorticoid steroids (GCs) enrolled in the phase 3 randomized controlled DELOS trial. We evaluated the effect of idebenone on the highest flow generated during an inspiratory FVC maneuver (maximum inspiratory flow; V'I,max(FVC)) and the ratio between the largest inspiratory flow during tidal breathing (tidal inspiratory flow; V'I,max(t)) and the V'I,max(FVC). The fraction of the maximum flow that is not used during tidal breathing has been termed inspiratory flow reserve (IFR). DMD patients in both treatment groups of DELOS (idebenone, n = 31; placebo: n = 33) had comparable and abnormally low V'I,max(FVC) at baseline. During the study period, V'I,max(FVC) further declined by -0.29 L/sec in patients on placebo (95%CI: -0.51, -0.08; P = 0.008 at week 52), whereas it remained stable in patients on idebenone (change from baseline to week 52: 0.01 L/sec; 95%CI: -0.22, 0.24; P = 0.950). The between-group difference favoring idebenone was 0.27 L/sec (P = 0.043) at week 26 and 0.30 L/sec (P = 0.061) at week 52. In addition, during the study period, IFR improved by 2.8% in patients receiving idebenone and worsened by -3.0% among patients on placebo (between-group difference 5.8% at week 52; P = 0.040). Although the clinical interpretation of these data is currently limited due to the scarcity of routine clinical practice experience with dynamic inspiratory function outcomes in DMD, these findings from a randomized controlled study nevertheless suggest that idebenone preserved inspiratory muscle function as assessed by V'I,max(FVC) and IFR in patients with DMD. Pediatr Pulmonol. © 2016 The Authors. Pediatric Pulmonology Published by Wiley Periodicals, Inc. ispartof: Pediatric Pulmonology vol:52 issue:4 pages:508-515 ispartof: location:United States status: published
Databáze: OpenAIRE
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