Characterization of pulmonary function in 10-18 year old patients with Duchenne muscular dystrophy. 2017

Thomas Meier, and Christian Rummey, and Mika Leinonen, and Paolo Spagnolo, and Oscar H Mayer, and Gunnar M Buyse, and
Santhera Pharmaceuticals, Liestal, Switzerland.

Pulmonary function loss in patients with Duchenne muscular dystrophy (DMD) is progressive and leads to pulmonary insufficiency. The purpose of this study in 10-18 year old patients with DMD is the assessment of the inter-correlation between pulmonary function tests (PFTs), their reliability and the association with the general disease stage measured by the Brooke score. Dynamic PFTs (peak expiratory flow [PEF], forced vital capacity [FVC], forced expiratory volume in one second [FEV1]) and maximum static airway pressures (MIP, MEP) were prospectively collected from 64 DMD patients enrolled in the DELOS trial (ClinicalTrials.gov, number NCT01027884). Baseline PEF percent predicted (PEF%p) was <80% and patients had stopped taking glucocorticoids at least 12 months prior to study start. At baseline PEF%p, FVC%p and FEV1%p correlated well with each other (Spearman's rho: PEF%p-FVC%p: 0.54; PEF%p-FEV1%p: 0.72; FVC%p-FEV1%p: 0.91). MIP%p and MEP%p correlated well with one another (MIP%p-MEP%p: 0.71) but less well with PEF%p (MIP%p-PEF%p: 0.40; MEP%p-PEF%p: 0.41) and slightly better with FVC%p (MIP%p-FVC%p: 0.59; MEP%p-FVC%p: 0.74). The within-subject coefficients of variation (CV) for successive measures were 6.97% for PEF%p, 6.69% for FVC%p and 11.11% for FEV1%p, indicating that these parameters could be more reliably assessed compared to maximum static airway pressures (CV for MIP%p: 18.00%; MEP%p: 15.73%). Yearly rates of PFT decline (placebo group) were larger in dynamic parameters (PEF%p: -8.9% [SD 2.0]; FVC%p: -8.7% [SD 1.1]; FEV1%p: -10.2% [SD 2.0]) than static airway pressures (MIP%p: -4.5 [SD 1.3]; MEP%p: -2.8 [SD 1.1]). A considerable drop in dynamic pulmonary function parameters was associated with loss of upper limb function (transition from Brooke score category 4 to category 5). In conclusion, these findings expand the understanding of the reliability, correlation and evolution of different pulmonary function measures in DMD patients who are in the pulmonary function decline phase.

UI MeSH Term Description Entries
D008137 Longitudinal Studies Studies in which variables relating to an individual or group of individuals are assessed over a period of time. Bogalusa Heart Study,California Teachers Study,Framingham Heart Study,Jackson Heart Study,Longitudinal Survey,Tuskegee Syphilis Study,Bogalusa Heart Studies,California Teachers Studies,Framingham Heart Studies,Heart Studies, Bogalusa,Heart Studies, Framingham,Heart Studies, Jackson,Heart Study, Bogalusa,Heart Study, Framingham,Heart Study, Jackson,Jackson Heart Studies,Longitudinal Study,Longitudinal Surveys,Studies, Bogalusa Heart,Studies, California Teachers,Studies, Jackson Heart,Studies, Longitudinal,Study, Bogalusa Heart,Study, California Teachers,Study, Longitudinal,Survey, Longitudinal,Surveys, Longitudinal,Syphilis Studies, Tuskegee,Syphilis Study, Tuskegee,Teachers Studies, California,Teachers Study, California,Tuskegee Syphilis Studies
D012129 Respiratory Function Tests Measurement of the various processes involved in the act of respiration: inspiration, expiration, oxygen and carbon dioxide exchange, lung volume and compliance, etc. Lung Function Tests,Pulmonary Function Tests,Function Test, Pulmonary,Function Tests, Pulmonary,Pulmonary Function Test,Test, Pulmonary Function,Tests, Pulmonary Function,Function Test, Lung,Function Test, Respiratory,Function Tests, Lung,Function Tests, Respiratory,Lung Function Test,Respiratory Function Test,Test, Lung Function,Test, Respiratory Function,Tests, Lung Function,Tests, Respiratory Function
D012140 Respiratory Tract Diseases Diseases involving the RESPIRATORY SYSTEM. Respiratory Diseases,Respiratory System Diseases,Disease, Respiratory System,Disease, Respiratory Tract,Respiratory System Disease,Respiratory Tract Disease
D002648 Child A person 6 to 12 years of age. An individual 2 to 5 years old is CHILD, PRESCHOOL. Children
D003430 Cross-Sectional Studies Studies in which the presence or absence of disease or other health-related variables are determined in each member of the study population or in a representative sample at one particular time. This contrasts with LONGITUDINAL STUDIES which are followed over a period of time. Disease Frequency Surveys,Prevalence Studies,Analysis, Cross-Sectional,Cross Sectional Analysis,Cross-Sectional Survey,Surveys, Disease Frequency,Analyses, Cross Sectional,Analyses, Cross-Sectional,Analysis, Cross Sectional,Cross Sectional Analyses,Cross Sectional Studies,Cross Sectional Survey,Cross-Sectional Analyses,Cross-Sectional Analysis,Cross-Sectional Study,Cross-Sectional Surveys,Disease Frequency Survey,Prevalence Study,Studies, Cross-Sectional,Studies, Prevalence,Study, Cross-Sectional,Study, Prevalence,Survey, Cross-Sectional,Survey, Disease Frequency,Surveys, Cross-Sectional
D006801 Humans Members of the species Homo sapiens. Homo sapiens,Man (Taxonomy),Human,Man, Modern,Modern Man
D000293 Adolescent A person 13 to 18 years of age. Adolescence,Youth,Adolescents,Adolescents, Female,Adolescents, Male,Teenagers,Teens,Adolescent, Female,Adolescent, Male,Female Adolescent,Female Adolescents,Male Adolescent,Male Adolescents,Teen,Teenager,Youths
D020388 Muscular Dystrophy, Duchenne An X-linked recessive muscle disease caused by an inability to synthesize DYSTROPHIN, which is involved with maintaining the integrity of the sarcolemma. Muscle fibers undergo a process that features degeneration and regeneration. Clinical manifestations include proximal weakness in the first few years of life, pseudohypertrophy, cardiomyopathy (see MYOCARDIAL DISEASES), and an increased incidence of impaired mentation. Becker muscular dystrophy is a closely related condition featuring a later onset of disease (usually adolescence) and a slowly progressive course. (Adams et al., Principles of Neurology, 6th ed, p1415) Becker Muscular Dystrophy,Duchenne Muscular Dystrophy,Muscular Dystrophy, Becker,Muscular Dystrophy, Pseudohypertrophic,Becker's Muscular Dystrophy,Cardiomyopathy, Dilated, 3B,Cardiomyopathy, Dilated, X-Linked,Childhood Muscular Dystrophy, Pseudohypertrophic,Childhood Pseudohypertrophic Muscular Dystrophy,Duchenne and Becker Muscular Dystrophy,Duchenne-Becker Muscular Dystrophy,Duchenne-Type Progressive Muscular Dystrophy,Muscular Dystrophy Pseudohypertrophic Progressive, Becker Type,Muscular Dystrophy, Becker Type,Muscular Dystrophy, Childhood, Pseudohypertrophic,Muscular Dystrophy, Duchenne Type,Muscular Dystrophy, Duchenne and Becker Types,Muscular Dystrophy, Pseudohypertrophic Progressive, Becker Type,Muscular Dystrophy, Pseudohypertrophic Progressive, Duchenne Type,Muscular Dystrophy, Pseudohypertrophic, Childhood,Progressive Muscular Dystrophy, Duchenne Type,Pseudohypertrophic Childhood Muscular Dystrophy,Pseudohypertrophic Muscular Dystrophy, Childhood,Duchenne Becker Muscular Dystrophy,Duchenne Type Progressive Muscular Dystrophy,Muscular Dystrophy, Becker's,Muscular Dystrophy, Duchenne-Becker,Pseudohypertrophic Muscular Dystrophy

Related Publications

Thomas Meier, and Christian Rummey, and Mika Leinonen, and Paolo Spagnolo, and Oscar H Mayer, and Gunnar M Buyse, and
February 2009, Pediatrics international : official journal of the Japan Pediatric Society,
Thomas Meier, and Christian Rummey, and Mika Leinonen, and Paolo Spagnolo, and Oscar H Mayer, and Gunnar M Buyse, and
April 2019, Paediatric respiratory reviews,
Thomas Meier, and Christian Rummey, and Mika Leinonen, and Paolo Spagnolo, and Oscar H Mayer, and Gunnar M Buyse, and
December 2020, Journal of clinical and translational hepatology,
Thomas Meier, and Christian Rummey, and Mika Leinonen, and Paolo Spagnolo, and Oscar H Mayer, and Gunnar M Buyse, and
February 1977, Israel journal of medical sciences,
Thomas Meier, and Christian Rummey, and Mika Leinonen, and Paolo Spagnolo, and Oscar H Mayer, and Gunnar M Buyse, and
August 2007, Nederlands tijdschrift voor geneeskunde,
Thomas Meier, and Christian Rummey, and Mika Leinonen, and Paolo Spagnolo, and Oscar H Mayer, and Gunnar M Buyse, and
May 2015, Pediatric pulmonology,
Thomas Meier, and Christian Rummey, and Mika Leinonen, and Paolo Spagnolo, and Oscar H Mayer, and Gunnar M Buyse, and
July 1983, Journal of pediatric orthopedics,
Thomas Meier, and Christian Rummey, and Mika Leinonen, and Paolo Spagnolo, and Oscar H Mayer, and Gunnar M Buyse, and
May 2009, Journal of neurology,
Thomas Meier, and Christian Rummey, and Mika Leinonen, and Paolo Spagnolo, and Oscar H Mayer, and Gunnar M Buyse, and
January 1989, Pediatric pulmonology,
Thomas Meier, and Christian Rummey, and Mika Leinonen, and Paolo Spagnolo, and Oscar H Mayer, and Gunnar M Buyse, and
October 1997, Chest,
Copied contents to your clipboard!