Abstract:
: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. 2017;52:508-515. © 2016 The Authors. Pediatric Pulmonology Published by Wiley Periodicals, Inc.
journal_name
Pediatr Pulmonoljournal_title
Pediatric pulmonologyauthors
Buyse GM,Voit T,Schara U,Straathof CS,D'Angelo MG,Bernert G,Cuisset JM,Finkel RS,Goemans N,Rummey C,Leinonen M,Mayer OH,Spagnolo P,Meier T,McDonald CM,DELOS Study Group.doi
10.1002/ppul.23547subject
Has Abstractpub_date
2017-04-01 00:00:00pages
508-515issue
4eissn
8755-6863issn
1099-0496journal_volume
52pub_type
杂志文章,多中心研究,随机对照试验abstract::To assess whether there is a maturation of the response to tube breathing during the first 10 days of life, we have measured the dynamic ventilatory responses of nine term infants (34 studies) to added tubes equivalent to two anatomical dead spaces. Such a method allows an assessment of the baby's ability to increase ...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.1950090203
更新日期:1990-01-01 00:00:00
abstract::Little attention has been focused on the progressive pulmonary deterioration which occurs in mechanically ventilated infants with normal or mildly abnormal lungs. We hypothesized that lung function would deteriorate over a 24-hr period in anesthetized neonatal piglets with normal lungs mechanically ventilated at 2 cm ...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.1950190306
更新日期:1995-03-01 00:00:00
abstract:RATIONALE:Nitric oxide (NO) produced in the lung is an important mediator of normal lung development, vascular smooth muscle relaxation, and ventilation perfusion matching. NO is synthesized from arginine by the action of NO-synthase (NOS). Asymmetric dimethylarginine (ADMA), an endogenous derivate of arginine, inhibit...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.20886
更新日期:2008-12-01 00:00:00
abstract::Respiratory input impedance (Zin) is a potentially informative test of pulmonary function in infants who are unable to perform standard tests commonly performed in children and adults Analysis of Zin in dogs using the six-element model of DuBois et al. (J Appl Physiol 8:587, 1956) provides estimates of airways resista...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/(SICI)1099-0496(199612)22:6<364::AID-PPUL5
更新日期:1996-12-01 00:00:00
abstract::The effects of current methods of bone marrow transplantation (BMT) on pulmonary function in children have not been extensively studied. We reviewed serial pulmonary function tests (PFTs) in 25 children (median age, 9 yr; range, 4-15) who received allogeneic (n = 14) or autologous (n = 11) BMT for neoplastic diseases ...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.1950180604
更新日期:1994-12-01 00:00:00
abstract:BACKGROUND:Treatment of bronchopulmonary dysplasia (BPD) remains as yet an unmet clinical need and recently stem cells have been proposed as a therapeutic tool in animal models. We investigated the role of amniotic fluid stem cells (AFS) in an adult rat model of hyperoxia lung injury. METHODS:Fifty Sprague-Dawley rats...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.22791
更新日期:2013-11-01 00:00:00
abstract::The purpose of this report is to describe the College of American Pathologists sweat testing (SW) proficiency testing program for cystic fibrosis, to evaluate its impact on test performance, and to describe the current practice of sweat testing in North America. The study analyzed participant summary reports of the SW...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/1099-0496(200012)30:6<476::aid-ppul7>3.0.c
更新日期:2000-12-01 00:00:00
abstract:OBJECTIVES:A pilot study to compare pulmonary mechanics in a neonatal intensive care unit (NICU) cohort, specifically, comparing lung elastance between male and female infants in the NICU. HYPOTHESIS:Anecdotally, male infants are harder to ventilate than females. We hypothesize that males have higher model-based elast...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.24538
更新日期:2020-01-01 00:00:00
abstract::We used an in vitro model system to examine the sites of deposition of aqueous therapeutic aerosols administered through 3-mm, 6-mm, and 9-mm endotracheal tubes (commonly used in infants, children, and adults) at clinically relevant inspiratory flow rates. Aerosol was delivered to the endotracheal tube via a "T" piece...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.1950020108
更新日期:1986-01-01 00:00:00
abstract::The cotton rat was evaluated as a model for anti-inflammatory and antiviral influenza therapy. Beginning 3 days after intranasal infection with 10(7) tissue culture infectious doses-50% (TCID)(50) of an H3N2 human influenza, animals were treated topically via intranasal lavage with a range of doses of triamcinolone ac...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.10320
更新日期:2003-10-01 00:00:00
abstract::We studied the effect of low-protein diet (8% casein) on lung growth in rats from 3 to 7 weeks of age. Their diet was isocaloric with that of control animals fed a diet of 20% casein. The calorie intake of experimental animals was increased during the first 3 weeks of the experiment, but they increased less (about 10%...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.1950070308
更新日期:1989-01-01 00:00:00
abstract::We tested a health education intervention program to reduce passive smoking in infancy. The aim was to develop an instrument for study of tobacco smoke exposure and childhood respiratory illness. One hundred and eighty-four women who had smoked during pregnancy were allocated by month of delivery to an intervention gr...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.1950030311
更新日期:1987-05-01 00:00:00
abstract::The free running test is a useful method for evaluation of exercise-induced bronchospasm in children. In young children this test simulates real-life circumstances and can be done more easily than histamine or methacholine challenges. The interrupter technique is a noninvasive method for measuring airflow resistance d...
journal_title:Pediatric pulmonology
pub_type: 临床试验,杂志文章
doi:10.1002/(sici)1099-0496(199903)27:3<203::aid-ppul9
更新日期:1999-03-01 00:00:00
abstract:OBJECTIVE:To define the benefits of a flexible bronchoscopy (FB) service in a Paediatric Intensive Care Unit (PICU). DESIGN:Review of the first 200 FBs undertaken in a large PICU. SETTING:Large cardiac and medical PICU in the United Kingdom, also providing extra-corporeal life support. PATIENTS:129 patients (78 male...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.20910
更新日期:2008-12-01 00:00:00
abstract::To determine the extent of pulmonary dysfunction following primary closure of an abdominal wall defect, we obtained pulmonary function tests (PFT) in 11 newborn infants with gastroschisis and 6 with large omphaloceles admitted to a newborn ICU in a children's hospital. Patients were 1 to 30 days of age at the time of ...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.1950120309
更新日期:1992-03-01 00:00:00
abstract::Asthma has been found related to a higher risk of psychological problems. Inconsistent results have been reported with respect to the quality of life of children with asthma. So there may be a complex relationship between asthma severity, quality of life, and psychological factors. The aim of this study was to examine...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.20509
更新日期:2007-01-01 00:00:00
abstract:BACKGROUND:Most methods used for the assessment of severe steatorrhea in cystic fibrosis (CF) are sensitive. In fact, the tests show their usefulness in a borderline zone of the results. Yet, the existing data related to acid steatocrit (AS) are still contradictory. Therefore, in the present study we have aimed to asse...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.21149
更新日期:2010-03-01 00:00:00
abstract::We report on a 7-month old infant with severe respiratory distress secondary to a paratracheal bronchogenic cyst. Respiratory relief was achieved by transtracheal puncture of the cyst. Surgical removal of the cyst was performed 1 week later because of radiological evidence of reaccumulation of fluid. ...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.10408
更新日期:2004-12-01 00:00:00
abstract:BACKGROUND:Respiratory viral and atypical bacterial infections are associated with pulmonary exacerbations and hospitalisations in cystic fibrosis patients. We wanted to study the impact of such infections on children attending the outpatient clinic. METHODS:Seventy-five children were followed for 12 months at regular...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.20517
更新日期:2006-12-01 00:00:00
abstract::"Pulmonary Interstitial Glycogenosis: Diagnostic Evaluation and Clinical Course," written by Liptzin et al is a timely and insightful phenotypic summary of a rare pediatric interstitial lung disease. Twenty-four infants with biopsy-proven pulmonary interstitial glycogenosis (PIG) were reviewed at their center. Genetic...
journal_title:Pediatric pulmonology
pub_type: 评论,杂志文章
doi:10.1002/ppul.24167
更新日期:2018-12-01 00:00:00
abstract:BACKGROUND:Children with adenoid hypertrophy (AH) have impaired respiratory system defense mechanisms, such as mucociliary clearance. We hypothesized that AH negatively affects one of the most important aspects of mucociliary clearance-ciliary beat frequency (CBF) and that adenoidectomy could potentially restore this e...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.24622
更新日期:2020-03-01 00:00:00
abstract::A 14-year-old Korean boy was admitted with cough, hemoptysis, and fever. A chest X-ray showed a solitary pulmonary mass and pneumonitis. Bronchial biopsy by fiberoptic bronchoscopy revealed a poorly differentiated small cell carcinoma. All of the staging information indicated that the patient had limited disease. Duri...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/(sici)1099-0496(200004)29:4<317::aid-ppul1
更新日期:2000-04-01 00:00:00
abstract:OBJECTIVE:To determine the effect of changing electrode positions on vital signs and respiratory effort parameters measured with transcutaneous electromyography of the diaphragm (dEMG) in preterm infants. METHODS:In this observational study, simultaneous dEMG measurements were performed at the standard position and at...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.24585
更新日期:2020-02-01 00:00:00
abstract:BACKGROUND:sTREM-1 (soluble triggering receptor expressed on myeloid cells-1) is a novel inflammatory marker that may be of clinical use in cystic fibrosis (CF). Dysregulation of the TREM pathway has been demonstrated in other inflammatory diseases and modulation in animal models has therapeutic benefit. We hypothesise...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.23650
更新日期:2017-04-01 00:00:00
abstract::Pneumocyte apoptosis is implicated in the pathophysiology of acute inflammatory lung injuries in newborns and adults. Pulmonary angiotensin (ANG) II contributes to lung epithelial apoptosis in vitro, but its role in acute lung injury in vivo is unclear. We therefore studied the effects of ANG II receptor action on the...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.20187
更新日期:2005-04-01 00:00:00
abstract:OBJECTIVES:To investigate the effect of position on the strength of the Hering-Breuer reflex in prematurely born infants and determine whether any differences seen were related to differences in lung or tidal volume between positions. WORKING HYPOTHESIS:Position related differences in the strength of the Hering-Breuer...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.20855
更新日期:2008-08-01 00:00:00
abstract:OBJECTIVE:Lung ultrasound (LUS) is an emerging tool that may be used in the diagnosis and follow-up of children with viral bronchiolitis. In this study, we describe LUS abnormalities in children receiving invasive mechanical ventilation (IMV) for severe bronchiolitis in the pediatric intensive care unit (PICU). Our aim...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.24974
更新日期:2020-10-01 00:00:00
abstract::Twenty patients with cystic fibrosis (CF) were trained to mobilize intrabronchial secretions by a new method of positive expiratory pressure mask (PEP mask) physiotherapy (PT). Patients repeatedly expired forcefully through the PEP mask; expiratory resistance was varied by eight different internal diameter resistors a...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.1950020608
更新日期:1986-11-01 00:00:00
abstract::Maternal retinoid administration has beneficial effects on lung development in the nitrofen rodent toxic model of congenital diaphragmatic hernia (DH). We wanted to investigate the effects in a surgical model, where the retinoid signaling pathway is not primarily disrupted by the toxic agent. We created DH in fetal ra...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.20829
更新日期:2008-06-01 00:00:00
abstract::We present a 9-month-old infant with persistent cough refractory to conventional asthma therapy. An extensive evaluation eventually revealed a Chiari I malformation with syringohydromyelia. His cough resolved one month after surgical decompression, suggesting that brainstem compression from the Chiari malformation dir...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.20912
更新日期:2008-10-01 00:00:00