Abstract:
BACKGROUND:Mesenchymal stromal cell (MSC)-mediated therapeutic effects have been observed in the treatment of lung diseases. For the first time, this treatment was used as rescue therapy in a pediatric patient with a life-threatening respiratory syndrome associated with the filamin A (FLNA) gene mutation. METHODS:A child with a new pathogenic variant of the FLNA gene c.7391_7403del (p.Val2464AlafsTer5), at the age of 18 months, due to serious and irreversible chronic respiratory failure, was treated with repeated intravenous infusions of allogeneic bone marrow (BM)-MSCs. The child's respiratory condition was monitored. Immunologic studies before each MSC treatment were performed. RESULTS:No acute adverse events related to the MSC infusions were observed. After the second infusion, the child's respiratory condition progressively improved, with reduced necessity for mechanical ventilation support. A thorax computed tomography (CT) scan showed bilateral recovery of the basal parenchyma, anatomical-functional alignment and aerial penetration improvement. After the first MSC administration, an increase in Th17 and FoxP3+ T percentages in the peripheral blood was observed. After the second MSC infusion, a significant rise in the Treg/Th17 ratio was noted, as well as an increased percentage of CD20+ /CD19+ B lymphocytes and augmented PHA-induced proliferation. DISCUSSION:MSC infusions are a promising therapeutic modality for patients in respiratory failure, as observed in this pediatric patient with an FLNA mutation. MSCs may have an immunomodulatory effect and thus mitigate lung injury; although in this case, MSC antimicrobial effects may have synergistically impacted the clinical improvements. Further investigations are planned to establish the safety and efficacy of this treatment option for interstitial lung diseases in children.
journal_name
Pediatr Pulmonoljournal_title
Pediatric pulmonologyauthors
Pelizzo G,Avanzini MA,Lenta E,Mantelli M,Croce S,Catenacci L,Acquafredda G,Ferraro AL,Giambanco C,D'Amelio L,Giordano S,Re G,Zennaro F,Calcaterra Vdoi
10.1002/ppul.24497subject
Has Abstractpub_date
2020-01-01 00:00:00pages
190-197issue
1eissn
8755-6863issn
1099-0496journal_volume
55pub_type
杂志文章abstract::In 1994-1995 a cross-sectional epidemiological study investigating the respiratory health of school children in two Taiwan areas was conducted; one area was located in a region with petrochemical manufacturing complexes (Linyuan), and the other was situated in a reference area with no local industrial emissions (Taihs...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/(sici)1099-0496(199805)25:5<299::aid-ppul2
更新日期:1998-05-01 00:00:00
abstract:OBJECTIVE:Extremely low gestational age neonates (ELGANs) are at risk for pulmonary hypertension (PH). We hypothesized that PH, defined by echocardiogram at 36 weeks gestational age (GA), would associate with respiratory morbidity, increased oxidant stress, and reduced nitric oxide production. STUDY DESIGN:ELGANs in t...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.23913
更新日期:2018-03-01 00:00:00
abstract:OBJECTIVES:In hospitalized and nonhospitalized children with asthma exacerbations, we evaluated the determinants of (a) prolonged cough on day-14 and (b) asthma quality of life (QoL) questionnaires for parents (PACQLQ) on day-21. We hypothesized that children with more severe acute asthma are more likely to have prolon...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.25168
更新日期:2020-11-12 00:00:00
abstract:BACKGROUND:The purpose of this study was to evaluate the utility of the Cystic Fibrosis Questionnaire-Revised (CFQ-R) with toddlers and preschool-aged children. Clinically relevant relations between health-related quality of life (HRQOL), stress, and mealtime behaviors have not been examined. It was hypothesized that p...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.23149
更新日期:2015-06-01 00:00:00
abstract::The impulse oscillometry system (IOS) was introduced as a new technique to assess airflow obstruction in patients who are not able to perform forced breathing maneuvers, e.g., subjects with cerebral palsy or severe mental retardation, and young children. This study evaluates the sensitivity and specificity of IOS para...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.10235
更新日期:2003-03-01 00:00:00
abstract:OBJECTIVE:Alveolar concentration (C(A)NO) and bronchial flux (J(aw)NO) of nitric oxide (NO) characterize the contributions of peripheral and proximal airways to exhaled NO. Both parameters can be estimated using a two-compartment model if the fraction of NO in orally exhaled air (FE(NO)) is measured at multiple constan...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.22550
更新日期:2012-11-01 00:00:00
abstract::Follow-up studies have demonstrated that bronchiolitis caused by respiratory syncytial virus (RSV) is strongly associated with wheezing in the ensuing years. During the acute infection the immune response may induce long-lasting detrimental effects, thereby contributing to post-bronchiolitis wheezing (PBW). Therefore,...
journal_title:Pediatric pulmonology
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.1002/1099-0496(200008)30:2<92::aid-ppul3>3.0.co
更新日期:2000-08-01 00:00:00
abstract:BACKGROUND:Pressure rise time (PRT), also known as slope time to the peak inflating pressure can be set on some modern neonatal ventilators. On other ventilators, PRT is determined by the set circuit flow. Changing slope time can affect mean airway pressure (MAP), oxygenation, and carbon dioxide elimination. Our aim wa...
journal_title:Pediatric pulmonology
pub_type: 临床试验,杂志文章
doi:10.1002/ppul.24724
更新日期:2020-05-01 00:00:00
abstract::We assessed the ability of innovative lung function tests to detect bronchial obstruction induced by methacholine bronchial challenge. Fifty-five recurrently wheezy infants (mean age 16 +/- 5.2 months) free of respiratory symptoms underwent baseline lung function tests. Forty-two completed the methacholine challenge. ...
journal_title:Pediatric pulmonology
pub_type: 临床试验,杂志文章
doi:10.1002/ppul.1950180508
更新日期:1994-11-01 00:00:00
abstract::Our objective was to determine whether a simple method of maintaining positive pressure ventilation during nonbronchoscopic bronchoalveolar lavage (NB-BAL) would successfully reduce the incidence and/or severity of desaturation events. Our design was a clinical trial with historical controls. Seventy ventilated pediat...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.20082
更新日期:2004-09-01 00:00:00
abstract::We report on a 1-month-old boy who presented with a "horseshoe" lung, complete "O" rings of the trachea, and an unusual course of the left pulmonary artery mimicking a left pulmonary artery sling. Computed tomography, cardiac catheterization, and bronchoscopy were performed to confirm the rare coexistence of these ano...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.10172
更新日期:2002-11-01 00:00:00
abstract::In contrast to its effect on airway smooth muscle in the adult, in vitro studies have shown that caffeine significantly increases active tension in airway smooth muscle in the neonatal lamb. To determine if caffeine has a physiological effect on airway function during early development, we studied the effect of caffei...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.1950120106
更新日期:1992-01-01 00:00:00
abstract::In past decades, several chest radiograph scoring systems for cystic fibrosis were developed. This study was performed to compare interobserver variability of six different radiograph scores and to correlate them with clinical parameters. Thirty chest radiographs of 30 patients with cystic fibrosis were scored accordi...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.10280
更新日期:2003-06-01 00:00:00
abstract::We retrospectively reviewed the spectrum, course, and outcome of pulmonary diseases in 66 children with AIDS, hospitalized between 1982 and 1988, prior to the use of zidovudine. Fifty-two of the 66 (79%) patients developed pulmonary problems. In fifty-two percent of all patients, a pulmonary problem was the first symp...
journal_title:Pediatric pulmonology
pub_type: 评论,杂志文章
doi:10.1002/ppul.1950100402
更新日期:1991-01-01 00:00:00
abstract:OBJECTIVES:Childhood interstitial lung disease (chILD) is a group of rare chronic and complex disorders of variable pathology. There has been no systematic review of published chILD research. This study aimed to describe chILD classification systems, epidemiology, morbidity, treatments, outcomes, and the impact of chIL...
journal_title:Pediatric pulmonology
pub_type: 杂志文章,评审
doi:10.1002/ppul.23183
更新日期:2015-12-01 00:00:00
abstract::Central ventilation disorders(1) and airway obstruction(2) with chronic hypoxemia are causally related to cor pulmonale. Pulmonary vascular resistance is often reversible, and hypoxic pulmonary hypertension often responds to treatment with supplemental oxygen. Oxygen therapy during sleep may be useful as a temporary p...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.1037
更新日期:2001-03-01 00:00:00
abstract::We present a child with interstitial lung disease (ILD) who was diagnosed with desquamative interstitial pneumonitis following CT thorax and open lung biopsy aged 2 years. Subsequently, surfactant protein gene analysis revealed mutations in the ABCA3 gene. This case of ABCA3 deficient ILD describes a stable clinical c...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.22811
更新日期:2014-03-01 00:00:00
abstract::The term "atypical" pneumonia has been used to differentiate infections caused by Chlamydia pneumoniae, Mycoplasma pneumoniae, Legionella, and other related organisms from pneumonia caused by classic bacteria, the prototype being Streptococcus pneumoniae. However, recent studies demonstrated that the clinical presenta...
journal_title:Pediatric pulmonology
pub_type: 杂志文章,评审
doi:10.1002/ppul.10326
更新日期:2003-11-01 00:00:00
abstract:INTRODUCTION:Chest impedance (CI) is the current standard for cardio-respiratory monitoring in preterm infants but fails to provide direct and quantitative information on diaphragmatic activity. Transcutaneous electromyography (dEMG) is able to measure diaphragmatic activity, but its feasibility and repeatability to mo...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.23116
更新日期:2015-09-01 00:00:00
abstract:OBJECTIVES:Our therapeutic approach to a habit/tic cough is simple reassurance in a single consultation. To quality assure our practice, we followed up children to determine outcomes at least 3 months after diagnosis. DESIGN:Consecutive children diagnosed over 6 years were studied. Medical records were analyzed retros...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.23948
更新日期:2018-04-01 00:00:00
abstract::We have studied the effect of triamcinolone acetonide (TAC) on airway morphogenesis of the Sprague-Dawley fetal rat in whole organ lung cultures from day 15 to day 21 of equivalent gestational age (6 days in culture). TAC produced an increased number of peripheral buds from day 18 onward and the airway and airspaces h...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.1950140107
更新日期:1992-09-01 00:00:00
abstract:OBJECTIVE:Since preventive therapies for bronchopulmonary dysplasia (BPD) are limited we treated preterm infants with azithromycin to decrease the incidence of BPD. METHODS:Infants less than 1,250 g birth weight were randomized to azithromycin or placebo within 12 hr of beginning mechanical ventilation and within 72 h...
journal_title:Pediatric pulmonology
pub_type: 杂志文章,随机对照试验
doi:10.1002/ppul.21352
更新日期:2011-02-01 00:00:00
abstract::Eight patients with cystic fibrosis [CF] colonized with Pseudomonas aeruginosa (P. aeruginosa) had serial lung function, peripheral blood inflammatory markers, and serum IgG antibodies to Burkholderia cepacia (B. cepacia) lipopolysaccharide measured in the months preceding and following colonisation with B. cepacia. O...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/(sici)1099-0496(200001)29:1<8::aid-ppul2>3
更新日期:2000-01-01 00:00:00
abstract::In order to help specialists involved in CF care and clinical research to know the current best evidence about clinical effectiveness of interventions in CF, we designed and developed a web-based, free access tool called "CFDB"--Cystic Fibrosis DataBase (www.inetflow.it/CFDB). The database was built by searching in Me...
journal_title:Pediatric pulmonology
pub_type: 信件
doi:10.1002/ppul.22954
更新日期:2014-09-01 00:00:00
abstract::Tracheobronchomegaly (Mounier-Kuhn syndrome) is characterized by dilatation of the central airways, tracheobronchial outpouchings, and chronic tracheobronchitis. Most cases are diagnosed in adulthood. We report the clinical, radiographic, and bronchoscopic findings in a 14-year-old boy with tracheobronchomegaly, ptosi...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/(sici)1099-0496(200006)29:6<476::aid-ppul1
更新日期:2000-06-01 00:00:00
abstract:BACKGROUND:Juvenile dermatomyositis (JDM) is the main cause of chronic idiopathic inflammatory myopathy of autoimmune origin in children. The aim of this multicenter prospective study was to describe respiratory status and treatment of children followed for JDM. METHODS AND PATIENTS:Clinical manifestations, pulmonary ...
journal_title:Pediatric pulmonology
pub_type: 杂志文章,多中心研究
doi:10.1002/ppul.22742
更新日期:2013-10-01 00:00:00
abstract::Our purpose was to identify and further characterize physiologic mechanisms relevant to autoresuscitation from hypoxic apnea in infants dying suddenly and unexpectedly. We studied cardiorespiratory recordings of 24 infants (age range, 0.8-21 months) who died suddenly while being monitored at home. These recordings wer...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.10287
更新日期:2003-08-01 00:00:00
abstract::Respiratory inductive plethysmography (RIP) is a simple technique for an objective, noninvasive assessment of thoracoabdominal asynchrony, which in turn is an indirect measure of airway obstruction. We evaluated different indices of asynchrony obtained by RIP before and after methacholine-induced airway obstruction. B...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.1950200610
更新日期:1995-12-01 00:00:00
abstract::In adults abdominal obesity is related to lung dysfunction and waist circumference (WC) predicts pulmonary function. It is not known how WC affects pulmonary function in children. A cross-sectional study of 718 children 6-17 years of age was conducted in a rural community to determine the predictability of WC for pulm...
journal_title:Pediatric pulmonology
pub_type: 杂志文章
doi:10.1002/ppul.20854
更新日期:2009-03-01 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