Treatment of pediatric pulmonary arterial hypertension: A focus on the NO-sGC-cGMP pathway.


OBJECTIVE:While pulmonary arterial hypertension (PAH) is rare in infants and children, it results in substantial morbidity and mortality. In recent years, prognosis has improved, coinciding with the introduction of new PAH-targeted therapies, although much of their use in children is off-label. Evidence to guide the treatment of children with PAH is less extensive than for adults. The goal of this review is to discuss the treatment recommendations for children with PAH, as well as the evidence supporting the use of prostanoids, endothelin receptor antagonists (ERAs), and phosphodiesterase type 5 inhibitors (PDE5i) in this setting. DATA SOURCES:Nonsystematic PubMed literature search and authors' expertise. STUDY SELECTION:Articles were selected concentrating on the nitric oxide (NO)-soluble guanylate cyclase (sGC)-cyclic guanosine monophosphate (cGMP) pathway in PAH. The methodology of an ongoing study evaluating the sGC stimulator riociguat in children with PAH is also described. RESULTS:Despite recent medical advances, improved therapeutic strategies for pediatric PAH are needed. The efficacy and tolerability of riociguat in adults with PAH have been well trialed. CONCLUSION:The pooling of data across trials, supplemented by registry data, will help to confirm the safety and tolerability of prostanoids, ERAs, and PDE5i in children. Ongoing studies will clarify the place of sGC stimulators in the treatment strategy for pediatric PAH.


Pediatr Pulmonol


Pediatric pulmonology


Beghetti M,Gorenflo M,Ivy DD,Moledina S,Bonnet D




Has Abstract


2019-10-01 00:00:00












  • Small airway involvement in cystic fibrosis lung disease: routine spirometry as an early and sensitive marker.

    abstract:BACKGROUND:In young children with cystic fibrosis (CF) the forced expiratory volume in 1 second (FEV1 ) is often normal and a more sensitive measure to detect early obstructive lung disease is needed. AIM:To evaluate the progression of selected spirometry parameters with age in a cohort of CF patients and healthy chil...

    journal_title:Pediatric pulmonology

    pub_type: 杂志文章


    authors: Bakker EM,Borsboom GJ,van der Wiel-Kooij EC,Caudri D,Rosenfeld M,Tiddens HA

    更新日期:2013-11-01 00:00:00

  • Effects of birthweight and oxygen supplementation on lung function in late childhood in children of very low birth weight.

    abstract::Impaired respiratory function has been found frequently in ex-premature children, but it is unclear which specific factors influence this impairment the most. The aim of this study was to determine the importance of the contributions of birth weight, gestational age, neonatal respiratory disease, and its treatment on ...

    journal_title:Pediatric pulmonology

    pub_type: 杂志文章


    authors: Kennedy JD,Edward LJ,Bates DJ,Martin AJ,Dip SN,Haslam RR,McPhee AJ,Staugas RE,Baghurst P

    更新日期:2000-07-01 00:00:00

  • Diffuse panbronchiolitis in a 10-year-old boy.

    abstract::Diffuse panbronchiolitis is a progressive fatal respiratory disease of unknown cause seen predominantly in Southeast Asian adults. We report this condition in a 10-year-old child of Korean birth because of the uncommon presentation at this age and the favorable outcome associated with early diagnosis. Our objective wa...

    journal_title:Pediatric pulmonology

    pub_type: 杂志文章


    authors: Weinberger M,Fischer A,Kao S

    更新日期:2015-09-01 00:00:00

  • Lung function outcome at school age in very low birth weight children.

    abstract:OBJECTIVE:The aim of this study was to assess pulmonary function and its predictors in very low birth weight (birth weight ≤1,500 g) children (VLBWc) with or without bronchopulmonary dysplasia (BPD), born at gestational age ≤32 weeks at a single tertiary center during 1996-1999, after the introduction of surfactant the...

    journal_title:Pediatric pulmonology

    pub_type: 杂志文章


    authors: Cazzato S,Ridolfi L,Bernardi F,Faldella G,Bertelli L

    更新日期:2013-08-01 00:00:00

  • Assessment of passive respiratory compliance in healthy preterm infants: a critical evaluation.

    abstract::The airway occlusion techniques for assessing passive respiratory mechanics have become well established methods in fullterm neonates and older infants. The single breath technique (SBT) is frequently used for assessing lung function in intubated infants on neonatal intensive care units. However, less is known about t...

    journal_title:Pediatric pulmonology

    pub_type: 杂志文章


    authors: Gappa M,Rabbette PS,Costeloe KL,Stocks J

    更新日期:1993-05-01 00:00:00

  • Relief of central airways obstruction following spinal release in a patient with idiopathic scoliosis.

    abstract::SUMMARY. Patients with idiopathic scoliosis are reported to have a restrictive pattern on pulmonary function tests. A case is presented of a teenage girl with juvenile idiopathic scoliosis who had evidence of airways obstruction in addition to restriction on pulmonary function tests (PFT). Examination of flow-volume l...

    journal_title:Pediatric pulmonology

    pub_type: 杂志文章


    authors: Borowitz D,Armstrong D,Cerny F

    更新日期:2001-01-01 00:00:00

  • Resilience in adolescents and young adults with cystic fibrosis: A pilot feasibility study of the promoting resilience in stress management intervention.

    abstract:BACKGROUND:Disease burden in cystic fibrosis (CF) impacts quality of life, distress, and treatment adherence. The promoting resilience in stress management (PRISM), is a brief patient-focused intervention to promote resilience in adolescents and young adults (AYAs), which may mitigate the negative outcomes, and is prov...

    journal_title:Pediatric pulmonology

    pub_type: 临床试验,杂志文章


    authors: Toprak D,Nay L,McNamara S,Rosenberg AR,Rosenfeld M,Yi-Frazier JP

    更新日期:2020-03-01 00:00:00

  • Comparison of airway reactivity induced by cold air and metacholine challenges in asthmatic children.

    abstract::Bronchial responsiveness to isocapnic hyperventilation with cold air (CAH) and to inhaled methacholine (MCH) was compared in 17 children with bronchial asthma. The response to cold air was expressed as the percent drop in FEV1 from baseline at 4 min. after the challenge (delta % FEV1 CAH), and the response to methacho...

    journal_title:Pediatric pulmonology

    pub_type: 临床试验,杂志文章


    authors: de Benedictis FM,Canny GJ,MacLusky IB,Levison H

    更新日期:1995-06-01 00:00:00

  • Progressive lung and cardiac changes associated with pulmonary hypertension in the fetal rat.

    abstract::To determine the natural history of lung vascular remodeling and cardiac changes in the rat model of persistent pulmonary hypertension syndrome (PPHN) of the newborn, we studied fetal rats subjected to maternal indomethacin administration initiated on day 19 of gestation and continued for 2, 3, or 4 days. Animals rece...

    journal_title:Pediatric pulmonology

    pub_type: 杂志文章


    authors: Fabris VE,Pato MD,Belik J

    更新日期:2001-05-01 00:00:00

  • Measurement of single breath-hold carbon monoxide diffusing capacity in healthy infants and toddlers.

    abstract::We describe a method for measuring carbon monoxide diffusing capacity (DL(CO)) and alveolar volume (V(A)) in sleeping infants, using a single 4-sec breath-hold technique. The breath-hold maneuver is obtained by inducing a respiratory pause of the respiratory system. Several inflations of the respiratory system with ro...

    journal_title:Pediatric pulmonology

    pub_type: 杂志文章


    authors: Castillo A,Llapur CJ,Martinez T,Kisling J,Williams-Nkomo T,Coates C,Tepper RS

    更新日期:2006-06-01 00:00:00

  • CFTR, bicarbonate, and the pathophysiology of cystic fibrosis.

    abstract::The gene that encodes for the cystic fibrosis transmembrane regulator protein (CFTR) was identified in 1989, yet major pathophysiologic questions remain unanswered. There is emerging evidence that CFTR is a bicarbonate channel, a driver of chloride-bicarbonate exchange and through its action on local pH, a regulator o...

    journal_title:Pediatric pulmonology

    pub_type: 杂志文章,评审


    authors: Borowitz D

    更新日期:2015-10-01 00:00:00

  • Optimization of anti-pseudomonal antibiotics for cystic fibrosis pulmonary exacerbations: IV. colistimethate sodium.

    abstract::Patients with cystic fibrosis (CF) often experience acute pulmonary exacerbations (APE) and may be treated with a wide variety of intravenous antibiotics. The aim of this review is to provide an evidence-based summary of pharmacokinetic/pharmacodynamic (PK/PD), tolerability, and efficacy studies utilizing the intraven...

    journal_title:Pediatric pulmonology

    pub_type: 杂志文章


    authors: Young DC,Zobell JT,Waters CD,Ampofo K,Stockmann C,Sherwin CM,Spigarelli MG

    更新日期:2013-01-01 00:00:00

  • Down-regulation of regulatory proteins for differentiation and proliferation in murine fetal hypoplastic lungs: altered mesenchymal-epithelial interactions.

    abstract::We compared proliferation (growth) and differentiation (development) related proteins in normal and hypoplastic fetal murine lungs. The hypoplastic lungs were created in CD-1 fetal mice by nitrofen exposure (25 mg per pregnant mouse given intragastrically on gestational day 8 [Gd8]), as published earlier. The lungs we...

    journal_title:Pediatric pulmonology

    pub_type: 杂志文章


    authors: Chinoy MR,Chi X,Cilley RE

    更新日期:2001-08-01 00:00:00

  • Early elevations in B-type natriuretic peptide levels are associated with poor clinical outcomes in pediatric acute lung injury.

    abstract:OBJECTIVES:To determine B-type natriuretic peptide (BNP) levels in infants and children with acute lung injury (ALI), and to investigate associations between BNP levels and clinical outcome. DESIGN:Prospective observational study. SUBJECTS:After informed consent, plasma was collected from 48 pediatric patients on day...

    journal_title:Pediatric pulmonology

    pub_type: 杂志文章


    authors: Reel B,Oishi PE,Hsu JH,Gildengorin G,Matthay MA,Fineman JR,Flori H

    更新日期:2009-11-01 00:00:00

  • Effectiveness and tolerability of high-dose salmeterol in cystic fibrosis.

    abstract::The efficacy and tolerability of high-dose salmeterol (100 mcg, BID) and albuterol (2.5 mg, BID) were compared with those of albuterol (2.5 mg, BID) in outpatients with cystic fibrosis in a randomized, double-blind, double-dummy, placebo-controlled, crossover study with both short- (4 weeks of each) and long-term (24 ...

    journal_title:Pediatric pulmonology

    pub_type: 临床试验,杂志文章,随机对照试验


    authors: Hordvik NL,Sammut PH,Judy CG,Colombo JL

    更新日期:2002-10-01 00:00:00

  • Pulmonary function, serum markers of inflammation, and IgG antibodies to core lipopolysaccharide of Burkholderia cepacia in adults with cystic fibrosis, following colonization with Burkholderia cepacia.

    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


    authors: Hendry J,Nixon L,Dodd M,Elborn JS,Govan J,Shale DJ,Webb AK

    更新日期:2000-01-01 00:00:00

  • Current understanding of the inflammatory process in cystic fibrosis: onset and etiology.

    abstract::Although airway obstruction and chronic endobronchial infection have long been recognized as major factors in the pathogenesis of lung disease in cystic fibrosis (CF), only recently has it been recognized that the inflammatory process itself may be responsible in a major way for destroying the lungs. The most characte...

    journal_title:Pediatric pulmonology

    pub_type: 杂志文章


    authors: Konstan MW,Berger M

    更新日期:1997-08-01 00:00:00

  • Associations between age and sleep apnea risk among newborn infants.

    abstract:OBJECTIVE:Among older children, sleep-disordered breathing (SDB) is associated with measurable neurocognitive consequences. However, diagnostic SDB thresholds are lacking for infants < 12 months. We sought to evaluate the relationship between SDB indices, gestational age (GA), and postmenstrual age (PMA) for infants wh...

    journal_title:Pediatric pulmonology

    pub_type: 杂志文章


    authors: Matlen LB,Hassan F,Shellhaas RA

    更新日期:2019-08-01 00:00:00

  • NAVA-synchronized compared to nonsynchronized noninvasive ventilation for apnea, bradycardia, and desaturation events in VLBW infants.

    abstract::Neurally adjusted ventilatory assistance (NAVA) can overcome technical difficulties with synchronizing noninvasive ventilation breaths with the patient, a modality often used in very low birthweight infants (VLBW) with apnea of prematurity (AOP). This study is a retrospective single-center investigation into whether N...

    journal_title:Pediatric pulmonology

    pub_type: 杂志文章


    authors: Tabacaru CR,Moores RR Jr,Khoury J,Rozycki HJ

    更新日期:2019-11-01 00:00:00

  • Correlation of six different cystic fibrosis chest radiograph scoring systems with clinical parameters.

    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


    authors: Terheggen-Lagro S,Truijens N,van Poppel N,Gulmans V,van der Laag J,van der Ent C

    更新日期:2003-06-01 00:00:00

  • Cold air challenge in children with asthma.

    abstract::A study was designed to determine the sensitivity and specificity of a cold air bronchial provocation test. A total of 18 children with asthma (mean age 12 years) and 18 normal children (mean age 14 years) were studied. The cold air challenge consisted of a 4 min period of isocapnic hyperventilation of subfreezing air...

    journal_title:Pediatric pulmonology

    pub_type: 杂志文章


    authors: Reisman J,Mappa L,de Benedictis F,McLaughlin J,Levison H

    更新日期:1987-07-01 00:00:00

  • Infrared end-tidal CO2 measurement does not accurately predict arterial CO2 values or end-tidal to arterial PCO2 gradients in rabbits with lung injury.

    abstract::End-tidal PCO2 (PETCO2) measurements from two commercially available neonatal infrared capnometers with different sampling systems and a mass spectrometer were compared with arterial PCO2 (PaCO2) to determine whether the former could predict the latter in mechanically ventilated rabbits with and without lung injury. T...

    journal_title:Pediatric pulmonology

    pub_type: 杂志文章


    authors: Hopper AO,Nystrom GA,Deming DD,Brown WR,Peabody JL

    更新日期:1994-03-01 00:00:00

  • Advance care planning in adolescents with cystic fibrosis: A quality improvement project.

    abstract:INTRODUCTION:Advance care planning (ACP), though recommended, has not been studied in adolescents with cystic fibrosis (CF). This quality improvement project engaged adolescents with advanced CF disease in ACP and assessed patient and CF provider attitudes and preferences regarding ACP discussions and tools. MATERIALS...

    journal_title:Pediatric pulmonology

    pub_type: 杂志文章


    authors: Kazmerski TM,Weiner DJ,Matisko J,Schachner D,Lerch W,May C,Maurer SH

    更新日期:2016-12-01 00:00:00

  • Evaluation of the interrupter technique for the use of assessing airway obstruction in children.

    abstract::The purpose of this study was to determine if the interrupter technique, a noninvasive method for measuring airflow resistance, could be used to assess airway obstruction in children. In 107 children (74 with asthma, 12 with cystic fibrosis, and 21 without lung disease) conductance (mostly of airways) measured with th...

    journal_title:Pediatric pulmonology

    pub_type: 杂志文章


    authors: Carter ER,Stecenko AA,Pollock BH,Jaeger MJ

    更新日期:1994-04-01 00:00:00

  • The association between birth season and future development of childhood asthma.

    abstract::The aim of this study was to examine a possible association between birth season (date of birth) and future development of asthma in children. A case-control study was designed to include asthmatic children aged 2-7 years, living in the city of Beer-Sheva, in southern Israel, registered in one pediatric center. Contro...

    journal_title:Pediatric pulmonology

    pub_type: 杂志文章


    authors: Gazala E,Ron-Feldman V,Alterman M,Kama S,Novack L

    更新日期:2006-12-01 00:00:00

  • Salivary biomarkers of obstructive sleep apnea syndrome in children.

    abstract:OBJECTIVES:The present pilot study was performed to evaluate the HPA axis and ANS activity by measuring salivary cortisol and α-amylase diurnal trajectory and production, respectively, in mild or moderate-to-severe (MS) OSA-affected, but otherwise healthy, children. Moreover, a correlative analysis was performed betwee...

    journal_title:Pediatric pulmonology

    pub_type: 杂志文章


    authors: Patacchioli FR,Tabarrini A,Ghiciuc CM,Dima-Cozma LC,Prete A,Bianchini C,Nicoletti F,Gozal D,Villa MP

    更新日期:2014-11-01 00:00:00

  • Doppler echocardiographic evaluation of pulmonary artery pressure in pneumonia of infants and children.

    abstract::Previously, there was insufficient evidence to confirm that pneumonia in infants and children might lead to the development of pulmonary hypertension. Recently, it has been shown that acceleration time corrected for heart rate (ATc) and the ratios of right ventricular preejection period to right ventricular ejection t...

    journal_title:Pediatric pulmonology

    pub_type: 杂志文章


    authors: Du JB,Li SZ,Wang BL,Li YA

    更新日期:1991-01-01 00:00:00

  • Disseminated aspergillosis in a patient with cystic fibrosis and allergic bronchopulmonary aspergillosis.

    abstract::Allergic bronchopulmonary aspergillosis (ABPA) is a well-known complication of cystic fibrosis (CF), with an estimated incidence of up to 11%. In patients with CF, the diagnosis of ABPA must be based on significant elevation of Aspergillus fumigatus (Af) antibody and total serum IgE, since it is common to already have...

    journal_title:Pediatric pulmonology

    pub_type: 杂志文章


    authors: Chung Y,Kraut JR,Stone AM,Valaitis J

    更新日期:1994-02-01 00:00:00

  • Influence of jacket tightness and pressure on raised lung volume forced expiratory maneuvers in infants.

    abstract::While the use of the raised volume rapid thoraco-abdominal compression (RVRTC) technique has been shown to provide new insights into airway and pulmonary pathophysiology in infants, and appears to resemble the spirometric techniques used in older subjects, there is as yet no consensus regarding measurement procedures,...

    journal_title:Pediatric pulmonology

    pub_type: 杂志文章


    authors: Lum S,Hoo AF,Stocks J

    更新日期:2002-11-01 00:00:00

  • Care recommendations for the respiratory complications of esophageal atresia-tracheoesophageal fistula.

    abstract::Tracheoesophageal fistula (TEF) with esophageal atresia (EA) is a common congenital anomaly that is associated with significant respiratory morbidity throughout life. The objective of this document is to provide a framework for the diagnosis and management of the respiratory complications that are associated with the ...

    journal_title:Pediatric pulmonology

    pub_type: 杂志文章


    authors: Koumbourlis AC,Belessis Y,Cataletto M,Cutrera R,DeBoer E,Kazachkov M,Laberge S,Popler J,Porcaro F,Kovesi T

    更新日期:2020-10-01 00:00:00