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1.
Phys Med ; 81: 155-161, 2021 Jan.
Artigo em Inglês | MEDLINE | ID: mdl-33461028

RESUMO

PURPOSE: The use of coronary computed tomography angiography (CCTA) in children remains limited by patient's irradiation, and motion artefacts impairing image quality. Triggering the acquisition at the appropriate moment, and acquiring only necessary components of the cardiac cycle could overcome these limitations. Yet, optimal cardiac intervals to perform CCTA as a function of heart rate (HR) have not yet been addressed in pediatrics. METHODS: Fifty children with coronary artery anomalies underwent a CCTA on a wide-coverage single-beat CT scanner. Multiple phases from 25% to 85% of the R-R interval were acquired and reconstructed with 10% increments. Two radiologists independently assessed motion artifacts on each cardiac phase using a 4-point semi-quantitative scale. RESULTS: At patient level, the best phase for acquisition was found in diastole for patients with HR ≤ 75 bpm and in systole for patients with HR > 85 bpm. At coronary segments and structures level, median optimal phases were reported at 70%, 80%, 47%, 50%, and 54% of the R-R interval for patients with HR ≤ 60, 61-75, 86-100, 101-130, and >130 bpm respectively. For patients with HR between 76 and 85 bpm, no clear trend could be observed. Optimal acquisition durations represented 10% (2 phases), 20% (3 phases), 50% (multiphase), 20% (3 phases), and 10% (2 phases) of the R-R interval for patients with HR ≤ 60, 61-75, 76-100, 101-130, and >130 bpm, respectively. CONCLUSIONS: Optimal positioning and duration of CCTA acquisition intervals were investigated as a function of children's HR, to reduce motion artifacts and patient's irradiation.

2.
Health Qual Life Outcomes ; 19(1): 23, 2021 Jan 19.
Artigo em Inglês | MEDLINE | ID: mdl-33468144

RESUMO

BACKGROUND: Recent advances in the field of congenital heart disease (CHD) led to an improved prognosis of the patients and in consequence the growth of a new population: the grown up with congenital heart disease. Until recently, more than 50% of these patients were lost to follow up because of the lack of specialized structures. The critical moment is the transition between paediatric and adult unit. Therapeutic education is crucial to solve this issue by helping patients to become independent and responsible. The TRANSITION-CHD randomized trial aims to assess the impact of a transition education program on health-related quality of life (HRQoL) of adolescents and young adults with CHD. METHODS: Multicentre, randomised, controlled, parallel arm study in CHD patients aged from 13 to 25 years old. Patients will be randomised into 2 groups (education program vs. no intervention). The primary outcome is the change in self-reported HRQoL between baseline and 12-month follow-up. A total of 100 patients in each group is required to observe a significant increase of the overall HRQoL score of 7 ± 13.5 points (on 100) with a power of 80% and an alpha risk of 5%. The secondary outcomes are: clinical outcomes, cardiopulmonary exercise test parameters (peak VO2, VAT, VE/VCO2 slope), level of knowledge of the disease using the Leuven knowledge questionnaire for CHD, physical and psychological status. DISCUSSION: As the current research is opening on patient related outcomes, and as the level of proof in therapeutic education is still low, we sought to assess the efficacy of a therapeutic education program on HRQoL of CHD patients with a randomized trial. TRIAL REGISTRATION: This study was approved by the National Ethics Committee (South-Mediterranean IV 2016-A01681-50) and was registered on Clinicaltrials.gov (NCT03005626).


Assuntos
Cardiopatias Congênitas/psicologia , Educação de Pacientes como Assunto , Qualidade de Vida , Transição para Assistência do Adulto , Adolescente , Adulto , Humanos , Ensaios Clínicos Controlados Aleatórios como Assunto , Projetos de Pesquisa , Adulto Jovem
3.
J Pediatr ; 2020 Dec 15.
Artigo em Inglês | MEDLINE | ID: mdl-33333115

RESUMO

OBJECTIVE: To assess the inspiratory demand in young infants with acute viral bronchiolitis to provide a physiological basis for initial flow setting for patients supported with high flow nasal cannula. STUDY DESIGN: Prospective study in 44 infants up to 6 months old with acute viral bronchiolitis, admitted to a pediatric intensive care unit from November 2017 to March 2019. Airflow measurements were performed using spirometry. The primary endpoint was the inspiratory demand as measured by peak tidal inspiratory flow (PTIF). The secondary endpoints were the relationships determined between PTIF, patient weight, and disease severity. RESULTS: Median (Q25-Q75) age and weight of the patients were 37 (20-67) days and 4.3 (3.5-5.0) kg, respectively. Mean PTIF was 7.45 (95% CI 6.51-8.39, min-max: 2.40-16.00) L/minute. PTIF indexed to weight was 1.68 (95% CI 1.51-1.85, min-max: 0.67-3.00) L/kg/minute. PTIF was <2.5 L/kg/minute in 89% (95% CI 75-96) of infants. PTIF was correlated with weight (ρ= 0 .55, P < .001) but not with markers of disease severity, including modified Woods clinical asthma score, Silverman-Andersen score, respiratory rate, fraction of inspired oxygen, and PCO2. CONCLUSIONS: High flow nasal cannula therapy is used commonly to support infants with acute viral bronchiolitis. The efficiency of the device is optimal if the flow setting matches the patient's inspiratory demand. According to our results, a flow rate of <2.5 L/kg/minute would be appropriate in most situations.

4.
Health Qual Life Outcomes ; 18(1): 366, 2020 Nov 12.
Artigo em Inglês | MEDLINE | ID: mdl-33183312

RESUMO

BACKGROUND: In the context of tremendous progress in congenital cardiology, more attention has been given to patient-related outcomes, especially in assessing health-related quality of life (HRQoL) of patients with congenital heart diseases (CHD). However, most studies have mainly focused on teenagers or adults and currently, few HRQoL controlled data is available in young children. This study aimed to evaluate HRQoL of children with CHD aged 5 to 7 y.o., in comparison with contemporary peers recruited in school, as well as the factors associated with HRQoL in this population. METHODS: This multicentre controlled prospective cross-sectional study included 124 children with a CHD (mean age = 6.0 ± 0.8 y, 45% female) during their outpatient visit and 125 controls (mean age = 6.2 ± 0.8 y, 54% female) recruited at school. A generic paediatric HRQoL instrument was used (PedsQL 4.0). RESULTS: Self-reported HRQoL in children with CHD was similar to controls, overall (73.5 ± 1.2 vs. 72.8 ± 1.2, P = 0.7, respectively), and for each dimension. Parents-reported HRQoL was significantly lower in the CHD group than in controls. HRQoL was predicted by the disease severity and by repeated invasive cardiac procedures (surgery or catheterization). CONCLUSION: HRQoL in young children with CHD aged 5 to 7 years old was good and similar to controls. This study contributed to the growing body of knowledge on HRQoL in congenital cardiology and emphasized the need for child and family support in the most complex CHD. Trial registration This study was approved by the institutional review board of Montpellier University Hospital (2019_IRB-MTP_02-19) on 22 February 2019 and was registered on ClinicalTrials.gov (NCT03931096) on 30 April 2019, https://clinicaltrials.gov/ct2/show/NCT03931096 .

5.
Stem Cell Res ; 49: 102094, 2020 Nov 19.
Artigo em Inglês | MEDLINE | ID: mdl-33246213

RESUMO

Duchenne Muscular Dystrophy (DMD) is a X-linked degenerative pathology with a prevalence of 1/3600-6000 boys due to the absence of functional dystrophin in muscles. This muscular disease leads to skeletal muscle damages, respiratory failure and in the later stages dilated cardiomyopathy (DCM) leading to heart failure. We generated iPSC lines from three different DMD patients carrying respectively deletions of exons 1, 52 and 55 in the dystrophin gene. The reprogrammed iPSC lines showed expression of pluripotent markers, capacity to differentiate in trilineage embryonic layers and a normal karyotype.

6.
Br J Clin Pharmacol ; 2020 Oct 28.
Artigo em Inglês | MEDLINE | ID: mdl-33118199

RESUMO

AIM: Infantile haemangioma (IH) is the most common benign tumour in children. Since 2014, propranolol has become the first-choice therapy and currently Hemangiol is the only approved drug for complicated haemangioma. This post-marketing study reports the use of Hemangiol for IH in paediatric practice. METHOD AND RESULTS: From January 2014 to November 2018, 94 children (median age 4 [0; 21] months; 75% female) treated with Hemangiol for proliferative IH were enrolled in the study. The systematic paediatric cardiology consultation never contraindicated beta-blockers. Two Hemangiol initiation protocols were used: a conventional ambulatory 3-week titration phase protocol (n = 76, 80.9%), and a rapid initiation protocol with a 48-hour dose escalation in conventional hospitalization for severe proliferative or ulcerated IH (n = 18, 19.1%). In both protocols, the haemodynamic tolerance was good. The mean maintenance dose of Hemangiol was 2.7 ± 0.8 mg/kg/day, with a median treatment duration of 7 [1.5; 19] months. Adverse events (AEs) have been found in 25 (26,6%) patients, including 8 (8.5%) patients with serious AEs (uncontrolled bronchial hyperreactivity, n = 5; serious hypoglycaemia, n = 3). Some patients had one or more AEs, a total of 24 nonserious AEs was reported in 19 patients (sleep disturbances, n = 9; respiratory disorders, n = 5; digestive disorders, n = 6). No cardiac adverse event was reported. CONCLUSION: This post-marketing surveillance drug study supports the good tolerance of Hemangiol in children with IH. A rapid initiation protocol is of interest when treatment is urgent. The pretherapeutic paediatric cardiology consultation should not be systematic but only indicated for specific patients. CLINICALTRIALS.GOV: NCT04105517.

7.
Blood Adv ; 4(19): 4632-4639, 2020 Oct 13.
Artigo em Inglês | MEDLINE | ID: mdl-33002131

RESUMO

Anticoagulant treatment of pediatric central venous catheter-related venous thromboembolism (CVC-VTE) has not been specifically evaluated. In EINSTEIN-Jr, 500 children with any VTE received rivaroxaban or standard anticoagulants. A predefined analysis of the CVC-VTE cohort was performed. Children with CVC-VTE (age, birth to 17 years) were administered rivaroxaban or standard anticoagulants during the 1-month (children <2 years) or 3-month (all other children) study period. Predefined outcomes were recurrent VTE, change in thrombotic burden on repeat imaging, and bleeding. Predictors for continuation of anticoagulant therapy beyond the study period were evaluated. One hundred twenty-six children with symptomatic (n = 76, 60%) or asymptomatic (n = 50, 40%) CVC-VTE received either rivaroxaban (n = 90) or standard anticoagulants (n = 36). There was no recurrent VTE (0%; 95% confidence interval [CI], 0.0%-2.8%). Three children had the principal safety outcome: none had major bleeding and 3 children had clinically relevant nonmajor bleeding (2.4%; 95% CI, 0.7%-6.5%), all in the rivaroxaban arm. Complete or partial vein recanalization occurred in 57 (55%) and 38 (37%) of 103 evaluable children, respectively. Results were similar for symptomatic and asymptomatic CVC-VTE. Continuation of anticoagulant therapy beyond the study period occurred in 61 (48%) of children and was associated with residual VTE but only in children <2 years (odds ratio [OR], 20.9; P = .003) and continued CVC use (OR, 6.7; P = .002). Anticoagulant therapy appeared safe and efficacious and was associated with reduced clot burden in most children with symptomatic or asymptomatic CVC-VTE. Residual VTE and continued CVC use were associated with extended anticoagulation. This trial was registered at www.clinicaltrials.gov as #NCT02234843.

8.
Arch Cardiovasc Dis ; 2020 Oct 14.
Artigo em Inglês | MEDLINE | ID: mdl-33069639

RESUMO

BACKGROUND: Managing oral anticoagulant therapy with vitamin K antagonists remains challenging in paediatric medicine. AIMS: This study aimed to assess the correlation between time in therapeutic range and quality of life in children participating in a non-selective International Normalised Ratio self-monitoring and vitamin K antagonist education programme. METHODS: Children aged from 2 to 18 years and receiving vitamin K antagonist therapy were eligible for this prospective multicentre study. Clinical and demographic data were collected. Health-related quality of life was assessed using the PedsQL™ 4.0 questionnaire. Correlations between quality of life scores and time in therapeutic range were measured. RESULTS: A total of 121 children were included in the study (mean age 9.6±4.9 years). Cardiac conditions were the predominant indication for vitamin K antagonists. The mean time in therapeutic range was 0.78±0.15 overall, and 0.76±0.24 over the 3-month period before quality of life assessment. The mean total quality of life score was 76.2±18 in self reports, 71.4±22 in mother reports and 73.5±19 in father reports. The time in therapeutic range correlated with the total quality of life scores in self reports (r=0.22; P=0.04), mother reports (r=0.23; P=0.02) and father reports (r=0.28; P=0.02). The time in therapeutic range predominantly correlated with school functioning in self reports (r=0.38; P=0.002) and mother reports (r=0.40; P<0.001), and with physical functioning in father reports (r=0.28; P=0.03). CONCLUSIONS: Time in therapeutic range correlated with quality of life in children participating in a non-selective International Normalised Ratio self-monitoring and vitamin K antagonist education programme. Regular assessment of quality of life in patient education programmes contributes towards understanding the concerns and needs of patients.

9.
J Appl Physiol (1985) ; 129(5): 1102-1110, 2020 Nov 01.
Artigo em Inglês | MEDLINE | ID: mdl-32909919

RESUMO

Cardiopulmonary exercise test (CPET) is becoming a key examination to assess physical capacity and disease severity in pediatric cardiology. The V̇e/V̇co2 slope has been increasingly used as a surrogate marker for morbidity and mortality in adult heart failure, pulmonary arterial hypertension, and for adult patients with congenital heart disease (CHD). Nevertheless, the use of the V̇e/V̇co2 slope in children remains limited in the absence of reference values and clearly identified clinical determinants. This study aimed to compare the V̇e/V̇co2 slope in a pediatric cohort with CHD to that of age- and gender-adjusted healthy controls. We also intended to identify the clinical and CPET variables associated with V̇e/V̇co2 slope in this population. This cross-sectional study was carried out between November 2010 and September 2015 in two tertiary care pediatric cardiology reference centers. A total of 700 children were enrolled (399 CHD and 301 healthy controls). The mean V̇e/V̇co2 slope was significantly higher in the CHD subjects than in healthy subjects (31.6 ± 4.8 vs. 29.3 ± 4.8; P < 0.001). The V̇e/V̇co2 slope was higher in children with significant pulmonary regurgitation, tricuspid regurgitation, right ventricular hypertension, and right ventricle outflow tract (RVOT) obstacle. In the CHD group, V̇e/V̇co2 slope increase was associated with body mass index, the presence of a RVOT obstacle, the number of cardiac catheter procedures, as well as low age, forced vital capacity, tidal volume, and [Formula: see text]. Increased V̇e/V̇co2 slope was predominantly in children with single ventricle and/or residual right heart abnormalities, suggesting that maldistribution of pulmonary blood flow during exercise is an important CHD-unique determinant of V̇e/V̇co2 slope.NEW & NOTEWORTHY Using V̇e/V̇co2 slope is useful for children with congenital heart disease. V̇e/V̇co2 slope is sensitive to pulmonary blood flow maldistribution during exercise, this concerns congenital heart disease with pulmonary regurgitation, tricuspid regurgitation, right ventricular hypertension, and right ventricle outflow tract obstacle. V̇e/V̇co2 slope is a good parameter to follow single ventricles and right heart residual lesions (tetralogy of Fallot; pulmonary atresia; truncus arteriosus…).

10.
Arch Dis Child ; 105(12): 1167-1174, 2020 12.
Artigo em Inglês | MEDLINE | ID: mdl-32732318

RESUMO

OBJECTIVES: Cardiopulmonary exercise test (CPET) provides accurate evaluation of physical capacity and disease severity in children with congenital heart disease (CHD). However, full participation to obtain optimal measure of VO2max may be difficult. As an alternative, the oxygen uptake efficiency slope (OUES) is a reproducible and reliable parameter measured during CPET, which does not require a maximal exercise to be interpretable. This study aimed to evaluate the OUES of a large cohort of children with CHD, in comparison with healthy controls. We also intended to identify, in this specific population, the clinical and CPET variables associated with the OUES. METHODS: This cross-sectional study was carried out between November 2010 and September 2015 in two tertiary care paediatric and congenital cardiology centres. RESULTS: 709 children were included (407 CHD and 302 healthy controls). The association of clinical characteristics with weight-normalised OUES (OUESkg) was studied using a multivariable analysis. The mean OUESkg was significantly lower in CHD than in healthy controls (38.6±8.5 and 43.9±8.5; p<0.001, respectively), especially in the most severe CHD. The OUESkg correlated with VO2max (r=0.85, p<0.001), with cut-off values for normal exercise capacity of 38.4 in boys and 31.0 in girls. The decrease of OUESkg was associated with increased age, increased Body Mass Index, number of cardiac catheter or surgical procedures, female gender and decreased forced vital capacity (Z-score). CONCLUSION: The OUES is significantly impaired in children with CHD and strongly correlates with VO2max. The OUES has the same clinical determinants as VO2max and therefore may be of interest in submaximal exercise. TRIAL REGISTRATION NUMBER: NCT01202916.

11.
Theranostics ; 10(18): 8130-8142, 2020.
Artigo em Inglês | MEDLINE | ID: mdl-32724462

RESUMO

Anthracyclines are key chemotherapeutic agents used in various adult and pediatric cancers, however, their clinical use is limited due to possible congestive heart failure (HF) caused by acute and irreversible cardiotoxicity. Currently, there is no method to predict the future development of the HF in these patients. In order to identify early biomarkers to predict anthracycline cardiotoxicity in long-term survivors of childhood cancer, this longitudinal study aimed to analyze early and late in-vivo regional myocardial anthracycline-induced cardiotoxicity, related to in-vitro cardiac myocytes dysfunction, in a juvenile rat model. Methods: Young male Wistar rats (4 weeks-old) were treated with different cumulative doses of doxorubicin (7.5, 10 or 12.5 mg/kg) or NaCl (0.9%) once a week for 6 weeks by intravenous injection. Cardiac function was evaluated in-vivo by conventional (left ventricular ejection fraction, LVEF) and regional two-dimensional (2D) speckle tracking echocardiography over the 4 months after the last injection. The animals were assigned to preserved (pEF) or reduced EF (rEF) groups at the end of the protocol and were compared to controls. Results: We observed a preferential contractile dysfunction of the base of the heart, further altered in the posterior segment, even in pEF group. The first regional alterations appeared 1 month after chemotherapy. Functional investigation of cardiomyocytes isolated from the LV base 1 month after doxorubicin treatment showed that early in-vivo contractile alterations were associated with both decreased myofilament Ca2+ sensitivity and length-dependent activation. Changes in post-translational modifications (phosphorylation; S-glutathionylation) and protein degradation of the cardiac myosin binding protein-C may contribute to these alterations. Conclusion: Our data suggest that screening of the contractile defaults of the base of the heart by regional 2D strain echocardiography is useful to detect subclinical myocardial dysfunction prior to the development of delayed anthracycline-induced cardiomyopathy in pediatric onco-cardiology.

12.
Arch Cardiovasc Dis ; 113(8-9): 492-502, 2020.
Artigo em Inglês | MEDLINE | ID: mdl-32461091

RESUMO

The population of patients with congenital heart disease (CHD) is continuously increasing, and a significant proportion of these patients will experience arrhythmias because of the underlying congenital heart defect itself or as a consequence of interventional or surgical treatment. Arrhythmias are a leading cause of mortality, morbidity and impaired quality of life in adults with CHD. Arrhythmias may also occur in children with or without CHD. In light of the unique issues, challenges and considerations involved in managing arrhythmias in this growing, ageing and heterogeneous patient population and in children, it appears both timely and essential to critically appraise and synthesize optimal treatment strategies. The introduction of catheter ablation techniques has greatly improved the treatment of cardiac arrhythmias. However, catheter ablation in adults or children with CHD and in children without CHD is more technically demanding, potentially causing various complications, and thus requires a high level of expertise to maximize success rates and minimize complication rates. As French recommendations regarding required technical competence and equipment are lacking in this situation, the Working Group of Pacing and Electrophysiology of the French Society of Cardiology and the Affiliate Group of Paediatric and Adult Congenital Cardiology have decided to produce a common position paper compiled from expert opinions from cardiac electrophysiology and paediatric cardiology. The paper details the features of an interventional cardiac electrophysiology centre that are required for ablation procedures in adults with CHD and in children, the importance of being able to diagnose, monitor and manage complications associated with ablations in these patients and the supplemental hospital-based resources required, such as anaesthesia, surgical back-up, intensive care, haemodynamic assistance and imaging. Lastly, the need for quality evaluations and French registries of ablations in these populations is discussed. The purpose of this consensus statement is therefore to define optimal conditions for the delivery of invasive care regarding ablation of arrhythmias in adults with CHD and in children, and to provide expert and - when possible - evidence-based recommendations on best practice for catheter-based ablation procedures in these specific populations.


Assuntos
Arritmias Cardíacas/cirurgia , Procedimentos Cirúrgicos Cardíacos , Cardiologistas/normas , Serviço Hospitalar de Cardiologia/normas , Ablação por Cateter/normas , Competência Clínica/normas , Criocirurgia/normas , Cardiopatias Congênitas/cirurgia , Adolescente , Adulto , Fatores Etários , Arritmias Cardíacas/diagnóstico , Arritmias Cardíacas/mortalidade , Arritmias Cardíacas/fisiopatologia , Procedimentos Cirúrgicos Cardíacos/efeitos adversos , Procedimentos Cirúrgicos Cardíacos/mortalidade , Ablação por Cateter/efeitos adversos , Ablação por Cateter/mortalidade , Criança , Pré-Escolar , Consenso , Criocirurgia/efeitos adversos , Criocirurgia/mortalidade , Técnicas Eletrofisiológicas Cardíacas/normas , Cardiopatias Congênitas/diagnóstico por imagem , Cardiopatias Congênitas/mortalidade , Cardiopatias Congênitas/fisiopatologia , Frequência Cardíaca , Humanos , Lactente , Recém-Nascido , Fatores de Risco , Sobreviventes , Resultado do Tratamento , Adulto Jovem
13.
J Thromb Haemost ; 18(7): 1672-1685, 2020 07.
Artigo em Inglês | MEDLINE | ID: mdl-32246743

RESUMO

BACKGROUND: Recently, the randomized EINSTEIN-Jr study showed similar efficacy and safety for rivaroxaban and standard anticoagulation for treatment of pediatric venous thromboembolism (VTE). The rivaroxaban dosing strategy was established based on phase 1 and 2 data in children and through pharmacokinetic (PK) modeling. METHODS: Rivaroxaban treatment with tablets or the newly developed granules-for-oral suspension formulation was bodyweight-adjusted and administered once-daily, twice-daily, or thrice-daily for children with bodyweights of ≥30, ≥12 to <30, and <12 kg, respectively. Previously, these regimens were confirmed for children weighing ≥20 kg but only predicted in those <20 kg. Based on sparse blood sampling, the daily area under the plasma concentration-time curve [AUC(0-24)ss ] and trough [Ctrough,ss ] and maximum [Cmax,ss ] steady-state plasma concentrations were derived using population PK modeling. Exposure-response graphs were generated to evaluate the potential relationship of individual PK parameters with recurrent VTE, repeat imaging outcomes, and bleeding or adverse events. A taste-and-texture questionnaire was collected for suspension-recipients. RESULTS: Of the 335 children (aged 0-17 years) allocated to rivaroxaban, 316 (94.3%) were evaluable for PK analyses. Rivaroxaban exposures were within the adult exposure range. No clustering was observed for any of the PK parameters with efficacy, bleeding, or adverse event outcomes. Results were similar for the tablet and suspension formulation. Acceptability and palatability of the suspension were favorable. DISCUSSION: Based on this analysis and the recently documented similar efficacy and safety of rivaroxaban compared with standard anticoagulation, we conclude that bodyweight-adjusted pediatric rivaroxaban regimens with either tablets or suspension are validated and provide for appropriate treatment of children with VTE.

14.
J Cardiovasc Pharmacol ; 76(1): 23-31, 2020 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-32168152

RESUMO

BACKGROUND: Add-on therapy with prostacyclin in pediatric refractory pulmonary hypertension (PH) poses a challenge, especially when considering continuous intravenous administration in younger children. A search for alternate routes of drug delivery has led to the clinical investigation of stable and long-acting prostacyclin analogues, such as subcutaneous treprostinil. We reported 2 pediatric cases of PH treated with subcutaneous treprostinil and reviewed the literature on treprostinil use in children. METHOD: The literature review used 3 electronic databases and a combination of terms (treprostinil, pediatric, PH, prostanoid, etc). We also searched for pediatric clinical trials on treprostinil registered on international clinical trial registries. RESULTS: The reported cases highlighted the multifactorial nature of PH in pediatrics: a female child with a giant omphalocele, and intracardiac and extracardiac shunts; and a male premature child with a congenital diaphragmatic hernia and long-term PH. The literature review identified 19 studies reporting treprostinil use in 421 children with various types of PH (groups 1 and 3). Subcutaneous treprostinil was the most administered formulation, at a mean dose of 40 ng/kg/min. Overall, 12 clinical trials on treprostinil for children with PH were registered on the clinical trial registries. Most authors concluded that subcutaneous treprostinil was effective, well tolerated, and represented an alternative to intravenous epoprostenol. CONCLUSIONS: Subcutaneous treprostinil may be a useful adjunct in the therapeutic algorithm for children with severe PH, refractory to oral drugs, and after a complete check-up for all PH etiologies.

15.
ESC Heart Fail ; 7(2): 747-756, 2020 04.
Artigo em Inglês | MEDLINE | ID: mdl-32147955

RESUMO

AIMS: In univentricular hearts, selective lung vasodilators such as phosphodiesterase type 5 (PDE5) inhibitors would decrease pulmonary resistance and improve exercise tolerance. However, the level of evidence for the use of PDE5 inhibitors in patients with a single ventricle (SV) remains limited. We present the SV-INHIBITION study rationale, design, and methods. METHODS AND RESULTS: The SV-INHIBITION trial is a nationwide multicentre, randomized, double blind, placebo-controlled, Phase III study, aiming to evaluate the efficacy of sildenafil on the ventilatory efficiency during exercise, in teenagers and adult patients (>15 years old) with an SV. Patients with a mean pulmonary arterial pressure >15 mmHg and a trans-pulmonary gradient >5 mmHg, measured by cardiac catheterization, will be eligible. The primary outcome is the variation of the VE/VCO2 slope, measured by a cardiopulmonary exercise test, between baseline and 6 months of treatment. A total of 50 patients are required to observe a decrease of 5 ± 5 points in the VE/VCO2 slope, with a power of 90% and an alpha risk of 5%. The secondary outcomes are clinical outcomes, oxygen saturation, 6 min walk test, SV function, NT-proBNP, peak VO2 , stroke volume, mean pulmonary arterial pressure, trans-pulmonary gradient, SF36 quality of life score, safety, and acceptability. CONCLUSIONS: The SV-INHIBITION study aims to answer the question whether PDE5 inhibitors should be prescribed in patients with an SV. This trial has been built focusing on the three levels of research defined by the World Health Organization: disability (exercise tolerance), deficit (SV function), and handicap (quality of life).

16.
Int J Cardiol Heart Vasc ; 27: 100489, 2020 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-32154361

RESUMO

Background: Recent advances in the field of congenital heart disease (CHD) have significantly improved the overall prognosis. Now more attention is being given to health-related quality of life (HRQoL) and promotion of physical activity. Non-invasive relaxation therapy may be effective in cardiac patients concerned with exercise-induced dyspnoea. The SOPHROCARE randomised trial aims to assess the impact of Caycedian Sophrology on cardiopulmonary fitness in adolescents and young adults with CHD. Methods: The SOPHROCARE trial is a nationwide, multicentre, randomised, controlled study in CHD patients aged from 13 to 25 years old. Patients will be randomised into 2 groups (8 Sophrology group sessions vs. no intervention). The primary outcome is the change in percent predicted maximum oxygen uptake (VO2max) between baseline and 12-month follow-up. A total of 94 patients in each group is required to observe a significant increase of 10% in VO2max with a power of 80% and an alpha risk of 5%. The secondary outcomes are: clinical outcomes, cardiopulmonary exercise test parameters (VE/VCO2 slope, ventilatory anaerobic threshold, oxygen pulse, respiratory response to hypercapnia), health-related quality of life score (PedsQL), physical and psychological status. Conclusion: After focusing on the survival in CHD, current research is opening on secondary prevention and patient-related outcomes. We sought to assess in the SOPHROCARE trial, if a Sophrology program, could improve exercise capacity and quality of life in youth with CHD. Trial registration: Clinicaltrials.gov (NCT03999320).

17.
Cardiol Young ; 30(3): 427-430, 2020 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-32036804

RESUMO

The incidence of paediatric venous thromboembolism has steadily increased in the past decade, by nearly 10% per year. Deep venous thrombosis may remain completely asymptomatic during the acute phase and symptoms may occur later, due to complications. We related the case of a 9-month-old child with increasing cyanosis. A computed tomography (CT) angiography showed a thrombosis of the superior vena cava (SVC) with the development of collateral flow from the systemic to the pulmonary veins. Transcatheter shunt occlusion after SVC recanalization was successfully performed. We discussed the characteristics of these cases and the consequence on our practice in term of treatment (anticoagulation, transcatheter, intervention) and screening.

18.
J Infect Public Health ; 13(2): 167-172, 2020 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-31378693

RESUMO

INTRODUCTION: Acute bronchiolitis epidemics are known to destabilize healthcare structures and stand as a major public health issue. Our tertiary care regional university hospital designed an organizational infrastructure paediatric plan (OIPP) to adapt to bronchiolitis epidemics. This study aimed to assess the impact of the OIPP on the length of stay and quality of care of children hospitalized for bronchiolitis. METHODS: This epidemiological study analyzed data from 2 epidemic seasons before and after the OIPP implementation. The OIPP used a standardized algorithm of patient orientation and a 4-level stratification of care. RESULTS: A total of 1636 children were included in the study, with 718 children before and 918 children after the OIPP implementation. The length of stay significantly decreased after the OIPP implementation, from 5.1±6.8 days to 3.9±3 days (P<0.001). The 30-day readmission for bronchiolitis remained stable (4.6% vs. 3.4%, P=0.2). The net annual patient service revenue increased from 1,292,532 € to 1,545,720 €. CONCLUSION: The implementation of the OIPP resulted in a significant decrease in the average hospital length of stay. This plan improved patients' quality of care while maintained a balanced budget. TRIAL REGISTRATION NUMBER: ClinicalTrials.gov NCT03663660.


Assuntos
Bronquiolite/epidemiologia , Epidemias , Tempo de Internação , Qualidade da Assistência à Saúde , Feminino , França/epidemiologia , Hospitalização , Humanos , Lactente , Recém-Nascido , Masculino , Infecções por Vírus Respiratório Sincicial/epidemiologia , Vírus Sinciciais Respiratórios , Estudos Retrospectivos , Estações do Ano
19.
Lancet Haematol ; 7(1): e18-e27, 2020 Jan.
Artigo em Inglês | MEDLINE | ID: mdl-31699660

RESUMO

BACKGROUND: Treatment of venous thromboembolism in children is based on data obtained in adults with little direct documentation of its efficacy and safety in children. The aim of our study was to compare the efficacy and safety of rivaroxaban versus standard anticoagulants in children with venous thromboembolism. METHODS: In a multicentre, parallel-group, open-label, randomised study, children (aged 0-17 years) attending 107 paediatric hospitals in 28 countries with documented acute venous thromboembolism who had started heparinisation were assigned (2:1) to bodyweight-adjusted rivaroxaban (tablets or suspension) in a 20-mg equivalent dose or standard anticoagulants (heparin or switched to vitamin K antagonist). Randomisation was stratified by age and venous thromboembolism site. The main treatment period was 3 months (1 month in children <2 years of age with catheter-related venous thromboembolism). The primary efficacy outcome, symptomatic recurrent venous thromboembolism (assessed by intention-to-treat), and the principal safety outcome, major or clinically relevant non-major bleeding (assessed in participants who received ≥1 dose), were centrally assessed by investigators who were unaware of treatment assignment. Repeat imaging was obtained at the end of the main treatment period and compared with baseline imaging tests. This trial is registered with ClinicalTrials.gov, number NCT02234843 and has been completed. FINDINGS: From Nov 14, 2014, to Sept 28, 2018, 500 (96%) of the 520 children screened for eligibility were enrolled. After a median follow-up of 91 days (IQR 87-95) in children who had a study treatment period of 3 months (n=463) and 31 days (IQR 29-35) in children who had a study treatment period of 1 month (n=37), symptomatic recurrent venous thromboembolism occurred in four (1%) of 335 children receiving rivaroxaban and five (3%) of 165 receiving standard anticoagulants (hazard ratio [HR] 0·40, 95% CI 0·11-1·41). Repeat imaging showed an improved effect of rivaroxaban on thrombotic burden as compared with standard anticoagulants (p=0·012). Major or clinically relevant non-major bleeding in participants who received ≥1 dose occurred in ten (3%) of 329 children (all non-major) receiving rivaroxaban and in three (2%) of 162 children (two major and one non-major) receiving standard anticoagulants (HR 1·58, 95% CI 0·51-6·27). Absolute and relative efficacy and safety estimates of rivaroxaban versus standard anticoagulation estimates were similar to those in rivaroxaban studies in adults. There were no treatment-related deaths. INTERPRETATION: In children with acute venous thromboembolism, treatment with rivaroxaban resulted in a similarly low recurrence risk and reduced thrombotic burden without increased bleeding, as compared with standard anticoagulants. FUNDING: Bayer AG and Janssen Research & Development.


Assuntos
Anticoagulantes/uso terapêutico , Rivaroxabana/uso terapêutico , Tromboembolia Venosa/tratamento farmacológico , Adolescente , Criança , Pré-Escolar , Feminino , Humanos , Lactente , Masculino , Fatores de Risco
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