Epidemiology of congenital heart defects in France from 2013 to 2022 using the PMSI-MCO (French Medical Information System Program in Medicine, Surgery, and Obstetrics) database.

BACKGROUND: Congenital heart defects are common and occur in approximately 0.9% of births. In France, the registries cover approximately 20% of the population but not the entirety of France; therefore, we aimed to update the incidence data for congenital heart defects in France from 2013 to 2022 using the medico-administrative database PMSI-MCO (French Medical Information System Program in Medicine, Surgery, and Obstetrics). We aimed to compare the frequency of risk factors in a population with congenital heart defects and a reference population. METHODS: From 2013 to 2022, we included children aged < 3 years diagnosed with congenital heart defects according to the International Classification of Diseases, 10th Revision, in the PMSI-MCO database. We compared them with a population without congenital defects on several medical data items (e.g., parity, gemellarity, and mortality rate). Bivariate and multivariate analyses compared children with congenital heart defects and children without congenital malformation. RESULTS: We identified 83,879 children with congenital heart defects in France from 2013 to 2022 in the PMSI-MCO database and 7,739,840 children without such defects, including 7,218,952 without any congenital defects. We observed more deaths (7.49% vs. 0.68%, d = 0.59) and more twinning (8.67% vs. 1.23%, d = 0.35) among children with congenital heart defects. Multivariate analysis revealed an increased risk of congenital heart defects in male individuals (OR [odds ratio] 1.056, 95% CI [confidence interval] [1.039-1.076]) and cases of medically assisted reproduction (OR 1.115, 95% CI [1.045-1.189]) and a reduced risk in the case of multiparity (OR 0.921, 95% CI [0.905-0.938]). CONCLUSIONS: According to the PMSI-MCO database, the incidence of congenital heart defects in France from 2013 to 2022 is 1% of births. Congenital heart defects are more frequent in cases of prematurity, twinning, primiparity, male sex, and maternal age > 40 years.

The RISE Study: Retrospective Registry for the International Safety and Efficacy Results of Patent Foramen Ovale Closure with Figulla Flex Il PFO and UNI Occluders.

Background: Transcatheter closure of a patent foramen ovale (PFO) is performed in cryptogenic stroke and other conditions. Information is lacking for some devices. Methods: We aimed to evaluate the Figulla Flex II PFO Occluder (FFP) and Figulla Flex UNI Occluder (FFU) through a retrospective multi-center registry. Results: 527 patients were included. Mean age was 48.9 (±13.8) years. The procedure was under transthoracic, transesophageal or intracardiac echocardiography in 185 (35.1%), 193 (36.6%) and 149 (28.3%) cases, respectively, and under general anesthesia in 191 patients (36.2%). The FFP and FFU were used in 408 (77.4%) and 119 (22.6%) cases, respectively. The success rate was 99.1%. Median follow-up was 1.1 (0.5-2.5) years. A new atrial fibrillation/flutter within six months occurred in 14 (2.7%) cases, with no difference between devices. One device embolization in the pulmonary artery was identified two years post-procedure. Residual shunts occurred in 18 (6.9%) cases at 1 year, with TIA in three (16.6%) patients. Out of 437 patients with stroke/TIA, 260 (59%) were followed more than one year after closure. Median follow-up was 2.1 (1.17-3.1) years, with four recurrent strokes/TIA. Conclusions: The FFP and FFU devices are safe and effective for PFO closure, with very few atrial fibrillation/flutter and neurologic events, except in cases with a residual shunt.

NT-proBNP course during MIS-C post-COVID-19: an observational study.

Multisystem inflammatory syndrome in children (MIS-C or PIMS-TS) is a severe disease. N-terminal pro-B-type natriuretic peptide (NT-proBNP) is used for positive and differential diagnosis, diagnosis of complications and severity, and cardiogenic shock prediction. However, contrasting cut-offs have been suggested. The aims of the present study were to compare NT-proBNP values depending on the time of measurement and to describe the NT-proBNP course during the MIS-C episode. The data from a single-centre cohort observational study on the impact of time to diagnosis, defined as the time from first symptom to diagnosis of MIS-C, were used for the purpose of this study, with an extended period of inclusion from May 2020 to April 2023. The timing and level of all NT-proBNP samples available for each patient were retrospectively collected. Thirty-seven children (18 (49%) females, median age 8.8 years, 14 (38%) with shock) were included. Until diagnosis, NT-proBNP increased with time and was significantly higher at 6 days from first symptoms than at 3 days (median (interquartile range) 32,933 (7773-61,592) versus 1994 (1291-4190) pg/mL, respectively, p = 0.031). From diagnosis, NT-proBNP decreased by at least 50% after 3.0 (2.1-5.3) days (n = 12) when NT-proBNP at diagnosis was low ≤ 11,000 pg/mL versus 1.8 (0.7-3.4) days (n = 16) when NT-proBNP at diagnosis was high (p = 0.040), and after 3.6 (2.4-5.9) days (n = 7) when fever persisted after 48 h versus 1.8 (0.8-3.0) days (n = 21) when fever resolved before 48 h (p = 0.004).  Conclusions: During the MIS-C episode, NT-proBNP increased over time until diagnosis and treatment. It dropped faster thereafter in children with high NT-proBNP at diagnosis > 11,000 pg/mL and slower in case of persistent fever. What is Known: • NT-proBNP is useful in MIS-C for positive and differential diagnosis, diagnosis of complications and severity, and cardiogenic shock prediction. • Contrasting cut-offs for differential diagnosis and severity assessment have been suggested. What is New: • Before diagnosis, NT-proBNP increases with time and is significantly higher at 6 days from first symptoms than at 3 days suggesting different cut-offs depending on the timing of measurement. • From diagnosis and treatment initiation, the 50% NT-proBNP drop occurs earlier in children with high NT-proBNP at diagnosis > 11,000 pg/mL and later in children with persistent fever.

Safety and efficacy of the Amplatzer™ Trevisio™ intravascular delivery system: Post-approval study results.

BACKGROUND: The Amplatzer™ Trevisio™ Intravascular Delivery System (Trevisio DS; Abbott Laboratories, Chicago, IL, USA) facilitates the delivery of Amplatzer™ Occluders and features an ultraflexible tip, which improves assessment of occluder position before release. AIMS: To assess the safety and efficacy of the Trevisio DS for transcatheter closure of patent foramen ovale and atrial septal defect. METHODS: The Amplatzer™ Trevisio™ Intravascular Delivery System Post-Approval Study was a prospective, postmarket, single-arm, multicentre, observational study of the Trevisio DS. Enrolled patients were indicated for transcatheter closure of patent foramen ovale or atrial septal defect. In all procedures, the Trevisio DS was used to deliver Amplatzer™ Occluders. Technical success was defined as successful deployment and release of at least one occluder. Device- or procedure-related serious adverse events were tracked until discharge or day 7, whichever occurred earlier. RESULTS: The study enrolled 144 patients with patent foramen ovale and 107 patients with atrial septal defect at 22 European sites; 53 patients with atrial septal defect (49.6%) were aged<18years. The rate of technical success was 98.4% (97.2% for atrial septal defect, 99.3% for patent foramen ovale). There was one serious adverse event (0.4%), an acute periprocedural device embolization that occurred after occluder release in a patient with atrial septal defect; the device was retrieved percutaneously. This was determined by the implanter to be unrelated to the performance of the Trevisio DS. CONCLUSIONS: The Trevisio DS exhibited a high rate of technical success and an excellent safety profile during transcatheter closure of patent foramen ovale and atrial septal defect.

Anomalous hepatic drainage and platypnea-orthodeoxia in cyanotic children.

The aetiology of cyanosis could be unclear in children, even for specialised paediatricians. Two cases were reported: first, a 6-year-old child with features of left isomerism and Fallot was fortuitously diagnosed with anomalous hepatic venous drainage before complete repair. Second, a newborn with an antenatal diagnosis of ductus venosus agenesis had an isolated intermittent right-to-left atrial shunt when upright, with favourable outcome, in contrast to the association with significant heart malformations including inferior caval vein interruption. Multimodality imaging and 3D printing helped to rule out extracardiac causes of persistent cyanosis and plan surgical repair.

Transcatheter Closure of Perimembranous Ventricular Septal Defects Including Multifenestrated and Gerbode-Type Defects Using the Lifetech Konar Device.

(1) Transcatheter closure of perimembranous ventricular septal defects (PmVSD) is becoming more attractive and effective with the development of new occluders. The aim of this study was to report a single-center experience in PmVSD closure using the Lifetech Konar-multifunctional occluder (MFO). (2) From March 2019 to October 2022, 43 consecutive patients were enrolled in the study. Among them, 13 had multifenestrated PmVSD including 5 Gerbode-type defects. (3) There were 23 males/20 females, and the median age was 17 years (range 2-68 years). Trivial aortic regurgitation was noticed in 19 patients. Implantation was successful in all patients under general anesthesia. A retrograde approach was used in 35 patients (81%). The retrograde approach was associated with a lower radiation dose (p = 0.042) and shorter fluoroscopy time (p = 0.002) compared to the antegrade approach. Full occlusion was observed immediately in 12 patients (28%) and in 33 patients (77%) at a median follow-up of 11 months. There were no complications such as embolization, complete atrioventricular block, device dislocation, new onset above grade I, or progression of tricuspid or aortic valve regurgitation. Seven of the thirteen patients with a multifenestrated defect had no residual shunt. The persistent shunts were all trivial intra-prosthetic leaks. (4) MFO is effective and safe for PmVSD closure including multifenestrated/Gerbode-type defects with no complication. However, a longer follow-up remains warranted to establish the safety of this technique.

Efficiency and Impact of Hypnoanalgesia for Cardiac Catheterisation in Paediatric Population.

Hypnoanalgesia is a promising non-pharmacologic adjunct technique in paediatric interventions. Its safety, efficiency, and impacts on paediatric cardiac catheterisation (CC) are unknown. METHODS: In a prospective study, patients aged <16 years who underwent CC under hypnoanalgesia from January to December 2021 were included. Pain and anxiety were assessed using the analgesia nociception index (ANI) and the visual analogue scale (VAS). RESULTS: Sixteen patients were included; the mean age was 10.5 years, and the mean weight was 37 kg. Catheterisations were interventional in 10 patients (62.5%). Hypnoanalgesia indications were general anaesthesia (GA) contraindication in four patients (25.0%), the need for accurate pressure measurements in three patients (18.7%), and interventionist/patient preferences in nine (56.3%). CC was accomplished in 15 patients (93.7%), even in complicated cases. In one case, pulmonary artery pressures were normalised compared to previous catheterisation under local anaesthesia alone. The VAS score was under 5/10 for all patients. The ANI remained above 50 (no painful zone) for all but one patient. There was no significant decrease in the ANI during the intervention compared to the baseline (p = 0.62). No complications were reported. CONCLUSION: Paediatric CC is feasible and safe under hypnoanalgesia, even in complicated cases. Hypnoanalgesia was efficient in managing pain and stress, and it ensures more reliable pressure measurements.

Pulmonary valve replacement in a large and tortuous right ventricle outflow tract with a 32†mm Myval valve under local anaesthesia: challenges and technical considerations: a case report.

Background: Pulmonary valve replacement in patients with congenital heart diseases and heart failure is challenging. Case summary: Here, we describe a case of a patient who had surgical fallot repair with chronic heart failure. Investigations found severe biventricular dysfunction and enlargement due to chronic pulmonary regurgitation. The right ventricle outflow tract was tortuous and large with a diameter of 35 mm. Percutaneous pulmonary valve implantation (PPVI) was done after a challenging pre-stenting. A 32 mm Myval valve over-sized to 35 mm was used for PPVI, which yielded a good result. Discussion: A 32 mm Myval valve is effective at extending the possibilities of PPVI in a large and tortuous right ventricle outflow tract not accessible for the other valves.

Lympho-hematopoietic malignancies risk after exposure to low dose ionizing radiation during cardiac catheterization in childhood.

Pediatric patients with congenital heart disease (CHD) often undergo low dose ionizing radiation (LDIR) from cardiac catheterization (CC) for the diagnosis and/or treatment of their disease. Although radiation doses from a single CC are usually low, less is known about the long-term radiation associated cancer risks. We aimed to assess the risk of lympho-hematopoietic malignancies in pediatric CHD patients diagnosed or treated with CC. A French cohort of 17,104 children free of cancer who had undergone a first CC from 01/01/2000 to 31/12/2013, before the age of 16 was set up. The follow-up started at the date of the first recorded CC until the exit date, i.e., the date of death, the date of first cancer diagnosis, the date of the 18th birthday, or the 31/12/2015, whichever occurred first. Poisson regression was used to estimate the LDIR associated cancer risk. The median follow-up was 5.9 years, with 110,335 person-years. There were 22,227 CC procedures, yielding an individual active bone marrow (ABM) mean cumulative dose of 3.0 milligray (mGy). Thirty-eight incident lympho-hematopoietic malignancies were observed. When adjusting for attained age, gender and predisposing factors to cancer status, no increased risk was observed for lympho-hematopoietic malignancies RR/mGy = 1.00 (95% CI: 0.88; 1.10). In summary, the risk of lympho-hematopoietic malignancies and lymphoma was not associated to LDIR in pediatric patients with CHD who undergo CC. Further epidemiological studies with greater statistical power are needed to improve the assessment of the dose-risk relationship.

Health-Related Quality of Life and Its Association With Outcomes in Adults With Congenital Heart Disease and Heart Failure: Insight From FRESH-ACHD Registry.

Background Quality of Life (QoL) is a prognostic factor in heart failure (HF) of patients with acquired cardiac disease. The aim of this study was to determine the predictive value of QoL on outcomes in adults with congenital heart disease (ACHD) and HF. Methods and Results Quality of life of 196 adults with congenital heart disease with clinical heart failure (HF) (mean age: 44.3±13.8 years; 51% male; 56% with complex congenital heart disease; 47% New York Heart Association class III/IV) included in the prospective multicentric registry FRESH-ACHD (French Survey on Heart Failure-Adult with Congenital Heart Disease) was assessed using the 36-Item Short Form Survey (SF-36), a patient-reported survey. The primary end point was defined by all-cause death, HF-related hospitalization, heart transplantation, and mechanical circulatory support. At 12 months, 28 (14%) patients reached the combined end point. Patients with low quality of life experienced major adverse events more frequently (logrank P=0.013). On univariate analysis, lower score at physical functioning (hazard ratio [HR], 0.98 [95% CI, 0.97-0.99]; P=0.008), role limitations related to physical health (HR, 0.98 [95% CI, 0.97-0.99]; P=0.008), and general health dimensions of the SF-36 (HR, 0.97 [95% CI, 0.95-0.99]; P=0.002) were significantly predictive of cardiovascular events. However, after multivariable analysis, SF-36 dimensions were no longer significantly associated with the primary end point. Conclusions Patients with congenital heart disease with HF and poor quality of life experience severe events more frequently, making quality of life assessment and rehabilitation programs essential to alter their trajectory.

Impact of time to diagnosis on the occurrence of cardiogenic shock in MIS-C post-COVID-19 infection.

BACKGROUND: In multisystem inflammatory syndrome in children (MIS-C), diagnostic delay could be associated with severity. This study aims to measure the time to diagnosis in MIS-C, assess its impact on the occurrence of cardiogenic shock, and specify its determinants. METHODS: A single-center prospective cohort observational study was conducted between May 2020 and July 2022 at a tertiary care hospital. Children meeting the World Health Organization MIS-C criteria were included. A long time to diagnosis was defined as six days or more. Data on time to diagnosis were collected by two independent physicians. The primary outcome was the occurrence of cardiogenic shock. Logistic regression and receiver operating characteristic curve analysis were used for outcomes, and a Cox proportional hazards model was used for determinants. RESULTS: Totally 60 children were assessed for inclusion, and 31 were finally analyzed [52% males, median age 8.8 (5.7-10.7) years]. The median time to diagnosis was 5.3 (4.2-6.2) days. In univariable analysis, age above the median, time to diagnosis, high C-reactive protein, and high N-terminal pro-B-type natriuretic peptide (NT-proBNP) were associated with cardiogenic shock [odds ratio (OR) 6.13 (1.02-36.9), 2.79 (1.15-6.74), 2.08 (1.05-4.12), and 1.70 (1.04-2.78), respectively]. In multivariable analysis, time to diagnosis ≥ 6 days was associated with cardiogenic shock [adjusted OR (aOR) 21.2 (1.98-227)]. Time to diagnosis ≥ 6 days had a sensitivity of 89% and a specificity of 77% in predicting cardiogenic shock; the addition of age > 8 years and NT-proBNP at diagnosis ≥ 11,254 ng/L increased the specificity to 91%. Independent determinants of short time to diagnosis were age < 8.8 years [aHR 0.34 (0.13-0.88)], short distance to tertiary care hospital [aHR 0.27 (0.08-0.92)], and the late period of the COVID-19 pandemic [aHR 2.48 (1.05-5.85)]. CONCLUSIONS: Time to diagnosis ≥ 6 days was independently associated with cardiogenic shock in MIS-C. Early diagnosis and treatment are crucial to avoid the use of inotropes and limit morbidity, especially in older children.

Prevalence and Associated Factors of Long-term Growth Failure in Infants with Congenital Heart Disease Who Underwent Cardiac Surgery Before the Age of One.

Long-term growth failure can have negative impact on health (by increasing morbidity and mortality) and on neurodevelopmental outcomes. Its prevalence among children with congenital heart disease (CHD) is not well described. The aim of our study was to evaluate the prevalence of growth failure in a population of infants with CHD away from cardiac surgery and identify associated factors. We conducted a retrospective and multicentric study that included infants from the North of France who underwent cardiac surgery before the age of one, between January 2013 and December 2017. 331 infants were included among which 48% had a prenatal diagnosis, 15% had a genetic syndrome, and 15% were premature infants. Mean birth weight was 3 ± 0.6 kg. At surgery, 35% presented feeding difficulties (need for enriched formula and/or feeding tube) and 14% had growth failure (defined by Z-score weight for age < -2SD). 6-12 months after surgery, 16% still presented growth failure. Several associated factors were identified: prenatal diagnosis, genetic syndrome association, birth weight ≤ 3 kg, complex CHD (≥ 2 significative lesions, or double outlet right ventricle or single ventricle physiology), surgery after 30 days, and need for diuretic drug before surgery and/or still needed 1 month after surgery. Growth failure persists between 6 and 12 months after surgery in 16% of infants with CHD. More studies are needed to link growth failure and neurodevelopment, which is the new challenge for this aging population.

Safety, Efficacy and Long-Term Outcomes of Patients Treated with the Occlutech Paravalvular Leak Device for Significant Paravalvular Regurgitation.

Between December 2014 and March 2021, 144 patients with aortic (Ao) or mitral (Mi) paravalvular leaks (PVLs) were enrolled at 21 sites in 10 countries. Safety data were available for 137 patients, who were included in the safety analysis fraction (SAF), 93 patients with Mi PVLs and 44 patients with Ao PVLs. The full analysis set (FAS) comprised 112 patients with available stratum (aortic/mitral leak) as well as baseline (BL), 180-day or later assessments (2 years). Procedural success (implantation of the device with a proper closure of the PVL, defined as reduction in paravalvular regurgitation of ≥one grade as assessed by echocardiography post implantation) was achieved in 91.3% of FAS patients with Mi PVLs and in 90.0% of those with Ao PVLs. The proportion of patients suffering from significant or severe heart failure (HF), classified as New York Heart Association (NYHA) class III/IV, decreased from 80% at baseline to 14.1% at 2-year follow-up (FAS). The proportion of FAS patients needing hemolysis-related blood transfusion decreased from 35.5% to 3.8% and from 8.1% to 0% in Mi patients and Ao patients, respectively. In total, 35 serious adverse events (SAEs) were reported in 27 patients (19.7%) of the SAF population. The SAEs considered possibly or probably related to the device included device embolization (three patients), residual leak (two patients) and vascular complication (one patient). During follow-up, 12/137 (8.8%) patients died, but none of the deaths was considered to be device-related. Patients implanted with the Occlutech Paravalvular Leak Device (PLD) showed long-lasting improvements in clinical parameters, including NYHA class and a reduced dependency on hemolysis-related blood transfusions.

Pulmonary Valve Replacement and Redo Pulmonary Valve Replacement via Ministernotomy.

Minimally invasive cardiac surgery is mainly dedicated to acquired left-sided valve diseases. Ministernotomy is widely used for aortic valve repair or replacement, whereas pulmonary valve repair via this approach has been reported only recently. This article aims to describe the use of ministernotomy for pulmonary valve replacement in adult congenital patients.

Cardiovascular events in perimembranous ventricular septal defect with left ventricular volume overload: a French prospective cohort study (FRANCISCO).

The long-term prospective multi-centre nationwide (French) observational study FRANCISCO will provide new information on perimembranous ventricular septal defect with left ventricular overload but no pulmonary hypertension in children older than 1 year. Outcomes will be compared according to treatment strategy (watchful waiting, surgical closure, or percutaneous closure) and anatomic features of the defect. The results are expected to provide additional guidance about the optimal treatment of this specific population, which is unclear at present. BACKGROUND: The management of paediatric isolated perimembranous ventricular septal defect (pmVSD) with left ventricle (LV) volume overload but no pulmonary arterial hypertension (PAH) remains controversial. Three therapeutic approaches are considered: watchful waiting, surgical closure, and percutaneous closure. We aim to investigate the long-term outcomes of these patients according to anatomic pmVSD characteristics and treatment strategy. METHODS: The Filiale de Cardiologie Pediatrique et Congénitale (FCPC) designed the FRANCISCO registry, a long-term prospective nationwide multi-centre observational cohort study sponsored by the French Society of Cardiology, which enrolled, over 2 years (2018­2020), patients older than 1 year who had isolated pmVSD with LV volume overload. Prevalent complications related to pmVSD at baseline were exclusion criteria. Clinical, echocardiographic, and functional data will be collected at inclusion then after 1, 5, and 10 years. A core lab will analyse all baseline echocardiographic data to depict anatomical pmVSD features. The primary outcome is the 5-year incidence of cardiovascular events (infective endocarditis, sub-aortic stenosis, aortic regurgitation, right ventricular outflow tract stenosis, tricuspid regurgitation, PAH, arrhythmia, stroke, haemolysis, heart failure, or death from a cardiovascular event). We plan to enrol 200 patients, given the 10% estimated 5-year incidence of cardiovascular events with a 95% confidence interval of ±5%. Associations linking anatomical pmVSD features and treatment strategy to the incidence of complications will be assessed. CONCLUSIONS: The FRANSCICO study will provide the long-term incidence of complications in patients older than 1 year with pmVSD and LV volume overload. The results are expected to improve guidance for treatment decisions.

Exposure to low-dose ionising radiation from cardiac catheterisation and risk of cancer: the COCCINELLE study cohort profile.

PURPOSE: The COCCINELLE study is a nationwide retrospective French cohort set up to evaluate the risk of cancer in patients who undergone cardiac catheterisation (CC) procedures for diagnosis or treatment of congenital heart disease during childhood. PARTICIPANTS: Children who undergone CC procedures from 1 January 2000 to 31 December 2013, before the age of 16 in one of the 15 paediatric cardiology departments which perform paediatric CC in mainland France were included. The follow-up started at the date of the first recorded CC procedure until the exit date, that is, the date of death, the date of first cancer diagnosis, the date of the 18th birthday or the 31 December 2015, whichever occurred first. The cohort was linked to the National Childhood Cancer Registry to identify patients diagnosed with cancer and with the French National Directory for the Identification of Natural Persons to retrieve the patients' vital status. FINDINGS TO DATE: A total of 17 104 children were included in the cohort and followed for 110 335 person-years, with 22 227 CC procedures collected. Among the patients, 81.6% received only one procedure. Fifty-nine cancer cases were observed in the cohort. Standardised incidence ratios (SIRs) were increased for all-cancer (SIR=3.8, 95% CI: 2.9 to 4.9), leukaemia (SIR=3.3, 95% CI: 2.0 to 5.4), lymphoma (SIR=14.9, 95% CI: 9.9 to 22.5) and solid cancers excluding central nervous system (CNS) tumours (SIR=3.3, 95% CI: 2.0 to 5.5) compared with the general population. FUTURE PLANS: Dose reconstruction is currently underway to estimate individual cumulative doses absorbed to relevant organs, including red bone marrow and brain for respectively haematologic disorders and CNS tumours risk estimation. A dose-response analysis will be conducted with consideration to confounding factors such as age at exposure, gender, predisposing factors to cancer and other sources of medical diagnostic low-dose ionising radiation.

Edwards SAPIEN XT transcatheter pulmonary valve implantation: 5-year follow-up in a French Registry.

OBJECTIVES: This study sought to investigate patient intermediate-term outcomes after transcatheter pulmonary valve replacement (TPVR) with Edwards SAPIEN valve. BACKGROUND: The Edwards SAPIEN valve, initially designed for percutaneous aortic valve replacement, has been approved for TPVR in patients with dysfunctional right ventricular outflow tracts (RVOT), but only short-term follow-up has been reported. METHODS: From 2011 to 2016, 62 patients undergoing successful TPVR using the SAPIEN XT valve were consecutively included into the study. Primary efficacy and safety endpoints were defined as freedom from valve-reintervention and freedom from infective endocarditis at last follow-up, respectively. RESULTS: The primary efficacy outcome was met for 87.1% patients after a mean follow-up of 4.6 ± 1.8 years, corresponding to a freedom of reintervention at 5 years of 89% (95% CI 74.8-95.6%). Reinterventions were exclusively due to recurrent obstruction, no significant valvular regurgitation was observed. One case of infective endocarditis was reported, corresponding to a rate of 0.35% per patient-year (95% CI 0.01-2.00%). At 5 years, freedom from infective endocarditis was 98.4% (95% CI 89.1-99.8%). Six patients died or were transplanted due to advanced cardiac failure, without relationship with TPVR. In univariate analysis, reintervention was associated with young age, a smaller tube-graft, a higher pulmonary valve gradient after the procedure and a ratio of largest implanted stent diameter to invasive balloon conduit diameter over 1.35. CONCLUSIONS: This study documents the mid-term safety and efficacy of the Edwards SAPIEN XT valve in patients with dysfunctional RVOT, and identifies a patient profile associated with an uncertain benefit-risk balance.

Association between prophylactic angiotensin-converting enzyme inhibitors and overall survival in Duchenne muscular dystrophy-analysis of registry data.

AIMS: To estimate the effect of prophylactic angiotensin-converting enzyme inhibitors (ACEi) on survival in Duchenne muscular dystrophy (DMD). METHODS AND RESULTS: We analysed the data from the French multicentre DMD Heart Registry (ClinicalTrials.gov: NCT03443115). We estimated the association between the prophylactic prescription of ACEi and event-free survival in 668 patients aged 8 to 13 years, with normal left ventricular function, using (i) a Cox model with intervention as a time-dependent covariate, (ii) a propensity-based analysis comparing ACEi treatment vs. no treatment, and (iii) a set of sensitivity analyses. The study outcomes were overall survival and hospitalizations for heart failure (HF) or acute respiratory failure. Among the 668 patients included in the DMD Heart Registry, 576 (mean age 6.1 ± 2.8 years) were eligible for this study, of whom 390 were treated with ACEi prophylactically. Death occurred in 53 patients (13.5%) who were and 60 patients (32.3%) who were not treated prophylactically with ACEi, respectively. In a Cox model with intervention as a time-dependent variable, the hazard ratio (HR) associated with ACEi treatment was 0.49 [95% confidence interval (CI) 0.34-0.72] and 0.47 (95% CI 0.31-0.17) for overall mortality after adjustment for baseline variables. In the propensity-based analysis, 278 patients were included in the treatment group and 834 in the control group, with 18.5% and 30.4% 12-year estimated probability of death, respectively. ACEi were associated with a lower risk of death (HR 0.39; 95% CI 0.17-0.92) and hospitalization for HF (HR 0.16; 95% CI 0.04-0.62). All other sensitivity analyses yielded similar results. CONCLUSION: Prophylactic ACEi treatment in DMD was associated with a significantly higher overall survival and lower rates of hospitalization for HF.

Screening for neurodevelopmental disorders in children with congenital heart disease.

The aim of this study was to evaluate the frequency of neurodevelopmental disorders (NDD) in children with significant congenital heart disease (CHD) and to determine associated factors to NDD and frequency of follow-up in developmental therapies. Two hundred and ten children with significant CHD aged from 6 to 66 months were enrolled over a period of six months. The Ages & Stages Questionnaire Third Edition in French (ASQ-3) was used to assess neurodevelopmental domains. NDD were defined if cut-off scores were ≤ - 1SD. - 1SD corresponded to "Monitor" range: children with minor or emerging disorders; - 2SD corresponded to "Refer" range: children exhibiting neurodevelopmental delays. Forty children were in "Monitor" range and 86 in "Refer" range. NDD rate was 60.0% (n = 126, 95% CI, 53.4 to 66.6%). There was no difference regarding CHD severity (p = 0.99). Only the presence of non-cardiac disease (OR = 2.14; 95% CI, 1.11 to 4.20) was associated with NDD. Forty-six children with NDD had no developmental follow-up (among them 21 were in "Refer" range (10%)) despite this being available.Conclusion: Children with significant CHD are at risk for NDD regardless of CHD severity. Systematic and early monitoring in a specific care program is required. Barriers that prevent access of care must be identified.Trial registration: Neurodevelopmental Disorders in Children With Congenital Heart Disease. NeuroDis-CHD. NCT03360370. https://clinicaltrials.gov/ct2/show/NCT03360370 What is Known: • Children with CHD are at risk for neurodevelopmental disorders and behavioural problems impacting their social adaptation, academic achievements and quality of personal and family life even in adulthood. What is New: • Children with CHD are at risk for neurodevelopmental disorders regardless of the complexity of the CHD. • Even with the availability of appropriate developmental services, children with CHD are not correctly followed, highlighting the need of a specific program of care for a better outcome. Local barriers that prevent access of care of those children must be identified.