142 results match your criteria: "University Hospital Necker-Enfants malades[Affiliation]"

Aorta Without Coronary Arteries: Anatomic Variants of a Rare Malformation.

Pediatr Cardiol

January 2024

Medical and Surgical Unit of Congenital and Paediatric Cardiology, Reference Centre for Complex Congenital Heart Defects - M3C, University Hospital Necker-Enfants Malades, Paris, France.

Absence of connection of both coronary arteries to the aorta is an extremely rare congenital malformation. Most cases reported are anatomic variants of anomalous left coronary artery to pulmonary artery, found in isolation or in association with other congenital heart defects. We describe here four cases of patients born without any coronary artery connected to the aorta, including two with an almost complete absence of epicardial coronary arteries, one with single coronary artery to the right pulmonary artery, and one with left ventricular connection of a single coronary artery.

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  • * It involved a Phase II/III trial where participants received different concentrations of CsA-CE over two periods, focusing on improvements in VKC symptoms and corneal staining scores.
  • * Although both CsA-CE doses improved symptoms and signs of VKC, the study did not meet its primary efficacy endpoint; however, results contributed to the planning of a Phase III trial for the treatment.
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Antiphospholipid syndrome (APS) is a chronic autoimmune disease involving vascular thrombosis and/or obstetric morbidity and persistent antibodies to phospholipids or certain phospholipid-associated proteins. It is a rare condition in adults and even rarer in children. The diagnosis of APS can be facilitated by the use of classification criteria based on a combination of clinical and biological features.

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  • * A series of visual tests and imaging techniques were conducted to examine the patients' eye conditions, confirming the presence of NARP syndrome symptoms in all three individuals.
  • * The findings highlight the variable progression of NARP syndrome and emphasize the need for a comprehensive approach that includes both neurological and eye evaluations for effective disease management.
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Intramuscular capillary-type hemangioma: Diagnosis, treatment, and outcomes. A French multicentric retrospective study of 66 cases.

Eur J Radiol

August 2023

CHRU Tours, Department of Dermatology, Unit of Pediatric Dermatology, Tours, France; Reference Center for Genodermatoses and Rare Skin Diseases (MAGEC-Tours), Tours, France; University of Tours, University of Nantes, INSERM 1246-SPHERE, Tours, France. Electronic address:

Purpose: Intramuscular capillary-type hemangiomas (ICTHs) are rare entities, belonging to the group of intramuscular "hemangiomas." The diagnosis remains challenging. We aimed to assess the diagnostic criteria, treatments and outcomes of ICTHs.

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The safety and effectiveness of a novel annular keratopigmentation technique; a cross-sectional survey of patients.

BMC Ophthalmol

June 2023

Clinique Espace Nouvelle Vision, 6 Rue de la Grande Chaumière, Paris, 75006, France.

Purpose: We investigated safety aspects and other patient experiences related to a novel Femtosecond Laser Assisted Annular Keratopigmentation technique (FLAAK).

Setting: Espace Nouvelle Vision Clinic in Paris.

Methods: Monocentric, post-operative, cross-sectional survey of patients who returned to the clinic for a color correction after the FLAAK procedure.

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Respiratory complications in pediatric epidermal necrolysis: A retrospective study of 22 cases.

J Am Acad Dermatol

October 2023

Department of Pediatric Pulmonology and Allergology, University Hospital Necker-Enfants Malades, Paris, France; Faculté de médecine, Université de Paris, Paris, France. Electronic address:

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Congenital disconnection of the pulmonary arteries.

Eur J Cardiothorac Surg

July 2023

Medical and surgical unit of Congenital and Paediatric Cardiology, Reference Centre for Complex Congenital Heart Defects-M3C, University Hospital Necker-Enfants Malades, Paris, France.

Objectives: Disconnected pulmonary artery (PA) is a rare anomaly that can be isolated or associated with complex intracardiac malformations. Early reimplantation of the disconnected PA is recommended to allow growth and satisfactory pulmonary perfusion while preventing collateral artery development. The aim of this study was to describe the characteristics of patients with disconnected PA and, for those who had surgical reimplantation, to determine the incidence, delay and predictive factors of reintervention for reconnected PA stenosis.

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Background: The FLURESP project is a public health research funded by the European Commission, with the objective to design a methodological framework to assess the cost-effectiveness of existing public health measures against human influenza pandemics. A dataset has been specifically collected in the frame of the Italian health system. As most of interventions against human influenza are relavant against other respiratory diseases pandemics, potential interests in COVID-19 are discussed.

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Future directions in managing aniridia-associated keratopathy.

Surv Ophthalmol

November 2023

MERLN Institute for Technology-Inspired Regenerative Medicine, Maastricht University, Maastricht, the Netherlands; University Eye Clinic Maastricht, Maastricht University Medical Center+, Maastricht, the Netherlands.

Congenital aniridia is a panocular disorder that is typically characterized by iris hypoplasia and aniridia-associated keratopathy (AAK). AAK results in the progressive loss of corneal transparency and thereby loss of vision. Currently, there is no approved therapy to delay or prevent its progression, and clinical management is challenging because of phenotypic variability and high risk of complications after interventions; however, new insights into the molecular pathogenesis of AAK may help improve its management.

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Purpose: Preoperative evaluation of Image Defined Risk Factors (IDRFs) in neuroblastoma (NB) is crucial for determining suitability for upfront resection or tumor biopsy. IDRFs do not all carry the same weighting in predicting tumor complexity and surgical risk. In this study we aimed to assess and categorize a surgical complexity (Surgical Complexity Index, SCI) in NB resection.

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Home monitoring in asthma: towards digital twins.

Curr Opin Pulm Med

July 2023

Department of Paediatrics/Paediatric Respiratory Medicine and Allergology, Erasmus University Medical Centre - Sophia Children's Hospital, Rotterdam, The Netherlands.

Purpose Of Review: We highlight the recent advances in home monitoring of patients with asthma, and show that these advances converge towards the implementation of digital twin systems.

Recent Findings: Connected devices for asthma are increasingly numerous, reliable and effective: new electronic monitoring devices extend to nebulizers and spacers, are able to assess the quality of the inhalation technique, and to identify asthma attack triggers when they include a geolocation function; environmental data can be acquired from databases and refined by wearable air quality sensors; smartwatches are better validated. Connected devices are increasingly integrated into global monitoring systems.

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Background: Lemierre syndrome is typically associated with ear, nose, and throat (ENT) infections caused by Fusobacterium necrophorum. Since 2002, cases of atypical Lemierre-like syndrome secondary to Staphylococcus aureus have been reported.

Cases: We report two pediatric cases of atypical Lemierre syndrome with a similar presentation: exophthalmia, absence of pharyngitis, metastatic lung infection, and intracranial venous sinus thrombosis.

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Background: Moyamoya angiopathy (MMA) is a rare cerebrovascular condition leading to stroke. Mutations in 15 genes have been identified in Mendelian forms of MMA, but they explain only a very small proportion of cases. Our aim was to investigate the genetic basis of MMA in consanguineous patients having unaffected parents in order to identify genes involved in autosomal recessive MMA.

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Characteristics, Natural Course and Treatment of Intramuscular Capillary-type Haemangioma: A Systematic Literature Review.

Acta Derm Venereol

March 2023

CHRU Tours, Department of Dermatology, Unit of Pediatric dermatology, Tours, France; Reference center for genodermatoses and rare skin diseases (MAGEC-Tours), Tours, France; University of Tours, University of Nantes, INSERM 1246-SPHERE, Tours, France.

Intramuscular capillary-type haemangiomas (ICTH) are rare vascular anomalies that can easily be misdiagnosed as other entities. A systematic review was performed of all cases of ICTH in the literature since its first description in 1972. An adjudication committee reviewed cases to include only ICTHs.

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Introduction: Vernal keratoconjunctivitis (VKC) is a rare, severe allergic ocular disease, typically occurring in children and adolescents, that can have a significant impact on quality of life and lead to visual impairment. Long-term treatment may be necessary to tackle chronic inflammation and topical corticosteroid dependency must be minimised due to the risk of complications. There is a need for unified clinical guidance to aid the assessment, diagnosis and management of VKC across Europe.

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Background:  rVIII-SingleChain is a recombinant factor VIII (FVIII) with increased binding affinity to von Willebrand factor compared with other FVIII products. rVIII-SingleChain is indicated for the treatment and prevention of bleeding episodes in patients with hemophilia A.

Objectives:  To collect real-world evidence data from patients treated with rVIII-SingleChain to confirm the efficacy and safety established in the clinical trial program and carry out a population pharmacokinetic (PK) analysis.

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Background: Continuous venovenous hemodiafiltration (CVVHDF) is one of the treatments of critically ill children presenting severe acute liver failure. This affliction might be induced by HSV infection requiring a treatment by acyclovir. Continuous kidney replacement therapy (CKRT) can alter its pharmacokinetics, according to its physicochemical properties and CVVHDF settings.

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Background: Acute intracranial large vessel occlusion (LVO) is an important cause of morbidity and mortality among children; however, unlike in adults, no clinical trial has investigated the benefit of mechanical thrombectomy (MT) in pediatric LVO. Thus, MT remains an off-label procedure for pediatric stroke.

Purpose: To investigate the efficacy and safety of MT in pediatric LVO.

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Article Synopsis
  • Researchers conducted two clinical trials (NOVATIVE and VEKTIS) to evaluate the effectiveness and safety of cyclosporine A cationic ophthalmic emulsion (CsA CE) in treating vernal keratoconjunctivitis (VKC) in children and adolescents.
  • The studies pooled data from 211 patients, comparing high-dose CsA CE, low-dose CsA CE, and vehicle (placebo), with results showing significant improvements in corneal fluorescein staining scores in both CsA groups versus the vehicle.
  • CsA CE was generally well-tolerated, with a similar rate of mild to moderate adverse events across all groups, supporting its use as a safe treatment
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New technologies enable the creation of digital twin systems (DTS) combining continuous data collection from children's home and artificial intelligence (AI)-based recommendations to adapt their care in real time. The objective was to assess whether children and adolescents with asthma would be ready to use such DTS. A mixed-method study was conducted with 104 asthma patients aged 8 to 17 years.

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Incidence of iatrogenic withdrawal syndrome and associated factors in surgical pediatric intensive care.

Arch Pediatr

January 2023

University of Paris, Paris, France; Department of Pediatric Anesthesia and Intensive Care, University Hospital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris, Paris, France; EA7323: Pharmacologie et évaluation des thérapeutiques chez l'enfant et la femme enceinte, Hôpitaux Universitaires Paris Centre, University of Paris, Paris, France.

Background: Iatrogenic withdrawal syndrome (IWS) is a complication of prolonged sedation/analgesia in pediatric intensive care unit (PICU) patients. The epidemiology of IWS is poorly understood, as validated diagnostic tools are rarely used. The main objective of our study was to use the WAT-1 score to assess the incidence of IWS in our unit.

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