107 results match your criteria: "Hôpitaux Universitaires Paris Centre site Hôtel Dieu[Affiliation]"

A pleiotropic recurrent dominant variant causes a complex multisystemic disease.

Sci Adv

September 2024

Laboratoire d'ImmunoRhumatologie Moléculaire, Institut national de la santé et de la recherche médicale (INSERM) UMR_S 1109, Plateforme GENOMAX, Centre de Recherche d'Immunologie et d'Hématologie and Centre de Recherche en Biomédecine de Strasbourg (CRBS), Faculté de Médecine, Fédération Hospitalo-Universitaire OMICARE, Fédération de Médecine Translationnelle de Strasbourg (FMTS), Université de Strasbourg, Strasbourg, France.

Article Synopsis
  • The study investigates a specific genetic variant in the IP3 receptor that results in a significant disorder affecting multiple systems, characterized by immunodeficiency and disturbed calcium release in cells.
  • The variant (c.7570C>T, p.Arg2524Cys) leads to cellular defects, particularly impacting T cells, and is shown to affect calcium regulation and mitochondrial function, evidenced in laboratory models.
  • Patients exhibited a range of symptoms beyond immunodeficiency, such as ectodermal dysplasia and short stature, suggesting that this genetic mutation plays a unique and broader role in disease compared to previously documented cases.
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Background: CASSIOPEIA part 1 demonstrated superior depth of response and prolonged progression-free survival with daratumumab in combination with bortezomib, thalidomide, and dexamethasone (D-VTd) versus bortezomib, thalidomide, and dexamethasone (VTd) alone as an induction and consolidation regimen in transplant-eligible patients newly diagnosed with myeloma. In CASSIOPEIA part 2, daratumumab maintenance significantly improved progression-free survival and increased minimal residual disease (MRD)-negativity rates versus observation. Here, we report long-term study outcomes of CASSIOPEIA.

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Article Synopsis
  • A study was conducted across 49 ICUs in France to investigate the clinical outcomes and severity of severe Pneumocystis jirovecii pneumonia (PJP), focusing on how delayed antibiotic treatment and corticosteroid therapy affect patient survival.
  • The study included 158 patients, mainly admitted due to acute respiratory failure, and found high mortality rates (31.6% in ICU, 40.5% at 6 months), with delayed antibiotic treatment beyond 96 hours linked to a significantly higher risk of death.
  • The results indicate that most patients did not receive preventive antibiotic therapy before needing intensive care, and both delayed antibiotic treatment and the use of corticosteroids worsened mortality rates in those with severe PJP.
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Background: The European Reference Network for rare Inherited Congenital Anomalies, ERNICA, guidelines for gastroschisis cover perinatal period to help teams to improve care.

Method: A systematic literature search including 136 publications was conducted. Research findings were assessed following the GRADE methodology.

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Article Synopsis
  • Ventilator associated pneumonia (VAP) caused by wild-type AmpC-producing Enterobacterales is common in ICU patients, and the study explores the effectiveness of different antimicrobial therapies (AMT), specifically piperacillin ± tazobactam (PTZ) and third-generation cephalosporins (3GCs).
  • In a study involving 274 ICU patients, no significant differences in treatment success at day 7 were found between the groups receiving PTZ, 3GCs, or other control treatments, with all groups showing success rates around 68-74%.
  • However, patients treated with 3GCs had a higher likelihood of pneumonia recurrence by day 28 compared to those on PTZ
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Hydrocortisone plus fludrocortisone for community acquired pneumonia-related septic shock: a subgroup analysis of the APROCCHSS phase 3 randomised trial.

Lancet Respir Med

May 2024

Department of Intensive Care, Raymond Poincaré Hospital, APHP University Versailles Saint Quentin-University Paris Saclay, Garches, France; Institut Hospitalo Universitaire PROMETHEUS, Garches, France; Laboratory of Infection & Inflammation-U1173, School of Medicine, INSERM, University Versailles Saint Quentin-University Paris Saclay, Garches, France; FHU SEPSIS, Garches, France. Electronic address:

Article Synopsis
  • The study explores the effectiveness of hydrocortisone and fludrocortisone treatments in patients with septic shock caused by community-acquired pneumonia (CAP) compared to non-CAP cases.
  • It includes data from the phase 3 APROCCHSS trial, which initially tested these treatments across multiple centers in France, focusing specifically on how they impact mortality outcomes.
  • Results indicate that patients with CAP may respond differently to these treatments, and various mortality rates and recovery metrics were analyzed to determine the overall benefit of the steroid regimen.
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Article Synopsis
  • In 2017, nusinersen, an injectable treatment for spinal muscular atrophy (SMA), was introduced, followed by the oral treatment risdiplam in 2020, leading to questions about appropriate care for adults with SMA due to limited data.
  • To standardize treatment access in France, a national SMA multidisciplinary team meeting (SMDT) was established in 2018 to support decision-making for adult patients.
  • An analysis of 107 patient cases showed that the SMDTs provided various treatment recommendations based on consultations, with most requests aimed at starting new treatments (nusinersen or risdiplam).
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Article Synopsis
  • New evidence regarding the use of corticosteroids in sepsis, ARDS, and CAP led to an updated guideline aimed at improving treatment recommendations for hospitalized adults and children.
  • A diverse 22-member panel of experts, including intensivists, doctors, and nurses, followed strict conflict of interest policies to develop evidence-based clinical practice guidelines.
  • The panel reviewed five key questions and provided four recommendations, including conditional use of corticosteroids in septic shock and ARDS, a strong recommendation for severe CAP, and advised against high-dose/short-duration steroid use in septic shock.
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Completeness and Mismatch of Patient-Important Outcomes After Trauma.

Ann Surg Open

December 2022

Centre d'Epidémiologie Clinique, AP-HP (Assistance Publique des Hôpitaux de Paris), Hôpital Hôtel Dieu, Paris, France.

Unlabelled: To assess the completeness of the collection of patient-important outcomes and the mismatch between outcomes measured in research and patients' important issues after trauma.

Summary Background Data: To date, severe trauma has mainly been assessed using in-hospital mortality. Yet, with 80 to 90% survivors discharged from hospital, it is critical to assess the collection of patient important long-term outcomes of trauma.

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Characteristics of Patients With Late-Onset Pompe Disease in France: Insights From the French Pompe Registry in 2022.

Neurology

August 2023

From the Neurology Department (C.L., N.T., P.L.), Raymond Poincaré University Hospital, Garches, APHP; Nord-Est-Ile-de-France Neuromuscular Reference Center (C.L., C.T., A.B., A.N.-P., M.M., H.P., N.T., A.A., P.L.), FHU PHENIX; Biostatistics Unit (DRCI) (M.D.A.), Clermont-Ferrand University Hospital; Service d'Electroneuromyographie et Pathologies Neuromusculaires (F.B.), Hospices Civils de Lyon; Inserm (C.T.), Lille University Hospital Center, U1172, Lille Neuroscience & Cognition, University of Lille; Centre de Référence des Maladies Neuromusculaires (E.S.-C., S.A.), Hôpital Timone Adultes, Assistance Publique Hôpitaux de Marseille; PACA Réunion Rhône Alpes Reference Center for Neuromuscular Diseases (E.S.-C., S.A.), FILNEMUS; Department of Neurology (E.L.), Grenoble University Hospital; APHP (A.B., A.A.), Service de Neuromyologie, Institut de Myologie, GH Pitié Salpêtrière, Paris; Neuromuscular Reference Center (G.S.), Bordeaux University Hospital (Pellegrin), University of Bordeaux; Neurology Department (J.-B.N.), Neuromuscular Center, CHRU Cavale Blanche, Brest; Peripheral Nervous System and Muscle Department (S.S.), Université Cote d'Azur, CHU de Nice; Centre de Référence des Maladies Neuromusculaires AOC (A.M.), CHU Hôtel Dieu, Nantes; Department of Neurology (A.N.-P.), University Hospital, Strasbourg; Centre de Référence des Maladies Neuromusculaires Rares Rhône-Alpes (A.L.), Hôpital Nord, CHU de Saint-Etienne; ALS Center (S.B.), Francois-Rabelais University, Tours, Centre-Val de Loire; Neuromuscular Reference Center (M.S.), Department of Neurology, University Hospital, Angers; Département de Neurologie (P.C.), Hôpital Purpan, CHU Toulouse; Department of Neurology (D.R.), CHU Nîmes, University of Montpellier, Nîmes; Department of Neurology (M.M.), Nancy University Hospital; Neuromuscular Reference Center (A.-L.B.-M.), Rouen University Hospital; Service de Physiologie et Explorations Fonctionnelles (H.P.), GH Paris Ile de France Ouest, Site Raymond Poincaré, APHP, Garches; U1179 INSERM (H.P., P.L.), Université Versailles Saint Quentin en Yvelines, Paris-Saclay; and Centre Hospitalo-Universitaire de Montpellier (D.H.), Hôpital Arnaud-de-Villeneuve, France.

Article Synopsis
  • The French Pompe Disease Registry was established in 2004 to track the progress of the disease in patients and evaluate the long-term effects of enzyme replacement therapy (ERT) with alglucosidase-alfa.
  • An update on 210 patients from the registry reveals that the median age of participants was almost 49, with many initially presenting symptoms like muscle weakness and respiratory issues, affecting their mobility significantly.
  • The findings indicate that awareness among doctors has improved, leading to earlier diagnoses and a decrease in the severity of cases at the time of inclusion, highlighting the registry's role in understanding and managing Pompe disease effectively.
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Importance: Optimal transfusion strategies in traumatic hemorrhage are unknown. Reports suggest a beneficial effect of 4-factor prothrombin complex concentrate (4F-PCC) on blood product consumption.

Objective: To investigate the efficacy and safety of 4F-PCC administration in patients at risk of massive transfusion.

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Asthma, rhinitis, and atopic dermatitis (AD) are interrelated clinical phenotypes that partly overlap in the human interactome. The concept of "one-airway-one-disease," coined over 20 years ago, is a simplistic approach of the links between upper- and lower-airway allergic diseases. With new data, it is time to reassess the concept.

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Objectives: To describe early electrocardiogram (ECG) abnormalities after status epilepticus (SE) and evaluate their association with 90-day neurological outcomes.

Design: Retrospective analysis of a multicenter, national prospective registry between February 2018 and June 2020.

Setting: Sixteen ICUs in France, IctalGroup Research Network.

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Characterisation of a high-risk profile for maternal thrombotic and severe haemorrhagic complications in pregnant women with antiphospholipid syndrome in France (GR2): a multicentre, prospective, observational study.

Lancet Rheumatol

December 2022

Assistance Publique-Hôpitaux de Paris, Service de Médecine Interne, Centre de Référence Maladies Auto-immunes et Systémiques Rares, Hôpital Cochin, Université de Paris, Paris, France; Assistance Publique-Hôpitaux de Paris, Centre for Clinical Epidemiology, Hôpital Hôtel-Dieu, Université de Paris, Centre of Research in Epidemiology and Statistics, Paris, France. Electronic address:

Background: Prospective data about the risks of thrombotic and severe haemorrhagic complications during pregnancy and post partum are unavailable for women with antiphospholipid syndrome. We aimed to assess thrombotic and haemorrhagic events in a prospective cohort of pregnant women with antiphospholipid syndrome.

Methods: This multicentre, prospective, observational study was done at 76 centres in France.

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Nestin contributes to laser choroidal and retinal neovascularization.

Mol Vis

October 2022

Centre de Recherches des Cordeliers, UMR_S INSERM 1138, Équipe 17, Université Paris Cité, Université Paris Sorbonne Cité, Paris, France.

Purpose: Choroidal and retinal neovascularization plays an essential role in various ocular diseases. In this study, we examined the role of nestin in this process. Nestin is an intermediate filament protein known to play several roles, including as a marker of neural progenitor and proliferating endothelial cells.

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Objectives: Despite international efforts to identify biomarkers of depression, none has been transferred to clinical practice, neither for diagnosis, evolution, nor therapeutic response. This led us to build a French national cohort (through the clinical and research network named SoPsy within the French biological psychiatry society (AFPBN) and sleep society (SFRMS)), to better identify markers of sleep and biological rhythms and validate more homogeneous subgroups of patients, but also to specify the manifestations and pathogeneses of depressive disorders. Before inclusions, we sought to provide a predefined, standardized, and robust set of data to be collected in all centers.

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Background: Dupilumab is the first biotherapy available for the treatment of moderate-to-severe childhood atopic dermatitis (AD).

Objective: The aim of this study was to evaluate the effectiveness and safety of dupilumab in daily practice.

Methods: Patients aged 6-11, who had received a first dose of dupilumab, were included in this multicentre retrospective cohort study.

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Article Synopsis
  • The study aims to evaluate how native joint septic arthritis (NJSA) is managed and the outcomes for patients in various rheumatology departments across France, covering cases from 2016 to 2017.
  • A total of 362 NJSA patients were analyzed, revealing that knee involvement is common and that Staphylococcus aureus is the leading pathogen; treatment varied and included prolonged antibiotic use, surgeries, and challenges related to complications and mortality.
  • The findings underscore a grim prognosis for NJSA, with significant morbidity and mortality rates, emphasizing the necessity for standardized management practices as outlined in new French guidelines released post-study.
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Purpose: To study prevalence of targeted therapy (TT)-related adverse events requiring ICU admission in solid tumor patients.

Methods: Retrospective multicenter study from the Nine-i research group. Adult patients who received TT for solid tumor within 3 months prior to ICU admission were included.

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Emergency vs Delayed Coronary Angiogram in Survivors of Out-of-Hospital Cardiac Arrest: Results of the Randomized, Multicentric EMERGE Trial.

JAMA Cardiol

July 2022

Department of Cardiology, European Hospital Georges Pompidou, Assistance Publique-Hôpitaux de Paris, Paris Cité University, Sudden Cardiac Death Expert Center, INSERM U 971, PARCC, Paris, France.

Importance: Although an emergency coronary angiogram (CAG) is recommended for patients who experience an out-of-hospital cardiac arrest (OHCA) with ST-segment elevation on the postresuscitation electrocardiogram (ECG), this strategy is still debated in patients without ST-segment elevation.

Objective: To assess the 180-day survival rate with Cerebral Performance Category (CPC) 1 or 2 of patients who experience an OHCA without ST-segment elevation on ECG and undergo emergency CAG vs delayed CAG.

Design, Setting, And Participants: The Emergency vs Delayed Coronary Angiogram in Survivors of Out-of-Hospital Cardiac Arrest (EMERGE) trial randomly assigned survivors of an OHCA without ST-segment elevation on ECG to either emergency or delayed (48 to 96 hours) CAG in 22 French centers.

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Article Synopsis
  • High-dose melphalan (HDM) combined with bortezomib (Bor-HDM) was tested in a phase 3 trial for patients with multiple myeloma to see if it was more effective than HDM alone.* -
  • The trial included 300 patients, and results showed no significant differences in complete response rates or minimum residual disease rates between the two treatment groups.* -
  • Although progression-free survival was slightly better in the Bor-HDM group (34.0 months) compared to HDM (29.6 months), the overall survival rates and serious adverse events were similar, indicating Bor-HDM did not provide a clear advantage.*
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