69 results match your criteria: "Centre de référence national pour les maladies auto-immunes systémiques rares[Affiliation]"

Allogeneic umbilical cord-derived mesenchymal stromal cells as treatment for systemic lupus erythematosus: a single-centre, open-label, dose-escalation, phase 1 study.

Lancet Rheumatol

December 2024

SITI, CHU Rennes, Etablissement Français du Sang Bretagne, Rennes, France; INSERM UMR 1236, Université Rennes, INSERM, Etablissement Français du Sang Bretagne, Rennes, France. Electronic address:

Background: Patients with systemic lupus erythematosus (SLE) with inadequate responses to standard therapies have unmet therapeutic needs. The immunomodulatory, proangiogenic, and antifibrotic properties of mesenchymal stromal cells support their use in treating patients with SLE. We aimed to assess the safety of a single intravenous infusion of allogeneic umbilical cord-derived mesenchymal stromal cells in patients with severe SLE.

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Preclinical in vivo assessment of the activity of AZD7442 anti-SARS-CoV-2 monoclonal antibodies against Omicron sublineages.

Biomed Pharmacother

August 2024

Unité des Virus Émergents (UVE: Aix-Marseille Univ, Università di Corsica, IRD 190, Inserm 1207, IRBA), France. Electronic address:

Article Synopsis
  • Therapeutic monoclonal antibodies have been effective against SARS-CoV-2 in at-risk groups, but their effectiveness is declining due to new variants.
  • Health authorities are using in vitro tests to guide treatment recommendations, but these tests may not accurately reflect clinical efficacy in real-world situations.
  • A study using hamsters shows that while AZD7442 retains some effectiveness against specific variants like BA.1 and BA.2, its reliability drops significantly against BA.5, underscoring the need for animal studies to enhance understanding of antibody performance in humans.
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Objectives: To investigate the frequency and factors associated with disease flare following vaccination against SARS-CoV-2 in people with inflammatory/autoimmune rheumatic and musculoskeletal diseases (I-RMDs).

Methods: Data from the European Alliance of Associations for Rheumatology Coronavirus Vaccine physician-reported registry were used. Factors associated with flare in patients with I-RMDs were investigated using multivariable logistic regression adjusted for demographic and clinical factors.

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[Indirect causes of maternal deaths (except stroke, cardiovascular diseases and infections) in France 2016-2018].

Gynecol Obstet Fertil Senol

April 2024

Service de gynécologie obstétrique, maternité Port Royal, hôpital Cochin, AP-HP, 27, rue du Faubourg-Saint-Jacques, 75014 Paris, France.

Maternal deaths from indirect obstetric cause result from a preexisting condition or a condition that occurred during pregnancy without obstetric causes but was aggravated by the physiological effects of pregnancy. Twenty-nine deaths with an indirect cause related to a preexisting condition, excluding circulatory diseases or infections, were analysed by the expert committee. Pre-pregnancy pathology was documented in 16 women (epilepsy, n=7; amyloid angiopathy, n=1; Dandy-Walker syndrome, n=1; autoimmune diseases, n=3; diffuse infiltrative pneumonitis, n=1; thrombotic thrombocytopenic purpura, n=1; ovarian cancer in fragile X, n=1; major sickle cell disease, n=1).

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Patterns of immunosuppressive drug use during pregnancy in women with systemic vasculitis: A nationwide population-based cohort study.

Eur J Intern Med

March 2023

Unité de Recherche Clinique, Hôpital Cochin, Assistance Publique-Hôpitaux de Paris, Paris, France; Département de Pharmacologie, Centre Régional de Pharmacovigilance, Hôpital Cochin, Assistance Publique-Hôpitaux de Paris, Paris, France. Electronic address:

Objective: Systemic vasculitis (SV) rarely affects women of childbearing age and only small series have been reported to date in pregnant patients. The discovery of an unplanned pregnancy can be an urgent cause for modifying treatments. This study aimed to describe immunosuppressive drugs use before, during and after pregnancy in women with SV.

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Vasculitis and familial Mediterranean fever: Description of 22 French adults from the juvenile inflammatory rheumatism cohort.

Front Med (Lausanne)

October 2022

Département de Médecine Interne, Centre de Référence des Maladies Auto-Inflammatoires et des Amyloses d'Origine Inflammatoire (CEREMAIA), Hôpital Tenon, Sorbonne Université, AP-HP, Paris, France.

Objective: The frequency of vasculitis may be increased in patients with Familial Mediterranean Fever (FMF), according to several studies. Our aim was to assess the characteristics of French adult patients with both diseases.

Methods: Patients with vasculitis were selected from patients followed for FMF in the French JIR-cohort.

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Anakinra in Patients With Systemic Juvenile Idiopathic Arthritis: Long-term Safety From the Pharmachild Registry.

J Rheumatol

April 2022

J.F. Swart, MD, N. Wulffraat, Professor, Department of Pediatric Immunology and Rheumatology, Wilhelmina Children's Hospital, Utrecht, the Netherlands.

Objective: To evaluate the long-term safety profile of anakinra in patients with systemic juvenile idiopathic arthritis (sJIA).

Methods: Data from patients with sJIA enrolled in the Pharmachild registry (ClinicalTrials.gov: NCT03932344) prior to September 30, 2018, and treated with anakinra were analyzed.

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Objective: To develop and validate new Juvenile Arthritis Disease Activity Score 10 (JADAS10) and clinical JADAS10 (cJADAS10) cutoffs to separate the states of inactive disease (ID), minimal disease activity (MiDA), moderate disease activity (MoDA), and high disease activity (HDA) in children with oligoarthritis and with rheumatoid factor-negative polyarthritis, based on subjective disease assessment by the treating pediatric rheumatologist.

Methods: The cutoffs definition cohort was composed of 1,936 patients included in the multinational Epidemiology, Treatment and Outcome of Childhood Arthritis (EPOCA) study. Using the subjective physician rating as an external criterion, 4 methods were applied to identify the cutoffs: mapping, Youden index, 90% specificity, and maximum agreement.

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Epidemiology, Mortality and Healthcare Resource Utilization Associated With Systemic Sclerosis-Associated Interstitial Lung Disease in France.

Front Med (Lausanne)

August 2021

Hôpital Louis Pradel, Centre Coordonnateur National de Référence des Maladies Pulmonaires Rares, Hospices Civils de Lyon, UMR754 INRAE and Université Claude Bernard Lyon 1, Member of ERN-LUNG, RespiFil, OrphaLung, Lyon, France.

To investigate the clinical characteristics, epidemiology, survival estimates and healthcare resource utilization and associated costs in patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD) in France. The French national administrative healthcare database, the Système National des Données de Santé (SNDS), includes data on 98.8% of the French population, including data relating to ambulatory care, hospitalizations and death.

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Objectives: Coxiella and Bartonella spp. display particular tropism for endothelial or endocardial tissues and an abnormal host response to infections with induced autoimmunity. We aimed, through a case series combined with a comprehensive literature review, to outline characteristics of Coxiella and Bartonella infections presenting as systemic vasculitis.

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[Professional representation of chronic idiopathic musculoskeletal pain in adolescents].

Soins Pediatr Pueric

June 2021

Maison de Solenn - Maison des adolescents de l'hôpital Cochin, AP-HP, 97 boulevard de Port-Royal, 75014 Paris, France; Université Paris-Saclay, UVSQ, Inserm, Centre de recherche en épidémiologie et santé des populations, Team DevPsy, 16 avenue Paul-Vaillant-Couturier, 94807 Villejuif, France.

Chronic idiopathic musculoskeletal pain is common in adolescence, and its impact is sometimes severe. The diagnostic process, which consists of eliminating other etiologies, can be long, complex, and at risk of medical nomadism. Specialists rely on many clinical elements to orient themselves.

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Estimates of epidemiology, mortality and disease burden associated with progressive fibrosing interstitial lung disease in France (the PROGRESS study).

Respir Res

May 2021

Hôpital Louis Pradel, Centre National de Référence des Maladies Pulmonaires Rares, Hospices Civils de Lyon, Lyon, OrphaLung, RespiFil, ERN-LUNG, Claude Bernard University Lyon 1, 28 Avenue du Doyen Lepine, 69677, Lyon Cedex, France.

Background: There is a paucity of data on the epidemiology, survival estimates and healthcare resource utilisation and associated costs of patients with progressive fibrosing interstitial lung disease (PF-ILD) in France. An algorithm for extracting claims data was developed to indirectly identify and describe patients with PF-ILD in the French national administrative healthcare database.

Methods: The French healthcare database, the Système National des Données de Santé (SNDS), includes data related to ambulatory care, hospitalisations and death for 98.

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[EBV and immunodeficiency].

Rev Med Interne

December 2021

Université de Strasbourg, Inserm UMR - S1109, 67000 Strasbourg, France; Hôpitaux universitaires de Strasbourg, service d'immunologie clinique et de médecine interne, centre national de référence des maladies auto-immunes et systémiques rares, Est/Sud-Ouest (RESO), centre de compétence pour les déficits immunitaires primitifs de l'adulte, 67000 Strasbourg, France; Université de Strasbourg, faculté de médecine, 67000 Strasbourg, France. Electronic address:

Epstein-Barr virus (EBV), discovered in 1964, is a double-stranded DNA virus belonging to the Herpesviridae family. EBV has a lymphoid tropism with transforming capacities using different oncogenic viral proteins. This virus has two replication cycles: a lytic cycle mainly occuring during primary infection and a latent cycle allowing viral persistence into host memory B cells.

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[Maternal mortality due to venous thromboembolism in France 2013-2015].

Gynecol Obstet Fertil Senol

January 2021

Médecine interne, centre de référence national pour les maladies auto-immunes rares d'Île de France, hôpital Cochin, AP-HP, 27, rue de Faubourg-Saint-Jacques, 75679 Paris, France.

Pregnancy and postpartum represent periods at very high risk of venous thromboembolism disease which appears to extend well beyond the classic 6-8 weeks after childbirth. Pulmonary embolism (PE) is still one of the three leading causes of direct maternal death (MM) in most developed countries. Between 2013 and 2015, 23 maternal deaths were caused by a venous thromboembolic complication (VTE) (20 pulmonary embolism and 3 cerebral thrombophlebitis), representing 8.

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Development and Testing of Reduced Versions of the Manual Muscle Test-8 in Juvenile Dermatomyositis.

J Rheumatol

June 2021

C. Malattia, MD, PhD, A. Consolaro, MD, PhD, UOC Clinica Pediatrica e Reumatologia, IRCCS Istituto Giannina Gaslini and Dipartimento di Neuroscienze, Riabilitazione, Oftalmologia, Genetica e Scienze Materno-Infantili (DiNOGMI), Università degli Studi di Genova, Genoa, Italy.

Objective: To develop and test shortened versions of the Manual Muscle Test-8 (MMT-8) in juvenile dermatomyositis (JDM).

Methods: Construction of reduced tools was based on a retrospective analysis of individual scores of MMT-8 muscle groups in 3 multinational datasets. The 4 and 6 most frequently impaired muscle groups were included in MMT-4 and MMT-6, respectively.

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[Indirect causes of maternal deaths (except stroke, cardiovascular diseases and infections) in France 2013-2015].

Gynecol Obstet Fertil Senol

January 2021

Département de gynécologie obstétrique, hôpital privé d'Antony, 1, rue Velpeau, 92160 Antony, France.

Maternal deaths from indirect obstetric causes are the result of a pre-existing disease or condition that appeared during pregnancy without obstetric causes, but which was aggravated by the physiological effects of pregnancy. Twenty-six deaths from indirect causes related to a pre-existing pathology, excluding disease of the circulatory system or infection, were analysed by the committee of experts. Pre-existing pathology during pregnancy was documented in 13 women (asthma, n=3, genetic diseases, n=3, previous breast cancer, n=2, major sickle cell syndrome, n=2, epilepsy, n=1 and brain tumour, n=1).

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Mediterranean Diet and Risk of Rheumatoid Arthritis: Findings From the French E3N-EPIC Cohort Study.

Arthritis Rheumatol

January 2021

Centre National de Référence pour les Maladies Auto-Immunes Systémiques Rares, AP-HP, Hôpital Bicêtre, Université Paris-Saclay, INSERM UMR1184, Le Kremlin Bicêtre, France.

Objective: The Mediterranean diet has been reported to be associated with a significant reduction in risk of noncommunicable diseases. We undertook this study to assess the relationship between adherence to the Mediterranean diet and the risk of rheumatoid arthritis (RA), especially in high-risk individuals.

Methods: The E3N study (Etude Epidémiologique Auprès des Femmes de la Mutuelle Générale de l'Education Nationale) is a French prospective cohort study that has included 98,995 women since 1990.

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