95 results match your criteria: "and Centre de Référence pour les Maladies Auto-Immunes Systémiques Rares[Affiliation]"

Efficacy of valaciclovir in preventing herpes zoster in patients receiving anifrolumab.

RMD Open

January 2025

Assistance Publique-Hôpitaux de Paris (AP-HP), Groupement Hospitalier Pitié-Salpêtrière, Centre de Référence des maladies auto-immunes et auto-inflammatoires systémiques rares de l'adulte d'Ile-de-France, Centre et Martinique, Service de Médecine Interne 2, Institut E3M, Paris, France, paris, France.

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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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Article Synopsis
  • First-line treatments for autoimmune systemic diseases (ARD) typically involve immunosuppressive drugs, but long-term use can lead to significant health risks.
  • Innovative therapies like mesenchymal stromal cells (MSCs) and Chimeric Antigen Receptor (CAR) T cell therapies are emerging as promising alternatives for severe or refractory cases of ARD.
  • A workshop by the French Speaking Society of Bone Marrow and Cell Transplantation focuses on establishing healthcare pathways and safety protocols for the deployment of MSCs and CAR-T therapies in ARD treatment, emphasizing patient safety and collaboration among specialists.
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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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Objectives: Alterations in tryptophan (Trp) metabolism have been reported in inflammatory diseases, including rheumatoid arthritis (RA). However, understanding whether these alterations participate in RA development and can be considered putative therapeutic targets remains undetermined.In this study, we combined quantitative Trp metabolomics in the serum from patients with RA and corrective administration of a recombinant enzyme in experimental arthritis to address this question.

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[Mobilization and conditioning protocols actualization for autologous stem cell transplantation for autoimmune diseases: Guidelines from MATHEC-SFGM-TC].

Bull Cancer

February 2024

AP-HP, hôpital Saint-Antoine, service d'hématologie clinique et thérapie cellulaire, 184, rue du Faubourg Saint-Antoine, 75012 Paris, France. Electronic address:

The Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC) organized the 13th workshop on hematopoietic stem cell transplantation clinical practices harmonization procedures in September 2022 in Lille, France. The aim of this workshop is to update the mobilization and conditioning protocols for autologous hematopoietic stem cell transplantation for autoimmune diseases, and to specify contraindications for transplant, conditioning regimen selection, immunosuppressive treatment discontinuation before mobilization and disease-specific surveillance.

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Advances in treatments of patients with classical and emergent neurological toxicities of anticancer agents.

Rev Neurol (Paris)

June 2023

Service de Neurologie, Hôpital d'Instruction des Armées Percy, Service de Santé des Armées, Clamart, France; UMR 9010 Centre Borelli, Université Paris-Saclay, École Normale Supérieure Paris-Saclay, CNRS, Service de Santé des Armées, Université Paris Cité, Inserm, Saclay, France; OncoNeuroTox Group: Center for Patients with Neurological Complications of Oncologic Treatments, Hôpitaux Universitaires Pitié-Salpêtrière - Charles-Foix et Hôpital d'Instruction des Armées Percy, Paris, France; École du Val-de-Grâce, Service de Santé des Armées, Paris, France. Electronic address:

The neurotoxicity associated to the anticancer treatments has received a growing body of interest in the recent years. The development of innovating therapies over the last 20years has led to the emergence of new toxicities. Their diagnosis and management can be challenging in the clinical practice and further research is warranted to improve the understanding of their pathogenic mechanisms.

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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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[The transitional care from pediatrics to adult medicine within the rare autoimmune and autoinflammatory diseases network].

Med Sci (Paris)

September 2022

Filière nationale des maladies auto-immunes et auto-inflammatoires rares (FAI²R), Hôpital Claude Huriez, CHU de Lille, Lille, France - Le Pass'âge, Hôpital-Femme-Mère-Enfant, Hospices civils de Lyon, Bron, France - Services de néphrologie, rhumatologie, dermatologie pédiatriques, Centre de référence des rhumatismes inflammatoires et maladies auto-immunes systémiques de l'enfant (RAISE), Hôpital femme-mère-enfant, Hospices civils de Lyon, Bron, France.

The committee involved in transitional care within the French network on rare autoimmune and auto-inflammatory diseases has developed tools and published recommendations for the care during the transition of adolescents and young adults suffering from these chronic diseases which often demonstrate flares. As the challenge is the compliance to the continuation of care in the adult world, the therapeutic alliance between the young patient and his/her pediatrician and then his/her adult doctor is particularly important. The working group thus carried out a survey on how doctors and young patients perceive their relationship during the period of transition in medical supervision.

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Anti-PM-Scl antibodies-positive patients encompass three different groups with distinct prognoses.

Rheumatology (Oxford)

April 2023

Département de Pneumologie, Hôpital Avicenne, Assistance Publique-Hôpitaux de Paris, INSERM U1272, Université Sorbonne Paris Nord, Bobigny, France.

Article Synopsis
  • The study aimed to identify homogeneous subgroups among patients with anti-PM-scleroderma-antibodies (PM-Scl-Abs) across various autoimmune diseases.
  • Data from 142 patients were analyzed and categorized into three distinct clusters based on their clinical and biological characteristics, focusing on skin and lung involvement.
  • The findings suggest that these subgroups exhibit differing clinical features and outcomes, with skin thickening and non-specific interstitial pneumonia (NSIP) being key factors for classification.
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Article Synopsis
  • The study aimed to assess the safety and effectiveness of filgotinib, lanraplenib, and tirabrutinib in patients with active Sjögren's Syndrome (SS).
  • In a double-blind trial involving 150 patients, primary outcomes were based on improvement criteria assessed at week 12, with no notable differences among the treatment groups compared to placebo.
  • Although primary and secondary endpoints were not met, some subgroup results and biomarker reductions suggest potential for these drugs, highlighting the need for further research.
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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.

Article Synopsis
  • The study aimed to assess the long-term safety of anakinra for treating patients with systemic juvenile idiopathic arthritis (sJIA) using data from the Pharmachild registry.
  • A total of 306 patients were analyzed, showing that while adverse events largely involved infections, the overall occurrence rate decreased over time, with most severe reactions occurring during the first six months of treatment.
  • Despite a high discontinuation rate (76%) primarily due to treatment inefficacy or achieving remission, no deaths or cancers were reported, affirming the medication's safety in the long term.
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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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