Agnès Veyradier

Natural History and Treatment Outcomes of Congenital Thrombotic Thrombocytopenic Purpura: A Retrospective Longitudinal Cohort Study

Paul Coppo, Marie Scully, Johanna A. Kremer Hovinga, Bérangère S. Joly et autres

Background: Congenital thrombotic thrombocytopenic purpura (cTTP) is an ultra-rare, life-threatening thrombotic disorder. The real-world clinical trajectory and management of cTTP are not well defined. Objectives: Describe patient characteristics, quantify the incidence and prevalence of clinical manifestations and disease-related complications, and describe treatment …

fr, gb, ch, us (code pays fourni par la source)

Health-related quality of life in adults with von Willebrand disease: results of the French real-life Willebrand study on health-related quality of life

A. Borel-Derlon, Agnès Veyradier, Y. Repesse, N. Itzhar-Baïkan et autres

Background: Hemorrhagic events in von Willebrand disease (VWD) impair patients' physical health, daily functioning, and psychological/emotional well-being. While few studies have assessed health-related quality of life (HRQoL) in VWD, no prospective evaluation had been conducted in France. Objectives: The Willebrand study on …

fr, au, de (code pays fourni par la source)

Trp1250, Lys1252, and Arg1367 of ADAMTS13 comprise a hot spot for anti-CUB domain antibodies in patients with iTTP

Tim Postmus, Paul H. P. Kaijen, Floris P. J. van Alphen, Carmen van der Zwaan et autres

ABSTRACT: Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare life-threatening thrombotic disorder, which results from the development of autoantibodies targeting ADAMTS13. Most patients (>90%) with iTTP display antibodies against a shared epitope in the spacer domain of ADAMTS13. Nevertheless, a smaller population …

nl, fr, be (code pays fourni par la source)

Anti‐ ADAMTS13 Antibodies Trajectory is Associated With ADAMTS13 Recovery in Immune‐Mediated TTP

Marie E. Robert, Arthur Mageau, Y. Benhamou, François Provôt et autres

Current triplet regimens associating therapeutic plasma exchange (TPE), immunosuppression with corticosteroids and rituximab, and caplacizumab have dramatically improved the outcome of immune-mediated thrombotic thrombocytopenic purpura (iTTP). However, nearly half of the patients require extended caplacizumab treatment (i.e., > 30 days) due to …

fr, us (code pays fourni par la source)

Caplacizumab use in immune-mediated thrombotic thrombocytopenic purpura: an international multicentre retrospective Cohort study (The Capla 1000+ project)

Paul Coppo, Michaël Bubenheim, Y. Benhamou, Linus A. Völker et autres

Background: The anti-Von Willebrand Factor (VWF) nanobody caplacizumab is licensed for adults with immune-mediated thrombotic thrombocytopenic purpura (iTTP) in association with therapeutic plasma exchange (TPE) and immunosuppression. However, whether caplacizumab reduces mortality, and its optimal timing of initiation, is not completely settled. …

fr, de, at, es, us, hu, ca, br, jp, it, gr, gb, ch (code pays fourni par la source)

Real-world evaluation of a novel automated method for measuring ADAMTS-13 activity using the Ceveron s100 analyzer

Bérangère S. Joly, Hélène Deniau, Chloé Doinel, Adeline Brouillard et autres

BACKGROUND: Thrombotic thrombocytopenic purpura (TTP) is a thrombotic microangiopathy characterized by a severe functional deficiency of ADAMTS-13. Measuring ADAMTS-13 activity is crucial for diagnosing TTP (<10 IU/dL), monitoring treatments, and detecting relapses (<20 IU/dL). The Technofluor assay (Technoclone) allows a rapid ADAMTS-13 …

fr (code pays fourni par la source)

Identification of novel cryptic epitopes uncovers a distinct and consistent open ADAMTS13 conformation in iTTP

Quintijn Bonnez, Febe Boudry, Liesbeth De Waele, Lode Vermeersch et autres

Introduction: Plasma ADAMTS13 (pADAMTS13) circulates in a closed conformation. Besides the well-described Spacer (S)-CUB domain interaction, recent AlphaFOLD2 predictions suggested additional interdomain interactions in the closed ADAMTS13 conformation. In immune-mediated thrombotic thrombocytopenic purpura (iTTP), patient IgG autoantibodies open ADAMTS13 to expose a …

be, fr (code pays fourni par la source)

N-glycan shielded CUB domains of ADAMTS13 prevent binding of C-terminal antibodies in patients with immune-mediated TTP

Tim Postmus, Natalia Schilder, Juliana Ferreira de Santana, Pieter Langerhorst et autres

ABSTRACT: In immune-mediated thrombotic thrombocytopenic purpura (iTTP), patients develop antibodies against ADAMTS13. Most patients exhibit inhibitory antispacer antibodies. Noninhibitory antibodies binding to the carboxy-terminal CUB domains have been suggested to enhance the clearance of ADAMTS13 in iTTP. Furthermore, anti-CUB antibodies induce an …

nl, fr, be (code pays fourni par la source)

Efficacy and Safety of Obinutuzumab in Immune‐Mediated Thrombotic Thrombocytopenic Purpura

Júlia Weisinger, Raïda Bouzid, Jehane Fadlallah, Christelle Barbet et autres

International audience

fr (code pays fourni par la source)

Evaluation of a semi-automated test for quantification of von Willebrand multimers

Christophe Peronino, Nadia Rivet, Nathalie Itzhar-Baïkian, Aurélien Philippe et autres

The assessment of von Willebrand factor (VWF) multimer distribution, particularly following the implantation of circulatory support devices, is a crucial parameter in hemostasis. Our study aimed to evaluate the semi-automated quantification of VWF multimers using the Sebia Hydrasys analyzer. Our analysis focused …

fr (code pays fourni par la source)

Type 2N von Willebrand disease: genotype drives different bleeding phenotypes and treatment needs

Mélanie Y. Daniel, Catherine Ternisien, Sabine Marie Castet, Céline Falaise et autres

fr (code pays fourni par la source)

Diagnosis of thrombotic thrombocytopenic purpura: easy-to-use fiber optic surface plasmon resonance immunoassays for automated ADAMTS-13 antigen and conformation evaluation

Quintijn Bonnez, Charlotte Dekimpe, Tim Bekaert, Edwige Tellier et autres

be, fr (code pays fourni par la source)