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Impact of Thrombopoietin Receptor Agonists on Pathophysiology of Pediatric Immune Thrombocytopenia.
Curr Issues Mol Biol
January 2025
Children & Adolescent Hematology-Oncology Unit, Second Department of Pediatrics, School of Medicine, Aristotle University of Thessaloniki, 54124 Thessaloniki, Greece.
Immune thrombocytopenia (ITP) in pediatric patients is a common cause of isolated thrombocytopenia. Various pathophysiological mechanisms are implicated in ITP pathogenesis, including the production of autoantibodies against components of platelets (PLTs) by B-cells, the activation of the complement system, phagocytosis by macrophages mediated by Fcγ receptors, the dysregulation of T cells, and reduced bone marrow megakaryopoiesis. ITP is commonly manifested with skin and mucosal bleeding, and it is a diagnosis of exclusion.
View Article and Find Full Text PDFA compound heterozygous mutation causes congenital thrombotic thrombocytopenic purpura: a case report.
Front Med (Lausanne)
January 2025
Department of Hematology, The Second Affiliated Hospital of Nanchang University, Nanchang, China.
Congenital thrombotic thrombocytopenic purpura (cTTP) is a thrombotic microangiopathy (TMA) characterized by severe hereditary ADAMTS13 (a disintegrin and metalloproteinase with thrombospondin type 1 motifs 13) deficiency caused by mutations. This rare autosomal recessive genetic disorder is often misdiagnosed as immune thrombocytopenia (ITP) or hemolytic uremic syndrome (HUS). Here, we report a 21-year-old male cTTP patient with a compound heterozygous mutation.
View Article and Find Full Text PDFEfficacy and Safety Results With Rilzabrutinib, an Oral Bruton Tyrosine Kinase Inhibitor, in Patients With Immune Thrombocytopenia: Phase 2 Part B Study.
Am J Hematol
January 2025
Hematology Division, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Current treatments for persistent or chronic immune thrombocytopenia (ITP) are limited by inadequate response, toxicity, and impaired quality of life. The Bruton tyrosine kinase inhibitor rilzabrutinib was evaluated to further characterize safety and durability of platelet response. LUNA2 Part B is a multicenter, phase 1/2 study in adults with ITP (≥ 3 months duration, platelet count < 30 × 10/L) who failed ≥ 1 ITP therapy (NCT03395210, EudraCT 2017-004012-19).
View Article and Find Full Text PDFRed flags to suspect inborn errors of immunity in patients with autoimmune diseases.
Biomedica
December 2024
Grupo de Inmunología Celular y Molecular - InmuBo, Universidad El Bosque, Bogotá, D. C., Colombia; Reumatología, Escuela de Medicina, Universidad Militar Nueva Granada, Bogotá, D. C., Colombia; Grupo de Inmunología, Escuela de Medicina, Universidad Militar Nueva Granada, Bogotá, D. C., Colombia; Departamento de Reumatología e Inmunología, Hospital Militar Central, Bogotá, D. C., Colombia.
Inborn errors of immunity are monogenic disorders that predispose patients to immune dysregulation, autoimmunity, and infection. Some autoimmune diseases, such as autoimmune cytopenias, systemic lupus erythematosus, and inflammatory bowel diseases, are increasingly recognized as phenotypes of inborn errors of immunity. The objective of this article was to identify red flags or clinical/laboratory markers to suspect inborn errors of immunity in patients with autoimmune cytopenias, systemic lupus erythematosus, and inflammatory bowel diseases through a systematic literature review.
View Article and Find Full Text PDFSuspected heparin-induced thrombocytopaenia in pulmonary thromboendarterectomy: retrospective cohort.
Interdiscip Cardiovasc Thorac Surg
December 2024
Department of Thoracic Surgery and Heart-Lung Transplantation, Paris-Saclay University, Marie-Lannelongue Hospital, Le Plessis-Robinson, France.
Objectives: Heparin is given for anticoagulation during and after pulmonary thromboendarterectomy for chronic thromboembolic pulmonary hypertension. Our objective was to add to the limited data available on the incidence, management and outcomes of suspected heparin-induced thrombocytopaenia after pulmonary thromboendarterectomy for chronic thromboembolic pulmonary hypertension.
Methods: This retrospective single-centre study included consecutive patients with suspected heparin-induced thrombocytopaenia after pulmonary thromboendarterectomy done in 2005-2018.
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