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Successful Use of TPO-Receptor Agonist Eltrombopag to Correct Thrombocytopenia for Unrelated-Donor Renal Transplant in an Adolescent with MYH-9-Related Disease.
Pediatr Blood Cancer
January 2025
Department of Pediatrics, Schulich School of Medicine & Dentistry, Western University, London, Ontario, Canada.
The Reconstitution of T-cells after Allogeneic Hematopoietic Stem Cell Transplant in a Pediatric Patient with Congenital Amegakaryocytic Thrombocytopenia (CAMT).
Endocr Metab Immune Disord Drug Targets
April 2024
Systematic Review and Meta-analysis Expert Group (SRMEG), Universal Scientific Education and Research Network (USERN), Tehran, Iran.
Background: Congenital amegakaryocytic thrombocytopenia (CAMT) is a bone marrow failure syndrome with autosomal recessive inheritance characterized by the lack of megakaryocytes and thrombocytopenia. The cause of the disease is a mutation in the c-Mpl gene, which encodes the thrombopoietin (TPO) receptor. The main treatment for this genetic disorder is an allogeneic hematopoietic stem cell transplant (allo-HSCT).
View Article and Find Full Text PDFInvestigational drugs for immune thrombocytopenia.
Expert Opin Investig Drugs
July 2022
Centre for Immunology, Blizard Institute, Barts and the London School of Medicine & Dentistry, Queen Mary University of London, London, UK.
Introduction: Primary immune thrombocytopenia (ITP) is an acquired autoimmune disease of unknown cause. Autoantibodies, self-reactive T cells and other immune abnormalities, with impairment of platelet production, lead to a reduced platelet count. Until recently, therapy was largely empirical using immune suppressants (none of which have undergone randomized clinical trials).
View Article and Find Full Text PDFCase Report: Successful Avatrombopag Treatment for Two Cases of Anti-PD-1 Antibody-Induced Acquired Amegakaryocytic Thrombocytopenia.
Front Pharmacol
January 2022
Department of Hematology, The Third Affiliated Hospital of Wenzhou Medical University, Wenzhou, China.
Anti-PD-1/PD-L1 immunotherapy has achieved impressive responses in multiple types of malignancies in recent years. However, immune-related adverse events (irAEs) occur and limit their continuous clinical use. Among these irAEs, acquired amegakaryocytic thrombocytopenia (AAT) is rare but often clinically serious, life-threatening and refractory to multiple treatment approaches.
View Article and Find Full Text PDFEltrombopag to allow chemotherapy in a patient with MYH9-related inherited thrombocytopenia and pancreatic cancer.
Int J Hematol
November 2020
Department of Medicine, Section of Internal and Cardiovascular Medicine, University of Perugia, Perugia, Italy.
Chemotherapy is the mainstay of treatment for advanced pancreatic cancer however, due to possible myelotoxicity, it is used with caution in patients with thrombocytopenia, especially when severe. TPO-receptor agonists have been employed for chemotherapy-induced thrombocytopenia, however treatment with TPO-receptor agonists to allow chemotherapy in patients with inherited thrombocytopenia has not been reported so far. We report the first successful use of eltrombopag to prevent chemotherapy-induced thrombocytopenia in a patient with MHY9-related disorder and pancreatic cancer.
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