• Promacta (eltrombopag olamine) is a thrombopoietin receptor agonist that is used to increase low blood platelet counts (thrombocytopenia) in certain people with chronic immune thrombocytopenia (ITP) and severe aplastic anemia. (drugs.com)
  • The thrombopoietin-receptor agonist eltrombopag is approved for use in patients with severe aplastic anemia who fail to respond adequately to immunosuppressive therapy. (medscape.com)
  • Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematological disorder characterized by complement-mediated intravascular hemolysis, bone marrow failure, and severe thrombophilia ( 1 ). (frontiersin.org)
  • Some individuals with acquired aplastic anemia also have another disorder at the same time, called paroxysmal nocturnal hemoglobinuria (PNH). (rarediseases.org)
  • True aplastic anemia (most common in adolescents and young adults) is idiopathic in about half of cases. (merckmanuals.com)
  • In this issue of Blood , 2 papers deal with HLA in patients with acquired idiopathic aplastic anemia (AA). (ashpublications.org)
  • Aplastic anemia is a disorder of the hematopoietic stem cell that results in a loss of blood cell precursors, hypoplasia or aplasia of bone marrow, and cytopenias in two or more cell lines (red blood cells, white blood cells, and/or platelets). (merckmanuals.com)
  • In acquired aplastic anemia, an almost complete absence of hematopoietic stem cells results in low levels of red and white blood cells and platelets (pancytopenia). (rarediseases.org)
  • Some pediatric patients with newly diagnosed acquired aplastic anemia may be more likely to have a complete response with immunosuppressive treatment combined with eltrombopag (ELTR) compared with immunosuppression alone, according to research published in Blood Advances . (thalassaemia.org.cy)
  • For this randomized phase 2 study, researchers compared the safety and efficacy of immunosuppression plus ELTR vs immunosuppression alone among pediatric patients with newly diagnosed severe or very severe aplastic anemia. (thalassaemia.org.cy)
  • The clinical presentation of patients with aplastic anemia includes signs and symptoms related to the decrease in bone marrow production of hematopoietic cells. (medscape.com)
  • The term aplastic anemia commonly implies a panhypoplasia of the bone marrow with cytopenias in at least two hematopoietic lineages. (merckmanuals.com)
  • The specific medications administered for aplastic anemia depend on the choice of therapy and whether it is supportive care only, immunosuppressive therapy, or hematopoietic cell transplantation (HCT). (medscape.com)
  • [ 6 ] The Pediatric Haemato-Oncology Italian Association has issued guidelines on diagnosis and management of acquired aplastic anemia in childhood. (medscape.com)
  • Patients with aplastic anemia require transfusion support until the diagnosis is established and specific therapy can be instituted. (medscape.com)
  • He is credited with contributing to understanding the pathophysiology of the disease as immune-mediated and with developing immunotherapy for aplastic anemia that has dramatically improved survival rates for the disease. (wikipedia.org)
  • Background: Severe aplastic anemia is a rare and serious blood disorder. (centerwatch.com)
  • However, it was not until 1904 that Anatole Chauffard named this disorder aplastic anemia. (medscape.com)
  • Pure Red Blood Cell Aplasia Acquired pure red blood cell aplasia is a disorder of erythroid precursors that results in an isolated normocytic anemia. (merckmanuals.com)
  • Individuals affected with acquired aplastic anemia are also at risk that it will evolve into another similar disorder known as myelodysplasia. (rarediseases.org)
  • Participants in this study must be 3 years or older and have a telomere disease and signs of aplastic anemia, lung disease such as pulmonary fibrosis, or liver disease. (nih.gov)
  • In combination with standard immunosuppressive therapy for the first-line treatment of severe aplastic anemia. (drugs.com)
  • It is unclear exactly how quickly Promacta + combination immunosuppressive therapy works in previously untreated patients with severe aplastic anemia. (drugs.com)
  • To treat patients with severe aplastic anemia who have had insufficient response to immunosuppressive therapy. (drugs.com)
  • It is unclear exactly how quickly Promacta works in patients with severe aplastic anemia who have not responded to previous immunosuppressive therapy. (drugs.com)
  • The British Committee for Standards in Haematology recommends treating infection or uncontrolled bleeding before administering immunosuppressive therapy, including in patients scheduled for HCT. (medscape.com)
  • [ 5 ] The Pediatric Haemato-Oncology Italian Association recommends HCT from a matched sibling donor for severe aplastic anemia, and if a matched donor is not available, options include immunosuppressive therapy or unrelated donor HCT. (medscape.com)
  • He is primarily known for his work in the pathophysiology and treatment of aplastic anemia, and is also known for his contributions to the pathophysiology of parvovirus B19 infection. (wikipedia.org)
  • This therapy is now the standard treatment for the disease worldwide. (wikipedia.org)
  • Although the anemia is often normocytic, mild macrocytosis can also be observed in association with stress erythropoiesis and elevated fetal hemoglobin levels. (medscape.com)
  • Overview of Decreased Erythropoiesis Anemia, a decrease in the number of red blood cells (RBCs), hemoglobin (Hb) content, or hematocrit (Hct), can result from decreased RBC production (erythropoiesis), increased RBC destruction. (merckmanuals.com)
  • The onset is insidious, and the initial clinical manifestation is frequently related to anemia or bleeding, although fever or infections may be noted at presentation. (medscape.com)
  • Importantly, the selection of the studies discussed here was made by PROF. DR. DOMINIQUE BRON (Haematologist at Institut Jules Bordet, Brussels and member of the BJH editorial board), based on the added value of the studies in daily clinical practice. (bjh.be)
  • Background: To date, fewer than 20 patients have been identified as having germline biallelic mutations in the coronin-1A gene (CORO1A) and its protein with clinical features of combined immunodeficiency characterized by T-cell lymphopenia ranging from the severe phenotype to the mild phenotype, recurrent infections, and lymphoproliferative disorders. (bvsalud.org)
  • At baseline, in the ELTR and immunosuppression-only groups, the median ages were 10.5 and 8.7 years, 71.4% and 61.2% of patients were male sex, and 36.7% and 42.9% had SAA within their respective group while 63.3% and 57.1% had vSAA, respectively. (thalassaemia.org.cy)
  • This study is testing whether low doses of the medicine danazol help prevent telomeres from getting shorter and reduce signs of damage from aplastic anemia or related conditions. (nih.gov)
  • In 2005, he and his postdoctoral fellow, Rodrigo Calado, described the first mutations in the telomerase gene, TERT, in patients with aplastic anemia. (wikipedia.org)
  • Conclusion: Collectively, we added new data about the CORO1A gene as a key player in actin cytoskeleton dynamics and cell signaling. (bvsalud.org)
  • Complete normalization of hemoglobin (complete and major hematological response), is seen in no more than one third of patients, while the remaining continue to experience some degree of anemia (good and partial hematological responses), in some cases requiring regular red blood cell transfusions (minor hematological response). (frontiersin.org)
  • In approximately one third of patients with aplastic anemia, there is no response to immunosuppression. (medscape.com)
  • [ 5 ] In the presence of severe infection, however, it may be necessary to proceed directly to HCT to provide the patient with the best chance for early neutrophil recovery. (medscape.com)
  • Furthermore, some aplastic anemia that is genetically inherited may, first manifest in adulthood, sometimes without a family history of blood disease. (rarediseases.org)
  • This registry seeks to collect information from patients who have Diamond-Blackfan anemia (DBA), in order to help researchers and doctors learn more about the condition. (nih.gov)
  • Acquired aplastic and PNH have a close relationship that is not fully understood by researchers. (rarediseases.org)
  • In the 1990s, Young initiated and implemented a formal epidemiologic study of aplastic anemia in Thailand, which revealed a much higher incidence rate than in the West. (wikipedia.org)
  • Participants in this study must be 2 years or older and have severe aplastic anemia. (nih.gov)
  • This study is testing whether adding the medicine eltrombopag to standard treatments is a better way to treat severe aplastic anemia. (nih.gov)
  • Severe or very severe aplastic anemia is a hematologic emergency, and care should be instituted promptly. (medscape.com)
  • Patients with aplastic anemia should be treated by physicians who are experts in the care of immunocompromised patients and in consultation with a hematologist and/or an HCT physician. (medscape.com)
  • Therefore, the phase III POLARIX trial evaluated the efficacy and safety of a modified regimen of R-CHOP (pola-R-CHP), in which vincristine was replaced with polatuzumab vedotin, as compared with standard R-CHOP. (bjh.be)
  • Some people who have aplastic anemia have very short telomeres, which protect the ends of DNA in chromosomes. (nih.gov)