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Anemia, AplasticAntilymphocyte SerumImmunosuppressionAnemiaBone Marrow TransplantationImmunosuppressive AgentsCyclosporineOxymetholoneTransplantation, HomologousGraft vs Host DiseaseTransplantation ConditioningHemoglobinuria, ParoxysmalHistocompatibility TestingHistocompatibilityTissue DonorsGraft vs Host ReactionGraft RejectionBlood TransfusionHematopoietic Stem Cell TransplantationCyclophosphamideHLA AntigensGraft SurvivalTreatment OutcomeBone MarrowFanconi AnemiaMyelodysplastic SyndromesPancytopeniaMethylprednisoloneDrug Therapy, CombinationHorsesSurvival AnalysisCord Blood Stem Cell TransplantationGranulocyte Colony-Stimulating FactorAnemia, HemolyticInfectionHematopoiesisLeukemiaFollow-Up StudiesSiblingsRetrospective StudiesVidarabineTime FactorsSurvival RateTransplantation ChimeraLiving DonorsRecurrenceAnemia, Hemolytic, AutoimmuneAnemia, RefractoryImmune ToleranceAnemia, HypochromicAnemia, MegaloblasticTacrolimusAnemia, MacrocyticAnemia, PerniciousKidney TransplantationFluoxymesteroneAnemia, Sickle CellBlood Cell Count

CyclosporineAntithymocyte globulinParoxysmal nocturnal hemoThrombocytopeniaIdiopathic aplasticRefractoryThrombopoietin-receptor agonist eltrombopagHemolytic AnemiaEltrombopagPediatric patientsPlateletsBoneStandard immunosuppressiveMyelodysplastic syndromesDiagnosisVSAAImmuneTreatmentClinicalTransplantationPancytopeniaDisorderHSCTWeaknessSymptomsHematologicalEfficacyTherapeuticConclusionsFanconiTelomereIncidenceInfectionsAbsentRejectionCongenitalOutcomesAcuteSignsErythropoiesisPatient educationBloodCytopeniasAdultsResponse

Cyclosporine7

• Treatment usually involves immunosuppression with equine antithymocyte globulin and cyclosporine , or bone marrow transplantation. (merckmanuals.com)
• Background A combination of horse anti-thymocyte globulin and cyclosporine produces responses in 60-70% of patients with severe aplastic anemia. (haematologica.org)
• We performed a phase II study of rabbit anti-thymocyte globulin and cyclosporine as first-line therapy for severe aplastic anemia. (haematologica.org)
• Are you or your child taking cyclosporine for aplastic anemia? (nih.gov)
• This study is testing whether the medicine sirolimus helps prevent the return of aplastic anemia after stopping cyclosporine. (nih.gov)
• The standards of care for aplastic anemia are allogeneic hematopoietic stem cell transplantation (HSCT) or immunosuppression with horse antithymocyte globulin and cyclosporine. (thalassaemia.org.cy)
• Only two-thirds of patients with acquired aplastic anaemia respond to standard immunosuppressive treatment with horse antithymocyte globulin plus cyclosporine. (bjh.be)

Antithymocyte globulin2

• He directed the first multicenter clinical trial using antithymocyte globulin for the treatment of aplastic anemia in the United States. (wikipedia.org)
• Inpatient care for patients with aplastic anemia may be needed during periods of infection and for specific therapies, such as antithymocyte globulin (ATG) or HCT. (medscape.com)

Paroxysmal nocturnal hemo2

• Some individuals with acquired aplastic anemia also have another disorder at the same time, called paroxysmal nocturnal hemoglobinuria (PNH). (rarediseases.org)
• Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematological disorder characterized by complement-mediated intravascular hemolysis, bone marrow failure, and severe thrombophilia ( 1 ). (frontiersin.org)

Thrombocytopenia6

• Symptoms are dependent on the severity of the anemia, leukopenia, and thrombocytopenia. (rarediseases.org)
• 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)
• This Phase 2, non-randomized pilot study of eltrombopag in aplastic anemia patients with immunosuppressive therapy refractory thrombocytopenia will test the safety and potential efficacy of eltrombopag treatment patients with refractory thrombocytopenia following immunosuppression for aplastic anemia. (drugpatentwatch.com)
• The primary objective is to assess the safety and efficacy of the oral thrombopoietin receptor agonist (TPO-R agonist) eltrombopag in aplastic anemia patients with immunosuppressive-therapy refractory thrombocytopenia. (drugpatentwatch.com)
• Consider discontinuing therapy if severe and unremitting thrombocytopenia or neutropenia occurs. (pfizermedicalinformation.com)
• Concurrent anemia, thrombocytopenia, and/or an abnormal result on a peripheral blood smear from a patient with neutropenia suggest an underlying hematologic disorder. (medscape.com)

Idiopathic aplastic2

• In this issue of Blood , 2 papers deal with HLA in patients with acquired idiopathic aplastic anemia (AA). (ashpublications.org)
• Nonetheless, the availability of such "genetic testing" has revealed increasing numbers of individuals who by clinical criteria appear to have idiopathic aplastic anemia (AA) and appear phenotypically normal yet have molecular hallmarks of congenital marrow failure syndromes. (ashpublications.org)

Refractory3

• While hATG has been the primary treatment used for severe AA, patients with relapsing or refractory AA often receive rATG after a first course of hATG. (haematologica.org)
• Eltrombopag and improved hematopoiesis in refractory aplastic anemia [published correction appears in N Engl J Med. (drugs.com)
• Haploidentical bone marrow transplantation in patients with relapsed or refractory severe aplastic anaemia in the USA (BMT CTN 1502): a multicentre, single-arm, phase 2 trial. (wustl.edu)

Thrombopoietin-receptor agonist eltrombopag1

• 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)

Hemolytic Anemia1

• The primary observed serious adverse side effect of ribavirin treatment is hemolytic anemia. (biomedcentral.com)

Eltrombopag4

• Eltrombopag Added to Immunosuppression in 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)
• 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)
• Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia. (drugs.com)

Pediatric patients1

• 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)

Platelets2

• 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)

Bone14

• Aplastic anemia is a syndrome of bone marrow failure characterized by peripheral pancytopenia and marrow hypoplasia (see the image below). (medscape.com)
• 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)
• Paul Ehrlich introduced the concept of aplastic anemia in 1888 when he reported the case of a pregnant woman who died of bone marrow failure. (medscape.com)
• The term aplastic anemia commonly implies a panhypoplasia of the bone marrow with cytopenias in at least two hematopoietic lineages. (merckmanuals.com)
• Aplastic anemia (AA) is a hematopoietic stem cell disorder characterized by pancytopenia and hypocellular bone marrow. (haematologica.org)
• Acquired aplastic anemia is a rare, serious blood disorder, due to failure of the bone marrow failure to produce blood cells. (rarediseases.org)
• Although bone marrow failure can occur secondary to other disorders, most aplastic anemia is due to the immune system mistakenly targeting the bone marrow (autoimmunity). (rarediseases.org)
• The symptoms of acquired aplastic anemia occur as a consequence of the bone marrow failing to produce enough blood cells. (rarediseases.org)
• It is believed that PNH arises in the setting of autoimmune acquired aplastic anemia and bone marrow failure. (rarediseases.org)
• Different factors contribute to residual anemia during eculizumab treatment: underlying bone marrow dysfunction, residual intravascular hemolysis and the emergence of C3-mediated extravascular hemolysis. (frontiersin.org)
• Aplastic anemia (moderate to severe) in patients unsuitable for bone marrow transplantation. (pfizermedicalinformation.com)
• The usefulness of ATGAM has not been demonstrated in patients with aplastic anemia who are suitable candidates for bone marrow transplantation or in patients with aplastic anemia secondary to neoplastic disease, storage disease, myelofibrosis, Fanconi's syndrome, or in patients known to have been exposed to myelotoxic agents or radiation. (pfizermedicalinformation.com)
• Hepatitis-associated aplastic anemia is a common syndrome in patients with bone marrow failure. (biomedcentral.com)
• Furthermore, due to its inhibition of cellular growth, interference with oncogene expression and augmentation of lymphocyte cytotoxicity for target cells, IFN-α may cause bone marrow suppression, including potentially severe cytopenias and, very rarely, AA [ 11 ]. (biomedcentral.com)

Standard immunosuppressive1

• In combination with standard immunosuppressive therapy for the first-line treatment of severe aplastic anemia. (drugs.com)

Myelodysplastic syndromes1

• Among the conditions HSCT can treat are: acute myeloid leukemia, acute lymphoblastic leukemia, chronic myeloid leukemia, chronic lymphocytic leukemia, myeloproliferative disorders, myelodysplastic syndromes, multiple myeloma, non-Hodgkin lymphoma, Hodgkin disease, aplastic anemia and pure red-cell aplasia-but this list is not exhaustive. (drugdiscoverynews.com)

Diagnosis6

• [ 6 ] The Pediatric Haemato-Oncology Italian Association has issued guidelines on diagnosis and management of acquired aplastic anemia in childhood. (medscape.com)
• Optimal management of aplastic anemia (AA) is not confined to immediate diagnosis, early decision making and timely initiation of major treatment strategies (immunosuppression or SCT) but also involves supportive treatment as a crucial part of patient care. (aamds.org)
• Patients with aplastic anemia require transfusion support until the diagnosis is established and specific therapy can be instituted. (medscape.com)
• It is characterized by pancy- lowing a diagnosis of aplastic anaemia. (who.int)
• Centre from the West Bank of Jordan with ed in the literature and most of them had the diagnosis of aplastic anaemia at 25 unsuccessful pregnancies [ 4 , 5 ]. (who.int)
• This was con- closporin 300 mg/day and was discharged sistent with the diagnosis of aplastic home on the 4th day after delivery. (who.int)

VSAA1

• 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)

Immune4

• 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)
• However, hepatitis-associated aplastic anemia is an immune-mediated disease that does not appear to be caused by any of the known hepatitis viruses including hepatitis C virus. (biomedcentral.com)
• The 3 groups are people who have 1) medical conditions without significant immunologic compromise, 2) medical conditions and treatments associated with limited immune deficits, and 3) medical conditions or treatments associated with severe immune compromise. (unboundmedicine.com)
• In general, the more severe and prolonged the immune defect, the greater the range of possible causative pathogens and the less typical the clinical presentation for a particular pathogen, prompting the need for early invasive investigation if the initial therapy is failing to effect improvement. (thoracickey.com)

Treatment10

• 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)
• Conclusions Despite reports suggesting differences in biological activity of different anti-thymocyte globulin preparations, rabbit and horse anti-thymocyte globulin appear to have a similar efficacy for up-front treatment of severe aplastic anemia. (haematologica.org)
• 2 , 3 When used as a first-line treatment for severe AA, 60-70% of patients have a hematologic response. (haematologica.org)
• On the other hand, the ongoing immunosuppression may have to be adapted to the perioperative requirements in terms of the active substance and the route of administration, the resulting increased risk of infection and possible side effects (e.g., myelosuppression, nephrotoxicity and impairment of wound healing) must be included in the perioperative treatment concept. (bvsalud.org)
• The PAS was supported by positive topline data from the REFLECTIONS B538-12 study which evaluated multiple switches between treatment with ABRILADA and its reference product, Humira, both of which were administered with methotrexate in adult patients with moderate to severe rheumatoid arthritis (RA). (pfizer.com)
• Treatment of chronic severe plaque psoriasis in children and adolescents from the age of 6 years who are inadequately controlled by, or are intolerant to, other systemic therapies or phototherapies. (medicines.org.uk)
• In addition, to the best of our knowledge there are no reported cases of patients with chronic hepatitis C virus infection developing aplastic anemia associated with pegylated interferon alpha 2a treatment. (biomedcentral.com)
• We report the case of a 46-year-old Greek man who developed severe aplastic anemia during treatment with pegylated interferon alpha 2a for chronic hepatitis C virus infection. (biomedcentral.com)
• The combination of a specific environmental precipitant represented by the hepatitis C virus infection, an altered metabolic detoxification pathway due to treatment with pegylated interferon alpha 2a and a facilitating genetic background such as polymorphism in metabolic detoxification pathways and specific human leukocyte antigen genes possibly conspired synergistically in the development of aplastic anemia in this patient. (biomedcentral.com)
• ENBREL is indicated for the treatment of patients 4 years or older with chronic moderate to severe plaque psoriasis who are candidates for systemic therapy or phototherapy. (enbrelpro.com)

Clinical4

• 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)
• Design and Methods Twenty patients with severe aplastic anemia treated with rabbit anti-thymocyte globulin were compared to 67 historical control cases with matched clinical characteristics treated with horse anti-thymocyte globulin. (haematologica.org)
• Clinical response was 63% (n=215) for CIMZIA patients with moderate-to-severe CD vs. 36% (n=210) for placebo patients, based on a 26-week study in which all patients received CIMZIA 400 mg at 0, 2, and 4 weeks, and Week 6 responders were randomized to CIMZIA 400 mg or placebo every 4 weeks. (cimziahcp.com)
• Clinical and laboratory observations suggest that acquired aplastic anemia is an autoimmune disease. (medscape.com)

Transplantation3

• Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation. (nih.gov)
• 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)
• Severe aplastic anemia (SAA) is a life-threatening blood disease which can be effectively treated with immunosuppressive drug regimens or allogeneic stem cell transplantation. (drugpatentwatch.com)

Pancytopenia2

• Aplastic anemia is suspected in patients, particularly young patients, with pancytopenia. (merckmanuals.com)
• Aplastic anemia is a term describing the common findings of pancytopenia and marrow hypoplasia arising from a variety of disease states, including acquired aplastic anemia and a variety of congenital marrow failure states. (ashpublications.org)

Disorder4

• 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)

HSCT1

• However, for several reasons an increasing number of non-malignant diseases (such as primary immunodeficiencies, aplastic anemia, metabolic diseases) can be cured with HSCT. (swisshealthmagazine.ch)

Weakness1

• Aplastic anaemia may develop during was of general weakness, loss of appetite pregnancy and sometimes improve sponta- and easy bruising at 16 weeks gestation. (who.int)

Symptoms1

• Symptoms of aplastic anemia are those of anemia, bleeding, and infection. (rarediseases.org)

Hematological2

• 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)
• The remainder consists of patients with hematological diseases (such as aplastic anemias or red blood cell disorders (e.g. sickle cell disease)) and primary immunodeficiencies (in particular, CGD). (swisshealthmagazine.ch)

Efficacy1

• 13 Only limited efficacy data are available for rATG used as initial therapy in severe AA. (haematologica.org)

Therapeutic1

• 1 consequently, therapeutic immunosuppression has been effective in a large proportion of patients. (haematologica.org)

Conclusions1

• CONCLUSIONS: Vigilant cancer and immunosuppression surveillance is warranted in OHT recipients at late-term follow-up. (bvsalud.org)

Fanconi1

• Diamond-Blackfan or Fanconi anaemia ) and acquired forms, depending on the age at which it occurs. (aa-pnh.org)

Telomere1

• 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)

Incidence2

• 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)
• The incidence of aplastic anaemia in Central Europe is 2-3 new cases per million people per year. (aa-pnh.org)

Infections1

• Generally, they are only diagnosed after one or more episodes of severe infections. (swisshealthmagazine.ch)

Absent1

• Cell production may be reduced during the course of the disease ( hypoplastic ) or completely absent ( aplastic ). (aa-pnh.org)

Rejection1

• While immunosuppression has been brought about in the past primarily to prevent rejection of transplanted organs, new applications involving mediation of the effects of INTERLEUKINS and other CYTOKINES are emerging. (lookformedical.com)

Congenital1

• Aplastic anaemia can be divided into congenital forms (e.g. (aa-pnh.org)

Outcomes1

• Providers also should assess whether health hazards at the destination could exacerbate any underlying conditions or cause more severe health outcomes in an immunocompromised traveler, and determine whether specific interventions are available to mitigate these risks. (cdc.gov)

Acute2

• The onset of aplastic anemia usually is insidious, often occurring over weeks or months after exposure to a virus, medication or toxin (eg, insecticides, benzene), though occasionally it can be acute. (merckmanuals.com)
• The attacks of colic may become more severe and may be associated with vomiting (see abdomen, acute). (zlibrary-global.se)

Signs1

• 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)

Erythropoiesis2

• 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)

Patient education1

• For patient education information, see What Is Aplastic Anemia? . (medscape.com)

Blood4

• Aplastic anemia is classified as severe according to blood counts. (rarediseases.org)
• Furthermore, some aplastic anemia that is genetically inherited may, first manifest in adulthood, sometimes without a family history of blood disease. (rarediseases.org)
• A low level of circulating red blood cells is called anemia. (rarediseases.org)
• Frequent outpatient follow-up for patients with aplastic anemia is needed to monitor blood counts and any adverse effects of various drugs. (medscape.com)

Cytopenias1

• However, 20-40% of patients without transplant options do not respond to immunosuppressive therapies, and have persistent severe cytopenias, requiring regular platelet transfusions, which are expensive and inconvenient, and are a risk for further serious bleeding complications. (drugpatentwatch.com)

Adults1

• True aplastic anemia (most common in adolescents and young adults) is idiopathic in about half of cases. (merckmanuals.com)

Response3

• To treat patients with severe aplastic anemia who have had insufficient response to immunosuppressive therapy. (drugs.com)
• In approximately one third of patients with aplastic anemia, there is no response to immunosuppression. (medscape.com)
• Constitutional aplastic anemia in response to hyper-tonicity, yet water retention clonazepam y alprazolam juntos sodium and water, atrial natriuretic factor, and interferon-gamma promote the table below provides catch-up schedules and other neural tumors, as well as in single-valve disease. (albionfoundation.org)