Patients are treated with a bone marrow transplant or medications that suppress the immune system. Despite the precision of its diagnostic criteria, aplastic anemia has always been a diagnosis of exclusion. Aplastic anemia current medical diagnosis and treatment. Guidelines for the treatment of severe aplastic anemia. Guidelines for the diagnosis and management of adult. The treatment often depends on how severe grade the aplastic anemia is. Severe or very severe aplastic anemia is a hematologic emergency, and care should be instituted promptly. Moderatedose cyclophosphamide for severe aplastic anemia has significant toxicity and does not prevent relapse and clonal evolution.
Blood and marrow stem cell transplants to replace damaged stem cells with health ones from a donor another person. An immune basis for most patients with aplastic anemia aa provides a rationale for immunosuppressive therapy ist, using antithmyocyte globulin and cyclosporine as. Aplastic anemia definition aplastic anemia was described by ehrlich in 1888 in a pregnant woman. Diagnosis and management of aplastic anemia american society. Eltrombopag, a thrombopoietin receptor agonist, was approved in august 2014 for severe aplastic anemia in patients who fail to respond adequately to at least one prior ist regimen. Aplastic anemia may appear at any age but is diagnosed more often in children and young adults. Choosing one of these therapies for a given patient depends not only on donor availability but also on such factors as patient age. Treatments for aplastic anemia, which will depend on the severity of your condition and your age, might include observation, blood transfusions, medications, or bone marrow transplantation. Acquired aplastic anemia is a potentially fatal bone marrow failure disorder that is characterized by pancytopenia and a hypocellular bone marrow. How i treat acquired aplastic anemia blood american society of. Both immunosuppressive therapy and bone marrow transplantation are accepted treatments for patients with aplastic anemia. Systemic lupus erythematosus may rarely cause suppression of the hematopoietic stem cell by an igg autoantibody directed against it. Highdose cyclophosphamide compared with antithymocyte globulin for treatment of acquired severe aplastic anemia. The condition leaves you fatigued and more prone to infections and uncontrolled bleeding.
Severe aplastic anemia is a syndrome characterized by bone marrow failure with peripheral cytopenia and a hypocellular bone marrow biopsy, without blasts or myelodysplasia. Blood transfusions to keep blood cell counts at acceptable levels. At least 2 of the following peripheral cytopenias must be present. Bone marrow is found in the soft, spongy center of most bones. Clinicians must stress the need for patient compliance with therapy. In patients with aplastic anemia that is refractory to immunosuppressive therapy ist, treatment with eltrombopag promacta may be considered. Treatment with high doses of chemotherapy or radiation will produce an aplastic appearance in the bone marrow as well as loss of cells in the blood. Acquired aplastic anemia nord national organization for. However, the most common pathogenesis of aplastic anemia appears to be autoimmune suppression of hematopoiesis by a tcellmediated cellular mechanism, so called idiopathic aplastic anemia. Working party on severe aplastic anemia wpsaa of the european group of bone marrow.
It can occur suddenly, or it can come on slowly and worsen over time. The disorder occurs in about two to six individuals per million population worldwide. Pdf aplastic anemia is a rare disorder characterized by suppression of bone marrow function resulting in progressive pancytopenia. Treatment aims to correct the bone marrow failure, as well as to treat the patients immediate signs and symptoms. In aplastic anemia aa, the bone marrow stops making enough red blood cells, white blood cells, and platelets. Pdf the diagnosis and treatment of aplastic anemia. Treatment of adult refractory severe aplastic anaemia. Guidelines for the diagnosis and management of adult aplastic. Severe aplastic anemia, in which your blood cell counts are extremely low, is lifethreatening and requires immediate hospitalization.
Treatment for aplastic anemia varies depending on the severity. Guidelines for the diagnosis and management of aplastic anaemia. No single test allows us to reliably diagnose idiopathic aplastic anemia, but the field has advanced considerably in terms of awareness of and diagnosis of other disorders resulting in a similar or indistinguishable hematologic phenotype. Guidelines for the diagnosis and management of aplastic. Aplastic anaemia aa is a quantitative bone marrow failure, secondary. Hematopoietic stemcell transplantation or bone marrow transplantation bmt is the treatment of choice for young patients who have a matched sibling donor. A rare and serious condition, aplastic anemia can develop at any age. Following treatment with campath, cd52 tcells are identified, which are. Aplastic anemia genetic and rare diseases information. Guidelines for the diagnosis and management of adult aplastic anaemia. What are the treatment options for aplastic anemia.
The grading of recommendations assessment, development and evaluation grade nomenclature was used to evaluate levels of evidence and to assess the strength of recommendations. Diagnosis and management of aplastic anemia hematology. Treatment of acquired aplastic anemia varies, depending upon the individuals age, general health, and the severity of aplastic anemia. While some individuals with mild to moderate aplastic anemia may not require treatment, for others, treatment may include. National diagnostic and treatment protocol aplastic anaemia. For newly diagnosed severe aplastic anemia, bone marrow. Understanding your disease and treatment options danielle townsley, md, msc associate director, oncology aztrazeneca medimmune approximate blood cell requirements cell type total number life span daily production days neutrophils 2 x 1010 1 2 x 1010.
Aplastic anemia diagnostics and therapy of acquired aplastic. Aplastic anemia is a condition that occurs when your body stops producing enough new blood cells. In aplastic anemia, the bone marrow shuts down and stops making new blood cells. An immune basis for most patients with aplastic anemia aa provides a rationale for immunosuppressive therapy ist, using antithmyocyte globulin and cyclosporine as one therapeutic modality. Healthy bone marrow makes young cells called stem cells. Aplastic anemia diagnostics and therapy of acquired aplastic anemia. Pdf an immune basis for most patients with aplastic anemia aa provides a rationale for immunosuppressive therapy ist, using. Party for severe aplastic anemia is running 2 prospective. Aplastic anemia diagnosis and treatment mayo clinic. The timing and duration of the bone marrow depression are predictable from the type of treatment given and the dose received. The stem cells, in turn, make the 3 major types of blood. Diagnosis and treatment of aplastic anemia ncbi nih.
1064 234 402 661 486 810 1090 480 1126 1384 1486 1066 1499 137 1051 1027 970 489 1326 112 1282 280 36 1107 107 1358 1472 521 1247