骨髓增生异常综合征诊治-英文课件.ppt

Myelodysplastic syndromes:A practical approach to diagnosis and treatment,Hong-ling Hao,M.D.Department of Hematology,Hebei General Hospital,Introduction,MDS are a heterogeneous group of disorders of blood cell production in the bone marrow that can transform into acute myeloid leukemia(AML).They are diagnosed most often in the elderly.The consequent cytopenias result in infections and bleeding complications.,The range of symptoms depends on the bone marrow cell type affected.They have symptoms stemming from anemia or thrombocytopenia,or have recurrent infections.,Subtypes of MDS have different pathologic and clinical presentations and different prognoses.They are often categorized as lower-risk or higher-risk,depending on the likelihood of transforming to AML.,Lower-risk MDS survive a median of 3 to 7 years.Higher-risk types are pathobiologically similar to AML in older adults,and patients either develop AML or die of complications of MDS,on average within 1.5 years.,Etiology,Genetic and environmental factors probably both play a role.Environmental factors:smoking;ionizing radiation;exposure to benzene,hair dyes,pesticides.Genetic factors:Down syndrome,Fanconi anemia,and Bloom syndrome are associated with MDS.MDS rarely run in families.,DiagnosisLaboratory tests,Complete blood cell count(CBC)Evaluation of anemia Tests for nutrient deficiencies Fecal occult blood testing Liver function tests Renal function tests,Thyroid function tests Reticulocyte count EPO level Screening for relevant infections:HIV,Hepatitis,Parvovirus B19 Review of drugs:MTX,Depakote,Luminal,Retrovir.,Evaluation of other cytopenias,Abdominal ultrasonography-for splenomegaly Coombs antiglobulin testsLDH levelAntinuclear antibody titerRheumatoid factor level,If tests are negative,Peripheral blood smearBone marrow aspiration and biopsySpecialized tests:1.Antiplatelet antibodies 2.Protein electrophoresis 3.Fluorescence in situ hybridization FISH Each has its prognostic and therapeutic implications!,Classification Systems for MDS,MDS have two main classification systems,the FAB and the WHO.The WHO classification is widely accepted because it incorporates morphologic and cytogenetic factors and correlates with prognosis.,FAB classification for MDS,WHO classification for MDS,接上一页,Scoring systems for MDS,These systems are based on:The number of myeloblasts in the bone marrow.(The higher the count,the worse the prognosis.)The number or degree of cytopenias.Cytogenetic abnormalities.,The most widely used prognostic systems:,IPSS:International Prognostic Scoring SystemWPSS:WHO Classification-based Prognostic Scoring System,Blood,Vol 89,No 6(March 15),1997:pp 2079-2088,Cancer Control 2008;15(suppl 4):413.,Treatment of MDS,1.Supportive care Includes transfusion of blood products to minimize complications of cytopenias and to improve quality of life,as well as antibiotics to treat active infections.,Red blood cells typically are given when the hemoglobin level falls below 8.5 g/dL.Platelets are given when the platelet count is below 10109/L,in the absence of symptoms.,Iron chelation:It is debatable?Blood product transfusions can lead to iron overload,particularly with a lifetime administration of more than 20 units.This intervention should be reserved for patients with lower-risk disease who are expected to survive more than 1 year.,Antibiotics:,The risk of infection is significantly increased when the neutrophil count is below 0.5109/L.Fever(temperature 38C)in neutropenic patients is an emergency,requiring immediate initiation of broad-spectrum antibiotics.Prophylactic antibiotics have no proven role in MDS patients with neutropenia.,2.Treatment of lower-risk disease,(1)Erythropoiesis-stimulating agents:Epo 100 IU/L and have minimal transfusion needs.Addition of a colony-stimulating factor can be considered for patients with neutropenia.Response is measured as an improvement in hemoglobin or as independence from transfusions.,(2)Other treatments:(If ESA treatment is ineffective),Immunosuppressive therapy:ATGLenalidomide for MDS with 5q-can decrease the need for blood transfusion in approximately 2/3 of these patients.Azacitidine or decitabineStem cell transplantationExperimental treatments:clinical trials,3.Treatment of higher-risk disease,About 25%of patients with newly diagnosed MDS and 15%to 20%of patients with established MDS have higher-risk disease.Regardless of blood counts,given the high likelihood of transformation to AML or death within 1.5 years.,The treatment options for higher-risk disease include:,Methyltransferase inhibitors such as azacitidine and decitabine.Cytotoxic chemotherapy(similar to treatment of acute myeloid leukemia).Bone marrow-hematopoietic stem cell transplantation(HSCT).Experimental treatments in clinical trials.,Stem cell transplantation cures MDS,Hematopoietic stem cell transplantation is the only curative treatment for MDS.However,it is performed in fewer than 5%of patients,because the rate of transplant-related death is high.The balance between risks and benefits of this treatments must be justifiable.,Take-home Points,MDS are complex and heterogeneous,so treatment options range from supportive care to chemotherapy and allogeneic stem cell transplantation.,Thank you for your attention!E-mail:MP:,On the Stork TowerWang Zhi-huan,The sun beyond the mountains glows,The Yellow River seawards flows.You can enjoy a grander sight,By climbing to a greater height.,