骨髓增生异常综合征诊治英文公开课课件

Introduction n nMDS are a heterogeneous group of MDS are a heterogeneous group of disorders of blood cell production in the disorders of blood cell production in the bone marrow that can transform into acute bone marrow that can transform into acute myeloid leukemia(AML).myeloid leukemia(AML).n nThey are diagnosed most often in the They are diagnosed most often in the elderly.elderly.n n The consequent cytopenias result in The consequent cytopenias result in infections and bleeding complications.infections and bleeding complications.Introduction MDS are a heterog1n nThe range of symptoms depends on the bone marrow cell type affected.They have symptoms stemming from anemia or thrombocytopenia,or have recurrent infections.The range of symptoms depends 2n nSubtypes of MDS have different pathologic and clinical presentations and different prognoses.n nThey are often categorized as lower-risk or higher-risk,depending on the likelihood of transforming to AML.Subtypes of MDS have different3n nLower-risk MDS survive a median of 3 to 7 years.n nHigher-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.Lower-risk MDS survive a media4Etiologyn nGenetic and environmental factors Genetic and environmental factors probably both play a role.probably both play a role.n nEnvironmental factors:Environmental factors:smoking;ionizing radiation;exposure to smoking;ionizing radiation;exposure to benzene,hair dyes,pesticides.benzene,hair dyes,pesticides.n nGenetic factors:Down syndrome,Fanconi Genetic factors:Down syndrome,Fanconi anemia,and Bloom syndrome are anemia,and Bloom syndrome are associated with MDS.associated with MDS.n nMDS rarely run in families.MDS rarely run in families.EtiologyGenetic and environmen5 DiagnosisLaboratory testsn nComplete blood cell count(CBC)n nEvaluation of anemia Tests for nutrient deficiencies Fecal occult blood testing Liver function tests Renal function tests Diagno6 Thyroid function tests Thyroid function tests Reticulocyte count Reticulocyte count EPO level EPO level Screening for relevant infections:Screening for relevant infections:HIV,Hepatitis,Parvovirus B19 HIV,Hepatitis,Parvovirus B19 Review of drugs:Review of drugs:MTX,Depakote,Luminal,Retrovir.MTX,Depakote,Luminal,Retrovir.Thyroid function test7骨髓增生异常综合征诊治英文公开课课件8Evaluation of other cytopeniasn nAbdominal ultrasonography -for splenomegaly n nCoombs antiglobulin testsn nLDH leveln nAntinuclear antibody titern nRheumatoid factor level Evaluation of other cytopenias9If tests are negativen nPeripheral blood smearn nBone marrow aspiration and biopsyn nSpecialized tests:1.Antiplatelet antibodies 2.Protein electrophoresis 3.Fluorescence in situ hybridization FISH Each has its prognostic and therapeutic implicationsEach has its prognostic and therapeutic implications!If tests are negativePeriphera10Classification Systems for MDSn nMDS have two main classification systems,the FAB and the WHO.n nThe WHO classification is widely accepted because it incorporates morphologic and cytogenetic factors and correlates with prognosis.Classification Systems for MDS11FAB classification for MDSFAB classification for MDSFAB classification for MDS12WHO classification for MDSWHO classification for MDS13接上一页接上一页接上一页接上一页接上一页14Scoring systems for MDSThese systems are based on:n nThe number of myeloblasts in the bone The number of myeloblasts in the bone marrow.marrow.(The higher the count,the worse the prognosis.(The higher the count,the worse the prognosis.)n nThe number or degree of cytopenias.The number or degree of cytopenias.n nCytogenetic abnormalities.Cytogenetic abnormalities.Scoring systems for MDSThese s15The most widely used prognostic systems:n nIPSS:IPSS:I International nternational P Prognostic rognostic S Scoring coring S Systemystemn nWPSS:WPSS:WWHO Classification-based HO Classification-based P Prognosticrognostic S Scoring coring S System ystem The most widely used prognosti16Complete blood cell count(CBC)Review of drugs:Addition of a colony-stimulating factor can be considered for patients with neutropenia.MDS are complex and heterogeneous,so treatment options range from supportive care to chemotherapy and allogeneic stem cell transplantation.Hematopoietic stem cell transplantation is the only curative treatment for MDS.Antiplatelet antibodiesEpo Fever(temperature 3838 C C)in neutropenic)in neutropenic patients is an emergency,requiring immediate patients is an emergency,requiring immediate initiation of initiation of broad-spectrum antibioticsbroad-spectrum antibiotics.n nProphylactic antibiotics have no proven role in Prophylactic antibiotics have no proven role in MDS patients with neutropenia.MDS patients with neutropenia.Antibiotics:The risk of infec222.Treatment of lower-risk disease(1)(1)Erythropoiesis-stimulating agents:Erythropoiesis-stimulating agents:n nEpo Epo 100 IU/L100 IU/L and have minimal transfusion and have minimal transfusion needs.needs.n nAddition of a colony-stimulating factor can be Addition of a colony-stimulating factor can be considered for patients with neutropenia.considered for patients with neutropenia.n nResponse is measured as an improvement in Response is measured as an improvement in hemoglobin or as independence from hemoglobin or as independence from transfusions.transfusions.2.Treatment of lower-risk di23(2)Other treatments:(2)Other treatments:(If ESA treatment is ineffectiveIf ESA treatment is ineffective)n nImmunosuppressive therapy:Immunosuppressive therapy:ATGATGn nLenalidomideLenalidomide for MDS with for MDS with 5q-5q-can decrease the can decrease the need for blood transfusion in approximately 2/3 need for blood transfusion in approximately 2/3 of these patients.of these patients.n nAzacitidine or decitabineAzacitidine or decitabinen nStem cell transplantationStem cell transplantationn nExperimental treatments:Experimental treatments:clinical trialsclinical trials(2)Other treatments:(If ESA 243.Treatment of higher-risk disease n nAbout About 25%25%of patients with newly diagnosed of patients with newly diagnosed MDS and MDS and 15%to 20%15%to 20%of patients with of patients with established MDS have higher-risk disease.established MDS have higher-risk disease.n nRegardless of blood counts,given the high Regardless of blood counts,given the high likelihood of transformation to AML or death likelihood of transformation to AML or death within within 1.51.5 years.years.3.Treatment of higher-risk di25The treatment options for higher-risk disease The treatment options for higher-risk disease include:include:n nMethyltransferase inhibitorsMethyltransferase inhibitors such as azacitidine such as azacitidine and decitabine.and decitabine.n nCytotoxic chemotherapyCytotoxic chemotherapy(similar to treatment of (similar to treatment of acute myeloid leukemia).acute myeloid leukemia).n nBone marrow-hematopoietic stem cell Bone marrow-hematopoietic stem cell transplantation(transplantation(HSCTHSCT).).n nExperimental treatments in Experimental treatments in clinical trialsclinical trials.The treatment options for high26MDS are a heterogeneous group of disorders of blood cell production in the bone marrow that can transform into acute myeloid leukemia(AML).Stem cell transplantation cures MDSMDS have two main classification systems,the FAB and the WHO.However,it is performed in fewer than 5%of patients,because the rate of transplant-related death is high.HIV,Hepatitis,Parvovirus B19Coombs antiglobulin testsComplete blood cell count(CBC)The risk of infection is significantly increased when the neutrophil count is below 0.Abdominal ultrasonographyEpo 100 IU/L and have minimal transfusion needs.Regardless of blood counts,given the high likelihood of transformation to AML or death within 1.On the Stork TowerWang Zhi-huanThey are diagnosed most often in the elderly.Reticulocyte count(The higher the count,the worse the prognosis.Coombs antiglobulin testsRheumatoid factor levelEnvironmental factors:Antinuclear antibody titerCytotoxic chemotherapy(similar to treatment of acute myeloid leukemia).Stem cell transplantation cures MDSStem cell transplantation cures MDSn nHematopoietic stem cell transplantation is the Hematopoietic stem cell transplantation is the only curative treatment for MDS.only curative treatment for MDS.n nHowever,it is performed in fewer than However,it is performed in fewer than 5%5%of of patients,because the rate of transplant-related patients,because the rate of transplant-related death is high.death is high.n nThe balance between The balance between risksrisks and and benefitsbenefits of this of this treatments must be justifiable.treatments must be justifiable.MDS are a heterogeneous group 27Take-home Pointsn nMDS are complex and heterogeneous,so treatment options range from supportive care to chemotherapy and allogeneic stem cell transplantation.Take-home PointsMDS are comple28Thank you for your attention!E-mail:Thank you for your attention!29On the Stork TowerWang Zhi-huanWang Zhi-huan The sun beyond the mountains glows,The sun beyond the mountains glows,The Yellow River seawards flows.The Yellow River seawards flows.You can enjoy a grander sight,You can enjoy a grander sight,By climbing to a greater height.By climbing to a greater height.On the Stork TowerWang Zhi-hu30