Aplastic anemia treatment options Grard Soci MD Ph

Aplastic anemia; treatment options Gérard Socié, MD Ph. D Hospital Saint Louis G. S / AIH 2006

Aplastic anemia; treatment options SAA Hypo cellular marrow (<30%) & at least 2/3 criteria: ANC <0. 5 x 109/L Platelets <20 x 109/L Reticulocytes <20 x 109/L v. SAA ANC <0. 2 x 109/L moderate AA not fulfilling criteria of SAA ANC >0. 5 x 109/L G. S / AIH 2006

Aplastic anemia; treatment options Hemophagocytosis by an immature myeloid cell Dysplastic changes in the few scattered hematopoietic cells MDS or SAA? Dysplastic changes in the myeloid series Fibrosis Splenomegaly Blasts Dysplastic megakaryocyte myeloid Megaloblastic proerythroblast, coarse chromatin, multiple nucleoli cytoplasmic budding Cytogenetics? PNH? G. S / AIH 2006

Aplastic anemia; treatment options Does this guideline hold true? G. S / AIH 2006

Aplastic anemia; treatment options HSCT HLA-id. Sibling D Ades et al. Blood. 2004; 103: 2490 -2497 G. S / AIH 2006

Aplastic anemia; treatment options Better outcome - early BMT - few transfusions - age - Gv. HD prophylaxis CSA+MTX 5 year Survival <1985: 51% + 4 1985 -94: 63% + 2 1995 -04: 69% + 2 n= 741 n=1384 n=2044 G. S / AIH 2006

Aplastic anemia; treatment options Cy - TAI Cy - ATG Time from transplant (year) G. S / AIH 2006

Aplastic anemia; treatment options G. S / AIH 2006

Aplastic anemia; treatment options Immunosuppression Time period N <1980 Survival 5 years 10 years 577 54± 4% 46± 4% 19801990 413 67± 5% 61± 5% 19902000 1114 73± 3% 69± 4% >2000 160 77± 8% - G. S / AIH 2006

Aplastic anemia; treatment options Immunosuppressive treatment Best results with ATG + CSA Bacigalupo A. Blood 2000; 95(6): 1931 -1934. G. S / AIH 2006

Aplastic anemia; treatment options Frickhofen N. Blood. 2003 (101). 1236 -1242 Socié G. NEJM. 1993; (329). 1152 -1157 Bacigalupo A, Blood 2000; (95). 1931 -1934 G. S / AIH 2006

Aplastic anemia; treatment options Socié G. et al. , Submitted 2006 G. S / AIH 2006

Aplastic anemia; treatment options UD transplant SFGM-TC > 1998 < 1998 G. S / AIH 2006

Aplastic anemia; treatment options SFGM-TC In multivariate analysis: survival -HLA allelic matching (P<. 01) - younger age of (<17 years, P<. 0001). Survival reached 78%± 11% at 5 -year for the younger patients HLA-matched. G. S / AIH 2006

Aplastic anemia; treatment options Identical sibling As early as possible Bone marrow Cy + ATG CSA +MTX MUD after failed IS 1 - 2 courses? Conditioning? Gv. HD Prophylaxis? G. S / AIH 2006

Aplastic anemia; treatment options BMT good results with Id Sibling BMT MUD BMT (2 nd line) Older (Age old? ? ) Alternative donors? Cord? IS good results with H-ATG + CSA G-CSF still need randomized trial no upper age limit refractory patients? other immunosuppressant? MMF ineffective Campath? Sirolimus? Anti-CD 25? Rituximab? G. S / AIH 2006