G. S / AIH 2006
Aplastic anemia; treatment options
Gérard Socié, MD PhDHospital Saint Louis
G. S / AIH 2006
Aplastic anemia; treatment options
SAAHypo cellular marrow (<30%) &
at least 2/3 criteria:
ANC <0.5x109/L
Platelets <20x109/L
Reticulocytes <20x109/L
v.SAAANC <0.2x109/L
moderate AAnot fulfilling criteria of SAA
ANC >0.5x109/L
G. S / AIH 2006
Aplastic anemia; treatment options
Megaloblastic proerythroblast, coarse chromatin, multiple
nucleoli cytoplasmic buddingDysplastic changes in the myeloid series
Dysplastic changes in the few scattered hematopoietic cells
Hemophagocytosisby an immature myeloid cell
MDS or SAA?
FibrosisSplenomegalyBlastsDysplastic megakaryocyte myeloid
Cytogenetics?PNH?
G. S / AIH 2006
Aplastic anemia; treatment options
Does this guideline hold true?
G. S / AIH 2006
Aplastic anemia; treatment options
Ades et al. Blood. 2004;103: 2490-2497
HSCT HLA-id.
Sibling D
G. S / AIH 2006
Aplastic anemia; treatment options
0 1 2 3 4 5 6 7 8 9 10
0.0
0.2
0.4
0.6
0.8
1.0
5 year Survival<1985: 51% + 4 n= 7411985-94: 63% + 2 n=13841995-04: 69% + 2 n=2044
Better outcome - early BMT- few transfusions- age- GvHD prophylaxis
CSA+MTX
G. S / AIH 2006
Aplastic anemia; treatment options
0.0
0.2
0.4
0.6
0.8
1.0
0 5 10 15 20
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
10 years
5 years
-77±8%160>2000
69±4%73±3%11141990-2000
61±5%67±5%4131980-1990
46±4%54±4%577<1980
SurvivalNTime period
G. S / AIH 2006
Aplastic anemia; treatment options
Bacigalupo A. Blood 2000; 95(6):1931-1934.
Immunosuppressive treatmentBest results withATG + CSA
G. S / AIH 2006
Aplastic anemia; treatment options
Bacigalupo A, Blood 2000; (95). 1931-1934
Frickhofen N. Blood. 2003 (101). 1236-1242Socié G. NEJM. 1993; (329). 1152-1157
G. S / AIH 2006
Aplastic anemia; treatment options
Socié G. et al., Submitted 2006
G. S / AIH 2006
Aplastic anemia; treatment options
UD transplant
< 1998
> 1998
SFGM-TC
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 possibleBone marrowCy + ATGCSA +MTX
MUD
after failed IS1 - 2 courses?Conditioning?GvHD Prophylaxis?
G. S / AIH 2006
Aplastic anemia; treatment options
BMT
good results withId Sibling BMTMUD BMT (2nd line)
Older (Age old??)Alternative donors?Cord?
IS
good results withH-ATG + CSAG-CSF still need randomized trialno upper age limit
refractory patients?other immunosuppressant?
MMF ineffectiveCampath? Sirolimus?Anti-CD25? Rituximab?
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