Gérard Socié, MD PhD Hospital Saint Louis

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

description

AIH. Gérard Socié, MD PhD Hospital Saint Louis. SAA Hypo cellular marrow (

Transcript of Gérard Socié, MD PhD Hospital Saint Louis

Page 1: Gérard Socié, MD PhD Hospital Saint Louis

G. S / AIH 2006

Aplastic anemia; treatment options

Gérard Socié, MD PhDHospital Saint Louis

Page 2: Gérard Socié, MD PhD Hospital 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

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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?

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G. S / AIH 2006

Aplastic anemia; treatment options

Does this guideline hold true?

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Aplastic anemia; treatment options

Ades et al. Blood. 2004;103: 2490-2497

HSCT HLA-id.

Sibling D

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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

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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)

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G. S / AIH 2006

Aplastic anemia; treatment options

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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

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G. S / AIH 2006

Aplastic anemia; treatment options

Bacigalupo A. Blood 2000; 95(6):1931-1934.

Immunosuppressive treatmentBest results withATG + CSA

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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

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Aplastic anemia; treatment options

Socié G. et al., Submitted 2006

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Aplastic anemia; treatment options

UD transplant

< 1998

> 1998

SFGM-TC

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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.

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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?

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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?