OUTCOME AFTER IMMUNOSUPPRESSION
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1 OUTCOME AFTER IMMUNOSUPPRESSION EBMT Aplastic Anemia Educational Meeting Budapest November 1 st 2012 Antonio M. Risitano, M.D., Ph.D. Head of Bone Marrow Transplant Unit Hematology, DBBM Federico II University of Naples OUTCOME OF IMMUNOSUPPRESSION FOR SAA Improvement over the years EBMT Database N= Survival improved with years, mostly due to: Better supportive therapy Better salvage treatment (SCT) Courtesy of Jakob Passweg 1
2 2003 n=112 hatg x 4 (40mg/kg) + CsA x 6 m OS OR 3m, 6m, 1y 3m survivors 3m survivors Hematological response is the main predictor for outcome CID 2011 n=420 (174 non-responders) Infection-related mortality from 37% to 11% Incidence of IFIs from 49% to 8% Group 1: 12/ /1986 Group 2: 11/ /2002 Group 3: 11/ /2008 The most relevant breakthrough in AA treatment was the anti-infectious supportive care: keeping AA patients alive until they recover (IST or SCT) 2
3 REASONS FOR BAD OUTCOME IN SAA Primary failures Refractoriness (about a third: predicting factors and early identification) Partial responses Secondary failures CyA-dependent responses Relapses Recurrent diseases Late failures Clonal evolution Secondary malignancies Many AA patients are not cured by IST!!! PRIMARY FAILURES 3
4 Factors predicting survival after IST for SAA Disease characteristics Very severe Severe Significant: age interval between dx and tx IST other than ATG+CyA Multivariate analysis Not significant: Disease severity Year of tx Use of G-CSF TAPERING/WITHDRAWING CyA ATG+CyA+G-CSF Scheinberg et al, BJH
5 REASONS FOR TREATMENT FAILURE Pathophysiology other than immune-mediated Irreversible stem cell deficit Insufficient immunosuppression REFRACTORY PATIENTS Is the diagnosis correct? Hypo-MDS? Constitutional AA? Empty Bone Marrow Dyskeratosis congenita: clinical overlap with AA, and shared pathophysiology Hyper/hypo-pigmentation Nail dystrophy Leukoplakia 5
6 TELOMERASE REPAIR COMPLEX GENE MUTATION AND BONE MARROW FAILURE Calado and Young, Blood 2008 REASONS FOR TREATMENT FAILURE Pathophysiology other than immune-mediated Irreversible stem cell deficit Insufficient immunosuppression Don t forget the old androgens! 6
7 REASONS FOR TREATMENT FAILURE Pathophysiology other than immune-mediated Irreversible stem cell deficit Insufficient immunosuppression Eltrombopag??? 7
8 ELTROMBOPAG IN SAA Preliminary data in refractory patients Phase II study n=25 Refractory SAA Eltrombopag mg, orally, for 12 weeks 44% hematological response (at least 1 lineage) Plt response 36% Hb response 24% ANC response 36% Increased marrow cellularity (resp.) Minimal toxicity, no fibrosis REASONS FOR TREATMENT FAILURE Pathophysiology other than immune-mediated Irreversible stem cell deficit Insufficient immunosuppression Improve immunosuppressive therapies 8
9 SECONDARY FAILURES 2003 n=112 hatg x 4 (40mg/kg) + CsA x 6 m Relapse 35% at 5y Relapse is the main cause of treatment failure, affecting between a third and half of responding patients 9
10 RELAPSES AFTER IST The role of maintenance CyA therapy Maintenance CyA is required to sustain blood counts after initial response to IST Frickhofen N. Blood (101) TAPERING/WITHDRAWING CyA Children Relapse CSA Tapering 10
11 What to do with refractory/relapsed AA patients? SECOND LINE TREATMENT FOR SAA PATIENTS Second ATG Pts with relapse / non-response CR or PR 27/43 (63%) survival 52% at 10 yrs Relapse of SAA is re-treatable by further IST Tichelli A. Br J Haematol (100)
12 Scheinberg Br J Haematol Refractory: 30% response Relapsed: 65% response Retreatment by ratg is more effective in relapsed than in refractory patients OS not affected due to salvage therapy STRATEGIES OF IMMUNOSUPPRESSION Autoantigen HLA TCR APC Naive T-cell Cell-cell contact Cytokine release (IL-1, IL-6) APC (HLA+Ag) triggering IL-2 release T-cell activation IL-2 signaling Activated T-cell IL-12/IL-23 ABT-874 Steroids Anergy inductors CTLA4-Ig Anti-CD154 Anti-cytokines Haematopoietic niche Haematopoietic stem cells Marrow damage TNF-α Etanercept Infliximab Adalimumab IFN-γ Fontolizumab? Inflammation CNIs CyA FK506 Selective Rituximab Visilizumab Zanolimumab Apolizumab Alefacept Efalizumab TNF-α IL-2 R blockers Daclizumab Basiliximab Anti-lymphocyte agents Biologicals Cytostatics IFN-γ Broad ATGs Alemtuzumab Perforine/ granzyme Fas-L Inhibitory Cell-cell cytokines contact Effector mechanisms MToR inhibitors Sirolimus Everolimus CTX MMF MTX AZA Second signals (IL-2, IL-12, IL-23, IL-4, IL-6, IL-17) T-cell differentiation Effector T-cell Proliferation T-cell expansion Expanded effector T-cell pool 12
13 Improving IST: the issue of different ATG preparations NEJM 2011 Phase III prospective randomized study, first-line treatment hatg + CyA (n=60) vs ratg + CyA (n=60) 6m 68% vs 37% (p<0.001) ratg is inferior to hatg in first line treatment of SAA, as indicated by hematological response and survival 13
14 NEJM 2011 ratg leads to more profound and prolonged CD4 depletion, especially of the Treg compartment Blood 2012 Phase II pilot study ratg + CyA (n=35) Retrospective matched comparison (pair-matched) with hatg + CyA (n=105) Pilot ratg + CyA study: OR 6m (CR 3%, PR 37%) ratg is inferior to hatg in first line treatment of SAA, as indicated by hematological response and survival 14
15 Improving IST: intensification by a third drug n=104 (38% vsaa) hatg+csa+mmf Overall response 3m 56% (14CR + 43PR) Overall response 6m 62% (16CR + 48PR) Overall survival Relapse 15
16 Sirolimus (Rapamune ) Improving IST: alternative regimens (non h-atg-based) 16
17 CYCLOPHOSPHAMIDE FOR TREATMENT OF SAA The Johns Hopkins experience N=67 (44 naive, 23 refractory); 50 mg/kg/day for 4 days OR 71% in naive, 48% in refractory patients OS and FFS 88% and 58% in naive patients, 62% and 27% in refractory patients Overall Survival Failure Free Survival n=44 n=23 n=44 n=23 TFR CI of fungal infections: 21% (naive) and 39% (refractory) CR Slower but more robust and durable responses No clonal evolution Blood 2012? 17
18 Risitano et al, 2010 Phase II prospective study with s.c. alemtuzumab ( mg in 5 days) N=28 (AA=13, PRCA=13, PWCA=2) 1,0 0,9 0,8 Cumulative proportion of survival 0,7 0,6 0,5 0,4 0,3 0,2 0,1 0, Months from treatment s.c. alemtuzumab is feasible and safe (no increased infectious morbidity) Remarkably effective, especially in single lineage marrow failures Frequent relapses (maintenance IS or retreatment needed) Late failures due to refractory relapses (15%) or clonal evolution (15%) Risitano et al, 2010 Phase II prospective study with s.c. alemtuzumab ( mg in 5 days) N=28 (AA=13, PRCA=13, PWCA=2) 1,0 0,9 0,8 Cumulative proportion of survival 0,7 0,6 0,5 0,4 0,3 0,2 0,1 0, Months from treatment s.c. alemtuzumab is feasible and safe (no increased infectious morbidity) Remarkably effective, especially in single lineage marrow failures Frequent relapses (maintenance IS or retreatment needed) Late failures due to refractory relapses (15%) or clonal evolution (15%) 18
19 LATE FAILURES PNH MDS/AML Second tumours Consider oligoclonal hematopoiesis in AA due to HSC reduction Pre-existing vs subsequent? CLONAL EVOLUTION A matter of definition PNH MDS AML Neutral fixation of mutation vs true clonal complication
20 Haematologica 2010 A PNH population is present at diagnosis in about 40% of patients (usually <50%, without hemolysis) Subsequent fluctations are common Clinical PNH in 5-10% Blood 2002 The outcome of SAA patients developing karyotypic abnormalities after IST depends on the specific abnormality: very bad for 7-, more favorable for 8+ 20
21 CRYPTIC GENOMIC ABERRATION IN AA AND MDS Wlodarski et al., Manuscript submitted 100% hpnh n=21 AA n=69 hypomds n=23 hypermds n=83 patients 80% 60% 40% 20% 0% normal abnormal n.i./n.a. MC SNP- A MC SNP- A MC SNP- A MC SNP- A normal 71% hpnh UPD 29% Type of lesion AA Abnormal 32% hypomds Abnormal 43% UPD 26.1% Loss 1.5% UPD 21.7% Loss 26.1% normal 68% normal 57% Tri 4.7% Tri 1.5% Mono7 8.7% Mono7 8.7% Response to IST (AA patients) no lesions by SNP-A: n=10 70% 30% refractory responders lesions by SNP-A: n=8 62% 38% Courtesy of Jarek Maciejewski CLINICAL OVERLAP BETWEEN PNH AND BMF Hypoplastic PNH AA Florid PNH Subclinical AA/PNH PNH Others? 21
22 2003 n=112 hatg x 4 (40mg/kg) + CsA x 6 m Clonal evolution (3y) 11% MDS (especially 7-7 ) 10% PNH NEJM 2011 Evolution to MDS (3y) 21% hatg 14% ratg Blood 2012 In all recent studies, the incidence of clonal evolution is about 10%, regardless the specific treatment Can we predict clonal evolution? 22
23 THERAPY OF SAA: THE ROLE OF G-CSF A survey from the EBMT WPSAA n (%) 840 G-CSF 363 (43) No G-CSF 477 (57) HR AML 2.5 MDS/AML 1.9 OS n.s. reverse causality or confounding by the indication G-CSF used in more ill patients? Blood 2011 Phase III randomized study hatg + CyA ± G-CSF (n=192) 6y 76%, 6y 42%; no difference in the two arms No difference in response rate (but lack of ANC response to G-CSF may predict no response to IST) No difference in clonal evolution No G-CSF G-CSF G-CSF does not increase the OR to IST; however, it may be useful to identify poor responders to IST, without carrying any increased risk of clonal evolution 23
24 NEJM 1993 Post-IST SAA (n=860) MDS 28 AML 16 NHL 1 ST 7 Post-BMT SAA (n=748) MDS 0 AML 2 NHL 0 ST 7 Survivors of SAA after IST are at high risk of hematological cancers; the risk of solid cancer is only slightly increased (in BMT recipient) 24
25 Treatment options for aplastic anemia Locasciulli et al, Haematologica 2007 NHLBI, NIH, Bethesda Hematology Branch Neal S. Young Cleveland Clinic Foundation Hematopoiesis and Experimental Hematology Division Jaroslaw P. Maciejewski Severe Aplastic Anemia Working Party Judith Marsh Jakob Passweg Andrea Bacigalupo Carlo Dufour Gerard Socié Federico II University of Naples Division of Hematology Prof. Bruno Rotoli Prof. Carmine Selleri 25
Yes Antonio M. Risitano, M.D., Ph.D. Head of Bone Marrow Transplantation Unit Federico II University of Naples
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