Table 1

Patient characteristics

Patient no.Age, yTypeKaryotypePrevious therapyDisease status baselineCohortNo. of vaccBlasts in BM baselineBest response, IWGPFS, dBest response modified criteriaDuration of early progression, dmPFS, dClinical response commentsTetramer+ T cells in pB baselineTetramer response in pB following vaccTetramer response in BM following vaccCytokine response in pB following vacc
66 M4 Normal 2× ind. reind. 2. PR 12 10 PD 21 mCR (17 mo) 65 514 − − 
82 M2 na None PD 85 SD 136 SD − 136 Off protocol after 4 vacc due to soft tissue infectious complications − 
64 M6 Complex 5q− 2× ind. PD 60 PD 50 PD − 50 − − − − 
72 M2 Normal Ind. 1. PR PD 37 PD − 37 − − 
74 M1 47 XX + 11 46 XX (23) 2× ind.reind. 2. PR 12,5 SD 201 SD − 201 − − − 
45 M7 Normal 3× ind reind. PD 60 PD 49 PD − 49 − − − − − 
73 WHO II Normal None PD 12 70 SD 262 SD − 262 Erythroid response for 7 months − − − 
63 WHO II Normal None PD 18 na SD 101 SD − 101 − − 
80 M4 Normal None PD 80 PD 60 mSD 82 144 Decrease of peripheral blasts − 
10 59 M4 Normal 2× ind. reind. autoPBSCT 3. PR 5–10 PD 29 PD − 29 − na na na na 
11 65 M4 Normal Ind. PD 12 10 SD 134 SD − 134 − − − − − 
12 67 RAEBII Normal None PD 24 12 Major neutrophil response 112 mSD 231 339 − 
13 61 WHO II Normal None PD 11 60 SD 142 SD − 142 − − − − 
14 69 RAEB I Normal ATG, CsA PD 10 10 Major neutrophil response 69 Major neutrophil response − 69 − − − − − 
15 75 WHO II Complex None PD 10 60 SD 132 SD − 132 Decrease of marrow blasts (30% at 3 months) − − 
16 82 M4 Normal None PD 11 50 PD 66 mSD 112 137  − 
17 65 M2 Trisomy 8 1× ind. 2× cons. 1. PR 27+ SD ongoing  SD ongoing − 571 (+) Erythroid response after 4 months, molecular response with complete disappearance of trisomy 8 − 
18 58 M2 Normal None PD 23+ 40 SD ongoing 506 (+) SD ongoing − 506 (+) Decrease of marrow blasts (12% at 3 months) − − − − 
19 69 WHO II 46 XX (del5) Low araC 1. PR 11 30 SD 168 SD  168 Blast response at 3 months (15%) − − 
Patient no.Age, yTypeKaryotypePrevious therapyDisease status baselineCohortNo. of vaccBlasts in BM baselineBest response, IWGPFS, dBest response modified criteriaDuration of early progression, dmPFS, dClinical response commentsTetramer+ T cells in pB baselineTetramer response in pB following vaccTetramer response in BM following vaccCytokine response in pB following vacc
66 M4 Normal 2× ind. reind. 2. PR 12 10 PD 21 mCR (17 mo) 65 514 − − 
82 M2 na None PD 85 SD 136 SD − 136 Off protocol after 4 vacc due to soft tissue infectious complications − 
64 M6 Complex 5q− 2× ind. PD 60 PD 50 PD − 50 − − − − 
72 M2 Normal Ind. 1. PR PD 37 PD − 37 − − 
74 M1 47 XX + 11 46 XX (23) 2× ind.reind. 2. PR 12,5 SD 201 SD − 201 − − − 
45 M7 Normal 3× ind reind. PD 60 PD 49 PD − 49 − − − − − 
73 WHO II Normal None PD 12 70 SD 262 SD − 262 Erythroid response for 7 months − − − 
63 WHO II Normal None PD 18 na SD 101 SD − 101 − − 
80 M4 Normal None PD 80 PD 60 mSD 82 144 Decrease of peripheral blasts − 
10 59 M4 Normal 2× ind. reind. autoPBSCT 3. PR 5–10 PD 29 PD − 29 − na na na na 
11 65 M4 Normal Ind. PD 12 10 SD 134 SD − 134 − − − − − 
12 67 RAEBII Normal None PD 24 12 Major neutrophil response 112 mSD 231 339 − 
13 61 WHO II Normal None PD 11 60 SD 142 SD − 142 − − − − 
14 69 RAEB I Normal ATG, CsA PD 10 10 Major neutrophil response 69 Major neutrophil response − 69 − − − − − 
15 75 WHO II Complex None PD 10 60 SD 132 SD − 132 Decrease of marrow blasts (30% at 3 months) − − 
16 82 M4 Normal None PD 11 50 PD 66 mSD 112 137  − 
17 65 M2 Trisomy 8 1× ind. 2× cons. 1. PR 27+ SD ongoing  SD ongoing − 571 (+) Erythroid response after 4 months, molecular response with complete disappearance of trisomy 8 − 
18 58 M2 Normal None PD 23+ 40 SD ongoing 506 (+) SD ongoing − 506 (+) Decrease of marrow blasts (12% at 3 months) − − − − 
19 69 WHO II 46 XX (del5) Low araC 1. PR 11 30 SD 168 SD  168 Blast response at 3 months (15%) − − 

ATG indicates antithymocyte globulin; autoPBSCT, autologous peripheral blood stem cell transplantation; con, consolidation chemotherapy; CsA, cyclosporin A; CR, complete remission; cytokine response, intracellular cytokine response as detected by flow cytometry after stimulation with WT1 peptide in percent (%) of CD3+CD8+ T cells, a cytokine response was considered positive if the percentage of WT1 peptide–specific cytokine producing CD3+CD8+ T cells was at least 2-fold the percentage of an HIV control peptide; Ind, induction chemotherapy; low araC, low-dose cytarabin; mPFS, modified progression-free survival (in case progression occurred prior to 6th vaccination, subsequent time was estimated from first vaccination until subsequent progression); na, not available; Patient no., order of inclusion into study; PD, progressive disease; PFS, progression-free survival; PR, partial remission; reind, reinduction chemotherapy; SD, stable disease; Tetramer+ T cells, WT1-specific tetramer-positive T cells in percent (%) of CD3+CD8+ T cells, which were considered positive, if the frequency of tetramer-positive CD3+CD8+ T cells at baseline exceeded 0.3%, which was the mean + 2 SDs (0.16% + 0.14%) observed in 12 healthy control subjects; tetramer response, an increase of at least 2-fold in frequency as compared to baseline was considered as vaccine-induced response; type, AMLs were classified according to the WHO classification (patients belonging to the category ″AMLs not otherwise categorized″ are subclassified according to the old FAB classification): all other patients belonged to the second group of the WHO classification (WHOII = ″AML with multilineage dysplasia- with prior MDS″); and vacc, vaccination.

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