Table 1 Selected completed trials of MDS vaccines

WT-1 peptide vaccine [28]

I

16

NCT00665002

montanide+ GM-CSF

HR-MDS

Well tolerated

1/2 patients (HR-MDS) with prolonged transfusion independence

WT-1 and PR-1

Peptide vaccine [24]

I

8

NCT00313638

montanide+ GM-CSF

MDS-RAEB; ineligible for HSCT

Well tolerated

PR-1/WT-1-specific CD8+ T-cells

RHAMM

Peptide vaccine [48]

I/II

10

ISRCTN32763606

Incomplete Freud adjuvant+ GM-CSF

MDS < 20% BM blasts (RA, RAEB-1, RAEB-2), MM

RHAMM-specific T-cell responses

1 MDS patient with blast reduction and transfusion-independent

DEC-205/NY-ESO-1 fusion

Peptide vaccine [42, 118]

I

9

NCT01834248 NCT03358719

Decitabine

Addition of nivolumab

Intermediate and HR-MDS

NY-ESO-1-specific T cell responses

No results posted

NY-ESO-1, MAGE-A3, PRAME, WT-1

Peptide vaccine [46]

I

5

NCT02750995

Azacitidine

HR-MDS

All patients progressed to AML; mean time to progression of 4.9 months from inclusion in the study

Whole-cell endogenous tumor antigen dendritic cell vaccine [68]

I

12

NCT01373515

DCP-0001 vaccine

HR-MDS

7/12 patients with positive response (median OS 1090 days); 5/12 with progressive disease (Median OS 144 days)

TAA whole-cell vaccine [61]

I

11

NCT00840931

K562-GM-CSF-CD40L vaccine+ lenalidomide+ GM-CSF

Intermediate and HR-MDS (failed HMA treatment)

Well tolerated

CR in 2/11, marrow CR in 1/11, PR in 1/11

TAA whole-cell vaccine [57]

II

15

NCT01773395

GVAX vaccine

MDS-RAEB

No significant difference in 18-month PFS, OS and relapse incidence between GVAX vs placebo

PR-1

Peptide vaccine [36]

I/II

62 (11 MDS)

NCT00004918

Montanide+ GM-CSF

MDS-RAEB

Clinical response (CR, PR and hematological improvement) in 24% patients