Background
Methods
Patient selection
Study design and treatment
Gene expression analysis-WT1/ABL
WT1 vaccine preparation
Collection of peripheral blood mononuclear cells (PBMCs) and preparation of CD8+ T cells
ELISPOT assay
T cell receptor (TCR) α and β-chain deep sequencing
Results
Patient characteristics and treatment courses
Patient no | Diagnosis | Arm | Prior treatments | Disease status at vaccination | WT1 treatment | Treatment after WT1 | Severe toxicities | Current status | PFS (days) |
---|---|---|---|---|---|---|---|---|---|
1 | CML myeloid blast phase | WT1 in Montanide | Failed ponatinib, had MRD SCT with active disease | Morphologic residual disease with molecule disease | s/p 6 WT1 vaccination, tolerated well | DLI 40 days after last WT1 vaccination | No | Died from infection with GVHD | 463 |
2 HLA A0202 | Relapsed AML with normal karyotype | WT1 in poly ICLC | CR after induction, 2 cycle HiDAC consolidation, relapse 7 months later, CR2 with clinical trial | Morphologic residual disease, with cytogenetic changes | s/p 6 WT1 vaccination, tolerated well | Disease progressed, and died from disease | Admitted for viral infection (not related) | Died due to disease progression | 186 |
3 | t-AML, t (8,21), del 9q | WT1 in poly ICLC | CR1 after induction and consolidation | Morphologic and cytogenetic remission | s/p 9 WT1 vaccination; tolerated well | Off study due to FDA hold of the study | No | In remission | 1731 |
4 | MDS RAEB2 | WT1 in poly ICLC | CR after MUD SCT | Morphologic and cytogenetic remission | s/p 9 WT1 vaccination; tolerated well | Off study due to FDA hold of the study | No | In remission | 1675 |
5 | Relapsed AML after allo-SCT | WT1 in Montanide | CR after 2rd haplo-cord SCT | Morphologic and cytogenetic remission | s/p 7 WT1 vaccination; tolerated well | Off study with neurologic complication | Myelitis after bacterial meningitis | Died in remission | 239 |
6 | AML with normal karyotype | WT1 in Montanide | CR after induction/1 cycle consolidation, | Morphologic remission but cytogenetic revolution with 17p deletion | s/p 12 WT1 vaccine | Disease relapsed 18 months after vaccine, treated with decitabine | Developed aseptic ulcer at vaccine sites of 12, 11, 10, and erythema at the 1st vaccine site | Relapsed, Died from disease progression | 832 |
7 | AML with complex karyotype | WT1 in Montanide | CR1 after induction, 3 cycle consolidation | Morphologic and cytogenetic remission | s/p 8 WT1, tolerated well, but had disease relapse | Disease progressed after 4 cycle 10 day decitabine | No | Died from disease progression | 169 |
Toxicities
Adverse events | Total, n (%) | Grade 1 and 2 | Grade 3 | Grade 4 | Grade 5 |
---|---|---|---|---|---|
Related to WT1 vaccine (all with Montanide) | |||||
Injection sites ulceration | 1 (14%) | 1 | |||
Injection sites reaction | 4 (57%) | 4 | |||
SAEs unrelated to WT1 vaccine | |||||
Ascending myelitis due to infection | 1 (14%) | 1 | |||
Hyperglycemia due to uncontrolled DM | 1 (14%) | 1 | |||
Thrombocytopenia due to disease | 1 (14%) | 1 | |||
Anemia due to disease | 1 (14%) | 1 | |||
Depression | 1 (14%) | 1 | |||
Upper respiratory infection (viral) | 3 (43%) | 3 |