Table 3.
Ongoing clinical trials for immune checkpoint inhibitors in treatment of MDS/AML
| Phase | Inclusion | Therapy | Target | Primary objectives | Reference |
|---|---|---|---|---|---|
| Ib | Relapsed MDS/AML/ALL after allo-SCT | Pembrolizumab | PD-1 | CR, PR, SD, toxicity | NCT03286114 |
| Early I | Relapsed MDS/AML/Hodgkin and non-Hodgkin lymphoma after allo-SCT | Pembrolizumab | PD-1 | AEs | NCT02981914 |
| II | High risk for relapse AML | Nivolumab | PD-1 | RFS | NCT02532231 |
| Ib | Relapsed MDS/AML after allo-SCT | Nivolumab and/or lpilimumab | PD-1 and/or CTAL-4 | MTD, DLTs, toxicity | NCT03600155 |
| I | High risk for relapsed MDS/AML after allo-SCT | Nivolumab and/or lpilimumab | PD-1 and/or CTAL-4 | Safety | NCT02846376 |
| II | Non-favorable risk AML | Pembrolizumab + Flu/Mel + Allo-SCT | PD-1 + Lymphodepletion + Allo-SCT | RFS | NCT02771197 |
| I | Previously untreated and relapsed/refractory MDS/AML | Pembrolizumab + DAC | PD-1 + HMA | AEs, MTD, CR/CRi | NCT03969446 |
| II/III | Previously untreated higher-risk MDS | Nivolumab + AZA/AZA | PD-1 + HMA | OS | NCT03092674 |
| II | R/R AML | Camrelizumab (SHR-1210) + DAC | PD-1 + HMA | ORR, CR | NCT04353479 |
| 0 | Previously untreated higher-risk MDS | Sintilimab + DAC | PD-1 + HMA | ORR | ChiCTR2100044393 |
| II | Higher-risk MDS | Camrelizumab + DAC | PD-1 + HMA | ORR | ChiCTR1900028440 |
| IV | MDS-EB1/2 | PD-1 monoclonal antibody + AZA | PD-1 + HMA | Efficacy | ChiCTR2000034927 |
| IV | HMAs failure MDS | Camrelizumab + DAC | PD-1 + HMA | ORR | ChiCTR2100044210 |
| II | previously untreated AML/sAML | Pembrolizumab + Ara-C + IDA/DNR | PD-1 + chemotherapy | MDR-CR | NCT04214249 |
| II | R/R AML excluding relapsed after HSCT | Tislelizumab + CAG | PD-1 + chemotherapy | ORR | NCT04541277 |
| – | Higher-risk MDS | Tislelizumab + HMA + cytarabine | PD-1 + HMA + chemotherapy | Efficacy | ChiCTR2100045296 |
| III | R/R AML | Visilizumab + Azacytidine + HAG regimen | PD-1 + HMA + chemotherapy | CR, CRi, PR | NCT04722952 |
| II | R/R AML | Nivolumab + AZA + Relatlimab | PD-1 + LAG-3 | MTD, DLTs, ORR | NCT04913922 |
| II | Previously untreated or HMAs failure MDS | Nivolumab; nivolumab + lpilimumab; nivolumab + lpilimumab + AZA | PD-1 and/or CTAL-4 and/or HMA | ORR | NCT02530463 |
| I | AML and intermediate or high- risk MDS | PDR001 + DAC and/or MBG453 | PD-1 + TIM-3 and/or HMA | AEs, DLTs | NCT03066648 |
| Ib/II | R/R AML | Avelumab + AZA + GO/VEN/anti-OX40 antibody PF-04518600 | PD-1 + CD33/HDACi/OX40 + HMA | AEs | NCT03390296 |
| Ib | HMAs failure MDS | Pembrolizumab + entinostat | PD-1 + HDACi | MTD | NCT02936752 |
| I | Untreated AML with TP53-mutated | Nivolumab + decitabine + venetoclax | PD-1 + HMA + HDACi | CRc | NCT04277442 |
| II | previously untreated AML/sAML | Pembrolizumab + AZA + VEN | PD-1 + HMA + HDACi | MRD-CR | NCT04284787 |
| I | Higher-risk MDS or AML with ≤ 30% blasts | Nivolumab + DAC + NY-ESO-1 vaccinationa | PD-1 + HMA + tumor vaccine | Safety | NCT03358719 |
| I/II | Higher-risk MDS/CML with HMA-failure | Atezolizumab + guadecitabine | PD-L1 + HMA | DLTs, ORR | NCT02935361 |
| II | MDS with post injectable HMA-failure | Durvalumab and/or oral AZA | PD-L1 + HMA | ORR | NCT02281084 |
| I | AML | Atezolizumab + Guadecitabine | PD-L1 + HMA | AEs, CR, CRp, CRi, DOR | NCT02892318 |
| I | R/R AML | Atezolizumab + Hu5F9-G4 | PD-L1 + CD47 | AEs, CR, DOR | NCT03922477 |
Allo-SCT allogeneic stem cell transplantation, AML acute myeloid leukemia, ALL acute lymphoblastic leukemia, CRi complete remission with incomplete count recovery, CRc composite complete response, DLTs dose-limiting toxicities, DOR duration of response, MTD maximum tolerated dose, RFS recurrence-free survival, SD stable disease
aNY-ESO-1 vaccination: Anti-DEC-205-NY-ESO-1 fusion protein plus poly-ICLC