Table 1.
Checkpoint Inhibitors Currently Under Evaluation for the Treatment of MDS.
| Target | Population | Comments | Phase | n | Status | Clinicaltrials.org Trial Number |
|---|---|---|---|---|---|---|
| PD-1 or PD-L1 | HR-MDS | Pembrolizumab + HMA | Phase Ib | 54 | Recruiting | NCT03969446 |
| IR-MDS or HR-MDS | Atezolizumab + HMA | Phase I/II | Recruiting | NCT04214249 | ||
| Myeloid malignancies | Pembrolizumab | Phase I | 197 | Completed | NCT01953692 | |
| CTLA-4 | HR-MDS + AML | Ipilimumab | Phase I | 42 | Completed | NCT01757639 |
| Pre- and post- HCT allograft, MDS and AML | Ipilimumab + decitabine | Phase I | 48 | Recruiting | NCT02890329 | |
| CTLA-4 + PD-1 | Myeloid malignancies post-allogeneic HSCT | Ipilimumab + nivolumab | Phase I | 71 | Completed | NCT01822509 |
| AML or HR-MDS | Ipilimumab + nivolumab | Phase I | 8 | Active, not recruiting | NCT02846376 | |
| HR-MDS + AML s/p allogeneic HSCT | Ipilimumab + nivolumab | Phase I | 55 | Recruiting | NCT03600155 | |
| Myeloid malignancies | Nivolumab + AZA ± ipilimumab | Phase II | 182 | Recruiting | NCT02397720 | |
| All-risk MDS | Ipilimumab ± nivolumab ± AZA | Phase II | 160 | Recruiting | NCT02530463 | |
| TIM-3 | LR-MDS | MBG-453 | Phase II | 20 | Not yet recruiting | NCT04823624 |
| LR to IR-MDS | MBG-453, canakinumab, NIS793 (Anti-TGF-β) | Phase I | 90 | Recruiting | NCT04823624 | |
| AML + HR-MDS | MBG453+ HDM201 (MDM2 inhibitor) | Phase Ib | 80 | Recruiting | NCT03940352 | |
| HR-MDS | MBG453 + AZA or HMA | Phase II | 90 | Not yet recruiting | NCT04878432 | |
| HR-MDS | MBG453 + AZA + venetoclax | Phase II | 76 | Recruiting | NCT04812548 | |
| IR-HR MDS | MBG453 versus placebo with HMA | Phase II | 127 | Active, not yet recruiting | NCT03946670 | |
| Myeloid malignancies | MBG453 ± PDR001 (anti-PD-1) ± HMA | Phase I | 243 | Active, not yet recruiting | NCT03066648 | |
| IR-HR MDS | MBG453 | Phase III | 500 | Recruiting | NCT04266301 | |
| CD47 | HR-MDS | AK117 (monoclonal Ab targeting CD47) + AZA | Phase I/II | 190 | Recruiting | NCT04900350 |
| HR-MDS | Evorpacept (soluble CD47 inhibitor) + AZA | Phase I/II | 173 | Recruiting | NCT04417517 |
Abbreviations: allo-HSCT = allogeneic hematopoietic cell transplantation; AML = acute myeloid leukemia; CTLA-4 = cytotoxic T-lymphocyte-associated protein 4; HMA = hypomethylating agent; HR = higher-risk; IR = intermediate risk; LR = lower risk; MDS = myelodysplastic syndrome; PD-1 = programmed cell death 1; PD-L1 = programmed death ligand 1.