Table 1.
Summary of Immunotherapy Biomarkers in Acute Myeloid Leukemia.
| Study | Therapy | Type | Biomarker | Outcome | Commercial testing available |
|---|---|---|---|---|---|
| Castaigne et al. (8) Lambert et al. (9) |
Gemtuzumab ozogamicin (GO) | Monoclonal | CD33 | Prolonged EFS and RFS in high CD33+ with GO | Yes |
| Khan et al. (10) | Greater efficacy on relapse but not OS in high CD33+ with GO | ||||
| Fournier et al. (11) | Benefit of GO in several activating signaling mutations in AML blasts | ||||
| Ravandi et al. (12) | AMG 330 | BITE | CD33 | 3 CR, 4 CRi, 1 morphologic leukemia free state | Yes |
| Godwin et al. (13) | Flotetuzumab | DART | CD123 | 3 CR, 2 received HSCT in primary refractory AML. Higher CD3 and CD8 cell infiltrates in baseline BM in responders | Yes |
| Uy et al. (14) | Median OS 10.2 months. 10-gene signature predictive of CR | ||||
| Vadakekolathu et al. (15, 16) | Increased expression of IFN-γ-related genes and immune infiltrative tumor microenvironment associated with responders | ||||
| Wei et al. (17) | Flotetuzumab + MGA012 | Ongoing study | |||
| Ravandi et al. (18, 19) | Vibecotamab | DART | CD123 | 2 Cr, 3 Cri, 3 morphologic leukemia free survival with overall response rate of 14%. Lower PD-1 expression on CD4+ and CD8+ T cells in responders | Yes |
| Assi et al. (20) | Nivolumab + idarubicin + cytarabine | ICI | T-Cell repertoire | 34 (77%) CR/CRi (28 CR, 6 CRi) and 18/34 (53%) undetectable MRD. Responders had greater total infiltrative CD3+ vs nonresponders who had increased baseline CD4+ Teffs co-expressing PD-1 and TIM3 | No |
| Daver et al. (21) | Nivolumab + azacitidine | Overall response rate was 33%, with 58% ORR in HMA-naïve patients. Increased pretherapy BM CD3+, CD4+ T effecter, and CD8+ cells; and increased peripheral CD3+ in responders | |||
| Daver et al. (22) | Nivolumab + azacitidine+ Ipilimumab | Median OS of 7.6 months. Increasing peripheral CD8+ T cells to sites of disease over therapy duration | |||
| Zeidner et al. (23) | Pembrolizumab + HiDAC | ICI | T-Cell repertoire | Overall response rate 46%, CR/CRi 38%. Median OS was 11.1 months. Increased frequency of senescent T cells in BM and peripheral blood and increased terminally differentiated effector T cells in peripheral blood in nonresponders. Increased TCF1+ CD8+ T cells in CR |
No |