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| Study | Phase of study | Patient population | Number of patients | Treatment and dosing used | Median age, years (range) | # patients >75 years old | Response rate (CR + CRi) | CR | Median event-free survival | Median duration of CR + CRi | Median overall survival | Grade 3/4 adverse events |
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| Dinardo et al., Lancet Oncol, 2018 [2] | 1b | Age >65, treatment naïve, ineligible for standard induction | 57 | 3 + 3 dose escalation; venetoclax-group A: target doses 400, 800, and 1200 mg/m2; group B: target doses 400, 800, and 1200 mg/m2; group C: target dose 400 mg/m2; azacitidine-75 mg/m2; decitabine-20 mg/m2 | Group A: 74 (71.5–79.0); group B: 75 (71.0–80.0); group C: 74 (69.0–79.5) | 39 | 61.4% (35/57) | 24.6% (14/57) | Not reported | Group A: 8.4 mos; group B: 12.3 mos; group C: 4.3 mos | 12.3 months (all groups) | Thrombocytopenia; febrile neutropenia; neutropenia |
| Dinardo et al., Blood, 2019 [3] | 1b | Age >65, treatment naïve, ineligible for standard induction | 145 | Venetoclax-ramp-up C1 from 20 (escalation phase) or 100 (expansion); target doses: 400, 800, and 1200 mg/m2; in escalation expansion phase: 400 mg and 800 mg; azacitidine-42 mg/m2, d1-7; decitabine-20 mg/m2, d1-5 | 74 (65–86) | 52 | 67% | 37% | Not reported | 11.3 months | 17.5 months | Febrile neutropenia; anemia; thrombocytopenia; neutropenia; pneumonia |
| Dinardo et al., NEJM, 2020 [1] | 3 | Ineligible for intensive induction, age >75, all treatment naive | 286 in variable group | Aza 75 mg/m2, d1-7; VEN 400 mg daily, d1-28 w/3 day ramp-up C1 | 76 (49–91) | Not reported | 66.4% | 36.7% | 9.8 months | 17.5 months | 14.7 months | Thrombocytopenia neutropenia; febrile neutropenia; anemia; leukopenia |
| Maiti et al., Blood, 2018 [4] | II | Age >69, ineligible for OR relapse/refractory AML | 48 | VEN days 1–28, cycle 1, D1-21; C2 and onwards VEN 200 mg PO daily in pts needing cyp3a4 decitabine-20 mg/m2 IV daily D1–D10 until CR, followed by 5-day cycles | 71 (22–82) | Not reported | 71% (34/48) | CR alone not reported | Not reached | Not reached | Not reached | Infections; neutropenia; tumor lysis syndrome |
| Wei et al., Journal of Clinical Oncology, 2019 [5] | Ib/II | Age >60, treatment naïve, ineligible for intensive chemo | 82 | Venetoclax-initial 50 or 100 mg, titrated up to the target dose; phase II recommended dose: 600 mg; 800 mg in phase Ib w/prolonged myelosuppression; low-dose cytarabine-20 mg/m2 daily SC injection D1–10, 28-day cycles | 74 (63–90) | 40 | 54% | 26% | Not reported | 8.1 months | 10.1 months | Cytopenia, febrile neutropenia; infections |
| Wei et al., Blood, 2020 [6] | 3 | Adults ≥18 y/o w/ND AML ineligible for intensive chemo (38% had secondary AML, and 20% had received prior hypomethylating agent treatment) | 210 | Venetoclax + LDAC | 76 (33–93) | 82 | 48% | 27% | 4.7 months | Not reported | 7.2 months | Febrile neutropenia; neutropenia; thrombocytopenia |
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