Table 2.
Key results of randomized trials related to FDA-approved indications
| Treatment | Durability of benefit | Overall survival | Toxicity | |||||||
|---|---|---|---|---|---|---|---|---|---|---|
| PFS/EFS* | HR | P | HR | P | Nausea, % | ≥G3 febrile neutropenia, % | Pneumonia, % | Discontinued due to AE, % | ||
| CLL-relapsed27,59 | ||||||||||
| Ven-Ritux | 71% @ 3 y | 0.16 | <.001 | 88% @ 3 y | 0.50 (.3-.85) | .009 | 21 | 3.6 | 8.2 | 17 |
| Ben-Ritux | 15% @ 3 y | 80% @ 3 y | 34 | 9.6 | 8.0 | 16 | ||||
| CLL–first-line28 | ||||||||||
| Ven-Obin | 88% @ 2 y | 0.35 | <.001 | 92% @ 2 y | 1.24 (.64-2.4) | .52 | 19 | 17.5 | 4.7 | 22† |
| Chl-Obin | 64% @ 2 y | 93% @ 2 y | 22 | 15.0 | 4.2 | 23† | ||||
| AML first-line (including sAML pretreated with HMA)37 | ||||||||||
| Ven-LoDAC | 4.7* | 0.58 | .002 | 8.4 | 0.70 (.50-.99) | .04 | 42 | 32 | 13 | 9 |
| Pbo-LoDAC | 2.0* | 4.1 | 31 | 29 | 10 | 9 | ||||
| AML–first-line (no prior HMA)38 | ||||||||||
| Ven-Aza | 9.8* | <.001 | 14.7 | 0.66 (.52-.85) | <.001 | 44 | 30 | 16 | NR | |
| Pbo-Aza | 7.0* | 9.6 | 35 | 10 | 22 | NR | ||||
≥G3, grade 3 or higher; AE, adverse event; Aza, azacytidine; Ben, bendamustine; Chl, chlorambucil; EFS, event-free survival; FDA, US Food and Drug Administration; HMA, hypomethylating agent therapy; HR, hazard ratio; LoDAC, low-dose cytosine arabinoside; NR, not reported; Obin, obinutuzumab; Pbo, placebo; PFS, progression-free survival; Ritux, rituximab; sAML, secondary AML; Ven, venetoclax.
*
EFS for AML.
†All cause discontinuation excluding PD.