Table 1.
Efficacy data from phase II studies of investigational therapeutic regimens for WM
| Agent/regimen | WM study population (n) | ORR (%) | CR (%) | Median TTP (months) | Median PFS (months) |
|---|---|---|---|---|---|
| Rituximab [9] | Previously untreated (n = 17) | 35 | 0 | 13 | – |
| Rituximab [10] | Previously treated with rituximab-based induction (n = 86) | 98 | 16.3 | – | 56.3 |
| Ofatumumab [11] | Previously untreated (n = 9) Relapsed (n = 28) |
59 | 0 | – | – |
| Alemtuzumab [12] | Symptomatic (n = 28)a | 75 | 4 | 14.5 | – |
| Bortezomib/dexamethasone/rituximab [13] | Previously untreated (n = 23) | 96 | 13 (+9 nCR) | >30 | – |
| Bortezomib/dexamethasone/rituximab [14] | Previously untreated (n = 59) | 85 | 3 | – | 42.0 |
| Weekly bortezomib/rituximab [15] | Previously untreated (n = 26) | 88 | 4 (+ 4 nCR) | Not reached | – |
| Weekly bortezomib/rituximab [16] | Relapsed/refractory (n = 37) | 81 | 5 | 16.4 | 15.6 |
| Carfilzomib/rituximab/ dexamethasone [17] | Previously untreated (n = 31) | 87b | 3 | – | – |
| Thalidomide/rituximab [18] | Previously untreated (n = 20) Relapsed/refractory (n = 5) |
72 | 4 | 34.8 | – |
| Lenalidomide/rituximab [19] | Previously untreated (n = 12) Relapsed (n = 4) |
50 | 0 | 17.1 | – |
| Everolimus [20] | Previously untreated (n = 33) | 72 | 0 | – | – |
| Everolimus [21] | Relapsed/refractory (n = 60) | 73 | 0 | 25.0 | 21.0 |
| Enzastaurin [22] | Relapsed/refractory (n = 42) | 38 | 0 | 10.9 | – |
| Perifosene [23] | Relapsed/refractory (n = 37) | 35 | 0 | 12.6 | 12.6 |
| Ibrutinib [24] | Relapsed/refractory (n = 63) | 90.5 | 0 | 9.6 | Not reached |
| Panobinostat [25] | Relapsed/refractory (n = 36) | 47 | 0 | – | 6.6 |
aTotal of 27 patients with WM (trial enrolled patients with lymphoplasmacytic lymphomas).
bResponse not affected by MXD88 or CXCR4 mutation status.
CR, complete response; nCR, near complete response (defined as fulfilling all CR criteria in the presence of a positive immunofixation study) [13]; ORR, overall response rate (minimal response or higher); PFS, progression-free survival; PR, partial response; TTP, time to progression; VGPR, very good partial response.