Table 2.
Selected Therapeutic Trials in Waldenstrom Macroglobulinemia
| Author | Regimen | Number of patients | ORR (%) | CR (%) | OS (mo) | PFS | Comments |
|---|---|---|---|---|---|---|---|
| Gertz et al55 | Rituximab | 69 | 52 | 0 | NA | NA | Well-tolerated, possibility of IgM flare |
| Dimopoulos et al56 | Cladribine | 26 | 85 | (3/26) | NA | NA | High risk of viral infections |
| Dhodapkar et al19 | Fludarabine | 182 | 36 | 2 | 88 | 49 | OS at 5 years 58% |
| Dimopoulos et al31 | Fludarabine | 28 | 36 | 0 | NA | NA | |
| Tamburini et al57 | Fludarabine Cyclophosphamide | 49 | 78 | 0 | NA | NA | |
| Weber et al32 | Cladribine, Cyclophosphamide Rituximab | 27 | 94 | 17 | 73 | Grade 3/4 neutropenia in 71%, serious infections in 35% | |
| Tedeschi et al33 | Fludarabine Cyclophosphamide Rituximab | 19 | 79 | 5 | NA | NA | Neutropenia observed in 82% |
| Vargaftig et al34 | Fludarabine Cyclophosphamide Rituximab | 25 | 78 | 0 | NA | NA | Significant risk of infections |
| Leblond et al37 | CAP | 45 | 11 | 0 | 45 | NA | |
| Abonour et al39 | CHOP-R | 16 | 100 | 0 | NA | NA | Rapid median time to response |
| Ioakimidis et al40 | CHOP-R CVP-R CP-R |
23 16 19 |
96 88 95 |
17 12 0 |
NA | NA | P values not different. Higher incidence for neutropenic fever and treatment-related neuropathy for CHOP-R and CVP-R versus CP-R. |
| Dimopoulos et al38 | DRC | 72 | 83 | 7 | NA | 2 year PFS, 67% | Well-tolerated; grade 3/4 neutropenia in 9%. |
| Treon et al42 | Bortezomib | 27 | 85 | 0 | NA | NA | |
| Treon et al44,58 | Bortezomib Dexamethasone Rituximab | 23 | 96 | 13 | NA | NA | Most common toxicity: peripheral neuropathy (61%). Rapid reduction in IgM |
| Treon et al47 | Rituximab Thalidomide | 25 | 72 | 0 | NA | NA | May be considered in patients presenting with cytopenias |
CAP=cyclophosphamide, doxorubicin, prednisone; CHOP-R=rituximab, cyclophosphamide, doxorubicin, vincristine, prednisone; CP-R=cyclophosphamide, prednisone, rituximab; CR=complete response; CVP-R= cyclophosphamide, vincristine, prednisone; DRC=dexamethasone, rituximab, cyclophosphamide; NA=not applicable; ORR=overall response rate; OS=overall survival; PFS=progression-free survival.