Registrational trials of approved nonchemotherapy drugs
| Agent . | Patient population . | Study design . | Administration . | N . | Primary end point . | Secondary end points . |
|---|---|---|---|---|---|---|
| Bortezomib55 | 1-3 prior lines of therapy | Phase 2 | Bortezomib 1.3 mg/m2 days 1, 4, 8, 11 q21 d until progression | 155 | TTP, 6.2 mo (95% CI, 4.0-6.9 mo) | ORR, 33% |
| CR + CRu, 8% | ||||||
| Bortezomib14 | Previously untreated | Randomized phase 3 | VR-CAP vs R-CHOP ×6-8 cycles | 243 vs 244 | Median PFS, 24.7 mo vs 14.4 mo; HR, 0.63 (95% CI, 0.50-0.79) | Median OS, 56.3 mo vs NR; HR, 0.80 (95% CI, 0.59-1.10) |
| Temsirolimus33 | Previously treated | Randomized phase 3 | Temsirolimus 175/75 mg vs 175/25 mg vs investigator’s choice until progression | 54 vs 54 vs 53 | Median PFS, 4.8 mo vs 3.4 mo vs 1.9 mo | ORR temsirolimus, 22% vs investigator’s choice 2%; P = .0019 |
| Ibrutinib56 | 1-5 prior lines of therapy, prior bortezomib in 48 of 111 | Phase 2 | Ibrutinib 560 mg daily until progression | 111 | ORR, 68% | Median PFS, 13.9 mo (95% CI, 7.0 vs NR) |
| Lenalidomide57 | Prior bortezomib, anthracycline, cyclophosphamide, rituximab | Phase 2 | Lenalidomide 25 mg days 1-21 q28 d until progression | 134 | ORR, 28% | Median PFS, 4.0 mo (95% CI, 3.6-5.6 mo) |
| Agent . | Patient population . | Study design . | Administration . | N . | Primary end point . | Secondary end points . |
|---|---|---|---|---|---|---|
| Bortezomib55 | 1-3 prior lines of therapy | Phase 2 | Bortezomib 1.3 mg/m2 days 1, 4, 8, 11 q21 d until progression | 155 | TTP, 6.2 mo (95% CI, 4.0-6.9 mo) | ORR, 33% |
| CR + CRu, 8% | ||||||
| Bortezomib14 | Previously untreated | Randomized phase 3 | VR-CAP vs R-CHOP ×6-8 cycles | 243 vs 244 | Median PFS, 24.7 mo vs 14.4 mo; HR, 0.63 (95% CI, 0.50-0.79) | Median OS, 56.3 mo vs NR; HR, 0.80 (95% CI, 0.59-1.10) |
| Temsirolimus33 | Previously treated | Randomized phase 3 | Temsirolimus 175/75 mg vs 175/25 mg vs investigator’s choice until progression | 54 vs 54 vs 53 | Median PFS, 4.8 mo vs 3.4 mo vs 1.9 mo | ORR temsirolimus, 22% vs investigator’s choice 2%; P = .0019 |
| Ibrutinib56 | 1-5 prior lines of therapy, prior bortezomib in 48 of 111 | Phase 2 | Ibrutinib 560 mg daily until progression | 111 | ORR, 68% | Median PFS, 13.9 mo (95% CI, 7.0 vs NR) |
| Lenalidomide57 | Prior bortezomib, anthracycline, cyclophosphamide, rituximab | Phase 2 | Lenalidomide 25 mg days 1-21 q28 d until progression | 134 | ORR, 28% | Median PFS, 4.0 mo (95% CI, 3.6-5.6 mo) |
CI, confidence interval; CR, complete response rate; CRu, complete response rate unconfirmed; HR, hazard ratio; NR, not reported; ORR, overall response rate; PFS, progression-free survival; q21, every 21 days; q28, every 28 days; R-CHOP, rituximab, cyclophosphamide, adriamycin, vincristine, prednisone; TTP, time to progression; VR-CAP, bortezomib 1.3 mg/m2 days 1, 4, 8, 11 plus rituximab, cyclophosphamide, adriamycin, prednisone.