Table 2.
Key blinatumomab clinical outcomes
| Pivotal study | Study population | Primary outcome | Other key outcomes |
|---|---|---|---|
| NCT02013167 (TOWER) [43] | R/R Ph– BCP-ALL (Adult) | Median OS: 7.7 months (95% CI, 5.6–9.6 months) |
CR within 12 weeks of treatment initiation: 91/267 (34%) (95% CI, 28.0–39.5%; p < 0.001) CRh within 12 weeks of treatment initiation: 119/267 (44%) (95% CI, 37.9–50.0%; p < 0.001) EFS (6-month estimate): 31% MRD remission (defined as an MRD level below 0.0001): 76% Adverse events (grade ≥ 3): 231/267 (87%) |
| NCT01466179 (Study MT103-211) [40] | R/R Ph– BCP-ALL (Adult) | CR or CRh: 81/189 (43%) (95% CI, 36–50%) within the first two cycles of treatment |
Median RFS in patients with CR/CRh: 5.9 months (95% CI, 4.8–8.3 months) Median OS: 6.1 months (95% CI, 4.2–7.5 months) alloHSCT after blinatumomab-induced remission: 32/81 (40%) 100-day mortality following alloHSCT: 11% (95% CI, 0–23%) MRD response: 60/73 (82%) (95% CI, 72–90%) Adverse events (grade ≥ 3): 71 (38%) |
| NCT01207388 (BLAST) [39] | MRD-positive BCP-ALL (Adult) | Complete MRD response: 88/113 (78%) patients after one cycle of treatment |
Median OS: 36.5 months (95% CI, 19.8 months to not estimable) Median RFS: 18.9 months (95% CI, 12.3–35.2 months) Duration of hematologic remission: not reached |
| NCT02000427 (ALCANTARA) [49] | R/R Ph+ BCP-ALL (Adult) | CR or CRh: 16/45 (36%) (95% CI, 22–51%) within the first two cycles of treatment |
Complete MRD response: 14/16 (88%) (95% CI, 62–98%) during the first two cycles of treatment Median RFS: 6.7 months (95% CI, 4.4 months to not estimable) Median OS: 7.1 months (95% CI, 5.6 months to not estimable) alloHSCT after blinatumomab-induced remission: 4/16 (25%) (95% CI, 7–52%) Adverse events (grade ≥ 3): 37/45 (82%) |
| NCT01471782 (Study MT103-205) [29] | R/R BCP-ALL (Pediatric) |
Maximum-tolerated dosage: 15 mg/m2/day CR: 27/70 (39%) (95% CI, 27–51%) |
Median RFS in responders (n = 27): 4.4 months (95% CI, 2.3–7.6 months) Median OS (n = 70): 7.5 months (95% CI, 4.0–11.8 months) alloHSCT after blinatumomab treatment: 24/70 (34%) Complete MRD response (< 10–4): 14/27 (52%) (95% CI, 32–71%) Adverse events (grade ≥ 3): 61 (87%) |
alloHSCT allogeneic hematopoietic stem cell transplant, CI confidence interval, CR complete remission, CRh complete remission with partial hematologic recovery of peripheral blood counts, EFS event-free survival, MRD minimal residual disease, OS overall survival, RFS relapse-free survival, R/R BCP-ALL relapsed or refractory B cell precursor acute lymphoblastic leukemia, R/R Ph+ BCP-ALL relapsed or refractory Philadelphia chromosome-positive B cell precursor acute lymphoblastic leukemia, R/R Ph– BCP-ALL relapsed or refractory Philadelphia chromosome-negative B cell precursor acute lymphoblastic leukemia