. Dose adjustment was possible in case of adverse events. The treated population included 185 patients who received at least 1 infusion of BLINCYTO; the median number of treatment cycles was 2 (range: 1 to 5). Patients who responded to BLINCYTO but later relapsed had the option to be retreated with BLINCYTO. Among treated patients, the median age was 39 years (range: 18 to 79 years), 63 out of 185 (34.1%) had undergone HSCT prior to receiving BLINCYTO, and 32 out of 185 (17.3%) had received more than 2 prior salvage therapies.
The primary endpoint was the complete remission/complete remission with partial hematological recovery (CR/CRh*) rate within 2 cycles of treatment with BLINCYTO. Seventy-seven out of 185 (41.6%) eva luable patients achieved CR/CRh* within the first 2 treatment cycles, with the majority of responses (81%, 62 out of 77) occurring within cycle 1 of treatment. See Table 3 for efficacy results from this study. The HSCT rate among those who achieved CR/CRh* was 39% (30 out of 77).
Table 3. Efficacy Results in Patients ≥ 18 Years of Age With Philadelphia Chromosome-Negative Relapsed or Refractory B-cell precursor Acute Lymphoblastic Leukemia (ALL) * CR (complete remission) was defined as ≤ 5% of blasts in the bone marrow, no evidence of disease, and full recovery of peripheral blood counts (platelets > 100,000/microliter and absolute neutrophil counts [ANC] > 1,000/microliter).
† CRh* (complete remission with partial hematological recovery) was defined as ≤ 5% of blasts in the bone marrow, no evidence of disease, and partial recovery of peripheral blood counts (platelets > 50,000/microliter and ANC > 500/microliter).
‡ MRD (minimal residual disease) response was defined as MRD by PCR < 1 x 10‑4
§ n1: number of patients who achieved MRD response and the respective remission status; n2: number of patients who achieved the respective remission status. Six CR/CRh* responders with missing MRD data were considered as MRD-nonresponders.
¶ DOR (duration of response)/RFS (relapse-free survival) was defined as time since first response of CR or CRh* to relapse or death, whichever is earlier.
N = 185
CR* CRh*† CR/CRh*
n (%)
[95% CI]
60 (32.4)
[25.7–39.7]
17 (9.2)
[5.4–14.3]
77 (41.6)
[34.4–49.1]
MRD response‡
n1/n2 (%)§
[95% CI]
48/60 (80.0)
[67.7–89.2]
10/17 (58.8)
[32.9–81.6]
58/77 (75.3)
[64.2–84.4]
DOR/RFS¶
Median (months) (range)
6.7 (0.46–16.5)
5.0 (0.13–8.8)
5.9 (0.13–16.5)
16. HOW SUPPLIED/STORAGE AND HANDLING
16.1 How SuppliedEach BLINCYTO package (NDC 55513-160-01) contains:
One BLINCYTO 35 mcg single-use vial containing a sterile, preservative-free, white to off-white lyophilized powder and
One IV Solution Stabilizer 10 mL single-use glass vial containing a sterile, preservative-free, colorless to slightly yellow, clear solution. Do not use the IV Solution Stabilizer to reconstitute BLINCYTO.
16.2 Storage and HandlingStore BLINCYTO and IV Solution Stabilizer vials in the original package refrigerated at 2°C to 8°C (36°F to 46°F) an |
