Table 3.
Overview of results – Tisagenlecleucel vs. SOC.
| Tisagenlecleucel vs. SOC | ||
|---|---|---|
| FAS | Naïve comparison | Adjusted comparison |
| N(Tisagenlecleucel) = 209 | N(Tisagenlecleucel) = 201 | |
| N(SOC) = 302 | N(SOC) = 273 | |
| OS | 0.38 (0.29–0.49); <0.001 | 0.47 (0.35–0.62); <0.001 |
| Univariate Cox-Regression | ||
| HR (95 % CI); p-value | ||
| Defined as the time from the date of treatment start to the date of death due to any cause. | ||
| ORR* | 4.95 (3.30–7.43); <0.001 | 3.34 (2.14–5.19); <0.001 |
| Logistic Regression | ||
| OR (95% CI) | ||
| Defined as the proportion of patients with a best overall disease response of CR or CRi. | ||
| EFSa* | 0.67 (0.52–0.86); 0.001 | n.a. |
| Univariate Cox-Regression | ||
| HR (95 % CI); p-value | ||
| Defined as the time from the date of treatment start to the date of relapse, death due to any cause after remission or treatment failure, whatever occurs first. | ||
| RFSb | 0.77 (0.51–1.18); 0.233 | n.a. |
| Univariate Cox-Regression | ||
| HR (95 % CI); p-value | ||
| Defined as time from achievement of CR or CRi whatever occurs first to date of relapse or death due to any cause during CR or CRi. | ||
| ITT | Naïve comparison | Adjusted comparison |
| N(Tisagenlecleucel) = 243 | N(Tisagenlecleucel) = 236 | |
| N(SOC) = 302 | N(SOC) = 281 | |
| OS | 0.44 (0.34–0.56); <0.001 | 0.54 (0.41–0.71); <0.001 |
| Univariate Cox-Regression | ||
| HR (95% CI); p-value | ||
| SOC: Defined as the time from the date of treatment start to the date of death due to any cause | ||
| Tisagenlecleucel: Defined as the time from the date of enrollment to the date of death due to any cause. | ||
| ORR* | 2.76 (1.94–3.94); <0.001 | 1.99 (1.33–2.97); <0.001 |
| Logistic Regression | ||
| OR (95% CI) | ||
| Defined as the proportion of patients with a best overall disease response of CR or CRi. | ||
CI Confidence interval, CR Complete remission, CRi CR with incomplete blood count recovery, EFS Event-free survival, FAS Full analysis set, HR Hazard ratio, ITT Intention to treat, n.a. not available, OR Odds ratio, ORR Overall remission rate, OS Overall survival, RFS Relapse-free survival, SOC Standard of care.
aData not available in the GMALL dataset (Frankfurt). Patients from this registry were included but censored at day 1.
bOnly data from the ALL-REZ BFM registry (Berlin) was included.
*missings SOC, naïve N = 3.
p-value: based on Log-Rank Test; p-value (ORR): based on Z-test.