Table 1.

Baseline characteristics of tisa-cel (ELARA) vs usual care (ReCORD-FL) cohorts

ELARA enrolled (N = 97)Before weightingAfter weighting
ReCORD-FL (N = 143)|SMD|ReCORD-FL (N = 99)SMD
Included in the PS model 
Age at index treatment initiation, mean (SD), y 56.5 (10.40) 60.1 (11.7) 0.326 56.1 (11.5) 0.038 
Gender, n (%)      
Female 33 (34.0) 61 (42.7) 0.178 30.8 (31.1) 0.063 
Male 64 (66.0) 82 (57.3) 0.178 68.3 (68.9) 0.063 
Region, n (%)      
Europe 44 (45.4) 90 (62.9) 0.358 41.4 (41.8) 0.072 
Rest of world 53 (54.6) 53 (37.1) 0.358 57.6 (58.2) 0.072 
Previous auto-HSCT, n (%)      
Yes 36 (37.1) 53 (37.1) 0.001 36.1 (36.5) 0.013 
No 61 (62.9) 90 (62.9) 0.001 62.9 (63.5) 0.013 
Number of previous lines of systemic treatment      
Median 0.117 0.104 
Minimum-maximum 2-13 2-10  2-10  
Disease stage at initial FL diagnosis, n (%)      
Stage I 6 (6.2) 10 (7.0) 0.033 4.7 (4.7) 0.064 
Stage II 13 (13.4) 13 (9.1) 0.137 9.5 (9.6) 0.12 
Stage III 21 (21.6) 26 (18.2) 0.087 25.4 (25.7) 0.095 
Stage IV 57 (58.8) 94 (65.7) 0.144 59.4 (60.0) 0.026 
Mo between initial FL diagnosis and initiation of index treatment      
Median 66.2 61.7 0.099 69.7 0.005 
Minimum-maximum 6.4-355.4 2.8-255  2.8-255  
Number of involved nodal sites at index treatment initiation, n (%)      
≤ 4 39 (40.2) 74 (51.7) 0.233 38.1 (38.5) 0.035 
> 4 58 (59.8) 69 (48.3) 0.233 60.9 (61.5) 0.035 
Double refractory, n (%)      
Yes 66 (68.0) 97 (67.8) 0.004 67.8 (68.5) 0.01 
No 31 (32.0) 46 (32.2) 0.004 31.2 (31.5) 0.01 
POD24, n (%)      
Yes 61 (62.9) 86 (60.1) 0.056 62.7 (63.3) 0.009 
No 36 (37.1) 57(39.9) 0.056 36.3 (36.7) 0.009 
Not included in PS model  
Refractory status to last preceding therapy, n (%)      
Yes 75 (77.3) 112 (78.3) 0.024 79.2 (80.0) 0.066 
No 21 (21.6) 31 (21.7) 0.001 19.8 (20.0) 0.041 
Missing 1 (1.0) 0.144 0.144 
FLIPI at index treatment initiation, n (%)      
High 59 (60.8) 80 (55.9) 0.099 56.6 (57.2) 0.074 
Intermediate 20 (20.6) 21 (14.7) 0.156 14.3 (14.5) 0.162 
Low 18 (18.6) 15 (10.5) 0.23 9.7 (9.8) 0.252 
Missing 27 (18.9) 0.682 18.3 (18.5) 0.673 
ELARA enrolled (N = 97)Before weightingAfter weighting
ReCORD-FL (N = 143)|SMD|ReCORD-FL (N = 99)SMD
Included in the PS model 
Age at index treatment initiation, mean (SD), y 56.5 (10.40) 60.1 (11.7) 0.326 56.1 (11.5) 0.038 
Gender, n (%)      
Female 33 (34.0) 61 (42.7) 0.178 30.8 (31.1) 0.063 
Male 64 (66.0) 82 (57.3) 0.178 68.3 (68.9) 0.063 
Region, n (%)      
Europe 44 (45.4) 90 (62.9) 0.358 41.4 (41.8) 0.072 
Rest of world 53 (54.6) 53 (37.1) 0.358 57.6 (58.2) 0.072 
Previous auto-HSCT, n (%)      
Yes 36 (37.1) 53 (37.1) 0.001 36.1 (36.5) 0.013 
No 61 (62.9) 90 (62.9) 0.001 62.9 (63.5) 0.013 
Number of previous lines of systemic treatment      
Median 0.117 0.104 
Minimum-maximum 2-13 2-10  2-10  
Disease stage at initial FL diagnosis, n (%)      
Stage I 6 (6.2) 10 (7.0) 0.033 4.7 (4.7) 0.064 
Stage II 13 (13.4) 13 (9.1) 0.137 9.5 (9.6) 0.12 
Stage III 21 (21.6) 26 (18.2) 0.087 25.4 (25.7) 0.095 
Stage IV 57 (58.8) 94 (65.7) 0.144 59.4 (60.0) 0.026 
Mo between initial FL diagnosis and initiation of index treatment      
Median 66.2 61.7 0.099 69.7 0.005 
Minimum-maximum 6.4-355.4 2.8-255  2.8-255  
Number of involved nodal sites at index treatment initiation, n (%)      
≤ 4 39 (40.2) 74 (51.7) 0.233 38.1 (38.5) 0.035 
> 4 58 (59.8) 69 (48.3) 0.233 60.9 (61.5) 0.035 
Double refractory, n (%)      
Yes 66 (68.0) 97 (67.8) 0.004 67.8 (68.5) 0.01 
No 31 (32.0) 46 (32.2) 0.004 31.2 (31.5) 0.01 
POD24, n (%)      
Yes 61 (62.9) 86 (60.1) 0.056 62.7 (63.3) 0.009 
No 36 (37.1) 57(39.9) 0.056 36.3 (36.7) 0.009 
Not included in PS model  
Refractory status to last preceding therapy, n (%)      
Yes 75 (77.3) 112 (78.3) 0.024 79.2 (80.0) 0.066 
No 21 (21.6) 31 (21.7) 0.001 19.8 (20.0) 0.041 
Missing 1 (1.0) 0.144 0.144 
FLIPI at index treatment initiation, n (%)      
High 59 (60.8) 80 (55.9) 0.099 56.6 (57.2) 0.074 
Intermediate 20 (20.6) 21 (14.7) 0.156 14.3 (14.5) 0.162 
Low 18 (18.6) 15 (10.5) 0.23 9.7 (9.8) 0.252 
Missing 27 (18.9) 0.682 18.3 (18.5) 0.673 

Weights, such as those applied to our control group (ie, the ReCORD-FL sample), can reduce the precision of statistical estimates such that the analyses behave as though we have a smaller-than-actual sample of controls. The ESS quantifies this reduction of precision in the controls. Thus, the larger the reduction from actual to effective sample size, the more statistical precision that is lost via weighting.

Auto-HSCT, autologous HSCT; ESS, effective sample size; FLIPI, Follicular Lymphoma International Prognostic Index; SD, standard deviation.

Enrolled patients are those who met inclusion or exclusion criteria and had a leukapheresis product accepted for manufacturing, regardless of infusion status (only 1 enrolled patient was not included owing to missing data).

Sample size after weighting (ie, sum of weights) was 99 for the ReCORD-FL study, and effective sample size was 95.

Because double refractoriness and last prior therapy refractory status both capture refractoriness status, only 1 prognostic factor (double refractoriness) was included in the propensity model. Furthermore, refractory status to last preceding therapy was already very well balanced (SMD < 0.25) before weighting, suggesting that the inclusion or exclusion of this prognostic factor has a limited impact on the analysis. FLIPI was excluded from the model on the basis of missingness (27 [19%] additional patients would be excluded from the analysis if FLIPI were included in the model). Considering that 3 of the 5 risk factors of the FLIPI score are already included in the model (age, number of nodal sites, and disease stage), which achieved excellent balance with absolute SMDs < 0.25, FLIPI was also well balanced between the cohorts before weighting, and thus, to conserve sample size, FLIPI was excluded.

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