Table 2

Probability of rejecting at either 50 or 100 patients, and average arm size for a worthless treatment (no benefit), and power, and average arm size for a worthwhile treatment (15% improvement in CR) based on a baseline CR rate of 15%

Critical improvement in CR rate at 100 patients (Δ)2, %Critical improvement in CR rate after 50 patients (Δ1)
0%, % (no.)2.5%, % (no.)5%, % (no.)7.5%, % (no.)10%, % (no.)
2.5      
    Probability of rejecting treatment early if worthless* (mean size per arm) 72 (106) — — — — 
    Probability of accepting worthwhile treatment, power (mean size per arm) 93 (195) — — — — 
5      
    Probability of rejecting treatment early if worthless* (mean size per arm) 82 (96) 85 (82) — — — 
    Probability of accepting worthwhile treatment, power (mean size per arm) 92 (194) 89 (188) — — — 
7.5      
    Probability of rejecting treatment early if worthless* (mean size per arm) 93 (85) 93 (74) 94 (68) — — 
    Probability of accepting worthwhile treatment, power (mean size per arm) 88 (188) 85 (184) 83 (179) — — 
10      
    Probability of rejecting treatment early if worthless* (mean size per arm) 97 (81) 97 (70) 97 (65) 97 (61) — 
    Probability of accepting worthwhile treatment, power (mean size per arm) 81 (181) 79 (177) 78 (173) 75 (168) — 
15      
    Probability of rejecting treatment early if worthless* (mean size per arm) > 99.5 (78) > 99.5 (67) > 99.5 (62) > 99.5 (59) > 99.5 (56) 
    Probability of accepting worthwhile treatment, power (mean size per arm)      
72 (145) 71 (143) 70 (140) 68 (137) 64 (133)  
Critical improvement in CR rate at 100 patients (Δ)2, %Critical improvement in CR rate after 50 patients (Δ1)
0%, % (no.)2.5%, % (no.)5%, % (no.)7.5%, % (no.)10%, % (no.)
2.5      
    Probability of rejecting treatment early if worthless* (mean size per arm) 72 (106) — — — — 
    Probability of accepting worthwhile treatment, power (mean size per arm) 93 (195) — — — — 
5      
    Probability of rejecting treatment early if worthless* (mean size per arm) 82 (96) 85 (82) — — — 
    Probability of accepting worthwhile treatment, power (mean size per arm) 92 (194) 89 (188) — — — 
7.5      
    Probability of rejecting treatment early if worthless* (mean size per arm) 93 (85) 93 (74) 94 (68) — — 
    Probability of accepting worthwhile treatment, power (mean size per arm) 88 (188) 85 (184) 83 (179) — — 
10      
    Probability of rejecting treatment early if worthless* (mean size per arm) 97 (81) 97 (70) 97 (65) 97 (61) — 
    Probability of accepting worthwhile treatment, power (mean size per arm) 81 (181) 79 (177) 78 (173) 75 (168) — 
15      
    Probability of rejecting treatment early if worthless* (mean size per arm) > 99.5 (78) > 99.5 (67) > 99.5 (62) > 99.5 (59) > 99.5 (56) 
    Probability of accepting worthwhile treatment, power (mean size per arm)      
72 (145) 71 (143) 70 (140) 68 (137) 64 (133)  

— indicates not applicable.

*

A treatment is rejected early if the critical improvements are not seen.

Cut-offs giving good balance between power and futility size.

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