Background
Methods
Study design and participants
Ethics, consent, and permissions
Treatments and endpoints
HRQL assessments
HRQL statistical analyses
Results
Key patient characteristics
Summary of the efficacy and safety results
Patient-reported FACT-Leu and EQ-5D outcomes
Idelalisib/rituximab/bendamustine, N = 207 | n | Placebo/rituximab/bendamustine, N = 209 | n | |
---|---|---|---|---|
FACT-Leu total scorea | 125.27 (24.103) | 196 | 123.17 (27.540) | 202 |
Trial outcome index scoreb | 86.12 (18.662) | 196 | 84.71 (21.337) | 203 |
Physical well-being | 21.77 (5.012) | 197 | 21.39 (5.418) | 203 |
Social/family well-being | 21.51 (5.502) | 198 | 21.40 (5.392) | 203 |
Emotional well-being | 17.68 (4.215) | 198 | 16.93 (4.902) | 204 |
Functional well-being | 17.99 (6.068) | 199 | 17.32 (6.135) | 204 |
Leukemia-specific symptoms | 46.31 (10.312) | 199 | 45.95 (12.206) | 204 |
EQ-5D utility index | 0.78 (0.217) | 197 | 0.78 (0.228) | 195 |
EQ-VAS | 68.8 (17.81) | 190 | 67.4 (19.28) | 194 |
FACT-Leu
Idelalisib/bendamustine/rituximab, N = 207 | Placebo/bendamustine/rituximab, N = 209 | |||||||
---|---|---|---|---|---|---|---|---|
Patients with MID improvementa | Time to symptom improvementb | Proportion of patients with any symptom improvementc | Patients with MID improvementa | Time to symptom improvementb | Proportion of patients with any symptom improvementc | HR (95% CI) | P-value | |
PWB | 97 (69.3) | 12.3 (9.1, 16.1) N = 140 | 139 (67.1) | 89 (61.8) | 20.9 (12.9, 30.1) N = 144 | 141 (67.5) | 1.28 (0.96, 1.70) | 0.1026 |
S/FWB | 82 (59.0) | 20.4 (12.1, 39.9) N = 139 | 130 (62.8) | 79 (52.7) | 32.4 (16.3, 72.7) N = 150 | 139 (66.5) | 1.20 (0.88, 1.63) | 0.2663 |
EWB | 99 (62.7) | 16.1 (8.9, 23.9) N = 158 | 159 (76.8) | 103 (61.7) | 16.9 (12.4, 24.4) N = 167 | 147 (70.3) | 1.04 (0.79, 1.37) | 0.8357 |
FWB | 102 (60.0) | 20.9 (12.1, 39.9) N = 170 | 142 (68.6) | 100 (55.2) | 24.7 (16.1, 44.3) N = 181 | 145 (69.4) | 1.07 (0.81, 1.42) | 0.6321 |
LeuS | 142 (74.7) | 8.4 (6.3, 12.7) N = 190 | 168 (81.2) | 133 (68.6) | 12.3 (11, 16.3) N = 194 | 168 (80.4) | 1.22 (0.96, 1.55) | 0.1134 |
EQ-5D
Idelalisib/rituximab/bendamustine, N = 207 | Placebo/rituximab/bendamustine, N = 209 | |||||
---|---|---|---|---|---|---|
Dimensions | Baseline | Week 24 | Week 48 | Baseline | Week 24 | Week 48 |
Anxiety/Depression | ||||||
Level 1 | 113 (57.1) | 102 (65.8) | 85 (66.4) | 117 (59.1) | 91 (60.3) | 70 (66.7) |
Level 2 | 84 (42.4) | 50 (32.3) | 41 (32.0) | 75 (37.9) | 55 (36.4) | 32 (30.5) |
Level 3 | 1 (0.5) | 3 (1.9) | 2 (1.6) | 6 (3.0) | 5 (3.3) | 3 (2.9) |
Mobility | ||||||
Level 1 | 145 (73.6) | 119 (76.8) | 94 (73.4) | 142 (71.4) | 112 (74.7) | 84 (80.0) |
Level 2 | 52 (26.4) | 36 (23.2) | 34 (26.6) | 55 (27.6) | 38 (25.3) | 21 (20.0) |
Level 3 | 0 | 0 | 0 | 2 (1.0) | 0 | 0 |
Pain/Discomfort | ||||||
Level 1 | 105 (53.3) | 96 (61.9) | 77 (60.2) | 114 (57.0) | 79 (52.3) | 63 (60.6) |
Level 2 | 85 (43.1) | 54 (34.8) | 51 (39.8) | 81 (40.5) | 69 (45.7) | 37 (35.6) |
Level 3 | 7 (3.6) | 5 (3.2) | 0 | 5 (2.5) | 3 (2.0) | 4 (3.8) |
Self-Care | ||||||
Level 1 | 184 (92.9) | 136 (87.7) | 115 (89.8) | 184 (92.5) | 131 (86.8) | 92 (87.6) |
Level 2 | 14 (7.1) | 19 (12.3) | 13 (10.2) | 13 (6.5) | 20 (13.2) | 12 (11.4) |
Level 3 | 0 | 0 | 0 | 2 (1.0) | 0 | 1 (1.0) |
Usual Activities | ||||||
Level 1 | 126 (63.6) | 93 (60.4) | 83 (64.8) | 122 (61.3) | 87 (57.6) | 71 (68.3) |
Level 2 | 65 (32.8) | 52 (33.8) | 45 (35.2) | 70 (35.2) | 61 (40.4) | 31 (29.8) |
Level 3 | 7 (3.5) | 9 (5.8) | 0 | 7 (3.5) | 3 (2.0) | 2 (1.9) |
Treatment differencea LSM (95% CI) | ||
---|---|---|
EQ-5D UI | EQ-VAS | |
Week 4 | 0.03 (−0.01, 0.07) | 0.18 (−3.24, 3.61) |
P-value | 0.1302 | 0.9167 |
Week 8 | −0.01 (−0.05, 0.04) | −1.54 (−5.06, 1.98) |
P-value | 0.7570 | 0.3895 |
Week 12 | 0.00 (−0.04, 0.05) | −1.00 (−4.96, 2.97) |
P-value | 0.8730 | 0.6216 |
Week 16 | 0.02 (−0.03, 0.07) | −0.09 (−3.82, 3.65) |
P-value | 0.4325 | 0.9631 |
Week 20 | 0.02 (−0.03, 0.06) | 0.17 (−3.74, 4.09) |
P-value | 0.4514 | 0.9301 |
Week 24 | 0.04 (−0.01, 0.08) | −0.04 (−3.92, 3.85) |
P-value | 0.1433 | 0.9851 |
Week 30 | 0.03 (−0.02, 0.08) | 0.69 (−3.36, 4.73) |
P-value | 0.2915 | 0.7389 |
Week 36 | 0.02 (−0.03, 0.07) | 1.94 (−2.24, 6.13) |
P-value | 0.4630 | 0.3623 |
Week 42 | 0.00 (−0.06, 0.06) | −1.56 (−5.92, 2.80) |
P-value | 0.9995 | 0.4823 |
Week 48 | 0.02 (−0.04, 0.07) | 1.99 (−2.06, 6.03) |
P-value | 0.5601 | 0.3348 |
Week 60 | 0.03 (−0.03, 0.09) | 1.53 (−3.05, 6.11) |
P-value | 0.2981 | 0.5128 |
Week 72 | 0.04 (−0.04, 0.11) | −0.97 (−6.41, 4.48) |
P-value | 0.3131 | 0.7275 |
Week 84 | −0.04 (− 0.11, 0.02) | 0.03 (−6.14, 6.20) |
P-value | 0.2039 | 0.9921 |