Table 1.
Baseline demographics and clinical characteristics of the Asia region population and overall population for the nal‐IRI+5‐FU/LV and 5‐FU/LV treatment groups (ITT population)
| Asia region population | Overall population | |||||||
|---|---|---|---|---|---|---|---|---|
| Combination therapy | Monotherapy | Combination therapy | Monotherapy | |||||
|
nal‐IRI+ 5‐FU/LV n = 34 |
5‐FU/LV n = 35 |
nal‐IRI n = 50 |
5‐FU/LV n = 48 |
nal‐IRI+ 5‐FU/LV n = 117 |
5‐FU/LV n = 119 |
nal‐IRI n = 151 |
5‐FU/LV n = 149 |
|
| Gender, n (%) | ||||||||
| Female | 16 (47.1) | 13 (37.1) | 23 (46.0) | 18 (37.5) | 48 (41.0) | 52 (43.7) | 64 (42.4) | 68 (45.6) |
| Male | 18 (52.9) | 22 (62.9) | 27 (54.0) | 30 (62.5) | 69 (59.0) | 67 (56.3) | 87 (57.6) | 81 (54.4) |
| Age, y | ||||||||
| Median | 60.0 | 58.0 | 62.5 | 60.0 | 63.0 | 62.0 | 65.0 | 63.0 |
| Min, Max | 43, 81 | 34, 73 | 39, 87 | 34, 78 | 41, 81 | 34, 80 | 31, 87 | 34, 83 |
| Ethnicity, n (%) | ||||||||
| Asian | 34 (100.0) | 35 (100.0) | 50 (100.0) | 48 (100.0) | 34 (29.1) | 36 (30.3) | 52 (34.4) | 50 (33.6) |
| Caucasian | 0 | 0 | 0 | 0 | 72 (61.5) | 76 (63.9) | 89 (58.9) | 92 (61.7) |
| Other | 0 | 0 | 0 | 0 | 11 (9.4) | 7 (5.9) | 10 (6.7) | 7 (4.7) |
| Baseline KPS, n (%)a | ||||||||
| 90‐100 | 22 (64.7) | 22 (62.9) | 31 (62.0) | 29 (60.4) | 69 (59.0) | 57 (47.9) | 86 (57.0) | 76 (51.0) |
| 70‐80 | 12 (35.3) | 13 (37.1) | 19 (38.0) | 19 (39.6) | 45 (38.5) | 61 (51.3) | 65 (43.0) | 72 (48.3) |
| 50‐60 | 0 | 0 | 0 | 0 | 3 (2.6) | 0 | 0 | 0 |
| Missing | 0 | 0 | 0 | 0 | 0 | 1 (0.8) | 0 | 1 (0.7) |
| Baseline CA19‐9, n (%)b | ||||||||
| Median (U/mL) | 475.5 | 4945.0 | 1888.0 | 4101.5 | 1278.0 | 1292.0 | 2189.0 | 1019.0 |
| <40 U/mL | 7/32 (21.9) | 3 (8.6) | 9 (18.0) | 6 (12.5) | 22/114 (19.3) | 23/114 (20.2) | 21/146 (14.4) | 28/144 (19.4) |
| ≥40 U/mL | 25/32 (78.1) | 32 (91.4) | 41 (82.0) | 42 (87.5) | 92/114 (80.7) | 91/114 (79.8) | 125/146 (85.6) | 116/144 (80.6) |
| Missing | 2 (5.9) | 0 | 0 | 0 | 3 (2.6) | 5 (4.2) | 5 (3.3) | 5 (3.4) |
| Baseline albumin, n (%)a | ||||||||
| <4.0 g/dL | 18 (52.9) | 18 (51.4) | 25 (50.0) | 26 (54.2) | 64 (54.7) | 65 (54.6) | 88 (58.3) | 83 (55.7) |
| ≥4.0 g/dL | 16 (47.1) | 17 (48.6) | 25 (50.0) | 22 (45.8) | 53 (45.3) | 54 (45.4) | 63 (41.7) | 66 (44.3) |
| Median (g/dL) | 4.0 | 3.9 | 4.1 | 3.8 | 4.1 | 4.0 | 4.0 | 4.0 |
| Measurable metastatic lesions at baseline, n (%) | ||||||||
| 1 | 9 (26.5) | 8 (22.9) | 11 (22.0) | 8 (16.7) | 19 (16.2) | 22 (18.5) | 36 (23.8) | 26 (17.4) |
| 2 | 13 (38.2) | 15 (42.9) | 23 (46.0) | 22 (45.8) | 49 (41.9) | 58 (48.7) | 63 (41.7) | 72 (48.3) |
| 3 | 3 (8.8) | 6 (17.1) | 7 (14.0) | 9 (18.8) | 22 (18.8) | 15 (12.6) | 22 (14.6) | 21 (14.1) |
| >3 | 1 (2.9) | 2 (5.7) | 0 | 3 (6.3) | 7 (6.0) | 8 (6.7) | 7 (4.6) | 10 (6.7) |
| Anatomical location of lesions at baseline, n (%)c | ||||||||
| Liver | 18 (52.9) | 24 (68.6) | 30 (60.0) | 34 (70.8) | 75 (64.1) | 84 (71.8) | 101 (66.9) | 108 (72.5) |
| Homozygous for UGT1A1*28, n (%) | 1 (2.9) | 0 | 0 | 0 | 7 (6.0) | 9 (7.6) | 7 (4.6) | 13 (8.7) |
| Disease stage at diagnosis, n (%) | ||||||||
| IA | 1 (2.9) | 0 | 0 | 0 | 1 (0.9) | 2 (1.7) | 1 (0.7) | 2 (1.3) |
| IB | 0 | 0 | 2 (4.0) | 0 | 1 (0.9) | 3 (2.5) | 4 (2.6) | 3 (2.0) |
| IIA | 2 (5.9) | 3 (8.6) | 11 (22.0) | 4 (8.3) | 6 (5.1) | 9 (7.6) | 19 (12.6) | 11 (7.4) |
| IIB | 11 (32.4) | 7 (20.0) | 7 (14.0) | 7 (14.6) | 26 (22.2) | 22 (18.5) | 26 (17.2) | 25 (16.8) |
| III | 6 (17.6) | 3 (8.6) | 8 (16.0) | 6 (12.5) | 21 (17.9) | 19 (16.0) | 30 (19.9) | 24 (16.1) |
| IV | 14 (41.2) | 22 (62.9) | 22 (44.0) | 31 (64.6) | 61 (52.1) | 62 (52.1) | 70 (46.4) | 82 (55.0) |
| Primary tumor location, n (%) | ||||||||
| Head only | 23 (67.6) | 18 (51.4) | 30 (60.0) | 23 (47.9) | 70 (59.8) | 65 (54.6) | 92 (60.9) | 77 (51.7) |
| Body only | 3 (8.8) | 8 (22.9) | 6 (12.0) | 11 (22.9) | 12 (10.3) | 19 (16.0) | 16 (10.6) | 26 (17.4) |
| Tail only | 4 (11.8) | 6 (17.1) | 7 (14.0) | 9 (18.8) | 14 (12.0) | 19 (16.0) | 24 (15.9) | 24 (16.1) |
| Multi‐locations incl. head | 0 | 0 | 1 (2.0) | 0 | 6 (5.1) | 4 (3.4) | 7 (4.6) | 4 (2.7) |
| Multi‐locations excl. head | 3 (8.8) | 3 (8.6) | 3 (6.0) | 4 (8.3) | 9 (7.7) | 10 (8.4) | 7 (4.6) | 14 (9.4) |
| Unknown | 0 | 0 | 2 (4.0) | 1 (2.1) | 6 (5.1) | 2 (1.7) | 5 (3.3) | 4 (2.7) |
| Previous lines of metastatic therapy, n (%) | ||||||||
| 0 | 5 (14.7) | 6 (17.1) | 4 (8.0) | 8 (16.7) | 15 (12.8) | 15 (12.6) | 17 (11.3) | 19 (12.8) |
| 1 | 20 (58.8) | 21 (60.0) | 25 (50.0) | 28 (58.3) | 62 (53.0) | 67 (56.3) | 86 (57.0) | 86 (57.7) |
| ≥2 | 9 (26.5) | 8 (22.9) | 21 (42.0) | 12 (25.0) | 40 (34.2) | 37 (31.1) | 48 (31.8) | 44 (29.5) |
| Prior anticancer therapy, n (%)d | ||||||||
| Gemcitabine alone | 13 (38.2) | 12 (34.3) | 13 (26.0) | 33 (68.8) | 53 (45.3) | 55 (46.2) | 67 (44.4) | 66 (44.3) |
| Gemcitabine combination | 21 (61.8) | 23 (65.7) | 37 (74.0) | 33 (68.8) | 64 (54.7) | 64 (53.8) | 84 (55.6) | 83 (55.7) |
| Fluorouracil‐containing | 13 (38.2) | 16 (45.7) | 27 (54.0) | 22 (45.8) | 50 (42.7) | 52 (43.7) | 70 (46.4) | 63 (42.3) |
| Irinotecan‐containing | 1 (2.9) | 3 (8.6) | 3 (6.0) | 3 (6.3) | 12 (10.3) | 17 (14.3) | 17 (11.3) | 17 (11.4) |
| Platinum‐containing | 9 (26.5) | 14 (40.0) | 24 (48.0) | 18 (37.5) | 38 (32.5) | 41 (34.5) | 54 (35.8) | 45 (30.2) |
| Post‐study anticancer therapy, n (%)d | ||||||||
| Received post‐study anticancer therapy | 17 (50.0) | 18 (51.4) | 24 (48.0) | 24 (50.0) | 36 (30.8) | 45 (37.8) | 54 (35.8) | 55 (36.9) |
| Gemcitabine combination | 7 (20.6) | 6 (17.1) | 7 (14.0) | 9 (18.8) | 11 (9.4) | 12 (10.1) | 16 (10.6) | 17 (11.4) |
| Fluorouracil‐containing | 11 (32.4) | 14 (40.0) | 20 (40.0) | 18 (37.5) | 22 (18.8) | 30 (25.2) | 39 (25.8) | 37 (24.8) |
| Irinotecan‐containing | 4 (11.8) | 7 (20.0) | 6 (12.0) | 9 (18.8) | 8 (6.8) | 9 (7.6) | 7 (4.6) | 11 (7.4) |
| Platinum‐containing | 11 (32.4) | 8 (22.9) | 14 (28.0) | 13 (27.1) | 19 (16.2) | 22 (18.5) | 25 (16.6) | 27 (18.1) |
| Other non‐investigational agents | 5 (14.7) | 5 (14.3) | 7 (14.0) | 5 (10.4) | 13 (11.1) | 9 (7.6) | 15 (9.9) | 11 (7.4) |
| Investigational agents | 1 (2.9) | 0 | 0 | 0 | 3 (2.6) | 4 (3.4) | 5 (3.3) | 5 (3.4) |
| Not recorded | 17 (50.0) | 17 (48.6) | 26 (52.0) | 24 (50.0) | 81 (69.2) | 74 (62.2) | 97 (64.2) | 94 (63.1) |
| Median time since last prior anticancer therapy, months (1st and 3rd quartiles) | 1.1 (0.8, 1.4) | 1.0 (0.6, 1.3) | 1.1 (0.7, 1.5) | 1.0 (0.6, 1.4) | 1.4 (0.9, 2.1) | 1.1 (0.9, 2.2) | 1.3 (0.9, 1.9) | 1.2 (0.9, 2.0) |
| Median time since initial diagnosis, months (1st and 3rd quartiles) | 10.2 (5.1, 19.9) | 7.3 (4.8, 10.5) | 12.4 (6.6, 18.2) | 6.9 (3.9, 11.5) | 10.3 (6.2, 17.1) | 10.3 (6.2, 15.1) | 10.4 (6.1, 17.2) | 9.5 (5.7, 14.4) |
| Median time from last study drug exposure to first post‐study anticancer therapy, weeks (1st and 3rd quartiles) | 2.9 (2.4, 3.9) | 2.9 (1.9, 3.8) | 3.7 (3.0, 5.9) | 3.1 (2.9, 4.0) | 3.1 (2.6, 5.5) | 3.6 (2.9, 4.9) | 4.4 (3.1, 5.7) | 3.7 (2.9, 4.9) |
Baseline is defined as the last measurement obtained prior to study drug administration.
Abbreviations: 5‐FU, 5‐fluorouracil; CA19‐9, carbohydrate antigen 19‐9; ITT, intention‐to‐treat; KPS, Karnofsky performance status; LV, leucovorin (folinic acid); nal‐IRI, liposomal irinotecan.
a
KPS and albumin summaries are based on classification per randomization.
b
Includes only patients with a measured CA19‐9 level prior to treatment. In the overall intent‐to‐treat population, data were missing for three patients in the nal‐IRI+5‐FU/LV group and in five patients in the 5‐FU/LV group (enrolled under protocol version 2).
c
Based on lesion locations according to RECIST guidelines v1.1. Includes all measurable and non‐measurable lesions, and all metastatic and non‐metastatic lesions.
d
Columns add up to ≥100% as some patients received more than one prior line of therapy or more than one post‐study treatment anticancer therapy and may therefore be included in more than one category.