Table 1.
Baseline characteristics of patients treated with selpercatinib (LIBRETTO-001 trial) and the real-world control, before and after entropy balancing
| Characteristic | Selpercatinib cohort (LIBRETTO-001) | First-line setting, before entropy balancing |
First-line setting, after entropy balancinga |
||||
|---|---|---|---|---|---|---|---|
| Real-world control Analytic Strategy 1 (RET fusion positive)a | Real-world control Analytic Strategies 2 and 3 | Real-world control sensitivity analysis | Selpercatinib cohort (LIBRETTO-001) | Real-world control Analytic Strategies 2 and 3 | Real-world control sensitivity analysis | ||
| N | 48 | 29 | 2791 | 985 | 48 | 2791 | 985 |
| Sex, n (%) | |||||||
| Female | 29 (60.4) | 13 (44.8) | 1132 (40.6) | 369 (37.5) | 29 (60.4) | 1686 (60.4) | 595 (60.4) |
| Male | 19 (39.6) | 16 (55.2) | 1659 (59.4) | 616 (62.5) | 19 (39.6) | 1105 (39.6) | 390 (39.6) |
| Age, mean (SD) | 62.2 (14.1) | 65.6 (11.0) | 68.8 (9.5) | 68.1 (9.3) | 62.2 (14.1) | 62.2 (13.5) | 62.2 (14.1) |
| Body weight, mean kg (SD) | 71.7 (18.1) | 75.8 (16.3) | 75.6 (19.3) | 75.8 (18.6) | 71.7 (18.1) | 71.7 (19.7) | 71.7 (20.0) |
| ECOG performance status, n (%) | |||||||
| 0 | 20 (41.7) | 7 (24.1) | 733 (26.3) | 282 (28.6) | 20 (41.7) | 1163 (41.7) | 410 (41.7) |
| >0 | 28 (58.3) | 15 (51.7) | 1556 (55.8) | 528 (53.6) | 28 (58.3) | 1628 (58.3) | 575 (58.3) |
| Missing | 0 | 7 (24.1) | 502 (18.0) | 175 (17.8) | 0 | 0 | 0 |
| History of smoking, n (%) | |||||||
| Yes | 14 (29.2) | 13 (44.8) | 2537 (90.9) | 888 (90.2) | 14 (29.2) | 814 (29.2) | 287 (29.2) |
| No | 34 (70.8) | 16 (55.2) | 253 (9.1) | 97 (9.8) | 34 (70.8) | 1977 (70.8) | 698 (70.8) |
| Missing | 0 | 0 | 1 (0.0) | 0 | 0 | 0 | 0 |
| Stage at initial diagnosis, n (%) | |||||||
| I-III | 4 (8.3) | 8 (27.6) | 1126 (40.3) | 213 (21.6) | 4 (8.3) | 291 (10.4) | 103 (10.4) |
| IV | 43 (89.6) | 21 (72.4) | 1600 (57.3) | 754 (76.5) | 43 (89.6) | 2500 (89.6) | 882 (89.6) |
| Missing | 1 (2.1) | 0 | 65 (2.3) | 18 (1.8) | 1 (2.1) | 0 | 0 |
| Tumor histology, n (%) | |||||||
| Non-squamous | 43 (89.6) | 29 (100.0) | 1571 (56.3) | 647 (65.7) | 43 (89.6) | 2500 (89.6) | 882 (89.6) |
| Squamous | 0 | 0 | 1075 (38.5) | 285 (28.9) | 0 | 291 (10.4) | 103 (10.4) |
| NOS | 5 (10.4) | 0 | 145 (5.2) | 53 (5.4) | 5 (10.4) | 0 | 0 |
| Presence of brain metastases, n (%) | 3 (6.3) | 5 (17.2) | 415 (14.9) | 178 (18.1) | 3 (6.3) | 174 (6.2) | 62 (6.3) |
| Time from advanced/metastatic diagnosis to index therapy, mean (SD) days | 62.2 (46.5) | 115.7 (277.3) | 105.2 (327.7) | 71.0 (235.7) | 62.2 (46.5) | 62.2 (127.0) | 62.2 (271.2) |
| Characteristic | Selpercatinib cohort (LIBRETTO-001, PAS) | Post-progression setting, before entropy balancing |
Post-progression setting, after entropy balancinga |
||||
|---|---|---|---|---|---|---|---|
| Real-world control Analytic Strategy 1a | Real-world control Analytic Strategies 2 and 3 | Real-world control sensitivity analysis | Selpercatinib cohort (LIBRETTO-001, PAS) | Real-world control Analytic Strategies 2 and 3 | Real-world control sensitivity analysis | ||
| N | 105 | 17 | 1503 | 720 | 105 | 1503 | 720 |
| Sex, n (%) | |||||||
| Female | 62 (59.0) | 10 (58.8) | 619 (41.2) | 305 (42.4) | 62 (59.0) | 887 (59.0) | 425 (59.0) |
| Male | 43 (41.0) | 7 (41.2) | 884 (58.8) | 415 (57.6) | 43 (41.0) | 616 (41.0) | 295 (41.0) |
| Age, mean (SD) | 58.6 (11.9) | 65.3 (11.8) | 68.2 (9.5) | 68.5 (9.4) | 58.6 (11.9) | 58.6 (15.9) | 58.6 (11.0) |
| Body weight, mean kg (SD) | 67.8 (17.1) | 70.2 (12.8) | 75.0 (18.7) | 75.1 (19.6) | 67.8 (17.1) | 67.8 (17.3) | 67.79 (17.0) |
| ECOG performance status, n (%) | |||||||
| 0 | 31 (29.5) | 2 (11.8) | 311 (20.7) | 145 (20.1) | 31 (29.5) | 444 (29.5) | 213 (29.5) |
| >0 | 74 (70.5) | 12 (70.6) | 1019 (67.8) | 499 (69.3) | 74 (70.5) | 1059 (70.5) | 507 (70.5) |
| Missing | 0 | 3 (17.6) | 173 (11.5) | 76 (10.6) | 0 | 0 | 0 |
| History of smoking, n (%) | |||||||
| Yes | 30 (28.6) | 9 (52.9) | 1336 (88.9) | 650 (90.3) | 30 (28.6) | 429 (28.6) | 206 (28.6) |
| No | 75 (71.4) | 8 (47.1) | 167 (11.1) | 70 (9.7) | 75 (71.4) | 1074 (71.4) | 514 (71.4) |
| Stage at initial diagnosis, n (%) | |||||||
| I-III | 4 (3.8) | 4 (23.5) | 659 (43.8) | 291 (40.4) | 4 (3.8) | 57 (3.8) | 27 (3.8) |
| IV | 101 (96.2) | 13 (76.5) | 807 (53.7) | 410 (56.9) | 101 (96.2) | 1446 (96.2) | 693 (96.2) |
| Missing | 0 | 0 | 37 (2.5) | 19 (2.6) | 0 | 0 | 0 |
| Tumor histology, n (%) | |||||||
| Non-squamous | 92 (87.6) | 17 (100.0) | 829 (55.2) | 382 (53.1) | 92 (87.6) | 1317 (87.6) | 631 (87.6) |
| Squamous | 1 (1.0) | 0 | 605 (40.3) | 307 (42.6) | 1 (1.0) | 186 (12.4) | 89 (12.4) |
| NOS | 12 (11.4) | 0 | 69 (4.6) | 31 (4.3) | 12 (11.4) | 0 | 0 |
| Presence of brain metastases, n (%) | 11 (10.5) | 3 (17.6) | 262 (17.4) | 129 (17.9) | 11 (10.5) | 157 (10.5) | 75 (10.5) |
| Time from metastatic diagnosis to index therapy, mean (SD) days | 856.5 (692.5) | 690.9 (665.3) | 470.5 (575.6) | 443.6 (557.6) | 856.5 (692.5) | 856.5 (1217.7) | 856.5 (941.0) |
Analytic Strategy 1: patients with RET fusion-positive disease; Analytic Strategy 2: all patients, regardless of RET fusion status; Analytic Strategy 3: all patients, regardless of RET fusion status and applying the RET adjustment factor; sensitivity analysis: all patients who had received prior platinum-based therapy.
ECOG, Eastern Cooperative Oncology Group; NOS, not otherwise stated; PAS, Primary analysis set; RET, REarranged during Transfection; SD, standard deviation.
a
Due to the small sample size, entropy balancing could not be applied to Analytic Strategy 1.