Table 1.
Summary of best available therapies received by real-world patients, overall and by line of therapy.
| Overalla | First line | Second line | Third or later lines | |
|---|---|---|---|---|
| Number of unique patients | N = 141 | N = 118 | N = 69 | N = 35 |
| Total number of lines of therapy included | N = 222 | N = 118 | N = 69 | N = 35 |
| Total number of lines of therapy contributed by patient | ||||
| Mean (SD) | 1.6 (0.9) | – | – | – |
| Median (min, max) | 1.0 (1.0, 7.0) | – | – | – |
| Number of lines of therapy contributed, n (%) | ||||
| 1 | 86 (61.0%) | – | – | – |
| 2 | 40 (28.4%) | – | – | – |
| ≥3 | 15 (10.6%) | – | – | – |
| Year of line of therapy start date, n (%) | ||||
| 2009–2013 | 66 (29.7%) | – | – | – |
| 2014–2017 | 99 (44.6%) | – | – | – |
| 2018–2021 | 57 (25.7%) | – | – | – |
| Agents used in each included line of therapy, n (%) | ||||
| TKI therapy | 120 (54.1%) | 71 (60.2%) | 34 (49.3%) | 15 (42.9%) |
| Cytoreductive therapy | 91 (41.0%) | 39 (33.1%) | 33 (47.8%) | 19 (54.3%) |
| Biologic therapy | 25 (11.3%) | 14 (11.9%) | 8 (11.6%) | 3 (8.6%) |
| Agent-level information availableb | N = 196 | N = 107 | N = 59 | N = 30 |
| TKI | ||||
| Midostaurin | 99 (50.5%) | 58 (54.2%) | 29 (49.2%) | 12 (40.0%) |
| Ripretinib | 4 (2.0%) | 2 (1.9%) | 0 (0.0%) | 2 (6.7%) |
| Ibrutinib | 3 (1.5%) | 3 (2.8%) | 0 (0.0%) | 0 (0.0%) |
| Dasatinib | 2 (1.0%) | 1 (0.9%) | 1 (1.7%) | 0 (0.0%) |
| Imatinib | 2 (1.0%) | 1 (0.9%) | 0 (0.0%) | 1 (3.3%) |
| Cytoreductive therapy | ||||
| Cladribine | 49 (25.0%) | 20 (18.7%) | 18 (30.5%) | 11 (36.7%) |
| Hydroxyurea | 17 (8.7%) | 10 (9.3%) | 5 (8.5%) | 2 (6.7%) |
| Azacitidine | 3 (1.5%) | 0 (0.0%) | 2 (3.4%) | 1 (3.3%) |
| Biologic | ||||
| Interferon-alfa | 11 (5.6%) | 9 (8.4%) | 2 (3.4%) | 0 (0.0%) |
| Pegylated interferon | 8 (4.1%) | 3 (2.8%) | 4 (6.8%) | 1 (3.3%) |
| Brentuximab vedotin | 4 (2.0%) | 2 (1.9%) | 2 (3.4%) | 0 (0.0%) |
| Gemtuzumab ozogamicin | 1 (0.5%) | 0 (0.0%) | 0 (0.0%) | 1 (3.3%) |
BAT best available therapy, ECOG Eastern Cooperative Oncology Group, max maximum, min minimum, SD standard deviation, TKI tyrosine kinase inhibitor.
aThe BAT cohort was restricted to patients with available ECOG score during any time before to 3 months after the index date.
bAgent-level information for prior treatments was reported among patients from all study sites except Medical University of Vienna (Austria) (N = 26 lines of therapy), where only treatment class information was collected per local regulations.