Table 1. Prospective trials of promising targeted therapies in BTC
| Targetable pathway | Study | Drug | Total N (N with FGFR fusions/alterations) | Results* (overall population, FGFR fusion population, FGFR alteration** population) |
|---|
| FGFR | Javle et al. 2018 (19) | Infigratinib (BGJ398) | 61 (48 FGFR2 fusions, 11 FGFR2 alterations**) | Overall population: |
| • ORR 14.8% |
| • DCR 75.4% |
| • PFS 5.8 months |
| FGFR2 fusion cohort: |
| • ORR 18.8% |
| • DCR 83.3% |
| FGFR altered** cohort: |
| • ORR 0% |
| Mazzaferro et al.2019 (20), Droz Dit Bussett, et al. 2019 (21) | Derazantinib (ARQ 087) | 44 (29 FGFR2 fusions, 6 FGFR alterations, 9 no alteration) | FGFR fusion cohort: |
| • ORR 20.7% |
| • DCR 82.8% |
| • PFS 5.7 |
| FGFR altered cohort: |
| • ORR 0% |
| • DCR 67% |
| • PFS 6.7 mo |
| No FGFR alteration** cohort: |
| • ORR 0% |
| • DCR 22% |
| • PFS 1.5 mo |
| Park et al. 2019 (22) | Erdafitinib (JNJ42756493) | 17 (11 FGFR 2/3 fusions, 6 FGFR alterations) | Overall population: |
| • ORR 47% |
| • DCR 80% |
| • PFS 5.6 months |
| FGFR2/3 fusion cohort: |
| • ORR 67% |
| • DCR 100% |
| • PFS 12.65 months |
| FGFR altered** cohort: |
| • ORR 16.67% |
| Vogel et al. 2019 (23) | Pemigatinib (INCB054828) | 146 (107 FGFR2 fusions, 20 FGFR alterations, 18 no alteration) | FGFR2 fusion cohort: |
| • ORR 35.5% |
| • DCR 82% |
| • PFS 6.9 months |
| • OS 21 months |
| FGFR altered** cohort: |
| • ORR 0% |
| • PFS 2.1 months |
| No FGF/FGFRalteration cohort: |
| • ORR 0% |
| • DCR 22% |
| • PFS 1.7 mo |
| Tran et al. 2018 (24) | TAS-120 | 45 (28 FGFR2 fusions, 17 FGF/FGFR alterations**) | FGFR2 fusion cohort: |
| • ORR 25% |
| • DCR 78.6% |
| • PFS 7.4 months |
| FGFR altered** cohort: |
| • ORR 17.6% |
| • DCR 76.4% |
| • PFS 6.8 months |
| IDH 1/2 | Lowery et al. 2019 (25) | Ivosidenib (AG-120) | 73 | • ORR 5% |
| • DCR 61% |
| • PFS 3.8 months |
| • OS 13.8 months |
| Abou-Alfa et al. 2019 (26) | Ivosidenib (AG-120) | 185 | • ORR 2.4% |
| • DCR 53% |
| • PFS 2.7 months |
| • OS 10.8 months |
| BRAFV600E | Wainberg et al. 2019 (27) | Dabrafenib/Trametinib | 33 | • ORR 41% |
| • PFS 7.2 months |
| • OS 11.3 months |
| PI3K/AKT/mTOR | Lau et al. 2018 (28) | Everolimus | 27 | • ORR 12% |
| • DCR 48% |
| • PFS 5.5 months |
| • OS 9.5 months |
| VEGF | Sun et al. 2019 (29) | Regorafenib | 43 | • ORR 11% |
| • DCR 56% |
| • PFS 15.6 weeks |
| • OS 31.8 weeks |
*, as reported per study population and publication; **, other alterations include mutations and amplifications in FGFR. N, number of enrolled subjects; ORR, overall response rate; DCR, disease control rate; PFS, median progression-free survival; OS, median overall survival; FGFR, fibroblast growth factor receptor; IDH, isocitrate dehydrogenase; BRAF, v-Raf murine sarcoma viral oncogene homolog B; MEK, mitogen-activated protein kinase; PI3K, phosphoinositide 3-kinase; AKT, protein kinase B; mTOR, mammalian target of rapamycin; VEGF, vascular endothelial growth factor.
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