Table 1.
Summary of main clinical trials evaluating the targeted therapies in advanced biliary tract cancer patients
|
Ref.
|
Country
|
Drug(s)
|
Number of patients
|
Study phase
|
ORR (%)
|
Mean OS (months)
|
Mean PFS (months)
|
Adverse events
|
| FGFR2 inhibitors | ||||||||
| Javle et al[44] | United States, Belgium, Spain, Germany, Singapore, Taiwan, and Thailand | Infigratinib (BGJ398) | 108 | 2 | 23.1 | 12.2 | 7.3 | Tiredness, baldness, hyperphosphatemia, and stomatitis |
| Abou-Alfa et al[45] | United States, France, Italy, Germany, Belgium, and South Korea | Pemigatinib (FIGHT-202) | 146 | 2 | 35.5 | 21.1 (FGFR2 fusion) | 6.9 (FGFR2 fusion) | Hypophosphatemia, arthralgia, stomatitis, hyponatremia, and abdominal pain |
| Mazzaferro et al[48] | United States and Italy | Derazantinib (AR087-101) | 29 | 1/2 | 20.7 | 12.7 | 5.7 | Fatigue, eye toxicity, hyperphosphatemia, and increase in ALT/AST |
| Bahleda et al[49] | United States, France, and Spain | Erdafitinib (JNJ-42756493) | 11 | 1 | 27.0 | 12.0 | 7.5 | Fatigue, eye toxicity, hyperphosphatemia, and increase in ALT/AST |
| Goyal et al[51] | United States, France, Spain, United Kingdom, Netherland, Japan, Germany, and South Korea | Futibatinib (TAS-120) | 103 | 2 | 42.0 | 21.7 | 9.0 | Hyperphosphatemia, diarrhea, fatigue, alopecia, and stomatitis |
| IDH inhibitors | ||||||||
| Zhu et al[54] | United States, China, Spain, United Kingdom, Ireland, and South Korea | Ivosidenib (AG-120) | 187 | 3 | 51.0 | 10.3 | 2.7 | Ascites, anemia, increase bilirubin level, and hyponatremia |
| BRAF inhibitors | ||||||||
| Subbiah et al[59] | United States, Denmark, United Kingdom, Austria, France, Italy, Spain, Germany, Netherland, Switzerland, Japan, and South Korea | Trametinib and Dabrafenib | 43 | 2 | 47.0 | 14.0 | 9.0 | Hypertension, reduced white blood cell count, and elevated gamma-glutamyl transferase |
| NTRK inhibitors | ||||||||
| Doebele et al[67] | United States, France, Italy, Spain, Germany, Australia, Hong Kong, Switzerland, Japan, and South Korea | Entrectinib | 54 | 1/2 | 57.0 | 21.0 | 11.0 | Anemia, increased weight, dyspnea, and fatigue |
BRAF: B-Raf gene; FGFR2: Fibroblast growth factor receptor 2; IDH: Isocitrate dehydrogenase; NTRK: Neurotrophic tyrosine receptor kinase; ORR: Objective response rate; OS: Overall survival; PFS: Progression-free survival; ALT: Alanine aminotransferase; AST: Aspartate aminotransferase.