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Yang et al. Hepatoma Res 2023;9:48 https://dx.doi.org/10.20517/2394-5079.2023.68 Page 7 of 21

Javle et al. 2a MyPathway/NCT02091141 39 BTCs (HER2- Pertuzumab plus 23 (95%CI, 11- 10.8 (95%CI, 0.7- 4.0 (95%CI, 1.8-5.7); 10.9 (95%CI, 5.2-15.6);
[54]
(2021) (7) positive) Trastuzumab 39); 25.4); iCCA: NA iCCA: 2.6 (95%CI, 1.0- iCCA: 3.9 (95%CI, 1.2-
(iCCA) iCCA: 0 5.3) 8.1)
Harding et al. 2 NCT01953926 25 BTCs (HER2 Neratinib 16 (95%CI, 4.5- Four PR: 3.0; 3.7 2.8 (95%CI, 1.1-3.7) 5.4 (95%CI, 3.7-11.7)
[56]
(2023) (6) mut) 36.1) (censored); 3.8;
(iCCA) 4.7.
Ohba et al. 2 JMA-IIA00423 32 BTCs (HER2- Trastuzumab HER2-positive: NA HER2-positive: 4.4 HER2-positive: 7.1
[55]
(2022) (24;8) positive; -low) deruxtecan 36.4 (95%CI, (95%CI, 2.8-8.3); (95%CI, 4.7-14.6);
(3) (iCCA) 19.6-56.1); HER2-low: 4.2 (95%CI, HER2-low: 8.9 (95%CI,
HER2-low: 12.5 1.3-6.2) 3.0-12.8)
(95%CI, 0.3-
52.7)
Meric- 1 NCT02892123 20 BTCs (HER2- Zanidatamab 47 (95%CI, 23- 6.6 (95%CI, 3.2- NA NA
Bernstam et al. (5) positive) 72) NE)
[57]
(2022) (iCCA)
RET
Subbiah et al. 1/2 ARROW/NCT03037385 29 solid tumors Pralsetinib 57 (95%CI, 35- 11.7 (95%CI, 5.5- 7.4 (95%CI, 5.1-13.6) 23.5 (95%CI, 19.8-23.9)
[69]
(2022) (3) (RET fus)(CCA) 77) 19.0)
Cocco et al. 1/2 LIBRETTO-001/NCT03157128 45 solid tumors Selpercatinib 43·9 (95%CI, 24.5 (95%CI, 9.2- 13.2 (95%CI, 7.4-26.2)* 18.0 (95%CI, 10.0-NE)*
[71]
(2018) (1) (RET fus)(CCA) 28.5-60.3); NR); CCA: 5.6
CCA: 100.0 (95%CI, NR-NR)*
(95%CI, 2.5-
100.0)*

NTRK
Hong et al. 1/2 NCT02122913; NCT02637687; 159 (2) solid tumors Larotrectinib 79 (95%CI, 72- 35.2 (95%CI, 22.8- 28·3 (95%CI 22-1-NE) 44·4 (95%CI 36·5-NE)
[75]
(2020) NCT02576431 (TRK fus) 85); CCA: 50 NE); CCA: 7.5
(CCA) (95%CI, 1-99)* (95%CI, NE-NE)*
Demetri et al. 2 ALKA-372-001/ EudraCT 2012-000148- 150 solid tumors Entrectinib 61.3 (95%CI, 20.0 (95%CI, 13.2- 13.8 (95%CI, 10.1-20.0) 37.1 (95%CI, 27.2-NE)
[76]
(2022) 88; STARTRK-1/ NCT02097810; (NTRK fus) 53.1-69.2) 31.1)
STARTRK-2/ NCT02568267

MSI-H/dMMR
Andre et al. 1 GARNET/NCT02715284 106 solid tumors Dostarlimab 38.7 (95%CI, NR NA NA
[83]
(2021) (1) (dMMR) 29.4-48.6);
(CCA) CCA: CR 100
Maio et al. 2 KEYNOTE-158/ NCT02628067 351 solid tumors Pembrolizumab 30.8 (95%CI, 47.5 (95%CI, 2.1+- NA NA-CCA: 19.4 (95%CI,
[82]
(2022) (22) (dMMR) 25.8-36.2); 51.1+); CCA: 4.2 (95%CI, 2.1- 6.5-NR)*
(CCA) CCA: 40.9 CCA: 30.6 (95%CI, 24.9)*
(95%CI, 20.7- 6.2-40.5+)*
63.6)*

*Evaluated by independent review committee. NE: not estimable; NA: not available; NR: not reached; IQR: interquartile range; CCA: cholangiocarcinoma; iCCA: intrahepatic cholangiocarcinoma; BTC: biliary tract
cancer; FGFR: fibroblast growth factor receptor; IDH: isocitrate dehydrogenase-2; HER2 (ERBB2): human epidermal growth factor receptor 2; NTRK: neurotrophic tyrosine receptor kinase; dMMR: deficient DNA
mismatch repair; MSI-H: high microsatellite instability.