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Biliary Tract Cancer · Biomarker · Relapsed / Refractory

MyPathway (HER2-positive biliary tract cohort)

In previously treated HER2-positive metastatic biliary tract cancer, is pertuzumab plus trastuzumab active in a molecular basket cohort?

Trial design and population

Population / eligibility
39 evaluable patients with previously treated metastatic biliary tract cancer with HER2 amplification and/or overexpression.
Intervention vs comparator
Pertuzumab plus trastuzumab in a non-randomized phase IIa basket cohort.
Sample size
39
Primary endpoint
Objective response rate

Key results

Nine of 39 patients had a partial response: ORR 23% (95% CI 11–39). Median follow-up was 8.1 months. Grade 3–4 treatment-emergent adverse events occurred in 46%.

Source-reported outcome visualization

Objective response rate · Best overall response

Pertuzumab + trastuzumab
23%

Bars display source-reported values for the named endpoint; where the endpoint is a median, bars compare medians and do not represent probabilities. No unreported rates are inferred.

Absolute Effect

Single-arm response rate; no comparator-based absolute benefit or NNT can be calculated.

NNT: Not applicable — single-arm cohort. No NNT is derived from median survival/PFS differences.

NNH / harm: Grade 3–4 treatment-emergent adverse events 46%, commonly elevated ALT/AST; three patients had treatment-related grade 3 events. No treatment-related deaths reported.

NNT/NNH are only displayed where the endpoint and time horizon support a defensible absolute-risk calculation. Otherwise, the source-reported outcome is visualized.

Safety and treatment burden

Grade 3–4 treatment-emergent adverse events 46%, commonly elevated ALT/AST; three patients had treatment-related grade 3 events. No treatment-related deaths reported.

Follow-up, maturity and limitations

Small non-randomized molecularly selected cohort; HER2 testing definitions and tumour heterogeneity matter. This is a basket cohort, not a dedicated randomized BTC trial.

Clinical interpretation

MyPathway provides proof-of-activity for dual HER2 blockade in HER2-positive BTC; interpret alongside later HER2-directed studies and current regulatory/guideline status.

Current-practice context · reviewed 20 October 2026

This page separates original trial findings from contemporary treatment context. Confirm current disease-specific guidelines, local regulatory status, biomarker requirements, prior treatment exposure and patient-specific contraindications before applying the evidence. Historical comparators may no longer represent current standard practice; subgroup findings and immature endpoints should not be generalized.

Primary and authoritative sources

Javle M, et al. Lancet Oncol. 2021;22:1290–1300. Source ↗
ClinicalTrials.gov NCT02091141 Source ↗