- Nanoparticle albumin-bound (nab)-paclitaxel combined with gemcitabine is well-tolerated and shows efficacy in patients with metastatic cholangiocarcinoma.
- The combination failed to meet the alternative hypothesis of 70% improvement in PFS.
Why this matters
- Paclitaxel has the potential to improve gemcitabine efficacy by inhibiting the metabolizing enzyme; the nanoparticle formulation reduces toxicity.
- Findings suggest that the new combination could be an acceptable alternative to standard therapy with gemcitabine plus a platinum analog.
- Single-arm, phase 2 trial of 74 patients (median age, 62 [range, 36-87] years, 44 women) receiving nab-paclitaxel plus gemcitabine.
- Median number of cycles, 6 (range, 1-18).
- Median follow-up, 10.2 (range, 0.6-27.3) months.
- Primary endpoint: PFS (null and alternative hypotheses of 55% and 70%, respectively).
- Funding: Celgene.
- PFS at 6 months was 61% (95% CI, 48%-73%), failing to meet the alternate hypothesis of 70%.
- Median PFS, 7.7 months (95% CI, 5.4-13.1).
- Median OS, 12.4 months (95% CI, 9.2-15.9).
- Median time to progression, 7.7 months (95% CI, 6.1-13.1).
- Confirmed best overall response rate, 30%. Confirmed best disease control rate, 66%.
- 97% of patients experienced a treatment-related adverse event (AE) (grade ≥3, 88%).
- Most common AEs: fatigue (70%), neutropenia (68%), peripheral neuropathy (55%).
- Uncontrolled study.
- Small sample size.