Open Access

Pilot study of sirolimus in patients with PIK3CA mutant/amplified refractory solid cancer

  • Authors:
    • Ki Sun Jung
    • Jeeyun Lee
    • Se Hoon Park
    • Joon Oh Park
    • Young Suk Park
    • Ho Yeong Lim
    • Won Ki Kang
    • Seung Tae Kim
  • View Affiliations

  • Published online on: May 29, 2017     https://doi.org/10.3892/mco.2017.1272
  • Pages: 27-31
  • Copyright: © Jung et al. This is an open access article distributed under the terms of Creative Commons Attribution License.

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Abstract

In patients with refractory cancer, the effect of additional chemotherapy is very limited. Targeted agents for molecular pathways associated with cancer cell progression and survival have emerged as attractive options in several cancer types. The current pilot study assessed the efficacy and safety of sirolimus in patients with refractory cancer with PIK3CA mutation/amplification. Refractory cancer patients with PIK3CA mutation/amplification were enrolled, irrespective of tumor‑types. Enrolled patients received a daily dose of 1 mg sirolimus and one cycle defined as 28 days. An assessment of the efficacy and safety of sirolimus was performed. Overall, 4 patients were enrolled between October 2014 and April 2015. The median of 2.5 cycles of sirolimus was administered. Three patients had advanced gastric cancer and one had advanced cholangiocarcinoma. The overall response rate was 0%, three patients (75%) had stable disease following one cycle and one patient (25%) received sirolimus for 4 cycles without disease progression. The median progression free survival was 1.9 months [95% confidence interval (CI), 0.3‑3.5 months], and the median overall survival was 3.6 months (95% CI, 0.4‑6.8 months). Grade 3 or greater hematologic/non‑hematologic toxicity was not observed. Grade 1 nausea was reported in one patient each. There were no treatment‑associated mortalities. Sirolimus had modest efficacy and a tolerable toxicity‑profile in patients with refractory cancer with PIK3CA mutation/amplification.
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July 2017
Volume 7 Issue 1

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APA
Jung, K., Lee, J., Park, S., Park, J., Park, Y., Lim, H. ... Kim, S. (2017). Pilot study of sirolimus in patients with PIK3CA mutant/amplified refractory solid cancer. Molecular and Clinical Oncology, 7, 27-31. https://doi.org/10.3892/mco.2017.1272
MLA
Jung, K., Lee, J., Park, S., Park, J., Park, Y., Lim, H., Kang, W., Kim, S."Pilot study of sirolimus in patients with PIK3CA mutant/amplified refractory solid cancer". Molecular and Clinical Oncology 7.1 (2017): 27-31.
Chicago
Jung, K., Lee, J., Park, S., Park, J., Park, Y., Lim, H., Kang, W., Kim, S."Pilot study of sirolimus in patients with PIK3CA mutant/amplified refractory solid cancer". Molecular and Clinical Oncology 7, no. 1 (2017): 27-31. https://doi.org/10.3892/mco.2017.1272