ArticleMolecular and clinical oncology2017
Pilot study of sirolimus in patients with PIK3CA mutant/amplified refractory solid cancer.
Article in Molecular and clinical oncology, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.
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Who cites it
12 citing papers in PubMed, 18 citations in OpenAlex.
- Molecular Alterations and Pathways in Intrahepatic Cholangiocarcinoma: Available Evidence and New Perspectives.International journal of molecular sciences · 2025Review
- Immunosuppression in liver transplant oncology: position paper of the Italian Board of Experts in Liver Transplantation (I-BELT).Updates in surgery · 2024Review
- Molecular Targeting of the Phosphoinositide-3-Protein Kinase (PI3K) Pathway across Various Cancers.International journal of molecular sciences · 2024Review
- TMEM16A partners with mTOR to influence pathways of cell survival, proliferation, and migration in cholangiocarcinoma.American journal of physiology. Gastrointestinal and liver physiology · 2023Article
- Identification of stem cell-related subtypes and risk scoring for gastric cancer based on stem genomic profiling.Stem cell research & therapy · 2021Article
- Clinical and Genomic Characteristics of Adult Diffuse Midline Glioma.Cancer research and treatment · 2021Article
- Promising Molecular Targets for the Targeted Therapy of Biliary Tract Cancers: An Overview.OncoTargets and therapy · 2021Review
- Antitumor activity and safety of sirolimus for solid tumors with PIK3CA mutations: A multicenter, open-label, prospective single-arm study (KM 02-01, KCSG UN17-16).Translational cancer research · 2020Article
- Review
- Transcriptomics analysis of sirolimus treatment in lupus nephritis.Molecular medicine reports · 2019Article
- mTOR Inhibitors in Advanced Biliary Tract Cancers.International journal of molecular sciences · 2019Review
- Targeted next generation sequencing identified clinically actionable mutations in patients with esophageal sarcomatoid carcinoma.BMC cancer · 2018Article
Corrections and comments
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Authors and funding
8 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
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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