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Efficacy and safety of pimitespib in gastrointestinal tumors
Kunihiko Kawai1, Tsuyoshi Takahashi2, Yukinori Kurokawa1
1Department of Gastroenterological Surgery, Osaka University Graduate School of Medicine, 2-2, Yamadaoka, Suita City, 565-0871, Osaka, Japan.
Background:
The aim of this study was to assess the efficacy and safety of pimitespib in real-world clinical settings for tyrosine kinase inhibitor-resistant GISTs.
Methods:
We retrospectively analyzed 15 patients at Osaka University Hospital. Patients treated with pimitespib as a fourth- or later-line treatment for unresectable or recurrent GISTs were included. Patient background, pimitespib dose, number of courses, adverse events, best response, progression-free survival, and overall survival were analyzed.
Results:
The median overall survival of patients treated with pimitespib was 24.4 months（95% confidence interval [CI] 7.2-not reached) and the one-year overall survival rate was 70.9%. The median progression-free survival was 4.6 months (95%CI 1.8-7.4), and the one-year progression-free survival rate was 15.2%. A partial response was observed in 0 patients, stable disease lasting for ≥ 12 weeks in 7 (46.7%), progressive disease in 7 (40.0%), and a non-evaluable state in 2 (13.3%). The disease control rate was 40%. Treatment-related adverse events were reported in all patients, with the most common being diarrhea (87%), followed by anorexia (27%), nausea (27%), and increased blood creatinine levels (27%). None of the patients discontinued the treatment because of adverse events.
Conclusion:
Pimitespib may offer a tolerable and effective treatment option for patients with unresectable or recurrent GISTs in clinical practice.
Insights
Pimitespib shows potential as a tolerable treatment for advanced gastrointestinal stromal tumors (GISTs) resistant to tyrosine kinase inhibitors. Real-world data suggest it offers survival benefits in patients with unresectable or recurrent GISTs.
Area of Science:
- Oncology
- Gastrointestinal Oncology
- Pharmacology
Background:
- Gastrointestinal stromal tumors (GISTs) often develop resistance to tyrosine kinase inhibitors (TKIs).
- Novel therapeutic strategies are needed for patients with advanced, unresectable, or recurrent GISTs.
- Pimitespib is a potential treatment option for TKI-resistant GISTs.
Purpose of the Study:
- To evaluate the real-world efficacy and safety of pimitespib.
- To assess pimitespib's role in patients with TKI-resistant GISTs.
- To analyze survival outcomes and adverse events associated with pimitespib treatment.
Main Methods:
- Retrospective analysis of 15 patients with unresectable or recurrent GISTs treated with pimitespib.
- Pimitespib was administered as a fourth- or later-line treatment.
- Data collected included patient demographics, pimitespib dosage, treatment duration, adverse events, and clinical response.
Main Results:
- Median overall survival was 24.4 months; one-year OS rate was 70.9%.
- Median progression-free survival was 4.6 months; one-year PFS rate was 15.2%.
- Disease control rate was 40%; 46.7% achieved stable disease for ≥ 12 weeks. All patients experienced treatment-related adverse events, primarily diarrhea (87%), but none discontinued treatment.
Conclusions:
- Pimitespib demonstrates tolerability and potential efficacy in clinical practice for GIST patients.
- It represents a viable treatment option for those with unresectable or recurrent GISTs.
- Further investigation into pimitespib's role in GIST treatment is warranted.
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