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Expanded Access Program Pralsetinib in Advanced Non-Small Cell Lung Cancer with Rearranged during Transfection (RET)
Youngkyung Jeon1, Hyun Ae Jung1, Sehhoon Park1
1Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
Purpose:
Rearranged during transfection (RET) gene rearrangement is a well-known driver event in non-small cell lung cancer (NSCLC). Pralsetinib is a selective inhibitor of RET kinase and has shown efficacy in oncogenic RET-altered tumors. This study evaluated the efficacy and safety of expanded access program (EAP) use of pralsetinib in pretreated, advanced NSCLC patients with RET rearrangement.
Materials And Methods:
Patients who received pralsetinib as part of the EAP at Samsung Medical Center were evaluated through a retrospective chart review. The primary endpoint was overall response rate (ORR) per the Response Evaluation Criteria in Solid Tumors (RECIST) ver. 1.1 guidelines. Secondary endpoints were duration of response, progression-free survival (PFS), overall survival (OS), and safety profiles.
Results:
Between April 2020 and September 2021, 23 of 27 patients were enrolled in the EAP study. Two patients who were not analyzed due to brain metastasis and two patients whose expected survival was within 1 month were excluded from the analysis. After a median follow-up period of 15.6 months (95% confidence interval [CI], 10.0 to 21.2), ORR was 56.5%, the median PFS was 12.1 months (95% CI, 3.3 to 20.9), and the 12-month OS rate was 69.6%. The most frequent treatment-related adverse events (TRAEs) were edema (43.5%) and pneumonitis (39.1%). A total of 8.7% of patients experienced extra-pulmonary tuberculosis. TRAEs with a common grade of three or worse were neutropenia (43.5%) and anemia (34.8%). Dose reduction was required in nine patients (39.1%).
Conclusion:
Pralsetinib presents a clinical benefit when used in patients with RET-rearranged NSCLC, consistent with a pivotal study.
Insights
Pralsetinib demonstrated significant clinical benefit in pretreated patients with advanced non-small cell lung cancer (NSCLC) harboring RET gene rearrangements. The study confirmed its efficacy and safety in this specific patient population.
Area of Science:
- Oncology
- Medical Genetics
- Pharmacology
Background:
- Rearranged during transfection (RET) gene rearrangement is a key driver in non-small cell lung cancer (NSCLC).
- Pralsetinib, a selective RET kinase inhibitor, has shown promise in treating tumors with oncogenic RET alterations.
Purpose of the Study:
- To evaluate the efficacy and safety of pralsetinib via an expanded access program (EAP).
- To assess pralsetinib use in pretreated, advanced NSCLC patients with RET rearrangement.
Main Methods:
- Retrospective chart review of patients receiving pralsetinib through an EAP.
- Primary endpoint: Overall Response Rate (ORR) using RECIST ver. 1.1.
- Secondary endpoints: Duration of response, Progression-Free Survival (PFS), Overall Survival (OS), and safety.
Main Results:
- An Overall Response Rate (ORR) of 56.5% was observed.
- Median Progression-Free Survival (PFS) was 12.1 months, with a 12-month Overall Survival (OS) rate of 69.6%.
- Common treatment-related adverse events included edema (43.5%) and pneumonitis (39.1%); Grade 3+ TRAEs included neutropenia (43.5%) and anemia (34.8%).
Conclusions:
- Pralsetinib shows clinical benefit in patients with RET-rearranged NSCLC.
- Findings are consistent with previous pivotal study results, supporting its use in this patient group.
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