Safety and effectiveness of selpercatinib in patients with RET fusion-positive non-small cell lung cancer in real-world clinical practice: a postmarketing study in Japan.

BACKGROUND

This study assessed the safety and effectiveness of selpercatinib, a selective rearranged during transfection (RET) kinase inhibitor, in patients with RET fusion-positive non-small cell lung cancer (NSCLC) in real-world clinical practice in Japan.

METHODS

This single-arm, multicenter, prospective observational study included patients with RET fusion-positive NSCLC who received at least one dose of selpercatinib between February 2022 and October 2023. Data were extracted from medical records and entered into an electronic data capture (eDC) system. Safety (adverse events [AEs] and AEs of special interest [AESIs]) and effectiveness (tumor response, overall survival [OS]) were assessed over 12 months.

RESULTS

Among 243 patients (median age: 67 years; 56% females), AEs occurred in 86.0% of patients, with Grade ≥ 3 AEs in 48.1% and serious AEs (SAEs) in 24.7%. The most common AEs (≥10%) included hypertension (23.5%), abnormal hepatic function (21.0%), rash (11.1%), aspartate aminotransferase increase (10.3%), and diarrhea (10.3%). AEs led to treatment modifications, including dose interruption (54.7%), dose reduction (14.8%), and discontinuation (6.2%). AESIs included liver injury (44.0%), hypertension-related events (23.9%), and hypersensitivity (MedDRA preferred term; 9.9%). The overall response rate was 58.8%, comprising complete response in 4.5% and partial response in 54.3% of patients. Median OS was not reached; the 12-month survival rate was 80.9% (95% CI, 74.9-85.6).

CONCLUSIONS

Real-world data showed selpercatinib to be effective in patients with RET fusion-positive NSCLC in Japan, with a favorable safety profile and no new safety concerns.