Real-World (rw) Early Clinical Endpoints for Patients with Resectable Non-Small Cell Lung Cancer (NSCLC): A Database Evaluation Study
Author(s)
Xiaohan Hu, MPH, PhD1, Ashwini Arunachalam, MPH, BDS1, Pragya Rai, PhD1, Chenan Zhang, PhD, MS2, Giovanna I. Cruz, PhD, MS2, Ashleigh McBratney, MS2, Sue Harp, AS2, Ronda Broome, MS, MSHMI2, Radha Krishnan, MD1, Ayman Samkari, MD1;
1Merck & Co., Inc., Rahway, NJ, USA, 2Syapse Holdings Inc., West Chester, PA, USA
1Merck & Co., Inc., Rahway, NJ, USA, 2Syapse Holdings Inc., West Chester, PA, USA
Presentation Documents
OBJECTIVES: With advances in perioperative therapy for resectable NSCLC, including immune checkpoint inhibitors (ICIs), there is need to identify early clinical endpoints in rw datasets analogous to clinical trial endpoints. Our aim was to develop and evaluate database-derived early clinical endpoints for resectable NSCLC.
METHODS: This retrospective database study drew on the Syapse Analytical Dataset, enriched with manual abstraction, comprising electronic medical records of patients treated at US community clinics. We included adults with AJCC v8 stage II/IIIA/IIIB(T3-4N2) or AJCC v7 stage IB/II/IIIA NSCLC first diagnosed 1-January-2016 to 31-December-2021, ECOG performance status 0-1/unknown, who had received neoadjuvant platinum-based chemotherapy (chemo). Patients in clinical trials, with unknown surgical history, or with prior ICI therapy were excluded. Endpoints derived from local pathology reports and clinician’s notes included major pathologic response (rwMPR: ≤10% residual viable tumor in resected tissue) and pathologic complete response (rwpCR: no residual invasive carcinoma in resected tissue [ypT0/Tis ypN0]). Patients were followed through 31-December-2023. Findings were evaluated with respect to published findings for chemo-treated control arms of three perioperative ICI trials for resectable NSCLC (NCT03425643, NCT03800134, NCT04025879).
RESULTS: Median age of 164 eligible patients (44.5% men) in the database was 65 years; most had clinical stage III NSCLC (IIIA/IIIB, 72.0%/6.1%); 53.0% also received neoadjuvant radiotherapy (neoRT). Of 145 (88.4%) who underwent surgery (86.2% lobectomy, 9.7% pneumonectomy, 4.1% other), 89.0% had no residual tumor. Fifty-eight patients (35.4%) had rwMPR; rwpCR occurred in 33 patients (20.1%). By neoRT receipt (yes/no), rwMPR was 50.6%/18.2%; rwpCR was 28.7%/10.4%. Median follow-up was 67.2 months. In the chemo-treated trial control arms, neoRT was not administered; MPR was 11.0%-12.3% and pCR was 4.0%-4.7%.
CONCLUSIONS: Differences in treatment regimens, dosage/duration of neoadjuvant therapy, and patient characteristics, in addition to small sample size, may have contributed to differences observed (versus the three trials) in rwMPR and rwpCR rates. Continued study is planned.
METHODS: This retrospective database study drew on the Syapse Analytical Dataset, enriched with manual abstraction, comprising electronic medical records of patients treated at US community clinics. We included adults with AJCC v8 stage II/IIIA/IIIB(T3-4N2) or AJCC v7 stage IB/II/IIIA NSCLC first diagnosed 1-January-2016 to 31-December-2021, ECOG performance status 0-1/unknown, who had received neoadjuvant platinum-based chemotherapy (chemo). Patients in clinical trials, with unknown surgical history, or with prior ICI therapy were excluded. Endpoints derived from local pathology reports and clinician’s notes included major pathologic response (rwMPR: ≤10% residual viable tumor in resected tissue) and pathologic complete response (rwpCR: no residual invasive carcinoma in resected tissue [ypT0/Tis ypN0]). Patients were followed through 31-December-2023. Findings were evaluated with respect to published findings for chemo-treated control arms of three perioperative ICI trials for resectable NSCLC (NCT03425643, NCT03800134, NCT04025879).
RESULTS: Median age of 164 eligible patients (44.5% men) in the database was 65 years; most had clinical stage III NSCLC (IIIA/IIIB, 72.0%/6.1%); 53.0% also received neoadjuvant radiotherapy (neoRT). Of 145 (88.4%) who underwent surgery (86.2% lobectomy, 9.7% pneumonectomy, 4.1% other), 89.0% had no residual tumor. Fifty-eight patients (35.4%) had rwMPR; rwpCR occurred in 33 patients (20.1%). By neoRT receipt (yes/no), rwMPR was 50.6%/18.2%; rwpCR was 28.7%/10.4%. Median follow-up was 67.2 months. In the chemo-treated trial control arms, neoRT was not administered; MPR was 11.0%-12.3% and pCR was 4.0%-4.7%.
CONCLUSIONS: Differences in treatment regimens, dosage/duration of neoadjuvant therapy, and patient characteristics, in addition to small sample size, may have contributed to differences observed (versus the three trials) in rwMPR and rwpCR rates. Continued study is planned.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
CO202
Topic
Clinical Outcomes
Topic Subcategory
Clinical Outcomes Assessment
Disease
SDC: Oncology