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Soo Park, MD
Soo Park, MD
Posted: Monday, March 17, 2025
Patients with melanoma brain metastases have historically been underrepresented in clinical trials, creating a need for standardized response assessment criteria tailored to brain metastases. In CheckMate 204, modified RECIST (mRECIST) and radiographic-only Response Assessment in Neuro-Oncology Brain Metastases (RANO-BM 5 mm) were determined to be reliable assessment scales for trials.
Raymond Y. Huang, MD, PhD, of Brigham and Women’s Hospital, Harvard Medical School, Boston, and colleagues compared multiple intracranial response criteria to determine the optimal method. mRECIST and volumetric criteria demonstrated higher intracranial objective response rates and stronger correlations with intracranial progression-free and overall survival than RANO-BM or RECIST v1.1.
A total of 119 patients with at least one unirradiated melanoma brain metastasis received the PD-1 inhibitor nivolumab and the CTLA-4 inhibitor ipilimumab every 3 weeks for four cycles, followed by maintenance nivolumab every 2 weeks for up to 24 months. Blinded independent central review was used to assess intracranial objective response rates with multiple response criteria—mRECIST, RECIST v1.1, RANO-BM (5-mm and 10-mm cutoffs), and volumetric criteria (5 mm and 10 mm). Intracranial progression-free survival and overall survival were analyzed based on response at a 6-week landmark.
Intracranial objective response rates were highest with mRECIST (45%) and volumetric (40%) criteria, whereas RECIST v1.1 and RANO-BM yielded lower response rates (27%–34%). Responders identified by mRECIST and volumetric criteria demonstrated the strongest correlation with improved intracranial progression-free and overall survival.
Of note, patients classified as responders by mRECIST—but nonresponders by RANO-BM 5 mm—exhibited overall survival comparable to that of RANO-BM responders. Volumetric response criteria further demonstrated a strong predictive value for survival outcomes.
Further research is needed to specifically evaluate the added value of mRECIST compared with RANO-BM and RECIST v1.1 in assessing brain metastases, particularly in symptomatic patients, noted the investigators. “These results also suggest that volumetric response assessments using spherical lesion assumptions have considerable potential and should be evaluated in future studies.”
Disclosure: The National Cancer Institute and Bristol Myers Squibb provided support. For full disclosures for the study authors, visit ascopubs.org.
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