Comparison of tumor assessments using RECIST 1.1 and irRECIST, and association with overall survival

Abstract

BackgroundPatients treated with immune checkpoint inhibitors (ICIs) may experience pseudoprogression, which can be classified as progressive disease (PD) by Response Evaluation Criteria in Solid Tumors (RECIST) V.1.1 and could lead to inappropriate treatment discontinuation. Immune-response criteria were developed to better capture novel response patterns seen with ICIs.MethodsWe pooled data from 1765 patients with 12 types of advanced solid tumors treated with avelumab (an anti-programmed death ligand 1 (PD-L1) monoclonal antibody) monotherapy in the JAVELIN Solid Tumor and JAVELIN Merkel 200 trials, conducted a comparative analysis of tumor assessments by investigators according to RECIST 1.1 and immune-related RECIST (irRECIST), and evaluated the correlation between progression-free survival (PFS) and overall survival (OS).ResultsIn total, 147 patients (8.3%) had a best overall response (BOR) of PD by RECIST 1.1 but had immune-related disease control by irRECIST (defined as immune-related BOR (irBOR) of immune-related stable disease or better). This discordance was seen irrespective of PD-L1 status and observed across all tumor types. Overall, PFS and immune-related PFS showed similar imputed rank correlations with OS.ConclusionsThe use of irRECIST identified a subset of patients with a BOR of PD by RECIST 1.1 but an irBOR of immune-related disease control by irRECIST with a distinctive survival curve, thereby providing more clinically relevant information than RECIST 1.1 alone. However, as a surrogate endpoint for OS in the whole population, immune-related PFS by irRECIST did not show improved predictive value compared with PFS by RECIST 1.1.

Occurrence of hyperprogressive disease following administration of immune checkpoint inhibitors in lung squamous cell carcinoma: A case report

“…However, unlike cytotoxic drugs, ICIs may lead to rapid disease progression rather than benefitting the patient during treatment, which in turn may result in reduced survival time and even mortality (20,21). At present, although no unified evaluation criteria for HPD have been published, the immune-related RECIST criteria are recommended to evaluate the efficacy of ICIs (22). Among the various standards describing HPD in available literature, the present study selected the method described by Russo et al (23), which is mostly used for patients with NSCLC receiving ICI treatment.…”

Expert consensus on perioperative treatment for non-small cell lung cancer

“…Therefore, appropriate alternative endpoints are needed to evaluate the efficacy of therapy in the early stage of cancer, which can be quickly introduced into the clinical practice ( 38 ). The most commonly used endpoints in neoadjuvant and adjuvant therapy studies include pCR, MPR, EFS, and DFS ( 39 - 41 ). One meta-analysis found DFS to be a valid surrogate endpoint for OS with adjuvant chemotherapy and radiotherapy in resectable early-stage NSCLC ( 42 ).…”

Assessment and Monitoring of Response to Systemic Treatment in Advanced Hepatocellular Carcinoma: Current Insights

“…However, despite being reported in up to 15.8% of the patients in trials with ICIs, pseudoprogression in HCC is overall a rare event (<10%). [36][37][38][39] The main differences between the RECIST v1.1, mRECIST, and iRECIST are summarized in Table 1.…”

“…[5][6][7]12,38 Therefore, such discrepancies between the endpoints recapitulating tumor response and survival improvement hamper their value as surrogate endpoints in advanced HCC. 39 The evaluation of treatment efficacy is strongly related to the assumption that changes in tumor burden would eventually translate into survival outcomes. Regardless of the criteria adopted, tumor growth and metastatic spread clearly indicate poor prognosis since they may eventually lead to death.…”

“… 5–7 , 12 , 38 Therefore, such discrepancies between the endpoints recapitulating tumor response and survival improvement hamper their value as surrogate endpoints in advanced HCC. 39 …”