Abstract
BACKGROUND: An interim analysis of ROSETTA (jRCT1021230006), a cohort study conducted in Japan, indicated favorable effectiveness and manageable safety of tirabrutinib for relapsed or refractory primary central nervous system lymphoma (r/rPCNSL) in a real-world setting. We analyzed patient characteristics influencing prolonged effectiveness of tirabrutinib. This is an encore presentation including new data of real-world progression-free survival (rwPFS) by the interval from the end of previous treatment. METHODS: We analyzed rwPFS by patient characteristics in 137 patients with r/rPCNSL who started tirabrutinib treatment between November 2020 and April 2022. RESULTS: Patients with one prior treatment (n = 90) had a numerically longer rwPFS compared to those with ≥2 prior treatments (n = 47; median, 16.3 vs 8.2 months), with a hazard ratio (HR) of 1.50 (1 vs ≥2; 95% confidence interval [CI], 0.94–2.39). The median rwPFS in patients with a Karnofsky Performance Status (KPS) score of 80–100 (n = 34; 11.0 months) was similar to that of 50–70 (n = 63; 10.7 months) with a HR of 0.87 (80–100 vs 50–70; 95% CI, 0.51–1.49) but numerically longer than that of 10–40 (n = 15; 2.3 months) with a HR of 1.68 (80–100 vs 10–40; 95% CI, 0.80–3.56). Additionally, the median rwPFS was numerically longer in patients with an interval of ≥1 year from the end of previous treatment to the start of tirabrutinib treatment (n = 46) than in patients with the interval of <1 year (n = 89; 15.6 vs 11.0 months), with a HR of 0.75 (<1 vs ≥1; 95% CI, 0.45–1.24). CONCLUSION: These data showed a tendency for prolonged rwPFS in patients with one prior treatment, a baseline KPS score of ≥50, or the interval of ≥1 year prior to the start of tirabrutinib treatment.