Summary
Among the 250 cases reviewed, 222 patients had confirmed MDS diagnosis and 192 received HMA therapy, including 57 treated with oral DEC-C, 98 with parenteral AZA, and 37 with parenteral DEC, as confirmed through chart review. The year of initial MDS diagnosis of the 192 patients ranged from 2005 to 2024, with 91 (47.3%) patients diagnosed on or after 2020. The median age was 71 years, with 38% of patients being female, 73% White, and 90% receiving care in a community setting. Approximately 13% of the 192 patients had an ECOG performance status of 2 or higher, and 23% had a Charlson Comorbidity Index (CCI) score of 2 or higher. Among the 57 patients who received oral DEC-C as their first HMA, 79% experienced dose modifications, with 65% undergoing delayed start of subsequent treatment cycle, 39% having decreased dosing durations, and 56% discontinuing treatment. Of the 98 patients treated with parenteral AZA as their first HMA, 95% had dose modifications, including 65% with delayed start of subsequent treatment cycle, 24% with decreased dosing durations, 9% with dose reductions, and 90% discontinuing treatment. Among the 37 patients who received parenteral DEC as their first HMA, 75% experienced dose modifications, with 51% having delayed start of subsequent treatment cycle, 16% having decreased dosing durations, 8% undergoing dose reductions, and 54% discontinuing treatment (Figure 1).
Methods
This study utilized data collected between 2012 and 2023 from 2 US databases (COTA NSCLC VANTAGE, ConcertAI Patient360 NSCLC) to create a real-world cohort. Patients aged 18 years or older with locally advanced/metastatic NSCLC, without prior systemic treatment, with ECOG PS 0-1 (when available), and confirmed exon20ins were included. Progression free survival (PFS), time to next treatment (TTNT), and overall survival (OS) of ACP from PAPILLON trial were compared to the pooled real-world physician choice (RWPC), using inverse probability weighting of the average treatment effect in the treated (IPW-ATT) adjustment for potential confounders.
Results
A total of 94 real-world patients were obtained. Median follow-up was 50.5 months; 59.6% were female, 54.3% were over 65 years, 36.2% reported a history of smoking, 26.6% had liver metastases, and 33.0% had brain metastases. 1L treatments included platinum-based chemotherapy + immunotherapy (35.1%), EGFR TKI alone (25.5%), platinum-based chemotherapies (16.0%), immunotherapy alone (6.4%), or others (17.0%). Based on IPW-ATT adjustment, ACP showed statistically significant improvement vs pooled RWPC with HRs (95% CI) of 0.37 (0.25, 0.55), 0.34 (0.22, 0.52), and 0.43 (0.26, 0.70) for PFS, TTNT, and OS, respectively.
Conclusions
Building on the findings from the PAPILLON trial, the superior efficacy demonstrated by ACP compared to RWPC in this study further supports its adoption as the new standard of care for 1L patients with advanced NSCLC harboring EGFR exon20ins mutations.
