Supplementary materials: Comparative effectiveness analysis between entrectinib clinical trial and crizotinib real-world data in ROS1+ NSCLC

dataset

posted on 2024-01-03, 15:59 authored by Robert C. Doebele, Laura Perez, Huong Trinh, Michael Martinec, Reynaldo Martina, Todd Riehl, Matthew G. Krebs, Neal J Meropol, William B Wong, Gracy Crane

These are supplementary materials for the article 'Comparative effectiveness analysis between entrectinib clinical trial and crizotinib real-world data in ROS1+ NSCLC' published in the Journal of Comparative Effectiveness Research.

Supplementary Materials
o Censoring
o Statistical methods
o Sensitivity analyses
o Unadjusted results
o Propensity score
Supplementary Table 1. Censoring rules for TTD, PFS, and OS.
Supplementary Table 2. Propensity score matching.
Supplementary Table 3. Comparison of results of the Cox pro-portional hazard model using BICR or investigator-assessed progression in the trial.
Supplementary Table 4. Landmark analyses at 1 and 3 months for TTD and PFS.
Supplementary Table 5. Sensitivity analysis of the TTD.
Supplementary Table 6. Subgroup analysis by CNS status: unad-justed TTD and PFS.
Supplementary Table 7. Multivariate Cox model of treatment effect by CNS status.
Supplementary Figure 1. Swimmer plot of patients receiving treatment beyond progression (TBP) in the crizotinib cohort (37 out of 65 patients received TBP).

Summary: Aim: Generating direct comparative evidence in prospective randomized trials is difficult for rare diseases. Real-world cohorts may supplement control populations. Methods: Entrectinib-treated adults with advanced ROS1 fusion-positive NSCLC (n = 94) from Phase I/II trials (ALKA-372-001 [EudraCT2012-00148-88], STARTRK-1 [NCT02097810], and STARTRK-2 [NCT02568267]) were compared with a real-world crizotinib-treated cohort (n = 65). Primary end point, time-to-treatment discontinuation (TTD); secondary end points, PFS and OS. Results: Median (95% CI) weighted TTD: 12.9 (9.9–17.4) months for entrectinib; 8.2 (6.2–9.9) months for crizotinib (weighted hazard ratio: 0.72 [0.51–1.02]). Median OS with entrectinib was not reached, weighted median OS with crizotinib was 18.5 (15.1–47.2) months. Conclusion: Entrectinib administered in clinical trials may be associated with longer TTD than a real-world crizotinib population.