EVALUATING THE TRANSPORTABILITY OF EFFICACY ESTIMATES IN RELAPSED/REFRACTORY MANTLE-CELL LYMPHOMA
Author(s)
Georg Hess, MD1, Lianne Barnieh, PhD2, Sonia Zebachi, PhD3, Antoine Movschin, MSc3, Paul Loustalot, MSc3, Anke Ohler, MD1, Leyla Mohseninejad, MSc, PhD4.
1Medical School of the Johannes Gutenberg-University, Mainz, Germany, 2BeOne Medicines, Versailles, France, 3Quinten Health, Paris, France, 4BeiGene, The Hague, Netherlands.
1Medical School of the Johannes Gutenberg-University, Mainz, Germany, 2BeOne Medicines, Versailles, France, 3Quinten Health, Paris, France, 4BeiGene, The Hague, Netherlands.
OBJECTIVES: Relapsed or refractory mantle cell lymphoma (R/R MCL) is a rare and aggressive B-cell malignancy associated with poor prognosis and limited treatment options after failure of prior lines of therapies (LoT). Zanubrutinib was evaluated in BGB-3111-206, a phase II single-arm trial, in 86 patients with previously treated MCL following at least one prior LoT (NCT03206970). As this trial was conducted exclusively in China, this current analysis evaluated the transportability of the efficacy outcomes from the 206 trial to a European population.
METHODS: The target population was derived from the European MCL Network Registry, a consortium of 15 national lymphoma study groups across Europe and defined by replicating the inclusion and exclusion criteria of the BGB-3111-206 trial. To evaluate efficacy of zanubrutinib in the target population, overall response rate (ORR) was estimated through a matching-adjusted indirect comparison (MAIC) by reweighting trial patients to match the baseline characteristics of the target population. A simulated treatment comparison (STC) was also performed by modeling the relationship between baseline covariates and patient response in the trial population and predicting response in the target population. Baseline covariates included age, sex, number of prior LoT, simplified MCL international prognostic index, blastoid morphology, and progressive disease to last LoT. Sensitivity analyses included using an extended set of covariates (for both the MAIC and STC), trimming of weights for MAIC and predictive performance metrics were computed for STC.
RESULTS: The ORR in BGB-3111-206 (84%; 95% confidence interval [CI]: 74%-91%) was comparable to both the estimated ORR for the target European population via the MAIC (79%; 95% CI: 55%-92%) and the STC (78%; 95% CI: 57%-94%). Findings were consistent across all sensitivity analyses.
CONCLUSIONS: This analysis provides evidence that the treatment effects of zanubrutinib observed in BGB-3111-206 are applicable to a European population with R/R MCL.
METHODS: The target population was derived from the European MCL Network Registry, a consortium of 15 national lymphoma study groups across Europe and defined by replicating the inclusion and exclusion criteria of the BGB-3111-206 trial. To evaluate efficacy of zanubrutinib in the target population, overall response rate (ORR) was estimated through a matching-adjusted indirect comparison (MAIC) by reweighting trial patients to match the baseline characteristics of the target population. A simulated treatment comparison (STC) was also performed by modeling the relationship between baseline covariates and patient response in the trial population and predicting response in the target population. Baseline covariates included age, sex, number of prior LoT, simplified MCL international prognostic index, blastoid morphology, and progressive disease to last LoT. Sensitivity analyses included using an extended set of covariates (for both the MAIC and STC), trimming of weights for MAIC and predictive performance metrics were computed for STC.
RESULTS: The ORR in BGB-3111-206 (84%; 95% confidence interval [CI]: 74%-91%) was comparable to both the estimated ORR for the target European population via the MAIC (79%; 95% CI: 55%-92%) and the STC (78%; 95% CI: 57%-94%). Findings were consistent across all sensitivity analyses.
CONCLUSIONS: This analysis provides evidence that the treatment effects of zanubrutinib observed in BGB-3111-206 are applicable to a European population with R/R MCL.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
RWD57
Topic
Real World Data & Information Systems, Study Approaches
Topic Subcategory
Reproducibility & Replicability
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Oncology