Lifetime treatment burden of patients with mantle cell lymphoma: simulation‐based analysis of real‐world data from 20 years

Background: Mantle cell lymphoma (MCL) is a rare subtype of B-cell lymphoma that is currently considered incurable, yet treatable condition with increasing amount of treatment options. Alas, the availability of novel therapies is often limited to later treatment lines due to their excessive cost. In this study, we aimed to conduct a baseline analysis on the lifetime treatment burden of MCL patients treated with conventional chemoimmunotherapy regimes. Specifically, we wanted to estimate the number of treatment lines received during 20-year follow-up to determine the number of MCL patients that could potentially receive novel, targeted therapies during the follow-up period. Methods: A real-world data set of 548 patients diagnosed and treated for MCL between the years 2000 and 2020 was collected from seven Finnish hospitals and one Spanish hospital. First, this data was analyzed using the traditional methods of survival analysis for overall survival, time to progression and follow-up time. From these initial results, we estimated the risk of progression from the first treatment line to subsequent treatment lines, to death from MCL, or death from other causes. These estimates were then applied in our competing risk analysis based on Weibull distribution and the data was completed by dynamic imputation of health event histories using discrete semi-Markov transitions in a limited time space to extrapolate the censored variables from our data set up to 20 years of follow-up for each patient. A state distribution plot from our analysis is shown below (Figure 1). Results: In our original data set, the median duration of follow-up was three years (range 0 to 18 years). At 10 years of simulated follow-up, 525 (95.8%) patients had received first-line therapy, 307 (56.0%) had received second-line therapy, 148 (27.0%) had received third-line therapy and 64 (11.7%) had received fourth-line therapy. A total of 262 (47.8%) patients had died from MCL and 113 (20.6%) from other causes. After 20 years of simulated follow-up, the respective figures were 525 (95.8%), 373 (68.1%), 192 (35.0%) and 85 (15.5 %). A total of 362 (66.1%) of patients had died from MCL and 166 (30.3%) from other causes. The median number of treatment lines received both at 10 and 20 years of follow-up was three. Figure 1. State distribution plot from 20-year follow-up The research was funded by: North Ostrobothnia Health Care District Keywords: Aggressive B-cell non-Hodgkin lymphoma, Cancer Health Disparities No conflicts of interests pertinent to the abstract.