Abstract
Background: Since 2020, several novel treatments (Txs) that differ in duration, dosing frequency, route of administration, efficacy, and/or safety, have been approved by the FDA for R/R FL, making Tx choices more complex. This study aimed to understand patient preferences (prefs) and perceived burden of different Tx features from patients (pts) with FL receiving Tx.
Methods: An online survey was completed between Dec 21, 2024 and Feb 24, 2025 by US adults recruited through physician referrals of pts with FL diagnosis (Dx), who had received ≥1 Tx. Tx prefs were assessed through: 1) direct elicitation, where pts were asked to choose from pairs of fixed Tx profiles defined by duration (fixed-duration [FD] vs treat to progression [TTP]), dosing frequency, administration route (subcutaneous [SC] vs intravenous [IV] vs oral ± IV), efficacy, and risk of cytokine release syndrome (CRS); and 2) an object-case best-worse scaling (BWS) to identify Tx attributes as least/most burdensome. The survey was pre-tested and refined based on input from 7 pts with FL to ensure relevance and comprehension. Descriptive analyses assessed the proportion of pts preferring each Tx profile, and Tx features ranked as least/most burdensome. A random-parameters logit model estimated the sample-level relative burden of each Tx feature. The BWS burden weights were then rescaled to fall between 0 and 10 to facilitate a comparison of relative burden between features. A multivariate logit model explored whether there was any preference heterogeneity at p=0.05.
Results: A total of 125 pts with FL responded to the survey (mean age: 60 yrs; 51% female; 57% White); most (48%) were based in a suburban setting (vs urban [35%] and rural [17%]). Almost all pts (99%) received conventional Tx (e.g. chemotherapy, R-CHOP, BR, G-Benda), while 29% received novel Tx (e.g. CAR T-cell therapy, bispecific antibodies, EZH2 inhibitor, BTK inhibitor combination); 96% reported experience with IV infusions, 34% with injections, and 53% with oral agents. Of the 41 pts receiving Tx at the time of survey completion, 66% were receiving conventional Tx and 34% were receiving novel Tx.
Among the different Tx profiles, pts' prefs were 62% for FD SC (less frequently [freq] dosed, moderate Tx response, low CRS risk) vs 23% for FD IV (less freq dosed, moderate Tx response, moderate CRS risk) vs 15% for TTP oral (more freq dosed, low Tx response, no CRS risk). Pts' prefs were 40% vs 27% vs 33% for TTP oral + IV (more freq dosed, low Tx response, no CRS risk) vs FD IV (less freq dosed, moderate Tx response, moderate CRS risk) vs FD SC (less freq dosed, moderate Tx response, low CRS risk), respectively. When considering potential for relapse within 1.5 years alongside Tx characteristics, pts' prefs were 51% for FD SC Tx with low CRS risk vs 34% for 1 time IV Tx with high CRS risk vs 15% for FD IV Tx with moderate CRS risk. When efficacy was equal, pts' prefs were 74% for FD SC (less freq dosed, low CRS risk) vs 14% for FD IV (less freq dosed, moderate CRS risk) vs 12% for TTP SC (more freq dosed, higher CRS risk).
The multivariate logit model outputs identified several characteristics associated with pts' prefs for FD SC/IV vs alternative Tx: longer time since Dx and living in an urban setting (vs rural/suburban) vs TTP oral Tx; currently taking Tx vs TTP oral + IV Tx; difficulty staying at a hospital several hours away from home and long travel time from the nearest major hospital vs the 1 time IV Tx; prior experience with IV Tx vs TTP SC Tx.
No mandatory monitoring after Tx was ranked as the least burdensome Tx attribute (BWS: 0.7) and staying close to a Tx center for 1 month and no driving for 2 months after Tx was ranked as the most burdensome (BWS: 10). Starting Tx later/having to wait was ~10 times more burdensome vs starting Tx immediately (BWS: 9.5 vs 1.0). The combination of TTP of IV + oral capsule was ~4 times more burdensome vs FD SC injection (BWS: 6.9 vs 1.7). More frequent dosing (28 times in 1 year) was ~3 times more burdensome vs less frequent dosing (10–19 times in 1 year [BWS: 6.2 vs 2.2]).
Conclusions: Pts expressed diverse prefs for Tx options in R/R FL. Tx attributes considered least burdensome included: starting Tx immediately, FD Tx, and/or less frequent dosing. These results may inform patient and provider shared decision making when selecting Tx. Future studies are warranted to ensure pts' perspectives are considered in the dynamic Tx landscape for R/R FL.
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