Analysis of Real-World Progression and Insufficient Response Variables and Related Endpoints Among Patients with Non-Hodgkin Lymphoma.

in Advances in therapy by Madeline Richey, Christina Fullerton, Qianyi Zhang, Tori Williams, Douglas Donnelly, Hannah C Wise, Aaron Dolor, Niquelle Wadé, Kelly Magee

TLDR

  • The study developed and assessed a novel approach to capture real-world progression and insufficient response events in NHL patients from EHR data, showing feasibility, reliability, and scalability.

Abstract

Methods for assessing change in tumor burden differ between clinical trial and routine clinical care settings, presenting a unique opportunity to design novel methods to capture clinical outcomes from electronic health record (EHR) data. We adapted a previously established approach for solid tumors and modified it to capture real-world progression (rwP) and real-world insufficient response (rwIR) events in non-Hodgkin lymphoma (NHL). This study used a nationwide EHR-derived deidentified database. The first phase assessed the rwP/rwIR approach in patients with follicular lymphoma (FL) and diffuse large B cell lymphoma (DLBCL). The second phase assessed the approach in a larger cohort of patients with mantle cell lymphoma (MCL). Performance was assessed through inter-abstractor agreement on event occurrence, date, and type, clinician assessment and source data completeness, downstream events after an rwP/rwIR event, and time-to-event analyses (real-world progression-free [rwPFS] and event-free [rwEFS] survival) and their correlation with real-world overall survival (rwOS). A total of 6162 patients with NHL were included in the study, comprising 672 patients with FL (median age, 64 years; female, 49%; male, 51%), 405 patients with DLBCL (median age, 70 years; female, 42%; male, 58%), and 5085 patients with MCL (median age, 69 years; female, 27%; male, 73%). Inter-abstractor agreement among all cohorts was 96-97% for event occurrence and 85-89% for event date within 30 days. The proportion of patients with an rwP/rwIR event was 26% in the FL cohort, 27% in the DLBCL cohort, and 42% in the MCL cohort. Clinically relevant downstream events were observed in 58% of the FL cohort, 63% of the DLBCL cohort, and 73% of the MCL cohort. In the MCL cohort, median rwPFS was 34.5 months, and median rwEFS was 32.2 months. Real-world OS correlated more strongly with rwPFS (85%) than with rwEFS (80%). The NHL-specific rwP/rwIR approach is feasible, reliable, and scalable. Observed inter-abstractor agreement and rwP/rwIR event frequency show applicability across NHL cohorts. Endpoint analyses and correlations with rwOS in a real-world population demonstrate the clinical relevance of this approach.

Overview

  • The study adapted an existing approach to capture real-world progression and insufficient response events in non-Hodgkin lymphoma (NHL) patients from electronic health record (EHR) data.
  • The approach was assessed in a nationwide EHR-derived deidentified database, with three cohorts: follicular lymphoma (FL), diffuse large B cell lymphoma (DLBCL), and mantle cell lymphoma (MCL).
  • The study aimed to develop a reliable and scalable method to capture clinical outcomes in NHL patients, which can be used in routine clinical care settings.

Comparative Analysis & Findings

  • The inter-abstractor agreement for event occurrence and date was 96-97% and 85-89%, respectively, across all cohorts.
  • The proportion of patients with an rwP/rwIR event was 26% in FL, 27% in DLBCL, and 42% in MCL.
  • Real-world progression-free (rwPFS) survival correlated more strongly with real-world overall survival (rwOS) than event-free (rwEFS) survival, with median rwPFS of 34.5 months and median rwEFS of 32.2 months in the MCL cohort.

Implications and Future Directions

  • The NHL-specific rwP/rwIR approach is feasible, reliable, and scalable, and can be applied across NHL cohorts.
  • Future studies can explore the use of this approach in other lymphoma subtypes and diseases, and investigate potential biases and confounding variables.
  • The findings of this study demonstrate the potential of EHR data to capture clinical outcomes and inform treatment decisions in real-world patient populations.
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