Defining treatment success in chronic lymphocytic leukemia: exploring surrogate markers, comorbidities, and patient-centered endpoints.

Abstract

Introduction: Traditionally, the success of chronic lymphocytic leukemia (CLL) treatment has been primarily assessed based on clinical outcomes, such as disease response, progression-free survival (PFS), and overall survival (OS). However, the evolution of treatment approaches recognizes the importance of a patient-centered perspective that includes factors directly affecting patients' quality of life and overall well-being.

Areas covered: Studies addressing the impact of targeted agents on improving either OS or other endpoint surrogates were selected using PubMed and MEDLINE platforms. Our search also included studies that considered patient-centric endpoints such as health-related quality of life and patient-reported outcomes (PROs).

Expert opinion: The changing landscape of CLL treatment underscores the importance of continually exploring various endpoints to thoroughly define treatment success. Beyond conventional metrics such as OS and surrogate endpoints, namely, PFS, time to next treatment (TTNT), and measurable residual disease (MRD) assessment, it becomes crucial to integrate enhanced comorbidity evaluations and patient-centered viewpoints into a CLL success roadmap.Subsequent investigations ought to concentrate on enhancing current surrogate endpoints, discerning their contextual significance, and exploring innovative indicators of treatment efficacy and safety. Given the dynamic nature of CLL and the heterogeneity among patient groups, personalized strategies are essential, taking into account individual traits and patient preferences.