Jianchun Li, Lichaoyue Sun, Shuo Liang, Zhiheng Yu
From 2001 to 2025, research activities on TDM in leukemia treatment have shown continuous growth, with substantial expansion in both scope and depth. TDM is gradually becoming an increasingly important component of leukemia pharmacotherapy and may play a key role in supporting precision medicine and optimizing treatment outcomes in leukemia patients.
INTRODUCTION: Leukemia treatment has evolved rapidly over the past decade, with the introduction of targeted therapies, immunotherapies, and increasingly complex pharmacotherapeutic regimens. Many antileukemic agents and supportive medications exhibit substantial pharmacokinetic variability and have narrow therapeutic windows. Therefore, therapeutic drug monitoring (TDM) has emerged as an important strategy for optimizing individualized pharmacotherapy, improving treatment efficacy, and reducing drug-related toxicity. However, the overall research landscape, development trajectory, and emerging trends of TDM in leukemia have not been systematically evaluated.
AIM: This study aimed to systematically analyze global research trends and hotspots in TDM for leukemia between 2001 and 2025 using bibliometric methods and to provide insights into future research directions and clinical pharmacotherapy applications.
METHOD: Publications related to TDM in leukemia published between January 1, 2001, and December 31, 2025, were retrieved from the Web of Science Core Collection database. A topic-based search strategy was used to identify the relevant articles. Bibliometric analysis and visualization were conducted using VOSviewer and CiteSpace to evaluate publication output, collaboration networks among countries, institutions, and authors, journal distribution, highly cited publications, citation bursts, and keyword co-occurrence.
RESULTS: A total of 859 papers were retrieved, with the highest output (58 papers) observed between 2024 and 2025. The United States and China contributed the most publications. The University of Texas MD Anderson Cancer Center ranked first among the institutions. Highly cited papers primarily focused on pharmacotherapy for hematologic malignancies, pharmacodynamics and pharmacokinetic studies, and pre-transplant medication regimens. Burst analysis indicates that TDM in leukemia treatment has progressively evolved from early preliminary studies on exposure-response relationships to the implementation of precision dosing strategies and clinical guidelines, continuously expanding to cover an increasing number of therapeutic agents in leukemia treatment. Current research hotspots include tyrosine kinase inhibitors (particularly Bcr-Abl inhibitors), asparaginase, drug interactions, and safety management issues.
CONCLUSION: From 2001 to 2025, research activities on TDM in leukemia treatment have shown continuous growth, with substantial expansion in both scope and depth. TDM is gradually becoming an increasingly important component of leukemia pharmacotherapy and may play a key role in supporting precision medicine and optimizing treatment outcomes in leukemia patients.