血液系统恶性肿瘤及血液系统恶性肿瘤新兴疗法概览：系统综述

Hematologic malignancies remain a major therapeutic challenge due to disease heterogeneity, treatment resistance, and significant toxicity associated with conventional therapies. In recent years, rapid advances in molecular oncology and immunotherapy have led to the development of multiple emerging therapeutic strategies.

This review synthesizes evidence on novel approaches, including targeted therapies such as BTK inhibitors (e. g. , ibrutinib), BCL-2 inhibitors (e. g. , venetoclax), FLT3 and JAK inhibitors, and next-generation monoclonal and bispecific antibodies that enhance immune-mediated cytotoxicity.

In addition, gene- and cell-based modalities-including CAR T-cell therapy and CRISPR-mediated gene correction-have demonstrated promising efficacy, particularly in relapsed or refractory leukemia and lymphoma. Despite these advances, significant limitations remain, including treatment-related toxicities, mechanisms of resistance, high relapse rates after CAR-T therapy, and substantial financial and accessibility barriers.

Gaps in predictive biomarkers, optimal sequencing of therapies, and long-term safety also limit widespread implementation. Precision medicine approaches, driven by molecular diagnostics and genomic profiling, continue to refine individualized treatment strategies and hold potential to overcome current challenges.

Overall, this review provides an updated and comprehensive evaluation of emerging therapeutic modalities in hematologic malignancies and outlines key areas where further research is critically needed.