Establishment of patient-derived xenograft models in Chinese patients with multiple myeloma: Insights into therapeutic responsiveness and molecular subtyping

Abstract

Multiple myeloma (MM), a malignant hematologic tumor characterized by the proliferation of monoclonal plasma cells, remains incurable with high relapse rates despite advances in treatment. Patient-derived xenograft (PDX) models have emerged as a promising tool for understanding MM's complex pathophysiology and testing therapeutic responses. In this study, we successfully developed PDX models from three patients with MM by subcutaneously engrafting their tumor cells into immunodeficient NCG mice. These models accurately mirrored the clinical drug responses of their corresponding patient cases, exhibiting similar drug sensitivities and resistance patterns. Omics profiling facilitated the alignment of PDX models with specific molecular subgroups identified in current MM research, enhancing the models' clinical relevance. The concordance between PDX models and clinical data confirms the utility of these models in simulating patient-specific responses and advancing personalized treatment strategies. This study validates the effectiveness of PDX models established by subcutaneous engraftment of tumor cells in replicating human disease and treatment responses, thus providing a robust platform for future personalized treatments and development of targeted interventions in Chinese MM patients.