BCMA CAR-T Therapy Paves New Path for Treating High-Risk Multiple Myeloma

**BCMA CAR-T Therapy Paves New Path for Treating High-Risk Multiple Myeloma**

Multiple Myeloma

Multiple Myeloma (MM) is a malignant tumor originating from plasma cells, known for its complex biology and treatment resistance, presenting significant challenges to patients’ survival. For high-risk MM, especially in cases of relapse and refractory disease after multiple lines of therapy, treatment options become critically important. Recently, a team led by Professor Qian from a Chinese Hospital successfully treated a high-risk relapsed/refractory MM (RRMM) patient using BCMA CAR-T cell therapy, offering new hope for difficult-to-treat cases.

**Patient Overview**

The patient, a 56-year-old woman, initially sought treatment for persistent anemia and back pain. Baseline examinations revealed significant abnormalities, including a hemoglobin level of 67g/L, thrombocytopenia, and abnormal bone marrow findings. She was diagnosed with IgG-kappa type MM at stage ISS III/R-ISS III, with cytogenetic abnormalities and high-risk factors such as t(14;16) and 1q21 amplification. The disease rapidly progressed despite multiple lines of therapy, including VRd and DVD regimens, with external manifestations of extramedullary disease (EMD), indicating a poor prognosis.

**Treatment Journey**

The patient initially underwent VRd induction therapy, followed by DVD treatment when the disease progressed. Despite efforts, she developed multiple extramedullary lesions, leading to the use of the Dara+DECP regimen and autologous stem cell transplantation (ASCT). While these treatments provided partial remission, the disease relapsed within months, with extramedullary involvement further complicating the prognosis.

Given the poor prognosis and lack of effective treatments for EMD in MM, the decision was made to pursue BCMA CAR-T cell therapy, specifically with **Equecabtagene Autoleucel**, China’s first CAR-T product for treating MM. This therapy has demonstrated an impressive overall response rate (ORR) of 100% in patients with extramedullary disease, with a complete remission (CR) rate of 78.6%.

**CAR-T Therapy and Outcomes**

Following preconditioning with FC regimen, the patient underwent BCMA CAR-T therapy. After CAR-T infusion, the patient experienced mild cytokine release syndrome (CRS), which was successfully managed with supportive care. Over the course of three months, significant clinical improvements were observed. PET-CT scans showed no residual disease, and bone marrow biopsies were negative for clonal plasma cells. The patient achieved stringent complete remission (sCR).

Eight months after CAR-T therapy, follow-up results continue to show no evidence of disease, with the patient maintaining CR. This case highlights the long-lasting anti-tumor effects of Equecabtagene Autoleucel, a fully human BCMA CAR-T product, which offers low immunogenicity and sustained CAR-T cell persistence in vivo.

**Implications for Future Treatment**

This success story offers new hope for patients with high-risk and refractory MM, especially those with extramedullary involvement. It also provides valuable clinical insights into the use of BCMA CAR-T therapy as a promising treatment strategy. The case demonstrates that for patients with high-risk MM, comprehensive risk assessment considering genetic characteristics, treatment responses, and future treatment plans is essential for personalized care.

In conclusion, the application of Equecabtagene Autoleucel CAR-T therapy represents a significant breakthrough in the treatment of high-risk, refractory MM, particularly in cases involving extramedullary disease. This innovative approach not only extends survival but also enhances the quality of life for patients who have exhausted other treatment options, marking a new chapter in the fight against multiple myeloma.

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