Multiple Myeloma(MM)-01

Good Recovery of a Domestic Multiple Myeloma Patient with CAR-T Therapy Despite Lack of BCMA Expression

A female patient diagnosed with stage IIIA IgD-λ type multiple myeloma in 2018 received first-line treatment mainly with bortezomib. After 3 cycles, she achieved complete remission (CR). In October 2018, she underwent autologous hematopoietic stem cell transplantation as consolidation therapy, followed by maintenance therapy with lenalidomide. In April 2020, the disease relapsed, and she underwent 7 cycles of second-line treatment, yielding poor efficacy. From December 2020 to April 2021, she received chemotherapy mainly with daratumumab, but bone marrow biopsy still showed 21.763% malignant monoclonal plasma cells, with serum free light chain λ at 1470 mg/L and urine free light chain λ at 5330 mg/L. By this time, she had exhausted major standard and novel treatments available domestically, including undergoing autologous stem cell transplantation, with entry into a CAR-T clinical trial being the best remaining option.

Referred by local physicians, she presented to Ludaopei Hospital on May 10, 2021, hoping to enroll in their clinical trial for BCMA CAR-T therapy in multiple myeloma (MM). Upon admission, she was in a weakened state with generalized pain and recurrent fevers. Comprehensive examinations confirmed "multiple myeloma, λ light chain type, ISS stage III, R-ISS stage III, mSMART high-risk group."

PET-CT scan revealed increased metabolic activity in soft tissue density within the bone marrow cavities of bilateral femurs and tibias, indicative of tumor involvement. Bone marrow biopsy showed 60.13% malignant monoclonal plasma cells with no expression of BCMA.

Ludaopei Hospital informed the patient and her family about the efficacy of current treatments for BCMA-negative multiple myeloma, which although potentially effective according to some literature, lacks definitive data. After careful consideration, the patient and her family chose to proceed with the treatment plan.

Following preconditioning with FC regimen, BCMA CAR-T cells were infused on June 1, 2021, at Ludaopei Hospital. The patient developed fever post-infusion, which was gradually controlled with aggressive anti-infective and symptomatic supportive treatment. Fourteen days post-infusion, bone marrow biopsy showed no residual malignant monoclonal plasma cells. Thirty-one days post-infusion, bone marrow biopsy remained negative. Serum immunofixation was negative, serum free light chain λ was within normal range, and serum M protein was negative, indicating complete remission of the disease.

Currently, more than 8 months after receiving BCMA CAR-T cell infusion, the patient remains in complete remission with good recovery and high satisfaction with the treatment outcome.