学术型 BCMA CAR-T（ARI0002h）在双次 ASCT 后复发的 POEMS 综合征中实现 MRD 阴性缓解并伴 sBCMA 生物标志物应答

Polyneuropathy, Organomegaly, Endocrinopathy, Monoclonal gammopathy, and Skin changes (POEMS) syndrome is a rare plasma cell disorder with diverse multisystem manifestations. Autologous stem cell transplantation (ASCT) is the preferred front-line therapy when feasible, but relapsed/refractory (RR) cases after ASCT failure lack effective salvage options.

B-cell maturation antigen (BCMA)-directed CAR-T cell therapy has revolutionised multiple myeloma (MM) treatment. Given the shared BCMA expression on clonal plasma cells between MM and POEMS, CAR-T cell therapy is also a biologically compelling strategy for POEMS syndrome [1].

However, there are only two previous case reports of CAR-T cell therapy in POEMS [3,4], none involving patients with prior ASCT failure.

We report a 54-year-old male with RR-POEMS progressing after two previous ASCT failures who received an academic point-of-care BCMA-directed CAR-T cell therapy (ARI0002h) via fractionated infusion. No cytokine release syndrome (CRS) or neurotoxicity (ICANS) occurred. At 3 months, the patient achieved a measurable residual disease (MRD)-negative complete haematological response (CR H ) by Next Generation Flow (NGF) with a sensitivity of 10 and a radiological CR.

Responses have been sustained for 10 months. Serum BCMA (sBCMA) levels declined dynamically in parallel with haematological response, suggesting potential utility as a novel monitoring biomarker in POEMS. This case supports BCMA-directed CAR-T therapy as a safe and effective salvage strategy in RR-POEMS following ASCT failure, even in an aggressive disease context. Clinical trial registration: Not aplicable.