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<p>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<sub>H</sub>) by Next Generation Flow (NGF) with a sensitivity of 10⁻⁶ 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. </p><p>Clinical trial registration: Not aplicable.</p>

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Academic BCMA CAR-T (ARI0002h) achieves MRD-negative remission with sBCMA biomarker response in relapsed POEMS syndrome after double ASCT

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年5月12日

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  • Víctor Torrecillas-Mayayo,
  • Aina Oliver-Caldes,
  • Nuria Martínez-Cibrian,
  • Nil Albiol,
  • Luis Gerardo Rodríguez-Lobato,
  • Marta Español-Rego,
  • Valentín Ortiz-Maldonado,
  • Laura Rosiñol,
  • Natalia Tovar,
  • Sergio Navarro-Velasquez,
  • Paola Charry,
  • Daniel Munárriz,
  • Jose Miguel Mateos,
  • Carlos Jimenez-Mira,
  • David F. Moreno,
  • Mercedes Montoro,
  • Pilar Ayora,
  • Julio Delgado,
  • Manel Juan,
  • Azucena González-Navarro,
  • Maria Teresa Cibeira,
  • Carlos Fernández de Larrea

摘要

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 (CRH) by Next Generation Flow (NGF) with a sensitivity of 10⁻⁶ 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.