BCMA CAR T-cell therapy in a patient with heavily pretreated relapsed/refractory POEMS Syndrome: a case report
TL;DR
Evidence is provided that BCMA CAR T-cell therapy is a safe and efficacious option for patients with relapsed/refractory POEMS syndrome who received equecabtagene autoleucel, a fully human anti-BCMA CAR T-cell product.
Abstract
B-cell maturation antigen (BCMA) chimeric antigen receptor T-cell (CAR T-cell) therapy is an established treatment for relapsed/refractory multiple myeloma. However, its application in POEMS syndrome remains largely unreported, despite overlapping pathogenesis involving plasma cell dyscrasia. Herein, we present a woman in her 30s with heavily pretreated relapsed/refractory POEMS syndrome who received equecabtagene autoleucel, a fully human anti-BCMA CAR T-cell product. The patient achieved a complete vascular endothelial growth factor (VEGF) response by day 14 post-infusion, with sustained remission through 12 months of follow-up. Clinical improvements included resolution of heart failure, pulmonary hypertension, and renal dysfunction, along with marked neurological recovery, enabling the patient to return to normal daily activities and occupational functioning, a status that persisted through the 16-month follow-up. Treatment-related toxicities comprised Grade 2 cytokine release syndrome and persistent hypogammaglobulinemia, without severe infectious complications. No delayed neurotoxicity or secondary malignancies were observed. This case provides evidence that BCMA CAR T-cell therapy is a safe and efficacious option for patients with relapsed/refractory POEMS syndrome.