[Recent advances in pathogenesis, diagnosis, and therapeutic strategies for POEMS syndrome]

POEMS syndrome is a rare multisystemic disorder characterized by polyneuropathy, organomegaly, endocrinopathy, monoclonal plasma cell dyscrasia, and skin changes. Markedly elevated serum vascular endothelial growth factor (VEGF) levels play a crucial role in its pathogenesis. Recent genomic studies have identified specific mutational patterns in the immunoglobulin lambda light chain gene (e.g., IGLV1-40 or IGLV1-44), providing new insights into the unique biology of POEMS syndrome. For clinical diagnosis, the Japanese diagnostic criteria are highly practical and accurate, designating polyneuropathy, monoclonal plasma cell proliferation, and elevated serum VEGF levels (>1,000 pg/ml) as mandatory major criteria. Treatment strategies are determined based on eligibility for autologous stem cell transplantation (ASCT). For eligible patients, high-dose melphalan followed by ASCT is the standard of care, yielding significant neurological improvement and favorable long-term survival. In contrast, for transplant-ineligible patients or as induction therapy before ASCT, novel agents such as thalidomide, lenalidomide, and bortezomib have demonstrated clinical efficacy. Serum VEGF levels serve as an essential biomarker that sensitively reflects disease activity and treatment response, making them indispensable for monitoring during follow-up. This review provides a comprehensive overview of recent advances in the pathogenesis, diagnostic refinements, and latest therapeutic strategies for POEMS syndrome based on recent scientific evidence.