POEMS syndrome

POEMS syndrome is a rare paraneoplastic disorder caused by an underlying plasma cell neoplasm. The acronym POEMS stands for its major features: Polyneuropathy, Organomegaly, Endocrinopathy, Monoclonal protein (M-protein), and Skin changes. It is also known as Crow-Fukase syndrome, Takatsuki syndrome, or osteosclerotic myeloma. The disorder is a multisystem disease that primarily affects the nervous system, endocrine system, blood, skin, and multiple organs including the liver, spleen, and lymph nodes. The hallmark feature is a chronic, progressive, predominantly motor polyneuropathy that typically begins in the feet and ascends, often leading to significant disability. Organomegaly most commonly involves hepatomegaly, splenomegaly, or lymphadenopathy. Endocrine abnormalities may include hypogonadism, hypothyroidism, adrenal insufficiency, and diabetes mellitus. A monoclonal plasma cell proliferative disorder is present, almost always with a lambda light chain restriction. Skin changes can include hyperpigmentation, hypertrichosis, hemangiomas, and thickening of the skin. Other important features not captured in the acronym include papilledema, extravascular volume overload (edema, ascites, pleural effusions), thrombocytosis or polycythemia, elevated vascular endothelial growth factor (VEGF) levels, and sclerotic bone lesions. Treatment depends on the extent of disease. For patients with limited bone lesions (typically 1-3 lesions), radiation therapy directed at the affected bone is often effective and can be curative. For patients with widespread disease, systemic therapy is required, including autologous stem cell transplantation (for eligible patients), or chemotherapy regimens such as lenalidomide-dexamethasone or melphalan-dexamethasone. Anti-VEGF agents have been explored but require caution due to potential complications. Early diagnosis and treatment are important, as the polyneuropathy can be partially reversible with effective therapy. The median survival has improved significantly with modern treatments, often exceeding 10 years.

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Common questions about POEMS syndrome