NORD gratefully acknowledges Eilis Eschweiler and Regan Sweeney, NORD Editorial Interns from the University of Notre Dame and Angela Dispenzieri, MD, Division of Hematology and Division of Clinical Chemistry, Mayo Clinic, for assistance in the preparation of this report.
POEMS syndrome is an extremely rare paraneoplastic syndrome. Paraneoplastic syndromes are caused by an abnormal immune response to a cancerous tumor (neoplasm) where the body accidently attacks normal cells in the nervous system. POEMS is an acronym that stands for the disorder’s five major signs and symptoms, which include Polyneuropathy, Organomegaly, Endocrinopathy, Monoclonal gammopathy and Skin abnormalities. Common symptoms include progressive weakness of the nerves in the arms and legs (sensorimotor polyneuropathy), an abnormally enlarged liver and/or spleen (hepatosplenomegaly), enlarged lymph nodes (lymphadenitis), darkening of the skin (hyperpigmentation), thickening of the skin and excessive hair growth (hypertrichosis).
POEMS syndrome occurs 1.5 times more frequently among men than women, and the typical age of onset is adults in their 50s. The exact cause of POEMS syndrome is unknown, but research suggests that a chemical called VEGF (vascular endothelial growth factor) plays an important role in this disease.
POEMS syndrome is a chronic disorder and the prognosis depends on the extent of the disease and an individual’s response to treatment. There is no standard treatment for POEMS syndrome. Treatment options for patients diagnosed with POEMS syndrome include radiation therapy, chemotherapy, and/or hematopoietic cell transplantation. The median survival for patients with POEMS syndrome is more than14 years.
POEMS syndrome is a rare, multisystem paraneoplastic syndrome. POEMS stands for some of the major signs and symptoms associated with the disease. The major criteria for POEMS syndrome include polyneuropathy, monoclonal gammopathy and either osteosclerotic lesion, Castleman disease, or elevated levels of vascular endothelial growth factor (VEGF) in addition to at least one minor criterion listed below.
Summary of symptoms identified in patients with POEMS syndrome:
Polyneuropathy is a disease affecting nerves throughout the body. Symptoms of polyneuropathy include:
Organomegaly is the abnormal enlargement of an organ or organs in the body. Symptoms of organomegaly in patients with POEMS syndrome include:
Endocrinopathy is when abnormal hormone levels are produced by the endocrine system (system of glands that secrete hormones into the blood). Symptoms of endocrinopathy in POEMS syndrome patients include:
Monoclonal gammopathy is when abnormal bone marrow cells (plasma cells) that produce a protein (monoclonal protein) can be found in the bloodstream. Plasma cells are a key component of the immune system but overproduction of these cells in individuals with POEMS syndrome may result in these symptoms:
Additional blood abnormalities
The exact cause of POEMS syndrome is unknown. POEMS syndrome is associated with an overproduction of molecules that promote inflammation through cell-to-cell communication (pro-inflammatory cytokines). Studies have demonstrated abnormally high levels of vascular endothelial growth factor (VEGF) and pro-inflammatory cytokines, interleukin-6, interleukin-1 and TNF-alpha in the blood of individuals with POEMS syndrome. Cytokines are proteins that are produced by the body’s immune system in response to inflammation. More research is needed to determine the exact role cytokines and VEGF play in the development of POEMS syndrome.
POEMS syndrome affects 1.5 times more men than women, and disease onset usually occurs during a patient’s 40s to 60s. POEMS syndrome often goes unrecognized, making it difficult to determine the number of people afflicted with the disease.
Patients suspected of having POEMS syndrome should undergo a thorough clinical evaluation to include a physical exam, medical history review and laboratory testing. The detailed physical exam includes examination of the eyes, skin and neurological status as well as overall function of organs and body.
Confirmation of certain immunologic abnormalities plays an essential role in establishing the diagnosis of POEMS syndrome. Laboratory tests conducted on the blood (serum) or cerebrospinal fluid (CSF) may reveal elevated levels of M-proteins and blood plasma may show high levels of vascular endothelial growth factor (VEGF). Skeletal imaging may be performed to detect osteosclerotic lesions characteristic of POEMS syndrome. In many patients, surgical removal (biopsy) and microscopic examination of small samples of tissue from an osteosclerotic lesion or a bone marrow biopsy will reveal the abnormal presence of monoclonal plasma cells.
In order to be diagnosed with POEMS syndrome, a person must present both polyneuropathy and monoclonal plasma cell proliferative disorder in addition to one major criterion and one minor criterion. The other major criteria for POEMS syndrome include osteosclerotic lesion, Castleman disease, and elevated levels of vascular endothelial growth factor (VEGF). Minor criteria for POEMS syndrome include enlargement of organs (organomegaly), extracellular fluid accumulation (peripheral edema, ascites, or pleural effusion), endocrinopathy, skin changes, swelling of the optic disc (papilledema) and elevated blood cell count (polycythemia or thrombocytosis).
Current treatments for POEMS syndrome focus on improvement of symptoms and can lead to good prognosis in patients.
The use of ionizing radiation (radiotherapy) or surgical removal (excision) of osteosclerotic lesions that are localized (not spread throughout the body) may temporarily or permanently lead to remission of symptoms associated with POEMS syndrome.
In many patients, including those with widespread unusual hardening of bone (osteosclerotic lesions) or diffuse bone marrow involvement, therapy with certain anticancer drugs (alkylating chemotherapy agents) may alleviate symptoms associated with POEMS syndrome.
Some patients with widespread disease may also receive high doses of chemotherapy followed by infusion of healthy blood stem cells from their own body (autologous stem cell transplant). Additional therapies used to treat POEMS syndrome include lenalidomide, thalidomide and bortezomib depending on a patient’s disease course. Other treatment such as physical therapy and medications to relieve swelling can help with improving mobility and other symptoms of POEMS syndrome.
Information on current clinical trials is posted on the Internet at www.clinicaltrials.gov. All studies receiving U.S. government funding, and some supported by private industry, are posted on this government web site.
For information about clinical trials being conducted at the NIH Clinical Center in Bethesda, MD, contact the NIH Patient Recruitment Office:
Tollfree: (800) 411-1222
TTY: (866) 411-1010
Email: [email protected]
Some current clinical trials also are posted on the following page on the NORD website: https://rarediseases.org/for-patients-and-families/information-resources/info-clinical-trials-and-research-studies/
For information about clinical trials sponsored by private sources, contact: www.centerwatch.com
For information about clinical trials conducted in Europe, contact:
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