Ocular Findings in Patients with Polyneuropathy, Organomegaly, Endocrinopathy, Monoclonal Gammopathy, and Skin Changes Syndrome

Kaushik, Megha; Pulido, José S.; Abreu, Rodrigo de; Amselem, Luis; Dispenzieri, Angela

doi:10.1016/j.ophtha.2010.08.013

Cited by 82 publications

(82 citation statements)

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“…Papilledema is present in at least one-third of patients. Of the 33 patients at our institution referred for a formal ophthalmologic examination during a 10-year period, 67% had ocular signs and symptoms, the most common of which was papilledema in 52% of those examined [52]. The most common ocular symptoms reported were blurred vision in 15, diplopia in 5, and ocular pain in 3.…”

Section: Diagnosismentioning

confidence: 99%

“…The course of POEMS syndrome is usually chronic with modern estimated median survivals of nearly 14 years [48,58] Only fingernail clubbing, extravascular volume overload-i.e., effusions, edema, and ascites [48], respiratory symptoms [58], pulmonary hypertension [80] impaired DLCO, and papilledema [52] have been associated with a significantly shorter overall survival. The number of POEMS features does not affect survival [5,10].…”

Section: Risk Stratificationmentioning

confidence: 99%

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POEMS syndrome: Update on diagnosis, risk‐stratification, and management

2015

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Disease overview: POEMS syndrome is a paraneoplastic syndrome due to an underlying plasma cell neoplasm. The major criteria for the syndrome are polyradiculoneuropathy, clonal plasma cell disorder (PCD), sclerotic bone lesions, elevated vascular endothelial growth factor, and the presence of Castleman disease. Minor features include organomegaly, endocrinopathy, characteristic skin changes, papilledema, extravascular volume overload, and thrombocytosis. Diagnoses are often delayed because the syndrome is rare and can be mistaken for other neurologic disorders, most commonly chronic inflammatory demyelinating polyradiculoneuropathy. POEMS syndrome should be distinguished from the Castleman disease variant of POEMS syndrome, which has no clonal PCD and typically little to no peripheral neuropathy but has several of the minor diagnostic criteria for POEMS syndrome.Diagnosis: The diagnosis of POEMS syndrome is made with three of the major criteria, two of which must include polyradiculoneuropathy and clonal plasma cell disorder, and at least one of the minor criteria.Risk stratification: Because the pathogenesis of the syndrome is not well understood, risk stratification is limited to clinical phenotype rather than specific molecular markers. The number of clinical criteria is not prognostic, but the extent of the plasma cell disorder is. Those patients with an iliac crest bone marrow biopsy that does not reveal a plasma cell clone are candidates for local radiation therapy; those with a more extensive or disseminated clone will be candidates for systemic therapy.Risk‐adapted therapy: For those patients with a dominant sclerotic plasmacytoma, first line therapy is irradiation. Patients with diffuse sclerotic lesions or disseminated bone marrow involvement and for those who have progression of their disease 3–6 months after completing radiation therapy should receive systemic therapy. Corticosteroids are temporizing, but alkylators are the mainstay of treatment, either in the form of low dose conventional therapy or high dose with stem cell transplantation. Lenalidomide shows promise with manageable toxicity. Thalidomide and bortezomib also have activity, but their benefit needs to be weighed against their risk of exacerbating the peripheral neuropathy. The benefit of anti‐VEGF antibodies is conflicting. Prompt recognition and institution of both supportive care measures and therapy directed against the plasma cell result in the best outcomes. Am. J. Hematol. 90:951–962, 2015. © 2015 Wiley Periodicals, Inc.

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Section: Diagnosismentioning

confidence: 99%

Section: Risk Stratificationmentioning

confidence: 99%

POEMS syndrome: Update on diagnosis, risk‐stratification, and management

2015

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“…Papilledema is present in at least one-third of patients. Of the 33 patients at our institution referred for a formal ophthalmologic examination during a 10 year period, 67% had ocular signs and symptoms, the most common of which was papilledema in 52% of those examined [42]. The most common ocular symptoms reported were blurred vision in 15, diplopia in 5, and ocular pain in 3.…”

Section: Diagnosismentioning

confidence: 99%

POEMS syndrome: 2011 update on diagnosis, risk‐stratification, and management

Dispenzieri

2011

American J Hematol

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119

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Disease overviewPOEMS syndrome is a paraneoplastic syndrome due to an underlying plasma cell neoplasm. The major criteria for the syndrome are polyradiculoneuropathy, clonal plasma cell disorder (PCD), sclerotic bone lesions, elevated vascular endothelial growth factor, and the presence of Castleman disease. Minor features include organomegaly, endocrinopathy, characteristic skin changes, papilledema, extravascular volume overload, and thrombocytosis. Diagnoses are often delayed because the syndrome is rare and can be mistaken for other neurologic disorders, most commonly chronic inflammatory demyelinating polyradiculoneuropathy. POEMS syndrome should be distinguished from the Castleman disease variant of POEMS syndrome, which has no clonal PCD and typically little to no peripheral neuropathy but has several of the minor diagnostic criteria for POEMS syndrome. Diagnosis: The diagnosis of POEMS syndrome is made with three of the major criteria, two of which must include polyradiculoneuropathy and clonal plasma cell disorder, and at least one of the minor criteria. Risk stratification: Because the pathogenesis of the syndrome is not well understood, risk stratification is limited to clinical phenotype rather than specific molecular markers. The number of clinical criteria is not prognostic, but the extent of the plasma cell disorder is. Those patients with an iliac crest bone marrow biopsy that does not reveal a plasma cell clone are candidates for local radiation therapy; those with a more extensive or disseminated clone will be candidates for systemic therapy. Risk‐adapted therapy: For those patients with a dominant sclerotic plasmacytoma, first line therapy is irradiation. Patients with diffuse sclerotic lesions or disseminated bone marrow involvement and for those who have progression of their disease 3 to 6 months after completing radiation therapy should receive systemic therapy. Corticosteroids are temporizing, but alkylators are the mainstay of treatment, either in the form of low dose conventional therapy or high dose with stem cell transplantation. The benefit of anti‐VEGF antibodies is conflicting. Lenalidomide shows promise with manageable toxicity. Thalidomide and bortezomib also have activity, but their benefit needs to be weighed against their risk of exacerbating the peripheral neuropathy. Prompt recognition and institution of both supportive care measures and therapy directed against the plasma cell result in the best outcomes. Am. J. Hematol. 86:592–601, 2011. © 2011 Wiley‐Liss, Inc.

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“…Of the 33 patients at our institution referred for a formal ophthalmologic examination during a 10-year period, 67% had ocular signs and symptoms, the most common of which was papilledema in 52% of those examined. 86 In most cases, the optic disc edema is asymptomatic, but when it is not and when pressures are high, treatment with acetazolamide and corticosteroids may control symptoms until definitive chemotherapy or radiation therapy directed at the underlying clone can control the disease. In rare cases, serial therapeutic lumbar puncture may be required.…”

Section: Targeting Other Features Of the Diseasementioning

confidence: 99%

How I treat POEMS syndrome

Dispenzieri

2012

Blood

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101

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POEMS syndrome is a paraneoplastic syndrome whose acronym stands for less than half of the defining features of the disease, that is, polyradiculoneuropathy, organomegaly, potentially including coexisting Castleman disease, endocrinopathy, monoclonal plasma cell neoplasm, and skin changes. The other important features include papilledema, extravascular volume overload, sclerotic bone lesions, thrombocytosis, elevated VEGF, and abnormal pulmonary function. The diagnosis is based on having both the polyradiculoneuropathy and the monoclonal plasma cell disorder, and at least 1 of the other 3 major criteria (Castleman disease, sclerotic bone lesions, or elevated VEGF) and at least one minor criterion. The diagnosis is often delayed with intervening incorrect diagnoses of chronic inflammatory demyelinating polyradiculoneuropathy, myeloproliferative disorder, and monoclonal gammopathy of undetermined significance. Prompt treatment directed at the underlying plasma cell clone produces dramatic responses in the majority of patients. Although there are no randomized clinical trial data to direct best therapy, for patients with disseminated disease, highdose chemotherapy with peripheral blood transplantation has yielded durable benefit, whereas radiation therapy is typically effective for patients with a more localized presentation. More universal recognition of and more scientific inquiry into the underpinnings of the disease will provide direction toward the best treatment strategies in the future. (Blood. 2012;119(24):5650-5658) Introduction POEMS syndrome, 1 also known as osteosclerotic myeloma, Takatsuki syndrome, 2 and Crow-Fukase syndrome, 3,4 is a rare paraneoplastic syndrome resulting from an underlying plasma cell disorder. There acronym POEMS refers to several, but not all, of the features of the syndrome: polyradiculoneuropathy, organomegaly, endocrinopathy, monoclonal plasma cell disorder, and skin changes. There are 3 important points that relate to this memorable acronym. First, not all of the features within the acronym are required to make the diagnosis (Table 1). Second, there are other important features not included in the POEMS acronym, including papilledema, extravascular volume overload, sclerotic bone lesions, thrombocytosis/erythrocytosis, elevated VEGF levels, abnormal pulmonary function tests, and a predisposition toward thrombosis. Lastly, there is a Castleman disease variant of POEMS syndrome that may not be associated with a clonal plasma cell disorder. 5,6 Table 1 outlines the range of expected frequencies of each of the features based on the largest published series. 2,[7][8][9][10][11] The pathogenesis of the syndrome is not well understood. To date, VEGF is the cytokine that correlates best with disease activity, [12][13][14][15][16][17][18][19][20] although it may not be the driving force of the disease based on the mixed results seen with anti-VEGF therapy. 5,21-29 VEGF, which is expressed by osteoblasts, macrophages, tumor cells 30 (including plasma cells), 31,32 and megakaryocytes/pla...

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Ocular Findings in Patients with Polyneuropathy, Organomegaly, Endocrinopathy, Monoclonal Gammopathy, and Skin Changes Syndrome

Cited by 82 publications

References 20 publications

POEMS syndrome: Update on diagnosis, risk‐stratification, and management

POEMS syndrome: Update on diagnosis, risk‐stratification, and management

POEMS syndrome: 2011 update on diagnosis, risk‐stratification, and management

How I treat POEMS syndrome

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