POEMS syndrome with severe neurological damage clinically recovered with lenalidomide

Tomás, José Francisco; Giraldo, Pilar; Nistal, Sara

doi:10.3324/haematol.2011.041897

Cited by 28 publications

(29 citation statements)

References 12 publications

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“…Lenalidomide use in POEMS syndrome has previously been described only in case reports and small series, in which efficacy was good and toxicity mild [12][13][14]17].…”

Section: Discussionmentioning

confidence: 99%

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Efficacy of lenalidomide in POEMS syndrome: A retrospective study of 20 patients

et al. 2013

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POEMS syndrome is a rare disorder characterized by polyneuropathy, monoclonal gammopathy, multiorgan involvement, and elevated vascular endothelial growth factor levels. Localized bone lesions require irradiation, whereas young patients with disseminated disease receive intensive treatment with stem cell support. Treatment of older and non responding patients is not yet standardized. We report the use of a combination of lenalidomide and dexamethasone in 20 patients with POEMS syndrome. Four patients were newly diagnosed, and 16 had relapsed or progressed after treatment. All but one of the patients responded: clinical improvements were noted in neuropathies (16/20) organomegaly (13/13), peripheral edema (14/15), and pulmonary hypertension (5/5). At least a very good partial response was noted in 68% of patients, with partial responses in 26%. Serum VEGF levels fell markedly in all 17 patients with available values. Twelve patients had 18‐FDG‐PET/CT at diagnosis (11 with positive findings), and nine patients during follow‐up. The number of lesions fell markedly in five cases and remained stable in two cases, while two patients became negative. During a median follow‐up of 22 months, four patients relapsed. Toxicity, predominantly hematological, was mild and manageable. Lenalidomide thus appears to be effective in POEMS syndrome, inducing high rate of clinical and biological responses. Am. J. Hematol. 88:207–212, 2013. © 2012 Wiley Periodicals, Inc.

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“…Lenalidomide use in POEMS syndrome has previously been described only in case reports and small series, in which efficacy was good and toxicity mild [12][13][14]17].…”

Section: Discussionmentioning

confidence: 99%

“…Dispenzieri et al first reported the efficacy of lenalidomide in a patient who did not qualify for HDM/ASCT [12]. In 2009, we reported French experience with lenalidomide in nine patients [13], and Tomas et al recently published a Spanish cohort of 10 patients with favorable outcomes and no major toxicity [14].…”

Section: Introductionmentioning

confidence: 99%

Efficacy of lenalidomide in POEMS syndrome: A retrospective study of 20 patients

et al. 2013

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“…Other promising treatments include lenalidomide (Table 3), [63][64][65][66] thalidomide, 25,67-69 and bortezomib (see Table 4), [70][71][72][73][74] drugs all of which can have a direct antiplasma cell effect as well as anti-VEGF and anti-TNF effects. Enthusiasm for the latter 2 therapies should be tempered by the high risk of peripheral neuropathy induced by these drugs.…”

Section: Org Frommentioning

confidence: 99%

“…In a retrospective review of 10 patients with previously treated POEMS syndrome who were treated with lenalidomide with or without dexamethasone in Spain, all patients improved. 65 Median time from last therapy to lenalidomide was 4 months (range, 1-36 months). Because the prior therapy in 7 patients was intravenous immunoglobulin (IVIG) with or without prednisone, this is a relatively ineffective regimen and the benefit observed in these patients most certainly would have been the result of the lenalidomide with or without dexamethasone.…”

Section: Org Frommentioning

confidence: 99%

How I treat POEMS syndrome

Dispenzieri

2012

Blood

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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“…11 More recently, two studies have demonstrated promising results in the phase II trial setting with favorable outcomes. [12][13][14] As with MM, the use of HDT/ASCT in patients with POEMS has yielded encouraging results, though most reported series are small and single center experiences. [15][16][17][18][19][20] We, therefore, sought to characterize the effect of ASCT as therapy for patients with POEMS syndrome, determining the effect on organ-specific dysfunction and delineating key patient-and disease-specific factors predicting for outcome.…”

Section: Introductionmentioning

confidence: 99%

High-dose therapy and autologous stem cell transplantation in patients with POEMS syndrome: a retrospective study of the Plasma Cell Disorder sub-committee of the Chronic Malignancy Working Party of the European Society for Blood & Marrow Transplantation

et al. 2016

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OEMS syndrome is a rare para-neoplastic syndrome secondary to a plasma cell dyscrasia. Effective treatment can control the diseaserelated symptom complex. We describe the clinical outcome of autologous stem cell transplantation for patients with POEMS syndrome, determining the impact of patient-and disease-specific factors on prognosis. One hundred and twenty-seven patients underwent an autologous stem cell transplantation between 1997-2010 with a median age of 50 years (range 26-69 years). Median time from diagnosis to autologous stem cell transplantation was 7.5 months with 32% of patients receiving an autologous stem cell transplantation more than 12 months from diagnosis. Engraftment was seen in 97% patients and engraftment syndrome was documented in 23% of autologous stem cell transplantation recipients. Hematologic response was characterized as complete response in 48.5%, partial response in 20.8%, less than partial repsonse in 30.7%. With a median follow up of 48 months (95%CI: 38.3, 58.6), 90% of patients are alive and 16.5% of patients have progressed. The 1-year non-relapse mortality was 3.3%. The 3-year probabilities of progression-free survival and overall survival are 84% and 94%, respectively, with 5-year probabilities of progression-free survival and overall survival of 74% and 89%. In a cohort of graft recipients, detailed organ-specific symptom response demonstrated clear symptom benefit after autologous stem cell transplantation especially in relation to neurological symptom control. The data analyzed in this study demonstrate the clinical utility of autologous stem cell transplantation for patients with POEMS syndrome.

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POEMS syndrome with severe neurological damage clinically recovered with lenalidomide

Cited by 28 publications

References 12 publications

Efficacy of lenalidomide in POEMS syndrome: A retrospective study of 20 patients

Efficacy of lenalidomide in POEMS syndrome: A retrospective study of 20 patients

How I treat POEMS syndrome

High-dose therapy and autologous stem cell transplantation in patients with POEMS syndrome: a retrospective study of the Plasma Cell Disorder sub-committee of the Chronic Malignancy Working Party of the European Society for Blood & Marrow Transplantation

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