Thalidomide for POEMS syndrome

Kim, So Yeon; Lee, Sun Ah; Ryoo, Hun Mo; Lee, Kyung Hee; Hyun, Myung Soo; Bae, Sung Hwa

doi:10.1007/s00277-006-0119-z

Cited by 51 publications

(33 citation statements)

References 6 publications

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“…Thalidomide in combination with dexamethasone has also shown to produce responses in terms of VEGF, peripheral neuropathy, and extravascular volume overload, but hematologic responses have not been reported [164][165][166]179]. Enthusiasm for this therapy should be tempered by the risk of peripheral neuropathy induced by this drug.…”

Section: Management Of Poems Syndrome With Disseminated Bone Marrow Imentioning

confidence: 99%

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: Management Of Poems Syndrome With Disseminated Bone Marrow Imentioning

confidence: 99%

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

2015

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“…A starting dose of prednisone ranging between 20 and 1,500 mg/day has been used. No evidence based recommendation can be given as to the appropriate dose, but personal experience would place the daily starting anywhere between 1 and 2 [39,[148][149][150][151] 50% of patients have significant clinical improvement Melphalan-Dexamethasone [146] 81% hematologic response rate; 100% with some neurologic improvement Corticosteroids [3,39,55,73,152] 15% of patients have significant clinical improvement High-dose chemotherapy with PBSCT [52,124,[130][131][132][133][134][135][136][137]139,[153][154][155][156][157][158] 100% of surviving patients have significant clinical improvement Thal after MP [159] No hematological response but improved ascites; stabilized PN, splenomegaly, pulmonary hypertension Thal 1 Dex after CAD [160] CD/POEMS: improved ascites, effusions, pulmonary hypertension, PN, renal function, IL-6 level Thal 1 Dex [161] N 5 9; VEGF improved in all; PN improved in 66%; stable in 33%; improved edema; no HCR Thal after VAD, CTX, bevacizumab [23] Improved cardiopulmonary status, but no improved PN and rising VEGF Lenalidomide 1 Dex [140] Improved ascites, PS, PN, VEGF, testosterone, pulmonary function tests Lenalidomide1 Dex [141] N59; all had hematologic response; clinical responses in all evaluable patients including PS, neurological syndrome, edema, and VEGF. Bortez1AD after VAD, CMP, and AD [142] Improved M-protein, VEGF, paresthesias, splenomegaly, effusions, muscle strength, gynecomastia, and skin changes...…”

Section: Management Of Poems Syndrome With Disseminated Bone Marrow Imentioning

confidence: 99%

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

Dispenzieri

2011

American J Hematol

120

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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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“…Immunomodulatory drugs such as thalidomide and lenalidomide are highly effective in multiple myeloma, and their use in the POEMS syndrome is tempting because of their antiangiogenic properties. Thalidomide has been used in Japanese patients with good results, but the frequent peripheral neuropathy caused by this drug is a major issue in a disease in which the predominant complaint is sensorimotor peripheral neuropathy [8][9][10][11]. Dispenzieri et al first reported the efficacy of lenalidomide in a patient who did not qualify for HDM/ASCT [12].…”

Section: Introductionmentioning

confidence: 99%

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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Thalidomide for POEMS syndrome

Cited by 51 publications

References 6 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

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

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