A prospective phase II study of low dose lenalidomide plus dexamethasone in patients with newly diagnosed polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes (POEMS) syndrome

Li, Jian; Huang, Xiao Ping; Cai, Qianqian; Wang, Chen; Cai, Hao; Zhao, Hao; Zhang, Lu; Cao, Xinxin; Gale, Robert Peter; Zhou, Daobin

doi:10.1002/ajh.25100

Cited by 50 publications

(49 citation statements)

References 25 publications

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“…Other promising treatments include lenalidomide, thalidomide, and bortezomib, drugs all of which can have anti‐VEGF and anti‐TNF effects. Li and colleagues reported a phase II trial of 12 cycles of lenalidomide and dexamethasone in 41 patients . The complete hematologic response rate was 46%, the neurologic response rate was 95%, and the VEGF response rate was 83%.…”

Section: Management Of Poems Syndrome With Disseminated Bone Marrow Imentioning

confidence: 99%

POEMS Syndrome: 2019 Update on diagnosis, risk‐stratification, and management

2019

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Disease Overview: Polyneuropathy, organomegaly, endocrinopathy, M-protein, skin changes (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 PCD, 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. Risk factors include low serum albumin, age, pleural effusion, pulmonary hypertension, and reduced eGFR. 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. 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.

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Section: Management Of Poems Syndrome With Disseminated Bone Marrow Imentioning

confidence: 99%

POEMS Syndrome: 2019 Update on diagnosis, risk‐stratification, and management

2019

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“…Among these, thalidomide is the only agent whose efficacy and safety have been demonstrated by a randomized placebo-controlled study (5). The efficacy and safety of lenalidomide in newly diagnosed or relapsed patients with POEMS syndrome have also been explored in a large case series (8,9) and in two one-arm clinical trials (10,11). Several new agents for myeloma, such as daratumumab, may be effective in POEMS syndrome as well (14).…”

Section: Discussionmentioning

confidence: 99%

Lenalidomide Treatment for Thalidomide-refractory POEMS Syndrome: A Prospective Single-arm Clinical Trial

et al. 2020

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Objective A randomized controlled trial has shown the efficacy of thalidomide against Polyneuropathy, organomegaly, endocrinopathy, M-protein, and skin changes (POEMS) syndrome; however, there are still refractory patients. We studied the effects of lenalidomide, a derivative of thalidomide, on patients refractory to thalidomide. Methods This prospective single-arm trial evaluated the safety and efficacy of lenalidomide plus dexamethasone in refractory or recurrent patients with POEMS syndrome. The regimen was administered as six 28-day cycles with lenalidomide on days 1-21 (15 mg in cycle 1, and 25 mg in cycle 2-6) plus dexamethasone once a week (20 mg). The primary endpoints were the rate of reduction in the serum vascular endothelial growth factor (VEGF) level at 24 weeks and the incidence of adverse events. This trial was registered with ClinicalTrial.gov, NCT02193698. Results Between July 2014 and December 2015, five men were enrolled. All patients had been refractory to thalidomide plus dexamethasone for more than 24 weeks. The mean rate of reduction in the serum VEGF level at 24 weeks was 59.6%±8.3% (p=0.0003). The mean serum VEGF level decreased from 2,466±771 pg/ mL to 974±340 pg/mL. No serious adverse events were observed, and all patients completed six cycles treatment. Discussion Lenalidomide is a therapeutic option for thalidomide-refractory patients with POEMS syndrome.

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“…Li et al. also reported a prospective clinical trial of low‐dose lenalidomide plus dexamethasone in 41 patients . The complete hematological response rate, VEGF response rate and neurological response rate was 46%, 83% and 95%, respectively.…”

Section: Treatmentmentioning

confidence: 97%

“…Lenalidomide is a derivative of thalidomide, and a promising therapeutic option for POEMS syndrome . Nozza et al.…”

Section: Treatmentmentioning

confidence: 99%

Polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy and skin changes syndrome: Diagnosis, treatment and the current status in Japan

Suichi

Misawa

2020

Clinical & Exp Neuroim

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Polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy and skin changes (POEMS) syndrome is a rare cause of demyelinating polyneuropathy associated with monoclonal proliferation of plasma cells. Because the disorder shows diverse symptoms, its diagnosis is often difficult. Previously reported diagnostic criteria had many items, and their accuracy was not confirmed. Recently, new diagnostic criteria have been proposed, which consist of just seven items and have better diagnostic accuracy. Since around 2000, treatments for myeloma, such as high‐dose chemotherapy with autologous stem cell transplantation, immunomodulatory drugs and proteasome inhibitors, have been applied to polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy and skin changes syndrome. A recent national survey carried out in Japan showed that the prognosis has been prominently improved by these plasma cell‐targeting therapeutic interventions: 10‐year overall survival was 93%. Conversely, 24% of the patients were still treated with corticosteroid alone as an initial treatment, suggesting that recognition of the disorder is not sufficient. Accurate diagnosis and proper treatment in the early phase of the disease are essential to further improve the prognosis of polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy and skin changes syndrome. The present review focuses on diagnosis, treatment and the current status of the disease in Japan.

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A prospective phase II study of low dose lenalidomide plus dexamethasone in patients with newly diagnosed polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes (POEMS) syndrome

Cited by 50 publications

References 25 publications

POEMS Syndrome: 2019 Update on diagnosis, risk‐stratification, and management

POEMS Syndrome: 2019 Update on diagnosis, risk‐stratification, and management

Lenalidomide Treatment for Thalidomide-refractory POEMS Syndrome: A Prospective Single-arm Clinical Trial

Polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy and skin changes syndrome: Diagnosis, treatment and the current status in Japan

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