scholarly journals How I treat POEMS syndrome

Blood ◽  
2012 ◽  
Vol 119 (24) ◽  
pp. 5650-5658 ◽  
Author(s):  
Angela Dispenzieri
Keyword(s):  
Plasma Cell ◽  
Cell Clone ◽  
Treatment Strategies ◽  
Castleman Disease ◽  
Poems Syndrome ◽  
The Other ◽  
Bone Lesions ◽  
High Dose ◽  
Sclerotic Bone ◽  
Sclerotic Bone Lesions

Abstract 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, high-dose 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.

POEMS Syndrome

Hematology ◽  
2005 ◽  
Vol 2005 (1) ◽  
pp. 360-367 ◽  
Author(s):  
Angela Dispenzieri
Keyword(s):  
Peripheral Neuropathy ◽  
Chronic Inflammatory Demyelinating Polyneuropathy ◽  
Early Recognition ◽  
Correct Diagnosis ◽  
Bone Lesion ◽  
High Dose Chemotherapy ◽  
Poems Syndrome ◽  
Bone Lesions ◽  
High Dose ◽  
Sclerotic Bone

Abstract POEMS syndrome is defined by the presence of a peripheral neuropathy (P), a monoclonal plasma cell disorder (M), and other paraneoplastic features, the most common of which include organomegaly (O), endocrinopathy (E), skin changes (S), papilledema, edema, effusions, ascites, and thrombocytosis. Virtually all patients will have either sclerotic bone lesion(s) or co-existent Castleman’s disease. Not all features of the disease are required to make the diagnosis, and early recognition is important to reduce morbidity. Other names for the syndrome include osteosclerotic myeloma, Crow-Fukase syndrome, or Takatsuki syndrome. Because the peripheral neuropathy is frequently the overriding symptom and because the characteristics of the neuropathy are similar to that chronic inflammatory demyelinating polyneuropathy (CIDP), patients are frequently misdiagnosed with CIDP or monoclonal gammopathy of underdetermined significance (MGUS)-associated peripheral neuropathy. Not until additional features of the POEMS syndrome are recognized is the correct diagnosis made and effective therapies initiated. Clues to an early diagnosis include thrombocytosis and sclerotic bone lesions on plain skeletal radiographs. Therapies that may be effective in patients with CIDP and MGUS-associated peripheral neuropathy (intravenous gammaglobulin and plasmapheresis) are not effective in patients with POEMS. Instead, the mainstays of therapy for patients with POEMS include irradiation, corticosteroids, and alkylator-based therapy, including high-dose chemotherapy with peripheral blood stem cell transplantation.

The Roel of High Dose Chemotherapy and Autologous Stem Cell Transplantation (ASCT) In PATIENTS with POEMS SYNDROME: A Retrospective study of the MM Subcommittee of the Chronic Leukemia Working Party of the EBMT,

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 4115-4115 ◽  
Author(s):  
Gordon Cook ◽  
Laurent Garderet ◽  
Anja van Biezen ◽  
Anja Henseler ◽  
Véronique Leblond ◽  
...  
Keyword(s):  
Plasma Cell ◽  
Conflicts Of Interest ◽  
Conditioning Regimen ◽  
Working Party ◽  
Poems Syndrome ◽  
Plasma Cell Dyscrasia ◽  
Bone Lesions ◽  
High Dose ◽  
Engraftment Syndrome ◽  
The Impact

Abstract Abstract 4115 Introduction: Polyneuropathy, organomegaly, endocrinopathy, skin changes associated with a paraproteinaemia (POEMS syndrome) is a rare paraneoplastic syndrome secondary to a plasma cell dyscrasia. Effective treatment, including ASCT, of the underlying plasma cell dyscrasia can control the disease and often dramatically control symptoms. Limited data is available for ASCT in POEMS. Specific Aim: The aim of this study was to describe the clinical outcome of ASCT for patients with POEMS syndrome, determining the impact of patient and disease-specific factors on prognosis. The incidence of engraftment syndrome and the presentation of relapse were examined. Methodology: Patient-, disease-, and transplant-related variables were collected according to the data entries in the EBMT database, including tracking incomplete data entries from participating centers. Results: 116 patients underwent an ASCT between 1997–2009 and satisfied the entry criteria. The median age was 50 yrs (range 26–69) with 56.8% of patients '50 year of age. 58.6% had peripheral neuropathy, 66.2% volume overload, 48.3% had organomegaly, 19.8% had papiloedema, 46.6% had dermopathies and 34.5% had sclerotic bone lesions at presentation. The median time from diagnosis to ASCT was 7.8 mns (range 1–346) with 34.5% of patients receiving an ASCT >12 months from diagnosis. The graft source was PBSC in 100% of patients. Disease status at ASCT was: 32% CR/PR, 30% SD/MR/untreated and 5 in PD. Missing information on stage in 33% of the cases. The conditioning regimen was Melphalan ≥200mg/m2 in 52.5%, Melphalan <200mg/m2 in 9.3% (38.1 of data on dose is missing) and TBI-containing only 1 patient. Engraftment was seen in 112 (96.6%) patients with failed engraftment reported in 3 patients (2.5%). Details of the occurrence of engraftment syndrome are currently under collection and analysis though peri-engraftment fever was reported in 23.4% and pulmonary infiltrates in 4.8%. Haematological response was characterized as CR in 31%, PR in 20.7%, <PR in 20.7% and currently unknown in 27.6%. Best disease response, in terms of end-organ response is under evaluation. With a median follow-up of 30.1 mns (range 0.1–161), 90.5 % of patients are alive and only 8.6% of patients have relapsed. The non-relapse mortality was 6.9%. Causes of death: 5 died of infection, 2 from graft failure, 1 from cardiac toxicity. The 3-year probabilities of PFS and OS are 82% and 94%, respectively. The 5-year probabilities of PFS and OS are 80% and 92%, respectively. The data analyzed in this study, to-date, demonstrates that ASCT can be an effective and safe therapeutic modality for patients with POEMS syndrome. The role of high dose therapy compared with more conventional dose therapies warrants further investigation. Disclosures: No relevant conflicts of interest to declare.

The Role Of High Dose Chemotherapy and Autologous Stem Cell Transplantation (ASCT) In Patients With POEMS Syndrome: A Retrospective study Of The Plasma Cell Disorders Subcommittee Of The Chronic Malignancy Working Party Of The EBMT

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 415-415
Author(s):  
Gordon Cook ◽  
Laurent Garderet ◽  
Simona Iacobelli ◽  
Curly Morris ◽  
Nikolaus Kroeger ◽  
...  
Keyword(s):  
Median Time ◽  
Plasma Cell ◽  
Poems Syndrome ◽  
Plasma Cell Dyscrasia ◽  
Bone Lesions ◽  
High Dose ◽  
Specific Response ◽  
Organ Specific ◽  
Engraftment Syndrome ◽  
The Impact

Abstract Introduction Polyneuropathy, organomegaly, endocrinopathy, dermopathy associated with a paraproteinaemia (POEMS syndrome) is a rare paraneoplastic syndrome secondary to a plasma cell dyscrasia. Effective treatment of the underlying plasma cell dyscrasia, including ASCT, can control the disease and often dramatically control symptoms though limited data is available for ASCT in POEMS. Specific Aim The aim of this study was to describe the clinical outcome of ASCT for patients with POEMS syndrome. We wish to determine the impact of patient and disease-specific factors on prognosis and effectively measure the extent of systemic disease involvement and organ-specific responsiveness of ASCT. Methodology Patient-, disease-, and transplant-related variables were collected according to the data entries in the EBMT database, including tracking incomplete data entries from participating centers. Systemic involvement and organ-specific response to ASCT was detailed utilizing an organ involvement tool pre- and post-ASCT. Results 127 patients underwent an ASCT between 1997-2010 and satisfied the entry criteria. The median age was 50 years (range 26-69) with 51.2% ≤50 years of age. The extent of systematic disease involvement was: peripheral neuropathy in 58.6%, volume overload in 66.2%, organomegaly in 48.3%, papilledema in 19.8%, dermopathy in 46.6% and 34.5% had sclerotic bone lesions at presentation. The median time from diagnosis to ASCT was 7.5 months (range 1-346) with 31.5% of patients receiving an ASCT >12 months from diagnosis. The graft source was PBSC in 100% of patients. Disease status at ASCT was: 47.5% CR/PR, 34% SD/MR/untreated and 18.4% in PD (missing information in 19% of patients). The conditioning regimen was Melphalan ≥200mg/m2 in 52.5%, Melphalan <200mg/m2 in 9.3% (38.1 of data on dose is missing) and TBI-containing in only 1 patient. The total rate of engraftment at 3 months was 96.8%, with 2 patients (1.6%) dying before engraftment. Engraftment syndrome was documented in 37% of ASCT recipients including peri-engraftment fever in 23.4% & pulmonary infiltrates in 4.8%. Haematological complete response (CHR) was characterized in 25.2% with 16.5% having progressive disease or died without attaining CHR. CHR was demonstrated in13.3% at 12 month, 20% at 36 months and 23% at 60 months (median time to CHR of 8.6 months) post-ASCT. Both organ-specific & overall systemic response data is currently under analysis, including time-to-maximum response. With a median follow-up of 47.7 months (95%CI 38.3, 58.6), the non-relapse mortality at 1, 2 & 5 years was 3.3% (95%CI 0.1-6.4%), 4.4% (95%CI 0.6-8.2%) & 7.7% (1.9-13.6%), respectively. The median progression-free survival (PFS) was 106 months (95% CI 87.8, NR) with a 5-year PFS of 73.5% (95%CI 63.2-83.7%). The 5-year overall survival (OS) was 88.6% (95% CI 81.5-95.8%). The data analyzed in this study, demonstrates that ASCT can be an effective & safe therapeutic modality for patients with POEMS syndrome. Due to the systemic inflammatory nature of the condition, due care should be taken to supporting these patients through this procedure, especially in relation to engraftment syndrome. Disclosures: Nagler: Teva: Honoraria, Travel grants, research grants Other.

Lenalidomide Therapy in Nine Patients with POEMS Syndrome.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 3872-3872 ◽  
Author(s):  
Arnaud Jaccard ◽  
Julie Abraham ◽  
Christian Recher ◽  
Remy Dulery ◽  
Isabelle Guichard ◽  
...  
Keyword(s):  
Peripheral Neuropathy ◽  
Board Of Directors ◽  
Plasma Cells ◽  
High Dose Chemotherapy ◽  
Poems Syndrome ◽  
Bone Lesions ◽  
High Dose ◽  
Advisory Committees ◽  
Sclerotic Bone Lesions ◽  
Vegf Level

Abstract Abstract 3872 Poster Board III-808 Introduction POEMS syndrome is a rare disease characterized by peripheral neuropathy, organomegaly, endocrinopathy, monoclonal plasma cells, skin changes, papilledema, volume overload, sclerotic bone lesions, thrombocytosis, and high serum VEGF level. Efficient treatments consist in irradiation for patients with localized solitary plasmocytoma and high-dose chemotherapy with autologous stem cell transplantation for appropriate candidates without a focal lesion. Conventional myeloma chemotherapy can only control the disease in a limited number of patients. Results of monoclonal anti-VEGF antibodies, which seem to be attractive due to the role of VEGF in this disease, are controversy with efficacy in 3 patients but treatment related deaths in 2 other patients. Thalidomide effectiveness has been reported in Japanese patients but enthusiasm for its use is tempered by the high incidence of thalidomide-induced peripheral neuropathy. Lenalidomide, which efficacy has been described in one observation (Dispenzieri, Blood 2007 110: 1075-1076), has the advantage of being anti-angiogenic, cytotoxic to malignant plasma cells and with a much lower risk of peripheral neuropathy. We reported here a multicentric French experience with this drug in POEMS syndrome. Patients and Methods There were 3 women and 6 men treated with Lenalidomide in 7 French centres. Median age was 60 (41-76). All patients had sensitive polyneuropathy with motor deficiency in 5 patients. A monoclonal component was present in all cases (IgA lambda in 7 patients, IgG lambda and lambda light chain only in 1 patient each). Other manifestations of POEMS syndrome included sclerotic bone lesions in 6 patients, endocrinopathies in 7 patients, skin changes in 8 patients, oedema in 7 patients, organomegaly in 5 patients, papilledema in 5 patients, thrombocytosis in 3 patients. VEGF serum level was elevated in 4 among 5 patients with a dosage. Previous treatments were high-dose chemotherapy with autologous stem cell transplantation in 3 patients, Melphalan-Prednisone in 3 patients because of advanced age, and prolonged steroid treatment in 2 patients. One patient received Lenalidomide as primary treatment before high-dose therapy. Lenalidomide was given during 21 days each month and sequentially associated with dexamethasone, 5 patients received 25 mg/day and 4 patients received 10 or 15 mg, for a median of 5 cycles (1 to 11). Results Serious side effects were noted in 3 patients with 2 hematologic toxicities (grade III and IV) and a cutaneous allergy. Six patients could be evaluated for hematologic response and all responded, complete response in 3 patients and partial in 3 (>25%). Clinical responses occurred early, before 3 months of treatment, in 6 cases among 8 (1 patient is not yet evaluable), with a marked improvement in performance and in neurological syndrome. Other manifestations of POEMS syndrome improved, especially oedema in 5 cases among 6. VEGF level (normal value < 500 pg/ml) could be serially measured in 4 patients with a normalization in 1 patient and a significant decrease in 3 patients, median 7100 pg/ml (2100-10100) before treatment to 887 pg/ml (304-3270). In 1 of these 3 patients VEGF level increased to initial value while he was still taking Lenalidomide. A second patient experimented a relapse 5 months after ending Lenalidomide, he is still in good response after Lenalidomide reintroduction. With a median follow-up of 12 months (1-26) all patients are alive. Conclusion Lenalidomide seems to be a very promising therapy in POEMS syndrome. It should be tested in larger studies in patients with a systemic disease, who are not able to receive high dose therapy, in relapsing patients and before high dose treatment to avoid transplant related morbidity, particularly engraftment syndrome. Disclosures: Jaccard: Celgene: Membership on an entity's Board of Directors or advisory committees. Facon:Celgene: Membership on an entity's Board of Directors or advisory committees and Speakers Bureau. Moreau:Celgene: Membership on an entity's Board of Directors or advisory committees. Fermand:Celgene: Speakers Bureau.

Durable Responses with Autologous Hematopoietic Stem Cell Transplantation in Patients with POEMS Syndrome

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 4522-4522
Author(s):  
Krina Patel ◽  
Robert Z. Orlowski ◽  
Nina Shah ◽  
Qaiser Bashir ◽  
Yvonne T Dinh ◽  
...  
Keyword(s):  
Castleman Disease ◽  
Poems Syndrome ◽  
Bone Lesions ◽  
High Dose ◽  
Hematopoietic Stem ◽  
Plasma Cell Disorder ◽  
One Year ◽  
Cell Disorder ◽  
Related Mortality

Abstract Abstract 4522 Introduction: POEMS syndrome (polyradiculoneuropathy, organomegaly, endocrinopathy, monoclonal plasma cell disorder, and skin changes) is a rare plasma cell disorder often associated with papilledema, extravascular volume overload, sclerotic bone lesions, Castleman disease, and high vascular endothelial growth factor (VEGF) levels. High-dose chemotherapy and autologous hematopoietic stem cell transplant (auto-HCT) can be used with good clinical response, but has significant post-transplant morbidity. Here we present our experience in 5 patients with POEMS syndrome who underwent auto-HCT at the MD Anderson Cancer Center (MDACC). Method: Between January 1999 and October 2010, 5 patients (4 males, 1 female) with POEMS syndrome received auto-HCT at MDACC. Diagnosis of POEMS was based on criteria of Dispenzieri et. al.1 All 5 patients had received systemic therapy prior to auto-HCT as follows: bortezomib+dexamethasone (dex) in 2, lenalidomide+dex in 1, cyclophosphamide+dex in 1, and pulse dex in 1. Two patients also received localized radiation for bone disease. Peripheral blood stem cells were collected with granulocyte colony-stimulating factor (G-CSF) in 4 patients and with cyclophosphamide+G-CSF in 1. The preparative regimen was melphalan 200 mg/m2 in 4 patients, while 1 patient received 180 mg/m2 due to renal insufficiency. We evaluated the response rate, toxicity, transplant-related mortality, progression-free survival (PFS) and overall survival (OS). Hematologic response was defined by the International Myeloma Working Group (IMWG) criteria2. Result: Median age at auto-HCT was 48 years (range: 39–58) and median time from diagnosis to auto-HCT was 16.6 months (6.4 – 89 months). All 5 patients had osteosclerotic bone lesions and monoclonal gammopathy: IgA lambda in 3, IgG lambda in 1, and IgG kappa in 1. Two patients had biopsy proven Castleman disease. Four patients had debilitating polyneuropathy. Other features were: skin involvement in 3, endocrinopathy in 2, ascites in 1, anasarca in 2, pulmonary hypertension in 1, and papilledema in 1. Median follow up after auto-HCT was 11.6 months (8.6 – 83.5 months). Median Karnofsky performance status was 80% (70–100) at the time of transplant and 90% (80–100) by the one year follow up visit. Two patients (40%) had complete response, 2 (40%) had a very good partial response, and 1 (20%) had a partial response by IMWG criteria. Engraftment syndrome was not seen in any of the 5 patients. Significant post auto-HCT complications were fungal pneumonia in 2 patients and pulmonary embolism in one. Patients fully recovered from these complications. One-year transplant-related mortality was 0%. All 5 patients had complete or significant resolution of their clinical symptoms after auto-HCT. Median PFS and OS have not yet been reached. With a median follow up of 11.6 months, 1-year PFS and OS were 100%. Four patients are alive and in remission, while one patient died 6 years after his auto-HCT secondary to gastrointestinal bleeding unrelated to his underlying disease. Conclusion: High-dose therapy followed by auto-HCT is safe and effective in selected patients with POEMS syndrome and is associated with durable responses and survival. Disclosures: No relevant conflicts of interest to declare.

Current Treatment Approaches to Newly Diagnosed Multiple Myeloma

2021 ◽  
Vol 44 (12) ◽  
pp. 690-699
Author(s):  
Susanne Ghandili ◽  
Katja C. Weisel ◽  
Carsten Bokemeyer ◽  
Lisa Beatrice Leypoldt
Keyword(s):  
Multiple Myeloma ◽  
Monoclonal Antibody ◽  
Plasma Cell ◽  
Cell Clone ◽  
Unmet Need ◽  
Continuous Treatment ◽  
High Dose ◽  
Line Treatment ◽  
First Line ◽  
First Line Treatment

<b><i>Background:</i></b> Multiple myeloma is a so far incurable malignant plasma cell disorder. During the past 2 decades, treatment paradigms substantially changed when novel drugs were introduced initially in treatment of relapsed disease and subsequently also in first-line treatment. <b><i>Summary:</i></b> Up to now, first-line treatment differs between patients initially classified as transplant eligible and those who are considered as nontransplant eligible. Transplant-eligible patients receive a primary proteasome inhibitor (PI)-based induction which is being combined with an immunomodulating agent and a CD38-directed monoclonal antibody followed by high-dose melphalan therapy and autologous stem cell transplantation with subsequent maintenance treatment with lenalidomide. Patients who are considered as nontransplant eligible receive upfront treatment preferentially with a continuous combination treatment either with a CD38-directed monoclonal antibody in combination with the immunomodulating agent lenalidomide or a lenalidomide-PI combination followed by lenalidomide maintenance. <b><i>Key Messages:</i></b> Primary goal of the initiated treatment is to induce a rapid and deep remission which ideally leads to an eradication of the residual plasma cell clone in sense of a minimal residual disease negativity. Achievement of long-term remission with limited toxicity despite continuous treatment strategies and maintenance or improvement of life-quality is key. Despite successful treatment options, specific difficult-to-treat subgroups, especially patients with high-risk myeloma remain with inferior prognosis and a clear unmet need for novel therapeutic strategies. Future concepts will evaluate cellular treatments and other innovative immunotherapies in first-line treatment in curative intention.

Proposal of new clinical diagnostic criteria for POEMS syndrome

2018 ◽  
Vol 90 (2) ◽  
pp. 133-137 ◽  
Author(s):  
Tomoki Suichi ◽  
Sonoko Misawa ◽  
Yasunori Sato ◽  
Minako Beppu ◽  
Emiko Sakaida ◽  
...  
Keyword(s):  
Diagnostic Criteria ◽  
Plasma Cell ◽  
Characteristic Curve ◽  
Specific Treatment ◽  
Poems Syndrome ◽  
Bone Lesions ◽  
Standard Group ◽  
Skin Changes ◽  
Immunoglobulin Light Chain Amyloidosis ◽  
Serious Disease

ObjectiveTo propose the optimal diagnostic criteria for polyneuropathy, organomegaly, endocrinopathy, M-protein and skin changes (POEMS) syndrome using appropriate statistical methods and disease controls.MethodsThis retrospective cohort study included 104 consecutive patients with suspected POEMS syndrome, among whom a gold standard group of 60 patients with definitive POEMS syndrome diagnosis were followed for at least 12 months to strictly exclude other disorders mimicking POEMS syndrome and to confirm response to POEMS syndrome-specific treatment. Thirty patients with chronic inflammatory demyelinating polyradiculoneuropathy (demyelinating polyradiculoneuropathy controls) and 30 with multiple myeloma or immunoglobulin light chain amyloidosis (monoclonal plasma cell proliferation controls) were also included. Logistic regression analyses were performed to determine optimal combination of clinical and laboratory abnormalities, characteristic of POEMS syndrome.ResultsThe diagnostic criteria were statistically defined as the presence of the three major criteria (polyneuropathy (typically demyelinating), monoclonal plasma cell proliferative disorder and elevated vascular endothelial growth factor) and at least two of the four minor criteria (oedema/effusion, skin changes, organomegaly and sclerotic bone lesions), based on best performance by area under the receiver operating characteristic curve analyses. The sensitivity and specificity were 100% and 100%, respectively; the diagnostic accuracy of the proposed criteria was equivalent to somewhat complicated previous criteria.ConclusionsThe statistically defined, simple diagnostic criteria for POEMS syndrome could accelerate early diagnosis and treatment, thereby contribute to better outcome in patients with this serious disease. Prospective larger studies are required to confirm the validity.

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