Abstract
POEMS syndrome (acronym of polyradiculoneuropathy, organomegaly, endocrinopathies, monoclonal protein, and dermopathy, skin) is a rare multisystemic disease due to an underlying plasma cell neoplasm.
You have full access to this open access chapter, Download chapter PDF
Similar content being viewed by others
1 POEMS Syndrome
1.1 Introduction
POEMS syndrome (acronym of polyradiculoneuropathy, organomegaly, endocrinopathies, monoclonal protein, and dermopathy, skin) is a rare multisystemic disease due to an underlying plasma cell neoplasm. The pathogenesis of the syndrome is not well understood. Other names of the POEMS syndrome that are less frequently used are osteosclerotic myeloma, Takatsuki syndrome, or Crow-Fukase syndrome.
1.2 Clinical and Laboratory Manifestations
POEMS predominate in male being the age of maximum incidence (50–60 years).
Characteristic manifestations are:
Polyneuropathy: Typically demyelinating. Peripheral, ascending, symmetrical and affecting both sensation and motor function. It is the dominant characteristic. |
Organomegaly: Hepatomegaly (50%), splenomegaly, or lymphadenopathy. |
Endocrinopathy: Present in 84% of patients: gonadal, thyroid, pituitary, parathyroid, pancreatic, adrenal (in order of frequency, and many times multiple). |
Monoclonal protein: Almost always λ light chain. Usually Ig A or IgG and ≤3 g/dL. Bone marrow smear <5 to 10% plasma cells. |
Skin changes: Hyperpigmentation, hypertrichosis, glomeruloid hemangiomata, white nails, plethora, acrocyanosis, flushing. |
Other important manifestations are: — Papilledema (in one third of patients) — Extravascular volume overload — Sclerotic bone lesionsa (95%) — Thrombocytosis (in 54%) — VEGF elevationb — Castleman disease (in 11–30%) |
1.3 Diagnosis
Not all the features within the acronym are required to make the diagnosis. There are other relevant features not included in the POEMS acronym also important: PEST (papilledema, extravascular volume overload, sclerotic bone lesions, thrombocytosis/erythrocytosis), elevated VEGF levels, abnormal pulmonary function tests, and a predisposition to thrombosis.
There is a Castleman variant of POEMS syndrome that may be associated with a clonal plasma cell disorder. When Castleman disease variant of POEMS syndrome occurs without evidence of plasma cell disorder, then this entity should be considered separately.
Clinical criteria for POEMS diagnostic are shown in Table 82.1.
1.4 Prognosis
Chronic course, median survival of nearly 14 years, rarely progression to multiple myeloma.
The number of POEMS features does not affect survival.
Risk factors associated to better survival are: albumin >3.2 g/dL, achievement of a complete hematological response and younger age. Lower VEGF levels, better response to treatment.
Risk factors associated to shorter survival are: clubbing, extravascular volume overload, respiratory symptoms, papilledema, and coexisting Castleman disease.
Thrombocytosis and high bone marrow infiltration are associated with risk of cerebrovascular accidents.
Patients candidates for radiation therapy have a better overall survival.
1.5 Standard Treatment
1.5.1 In Case of an Isolated Bone Lesion (or Multiple, But Localized)
Radiotherapy to affected site(s) improves the symptoms of POEMS syndrome and can be curative.
1.5.2 Rest of Patients (Disseminated Disease)
– MEL/DEX
– LENA/DEX, THAL/DEX, BOR (these last two agents are of limited use due to the intrinsic risk of peripheral neuropathy), CY/DEX.
– Plasmapheresis, IVIg, IFN-α, tamoxifen, ATRA, bevacizumab (anti-VEGF agent), argatroban, and strontium-89 (mostly single-case reports).
– Attention to supportive care is mandatory (physical therapy, orthotics, etc.).
– Auto-HSCT.
1.5.3 Response Criteria
Monitoring the response to treatment in POEMS syndrome is a challenge. Patients must be followed carefully comparing the deficits to baseline. VEGF is an imperfect marker due to discordances between disease activity and response. The size of monoclonal protein is typically small making standard MM response criteria inapplicable. Patients can present clinical benefit without M-protein response therefore a clinical scoring system which can focus on organ-specific response would be useful clinically. So, response criteria for POEMS syndrome could be done as follows: hematological response using a modified amyloid response criteria, VEGF response, CT/PET response, and a simplified organ response (polyneuropathy assessment, pulmonary function tests, and extravascular overload).
1.6 Autologous HSCT (Table 82.2)
2 Monoclonal Ig Deposition Disease
2.1 Introduction
Monoclonal Ig deposition is a clonal plasma cell dyscrasia in which light-chain and/or heavy-chain subunits of Igs form non-fibrillar deposits in various tissues, causing organ dysfunction. Light-chain deposition disease is the most common of these entities.
2.2 Clinical Manifestation/Laboratory
Kidney — Always affected: nephrotic syndrome, hypertension, and rapidly progressing renal insufficiency — Immunofluorescence shows deposition of light chains along glomerular and tubular basement membranes → nodular glomerulosclerosis — Deposits are non-fibrillar, almost always composed of κ chain, and do not stain with Congo red dye |
Heart and liver — Less frequently affected: restrictive cardiopathy, myocardial infarction, cholestatic jaundice, hepatic failure |
Monoclonal gammopathy — Electrophoresis, immunofixation of serum and/or urine, serum-free light chain measurement |
2.3 Diagnosis
Based on the biopsy of the affected organ (almost always kidney)
2.4 Treatment
Controversial, not standard due to the low incidence. Conventional chemotherapy commonly used for MM is unsatisfactory. Possible alternatives are:
– MEL + prednisone
– VAD (vincristine, doxorubicin, DEX)
– THAL/DEX, BOR/DEX
– Auto-HSCT (see Table 82.3)
Recommended References
Batalini F, Econimo L, Quillen K, et al. High-dose melphalan and stem cell transplantation in patients on dialysis due to immunoglobulin light-chain amyloidosis and monoclonal immunoglobulin deposition disease. Biol Blood Marrow Transplant. 2018;24:127–32.
Cook G, Iacobelli S, Van Biezen A, et al. High-dose therapy and autologous stem cell transplantation in patients with POEMS syndrome: a retrospective study of the Chronic Mailnancy Working Party of the European Society for Blood and Marrow Transplantation. Haematologica. 2017;102:160–7.
D’Souza A, Lacy M, Gertz M, et al. Long-term outcomes after autologous stem cell transplantation for patients with POEMS syndrome: a single-center experience. Blood. 2012;120:56–62.
Dispenzieri A. POEMS syndrome: 2017 update on diagnosis, risk-stratification, and management. Am J Hematol. 2017;92:814–29.
Dispenzieri A, Kyle RA, Lacy MQ, et al. POEMS syndrome: definitions and long-term outcome. Blood. 2003;101:2496–506.
Dispenzieri A, Lacy MQ, Hayman SR, et al. Peripheral blood stem cell transplant for POEMS syndrome is associated with high rates of engraftment syndrome. Eur J Haematol. 2008;80:397–406.
Girnius S, Seldin DC, Quillen K, et al. Long-term outcome of patients with monoclonal IgG deposition disease treated with high-dose melphalan and stem cell transplantation. Bone Marrow Transplant. 2011;46:161–2.
Hassoun H, Flombaum C, D’Agati VD, et al. High-dose melphalan and auto-SCT in patients with monoclonal Ig deposition disease. Bone Marrow Transplant. 2008;42:1–8.
Jaccard A, Royer B, Bordessoule D, et al. High-dose therapy and autologous blood stem cell transplantation in POEMS syndrome. Blood. 2002;99:3057–9.
Kourelis TV, Buadi FK, Kumar SK, et al. Long-term outcome of patients with POEMS syndrome: an update of the Mayo Clinic experience. Am J Hematol. 2016;91:585–9.
Li J, Zhang W, Jiao L, et al. Combination of melphalan and dexamethasone for patients with newly diagnosed POEMS syndrome. Blood. 2011a;117:6445–9.
Li J, Zhou DB, Huang Z, et al. Clinical characteristics and long-term outcome of patients with POEMS syndrome in China. Ann Hematol. 2011b;90:819–26.
Pozzi C, D’Amico M, Fogazzi GB, et al. Light chain deposition disease with renal involvement: clinical characteristics and prognostic factors. Am J Kidney Dis. 2003;42:1154–63.
Rovira M, Carreras E, Blade J, et al. Dramatic improvement of POEMS syndrome following autologous haematopoietic cell transplantation. Br J Haematol. 2001;115:373–5.
Tovar N, Cibeira MT, Rosiñol L, et al. Bortezomib/dexamethasone followed by stem cell transplantation as front line treatment for light-chain deposition disease. Eur J Haematol. 2012;89(4):340–4.
Author information
Authors and Affiliations
Corresponding author
Editor information
Editors and Affiliations
Rights and permissions
Open Access This chapter is licensed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license and indicate if changes were made.
The images or other third party material in this chapter are included in the chapter's Creative Commons license, unless indicated otherwise in a credit line to the material. If material is not included in the chapter's Creative Commons license and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder.
Copyright information
© 2019 EBMT and the Author(s)
About this chapter
Cite this chapter
Cook, G., Rovira, M. (2019). POEMS Syndrome and Disease Produced by Other Monoclonal Immunoglobulins. In: Carreras, E., Dufour, C., Mohty, M., Kröger, N. (eds) The EBMT Handbook. Springer, Cham. https://doi.org/10.1007/978-3-030-02278-5_82
Download citation
DOI: https://doi.org/10.1007/978-3-030-02278-5_82
Published:
Publisher Name: Springer, Cham
Print ISBN: 978-3-030-02277-8
Online ISBN: 978-3-030-02278-5
eBook Packages: MedicineMedicine (R0)