POEMS syndrome medical therapy: Difference between revisions

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==Overview==
==Overview==
 
[[Patient|Patients]] with isolated [[bone]] [[lesion]] without [[bone marrow]] clonal [[Plasma cell|plasma cells]] involvement can be treated with [[Radiation therapy|radiotherapy]]. [[Patient|Patients]] with a [[disseminated disease]] (more [[bone]] [[Lesion|lesions]] and/or [[bone marrow]] [[plasmacytosis]]) are the candidates for [[systemic therapy]]. [[Systemic therapy]] includes [[Corticosteroid|corticosteroids]], autologous [[stem cell transplantation]] (ASCT), induction therapy, alkylator-based therapy, [[thalidomide]], [[lenalidomide]], [[bortezomib]], and [[bevacizumab]].
 
==Medical Therapy==
==Medical Therapy==
*Patients with isolated bone lesion without bone marrow clonal plasma cells involvement can be treated with radiotherapy.<ref name="pmid22547581">{{cite journal |vauthors=Dispenzieri A |title=How I treat POEMS syndrome |journal=Blood |volume=119 |issue=24 |pages=5650–8 |date=June 2012 |pmid=22547581 |pmc=3425020 |doi=10.1182/blood-2012-03-378992 |url=}}</ref>
*[[Patient|Patients]] with isolated [[bone]] [[lesion]] without [[bone marrow]] clonal [[Plasma cell|plasma cells]] involvement can be treated with [[Radiation therapy|radiotherapy]].<ref name="pmid22547581">{{cite journal |vauthors=Dispenzieri A |title=How I treat POEMS syndrome |journal=Blood |volume=119 |issue=24 |pages=5650–8 |date=June 2012 |pmid=22547581 |pmc=3425020 |doi=10.1182/blood-2012-03-378992 |url=}}</ref>
*Patients with a disseminated disease (more bone lesions and/or bone marrow plasmacytosis) are the candidates for systemic therapy.<ref name="pmid22547581">{{cite journal |vauthors=Dispenzieri A |title=How I treat POEMS syndrome |journal=Blood |volume=119 |issue=24 |pages=5650–8 |date=June 2012 |pmid=22547581 |pmc=3425020 |doi=10.1182/blood-2012-03-378992 |url=}}</ref>
*[[Patient|Patients]] with a [[disseminated disease]] (more [[bone]] [[Lesion|lesions]] and/or [[bone marrow]] [[plasmacytosis]]) are the candidates for [[systemic therapy]].<ref name="pmid22547581">{{cite journal |vauthors=Dispenzieri A |title=How I treat POEMS syndrome |journal=Blood |volume=119 |issue=24 |pages=5650–8 |date=June 2012 |pmid=22547581 |pmc=3425020 |doi=10.1182/blood-2012-03-378992 |url=}}</ref>
===Radiotherapy===
===Radiotherapy===
*More than 50% of patients treated with radiotherapy show a significant improvement of neuropathy.<ref name="pmid12456500">{{cite journal |vauthors=Dispenzieri A, Kyle RA, Lacy MQ, Rajkumar SV, Therneau TM, Larson DR, Greipp PR, Witzig TE, Basu R, Suarez GA, Fonseca R, Lust JA, Gertz MA |title=POEMS syndrome: definitions and long-term outcome |journal=Blood |volume=101 |issue=7 |pages=2496–506 |date=April 2003 |pmid=12456500 |doi=10.1182/blood-2002-07-2299 |url=}}</ref>
*More than 50% of [[Patient|patients]] treated with [[Radiation therapy|radiotherapy]] show a significant improvement of [[neuropathy]].<ref name="pmid12456500">{{cite journal |vauthors=Dispenzieri A, Kyle RA, Lacy MQ, Rajkumar SV, Therneau TM, Larson DR, Greipp PR, Witzig TE, Basu R, Suarez GA, Fonseca R, Lust JA, Gertz MA |title=POEMS syndrome: definitions and long-term outcome |journal=Blood |volume=101 |issue=7 |pages=2496–506 |date=April 2003 |pmid=12456500 |doi=10.1182/blood-2002-07-2299 |url=}}</ref>
*Maximal response to radiotherapy, i.e., improvement of neuropathy, can not be attained until 2–3 years since the first evidence of the effect of therapy.<ref name="pmid25610609">{{cite journal |vauthors=Brewis MJ, Church AC, Peacock AJ, Thomson S, Tighe J, Johnson MK |title=Pulmonary hypertension in POEMS syndrome: resolution following radiotherapy |journal=Pulm Circ |volume=4 |issue=4 |pages=732–5 |date=December 2014 |pmid=25610609 |pmc=4278633 |doi=10.1086/678553 |url=}}</ref><ref name="pmid24532337">{{cite journal |vauthors=Dispenzieri A |title=POEMS syndrome: 2014 update on diagnosis, risk-stratification, and management |journal=Am. J. Hematol. |volume=89 |issue=2 |pages=214–23 |date=February 2014 |pmid=24532337 |doi=10.1002/ajh.23644 |url=}}</ref>
*Maximal response to [[Radiation therapy|radiotherapy]], i.e., improvement of [[neuropathy]], can not be attained until 2–3 years since the first evidence of the effect of [[therapy]].<ref name="pmid25610609">{{cite journal |vauthors=Brewis MJ, Church AC, Peacock AJ, Thomson S, Tighe J, Johnson MK |title=Pulmonary hypertension in POEMS syndrome: resolution following radiotherapy |journal=Pulm Circ |volume=4 |issue=4 |pages=732–5 |date=December 2014 |pmid=25610609 |pmc=4278633 |doi=10.1086/678553 |url=}}</ref><ref name="pmid24532337">{{cite journal |vauthors=Dispenzieri A |title=POEMS syndrome: 2014 update on diagnosis, risk-stratification, and management |journal=Am. J. Hematol. |volume=89 |issue=2 |pages=214–23 |date=February 2014 |pmid=24532337 |doi=10.1002/ajh.23644 |url=}}</ref>
*Improvement of anasarca, papilledema, pulmonary hypertension and skin changes may occur within months after the end of radiotherapy.<ref name="pmid25610609">{{cite journal |vauthors=Brewis MJ, Church AC, Peacock AJ, Thomson S, Tighe J, Johnson MK |title=Pulmonary hypertension in POEMS syndrome: resolution following radiotherapy |journal=Pulm Circ |volume=4 |issue=4 |pages=732–5 |date=December 2014 |pmid=25610609 |pmc=4278633 |doi=10.1086/678553 |url=}}</ref><ref name="pmid24532337">{{cite journal |vauthors=Dispenzieri A |title=POEMS syndrome: 2014 update on diagnosis, risk-stratification, and management |journal=Am. J. Hematol. |volume=89 |issue=2 |pages=214–23 |date=February 2014 |pmid=24532337 |doi=10.1002/ajh.23644 |url=}}</ref>
*Improvement of [[anasarca]], [[papilledema]], [[pulmonary hypertension]] and [[skin changes]] may occur within months after the end of [[Radiation therapy|radiotherapy]].<ref name="pmid25610609">{{cite journal |vauthors=Brewis MJ, Church AC, Peacock AJ, Thomson S, Tighe J, Johnson MK |title=Pulmonary hypertension in POEMS syndrome: resolution following radiotherapy |journal=Pulm Circ |volume=4 |issue=4 |pages=732–5 |date=December 2014 |pmid=25610609 |pmc=4278633 |doi=10.1086/678553 |url=}}</ref><ref name="pmid24532337">{{cite journal |vauthors=Dispenzieri A |title=POEMS syndrome: 2014 update on diagnosis, risk-stratification, and management |journal=Am. J. Hematol. |volume=89 |issue=2 |pages=214–23 |date=February 2014 |pmid=24532337 |doi=10.1002/ajh.23644 |url=}}</ref>
*Radiotherapy can be considered as primary therapy if the bone lesion is large despite an infiltrated bone marrow.
*[[Radiation therapy|Radiotherapy]] can be considered as primary [[therapy]] if the [[bone]] [[lesion]] is large despite an infiltrated [[bone marrow]].
*[[Radiation therapy|Radiotherapy]] can also be considered in [[Relapse|relapsed]] [[Patient|patients]] with 1 or 2 [[bone]] [[Lesion|lesions]] on [[Positron emission tomography|positron emission tomography (PET)]].
===Systemic Therapy===
===Systemic Therapy===
====Corticosteroids====
====Corticosteroids====
*Corticosteroids can attenuate the symptoms for a short time without affecting the progression of the syndrome.<ref name="pmid26331353">{{cite journal |vauthors=Dispenzieri A |title=POEMS syndrome: update on diagnosis, risk-stratification, and management |journal=Am. J. Hematol. |volume=90 |issue=10 |pages=951–62 |date=October 2015 |pmid=26331353 |doi=10.1002/ajh.24171 |url=}}</ref>
*[[Corticosteroid|Corticosteroids]] can attenuate the [[Symptom|symptoms]] for a short time without affecting the progression of the [[syndrome]].<ref name="pmid26331353">{{cite journal |vauthors=Dispenzieri A |title=POEMS syndrome: update on diagnosis, risk-stratification, and management |journal=Am. J. Hematol. |volume=90 |issue=10 |pages=951–62 |date=October 2015 |pmid=26331353 |doi=10.1002/ajh.24171 |url=}}</ref>
====Autologous Stem Cell Transplantation (ASCT)====
====Autologous Stem Cell Transplantation (ASCT)====
*It is considered the preferred initial therapy in young patients.<ref name="pmid22611150">{{cite journal |vauthors=D'Souza A, Lacy M, Gertz M, Kumar S, Buadi F, Hayman S, Dingli D, Zeldenrust S, Kyle R, Ansell S, Inwards D, Johnston P, Micallef I, Porrata L, Litzow M, Gastineau D, Hogan W, Dispenzieri A |title=Long-term outcomes after autologous stem cell transplantation for patients with POEMS syndrome (osteosclerotic myeloma): a single-center experience |journal=Blood |volume=120 |issue=1 |pages=56–62 |date=July 2012 |pmid=22611150 |doi=10.1182/blood-2012-04-423178 |url=}}</ref>
*It is considered the preferred initial therapy in young [[Patient|patients]].<ref name="pmid22611150">{{cite journal |vauthors=D'Souza A, Lacy M, Gertz M, Kumar S, Buadi F, Hayman S, Dingli D, Zeldenrust S, Kyle R, Ansell S, Inwards D, Johnston P, Micallef I, Porrata L, Litzow M, Gastineau D, Hogan W, Dispenzieri A |title=Long-term outcomes after autologous stem cell transplantation for patients with POEMS syndrome (osteosclerotic myeloma): a single-center experience |journal=Blood |volume=120 |issue=1 |pages=56–62 |date=July 2012 |pmid=22611150 |doi=10.1182/blood-2012-04-423178 |url=}}</ref>
*It is associated with a durable response even if patients experience a relapse of the disease.<ref name="pmid22611150">{{cite journal |vauthors=D'Souza A, Lacy M, Gertz M, Kumar S, Buadi F, Hayman S, Dingli D, Zeldenrust S, Kyle R, Ansell S, Inwards D, Johnston P, Micallef I, Porrata L, Litzow M, Gastineau D, Hogan W, Dispenzieri A |title=Long-term outcomes after autologous stem cell transplantation for patients with POEMS syndrome (osteosclerotic myeloma): a single-center experience |journal=Blood |volume=120 |issue=1 |pages=56–62 |date=July 2012 |pmid=22611150 |doi=10.1182/blood-2012-04-423178 |url=}}</ref>
*It is associated with a durable response even if [[Patient|patients]] experience a [[relapse]] of the [[disease]].<ref name="pmid22611150">{{cite journal |vauthors=D'Souza A, Lacy M, Gertz M, Kumar S, Buadi F, Hayman S, Dingli D, Zeldenrust S, Kyle R, Ansell S, Inwards D, Johnston P, Micallef I, Porrata L, Litzow M, Gastineau D, Hogan W, Dispenzieri A |title=Long-term outcomes after autologous stem cell transplantation for patients with POEMS syndrome (osteosclerotic myeloma): a single-center experience |journal=Blood |volume=120 |issue=1 |pages=56–62 |date=July 2012 |pmid=22611150 |doi=10.1182/blood-2012-04-423178 |url=}}</ref>
*A retrospective study of the Plasma Cell Disorder sub-committee of the Chronic Malignancy Working Party of the European Society for Blood & Marrow Transplantation showed the 1-year non-relapse mortality to be 3.3%.<ref name="pmid27634201">{{cite journal |vauthors=Cook G, Iacobelli S, van Biezen A, Ziagkos D, LeBlond V, Abraham J, McQuaker G, Schoenland S, Rambaldi A, Halaburda K, Rovira M, Sica S, Byrne J, Sanz RG, Nagler A, van de Donk NW, Sinisalo M, Cook M, Kröger N, De Witte T, Morris C, Garderet L |title=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 |journal=Haematologica |volume=102 |issue=1 |pages=160–167 |date=January 2017 |pmid=27634201 |pmc=5210246 |doi=10.3324/haematol.2016.148460 |url=}}</ref>
*A retrospective study of the Plasma Cell Disorder sub-committee of the Chronic Malignancy Working Party of the European Society for Blood & Marrow Transplantation showed the 1-year non-[[relapse]] [[Mortality rate|mortality]] to be 3.3%.<ref name="pmid27634201">{{cite journal |vauthors=Cook G, Iacobelli S, van Biezen A, Ziagkos D, LeBlond V, Abraham J, McQuaker G, Schoenland S, Rambaldi A, Halaburda K, Rovira M, Sica S, Byrne J, Sanz RG, Nagler A, van de Donk NW, Sinisalo M, Cook M, Kröger N, De Witte T, Morris C, Garderet L |title=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 |journal=Haematologica |volume=102 |issue=1 |pages=160–167 |date=January 2017 |pmid=27634201 |pmc=5210246 |doi=10.3324/haematol.2016.148460 |url=}}</ref>
 
*Young [[Patient|patients]] can be ineligible for ASCT in the presence of serous cavity effusion, [[Renal insufficiency|renal impairment]], [[pulmonary hypertension]],  and severe [[End organ damage|end-organ dysfunction]].
*It is a [[lethal]] procedure and can result in engraftment syndrome ([[fever]], [[rash]], [[diarrhea]], [[weight gain]], and [[Respiratory system|respiratory]] problems that occur 7–15 days since [[stem cell]] [[Intravenous therapy|infusion]]).<ref name="pmid18221391">{{cite journal |vauthors=Dispenzieri A, Lacy MQ, Hayman SR, Kumar SK, Buadi F, Dingli D, Litzow MR, Gastineau DA, Inwards DJ, Elliott MA, Micallef IN, Ansell SM, Hogan WJ, Porrata LF, Johnston PA, Afessa B, Bryce A, Kyle RA, Gertz MA |title=Peripheral blood stem cell transplant for POEMS syndrome is associated with high rates of engraftment syndrome |journal=Eur. J. Haematol. |volume=80 |issue=5 |pages=397–406 |date=May 2008 |pmid=18221391 |pmc=2327207 |doi=10.1111/j.1600-0609.2008.01037.x |url=}}</ref>
====Induction Therapy====
*Induction [[therapy]] involves [[cyclophosphamide]] or [[lenalidomide]], [[thalidomide]] or [[bortezomib]] in combination with high-dose [[dexamethasone]].<ref name="pmid22843279">{{cite journal |vauthors=Kanai K, Sawai S, Sogawa K, Mori M, Misawa S, Shibuya K, Isose S, Fujimaki Y, Noto Y, Sekiguchi Y, Nasu S, Nakaseko C, Takano S, Yoshitomi H, Miyazaki M, Nomura F, Kuwabara S |title=Markedly upregulated serum interleukin-12 as a novel biomarker in POEMS syndrome |journal=Neurology |volume=79 |issue=6 |pages=575–82 |date=August 2012 |pmid=22843279 |doi=10.1212/WNL.0b013e318263c42b |url=}}</ref>
*In [[Patient|patients]] with an overproduction of [[Interleukin 12|IL-12]] and [[Vascular endothelial growth factor|VEGF]], induction [[therapy]] can modify the hypercytokinemia status, improving clinical condition and can control [[disease]] severity making [[Patient|patients]] eligible for ASCT.<ref name="pmid22843279">{{cite journal |vauthors=Kanai K, Sawai S, Sogawa K, Mori M, Misawa S, Shibuya K, Isose S, Fujimaki Y, Noto Y, Sekiguchi Y, Nasu S, Nakaseko C, Takano S, Yoshitomi H, Miyazaki M, Nomura F, Kuwabara S |title=Markedly upregulated serum interleukin-12 as a novel biomarker in POEMS syndrome |journal=Neurology |volume=79 |issue=6 |pages=575–82 |date=August 2012 |pmid=22843279 |doi=10.1212/WNL.0b013e318263c42b |url=}}</ref>
*The use of induction [[therapy]] before ASCT can also decrease the [[incidence]] of peritransplant [[Complication (medicine)|complications]].<ref name="pmid21815185">{{cite journal |vauthors=Jimenez-Zepeda VH, Trudel S, Reece DE, Chen C, Rabea AM, Kukreti V |title=Cyclophosphamide and prednisone induction followed by cyclophosphamide mobilization effectively decreases the incidence of engraftment syndrome in patients with POEMS syndrome who undergo stem cell transplantation |journal=Am. J. Hematol. |volume=86 |issue=10 |pages=873–5 |date=October 2011 |pmid=21815185 |doi=10.1002/ajh.22115 |url=}}</ref><ref name="pmid22825426">{{cite journal |vauthors=Li J, Zhang W, Duan MH, Jiao L, Zhu TN, Han B, Zhang L, Gan J, Zhou DB |title=PBSC mobilization in newly diagnosed patients with POEMS syndrome: outcomes and prognostic factors |journal=Bone Marrow Transplant. |volume=48 |issue=2 |pages=233–7 |date=February 2013 |pmid=22825426 |doi=10.1038/bmt.2012.138 |url=}}</ref>
*Induction [[therapy]] before mobilization (i.e., stimulation of [[Stem cell|stem cells]] out of the [[bone marrow]] into the [[Blood|bloodstream]]) reduces the levels of [[Cytokine|cytokines]] and also reduces the risk of inadequate mobilization.
====Alkylator-base Therapy====
*It is the treatment of choice for [[Patient|patients]] who are not suitable for ASCT.
*It consists of [[melphalan]] or [[cyclophosphamide]] plus [[Corticosteroid|corticosteroids]].<ref name="pmid12456500">{{cite journal |vauthors=Dispenzieri A, Kyle RA, Lacy MQ, Rajkumar SV, Therneau TM, Larson DR, Greipp PR, Witzig TE, Basu R, Suarez GA, Fonseca R, Lust JA, Gertz MA |title=POEMS syndrome: definitions and long-term outcome |journal=Blood |volume=101 |issue=7 |pages=2496–506 |date=April 2003 |pmid=12456500 |doi=10.1182/blood-2002-07-2299 |url=}}</ref>
*It is associated with a 2-year overall survival (OS) rate of 78%.<ref name="pmid12456500">{{cite journal |vauthors=Dispenzieri A, Kyle RA, Lacy MQ, Rajkumar SV, Therneau TM, Larson DR, Greipp PR, Witzig TE, Basu R, Suarez GA, Fonseca R, Lust JA, Gertz MA |title=POEMS syndrome: definitions and long-term outcome |journal=Blood |volume=101 |issue=7 |pages=2496–506 |date=April 2003 |pmid=12456500 |doi=10.1182/blood-2002-07-2299 |url=}}</ref>
*[[Adverse effect (medicine)|Adverse effects]] of this [[therapy]] include [[Hematology|hematologic]] [[neoplasia]] such as [[leukemia]] and [[Myelodysplastic syndrome|myelodysplasia]].
*In the first prospective clinical trial, 81% of the [[Patient|patients]] showed [[Hematology|hematologic]] response and 100% of the [[Patient|patients]] showed improvement in [[Neurology|neurological]] [[Symptom|symptoms]] and reduction on [[Vascular endothelial growth factor|VEGF]] levels.<ref name="pmid21393478">{{cite journal |vauthors=Li J, Zhang W, Jiao L, Duan MH, Guan HZ, Zhu WG, Tian Z, Zhou DB |title=Combination of melphalan and dexamethasone for patients with newly diagnosed POEMS syndrome |journal=Blood |volume=117 |issue=24 |pages=6445–9 |date=June 2011 |pmid=21393478 |pmc=3123016 |doi=10.1182/blood-2010-12-328112 |url=}}</ref>
*Data on the long term effect of this [[therapy]] is still unavailable.<ref name="pmid21393478">{{cite journal |vauthors=Li J, Zhang W, Jiao L, Duan MH, Guan HZ, Zhu WG, Tian Z, Zhou DB |title=Combination of melphalan and dexamethasone for patients with newly diagnosed POEMS syndrome |journal=Blood |volume=117 |issue=24 |pages=6445–9 |date=June 2011 |pmid=21393478 |pmc=3123016 |doi=10.1182/blood-2010-12-328112 |url=}}</ref>
====Thalidomide====
*It has been tested in POEMS [[syndrome]] due to its antiangiogenic, anti-[[Inflammation|inflammatory]], and [[Immunomodulator|immunomodulating]] properties.
*A multicenter, randomized, double-blind study showed a reduction of [[Vascular endothelial growth factor|VEGF]] [[serum]] level with [[thalidomide]].<ref name="pmid27496680">{{cite journal |vauthors=Misawa S, Sato Y, Katayama K, Nagashima K, Aoyagi R, Sekiguchi Y, Sobue G, Koike H, Yabe I, Sasaki H, Watanabe O, Takashima H, Nishizawa M, Kawachi I, Kusunoki S, Mitsui Y, Kikuchi S, Nakashima I, Ikeda S, Kohara N, Kanda T, Kira J, Hanaoka H, Kuwabara S |title=Safety and efficacy of thalidomide in patients with POEMS syndrome: a multicentre, randomised, double-blind, placebo-controlled trial |journal=Lancet Neurol |volume=15 |issue=11 |pages=1129–37 |date=October 2016 |pmid=27496680 |doi=10.1016/S1474-4422(16)30157-0 |url=}}</ref><ref name="pmid18469028">{{cite journal |vauthors=Kuwabara S, Misawa S, Kanai K, Sawai S, Hattori T, Nishimura M, Nakaseko C |title=Thalidomide reduces serum VEGF levels and improves peripheral neuropathy in POEMS syndrome |journal=J. Neurol. Neurosurg. Psychiatry |volume=79 |issue=11 |pages=1255–7 |date=November 2008 |pmid=18469028 |doi=10.1136/jnnp.2008.150177 |url=}}</ref>
*[[Neurotoxicity]], a serious [[Adverse effect (medicine)|side effect]], limits the use of [[thalidomide]].<ref name="pmid15114600">{{cite journal |vauthors=Sinisalo M, Hietaharju A, Sauranen J, Wirta O |title=Thalidomide in POEMS syndrome: case report |journal=Am. J. Hematol. |volume=76 |issue=1 |pages=66–8 |date=May 2004 |pmid=15114600 |doi=10.1002/ajh.20051 |url=}}</ref><ref name="pmid18469028">{{cite journal |vauthors=Kuwabara S, Misawa S, Kanai K, Sawai S, Hattori T, Nishimura M, Nakaseko C |title=Thalidomide reduces serum VEGF levels and improves peripheral neuropathy in POEMS syndrome |journal=J. Neurol. Neurosurg. Psychiatry |volume=79 |issue=11 |pages=1255–7 |date=November 2008 |pmid=18469028 |doi=10.1136/jnnp.2008.150177 |url=}}</ref>
====Lenalidomide====
*It appears to be the most promising drug for the treatment of POEMS [[syndrome]].
*It has been reported to have a long-lasting response and good tolerability.<ref name="pmid17644745">{{cite journal |vauthors=Dispenzieri A, Klein CJ, Mauermann ML |title=Lenalidomide therapy in a patient with POEMS syndrome |journal=Blood |volume=110 |issue=3 |pages=1075–6 |date=August 2007 |pmid=17644745 |doi=10.1182/blood-2007-03-082354 |url=}}</ref><ref name="pmid23335406">{{cite journal |vauthors=Royer B, Merlusca L, Abraham J, Musset L, Haroche J, Choquet S, Leleu X, Sebban C, Decaux O, Galicier L, Roussel M, Recher C, Banos A, Guichard I, Brisseau JM, Godmer P, Hermine O, Deplanque G, Facon T, Asli B, Leblond V, Fermand JP, Marolleau JP, Jaccard A |title=Efficacy of lenalidomide in POEMS syndrome: a retrospective study of 20 patients |journal=Am. J. Hematol. |volume=88 |issue=3 |pages=207–12 |date=March 2013 |pmid=23335406 |doi=10.1002/ajh.23374 |url=}}</ref><ref name="pmid22488443">{{cite journal |vauthors=Vannata B, Laurenti L, Chiusolo P, Sorà F, Balducci M, Sabatelli M, Luigetti M, Giannotta C, De Stefano V, Leone G, Sica S |title=Efficacy of lenalidomide plus dexamethasone for POEMS syndrome relapsed after autologous peripheral stem-cell transplantation |journal=Am. J. Hematol. |volume=87 |issue=6 |pages=641–2 |date=June 2012 |pmid=22488443 |doi=10.1002/ajh.23195 |url=}}</ref><ref name="pmid21849771">{{cite journal |vauthors=Suyanı E, Yağcı M, Sucak GT |title=Complete remission with a combination of lenalidomide, cyclophosphamide and prednisolone in a patient with incomplete POEMS syndrome |journal=Acta Haematol. |volume=126 |issue=4 |pages=199–201 |date=2011 |pmid=21849771 |doi=10.1159/000329896 |url=}}</ref><ref name="pmid25401269">{{cite journal |vauthors=Cai QQ, Wang C, Cao XX, Cai H, Zhou DB, Li J |title=Efficacy and safety of low-dose lenalidomide plus dexamethasone in patients with relapsed or refractory POEMS syndrome |journal=Eur. J. Haematol. |volume=95 |issue=4 |pages=325–30 |date=October 2015 |pmid=25401269 |doi=10.1111/ejh.12492 |url=}}</ref>
*A [[retrospective]] pooled analysis of 51 subjects treated with [[lenalidomide]] showed a 12-month progression free survival (PFS) rate of 93% and a 24-month rate of 47%.<ref name="pmid24295131">{{cite journal |vauthors=Zagouri F, Kastritis E, Gavriatopoulou M, Sergentanis TN, Psaltopoulou T, Terpos E, Dimopoulos MA |title=Lenalidomide in patients with POEMS syndrome: a systematic review and pooled analysis |journal=Leuk. Lymphoma |volume=55 |issue=9 |pages=2018–23 |date=September 2014 |pmid=24295131 |doi=10.3109/10428194.2013.869329 |url=}}</ref>
*[[Lenalidomide]], as an induction [[therapy]] before the [[Organ transplant|transplant]], can improve the [[patient]] clinical status and decrease [[Organ transplant|transplant]]-related [[morbidity]].
====Bortezomib====
*[[Bortezomib]], alone or in combination, can be used in newly-[[Diagnosis|diagnosed]] and in [[Relapse|relapsed]] [[Patient|patients]].<ref name="pmid19674063">{{cite journal |vauthors=Tang X, Shi X, Sun A, Qiu H, Gu B, Zhou H, Xue S, Liu Y, Ruan C, Wu D |title=Successful bortezomib-based treatment in POEMS syndrome |journal=Eur. J. Haematol. |volume=83 |issue=6 |pages=609–10 |date=December 2009 |pmid=19674063 |doi=10.1111/j.1600-0609.2009.01330.x |url=}}</ref><ref name="pmid19732138">{{cite journal |vauthors=Kaygusuz I, Tezcan H, Cetiner M, Kocakaya O, Uzay A, Bayik M |title=Bortezomib: a new therapeutic option for POEMS syndrome |journal=Eur. J. Haematol. |volume=84 |issue=2 |pages=175–7 |date=February 2010 |pmid=19732138 |doi=10.1111/j.1600-0609.2009.01341.x |url=}}</ref><ref name="pmid22416898">{{cite journal |vauthors=Warsame R, Kohut IE, Dispenzieri A |title=Successful use of cyclophosphamide, bortezomib, and dexamethasone to treat a case of relapsed POEMS |journal=Eur. J. Haematol. |volume=88 |issue=6 |pages=549–50 |date=June 2012 |pmid=22416898 |doi=10.1111/j.1600-0609.2012.01780.x |url=}}</ref>
*It is associated with highly satisfactory responses on [[neuropathy]], [[serum]] [[Vascular endothelial growth factor|VEGF]] level, and extravascular overload.<ref name="pmid19674063">{{cite journal |vauthors=Tang X, Shi X, Sun A, Qiu H, Gu B, Zhou H, Xue S, Liu Y, Ruan C, Wu D |title=Successful bortezomib-based treatment in POEMS syndrome |journal=Eur. J. Haematol. |volume=83 |issue=6 |pages=609–10 |date=December 2009 |pmid=19674063 |doi=10.1111/j.1600-0609.2009.01330.x |url=}}</ref><ref name="pmid19732138">{{cite journal |vauthors=Kaygusuz I, Tezcan H, Cetiner M, Kocakaya O, Uzay A, Bayik M |title=Bortezomib: a new therapeutic option for POEMS syndrome |journal=Eur. J. Haematol. |volume=84 |issue=2 |pages=175–7 |date=February 2010 |pmid=19732138 |doi=10.1111/j.1600-0609.2009.01341.x |url=}}</ref><ref name="pmid21153416">{{cite journal |vauthors=Ohguchi H, Ohba R, Onishi Y, Fukuhara N, Okitsu Y, Yamamoto J, Ishizawa K, Ichinohasama R, Harigae H |title=Successful treatment with bortezomib and thalidomide for POEMS syndrome |journal=Ann. Hematol. |volume=90 |issue=9 |pages=1113–4 |date=September 2011 |pmid=21153416 |doi=10.1007/s00277-010-1133-8 |url=}}</ref><ref name="pmid22416898">{{cite journal |vauthors=Warsame R, Kohut IE, Dispenzieri A |title=Successful use of cyclophosphamide, bortezomib, and dexamethasone to treat a case of relapsed POEMS |journal=Eur. J. Haematol. |volume=88 |issue=6 |pages=549–50 |date=June 2012 |pmid=22416898 |doi=10.1111/j.1600-0609.2012.01780.x |url=}}</ref>
*It is, however, also associated with a potential risk of progression of existing [[neuropathy]].
====Bevacizumab====
*[[Bevacizumab]] has anti-[[Vascular endothelial growth factor|VEGF]] properties and can result in a rapid decrease in the [[serum]] [[Vascular endothelial growth factor|VEGF]] levels.<ref name="pmid23463868">{{cite journal |vauthors=Sekiguchi Y, Misawa S, Shibuya K, Nasu S, Mitsuma S, Iwai Y, Beppu M, Sawai S, Ito S, Hirano S, Nakaseko C, Kuwabara S |title=Ambiguous effects of anti-VEGF monoclonal antibody (bevacizumab) for POEMS syndrome |journal=J. Neurol. Neurosurg. Psychiatry |volume=84 |issue=12 |pages=1346–8 |date=December 2013 |pmid=23463868 |doi=10.1136/jnnp-2012-304874 |url=}}</ref>
*It is not, however, associated with clinical improvement.<ref name="pmid23463868">{{cite journal |vauthors=Sekiguchi Y, Misawa S, Shibuya K, Nasu S, Mitsuma S, Iwai Y, Beppu M, Sawai S, Ito S, Hirano S, Nakaseko C, Kuwabara S |title=Ambiguous effects of anti-VEGF monoclonal antibody (bevacizumab) for POEMS syndrome |journal=J. Neurol. Neurosurg. Psychiatry |volume=84 |issue=12 |pages=1346–8 |date=December 2013 |pmid=23463868 |doi=10.1136/jnnp-2012-304874 |url=}}</ref>
*[[Bevacizumab]] causes sudden [[Vascular endothelial growth factor|VEGF]] removal, causing the collapse of newly-formed fragile [[Blood vessel|vessels]] and resulting in an increase in [[capillary]] leakage.<ref name="pmid16754779">{{cite journal |vauthors=Straume O, Bergheim J, Ernst P |title=Bevacizumab therapy for POEMS syndrome |journal=Blood |volume=107 |issue=12 |pages=4972–3; author reply 4973–4 |date=June 2006 |pmid=16754779 |doi=10.1182/blood-2005-12-5045 |url=}}</ref>


==References==
==References==

Latest revision as of 02:34, 11 February 2019

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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]Associate Editor(s)-in-Chief: Sabawoon Mirwais, M.B.B.S, M.D.[2]

Overview

Patients with isolated bone lesion without bone marrow clonal plasma cells involvement can be treated with radiotherapy. Patients with a disseminated disease (more bone lesions and/or bone marrow plasmacytosis) are the candidates for systemic therapy. Systemic therapy includes corticosteroids, autologous stem cell transplantation (ASCT), induction therapy, alkylator-based therapy, thalidomide, lenalidomide, bortezomib, and bevacizumab.

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References

  1. ↑ 1.0 1.1 Dispenzieri A (June 2012). "How I treat POEMS syndrome". Blood. 119 (24): 5650–8. doi:10.1182/blood-2012-03-378992. PMC 3425020. PMID 22547581.
  2. ↑ 2.0 2.1 2.2 Dispenzieri A, Kyle RA, Lacy MQ, Rajkumar SV, Therneau TM, Larson DR, Greipp PR, Witzig TE, Basu R, Suarez GA, Fonseca R, Lust JA, Gertz MA (April 2003). "POEMS syndrome: definitions and long-term outcome". Blood. 101 (7): 2496–506. doi:10.1182/blood-2002-07-2299. PMID 12456500.
  3. ↑ 3.0 3.1 Brewis MJ, Church AC, Peacock AJ, Thomson S, Tighe J, Johnson MK (December 2014). "Pulmonary hypertension in POEMS syndrome: resolution following radiotherapy". Pulm Circ. 4 (4): 732–5. doi:10.1086/678553. PMC 4278633. PMID 25610609.
  4. ↑ 4.0 4.1 Dispenzieri A (February 2014). "POEMS syndrome: 2014 update on diagnosis, risk-stratification, and management". Am. J. Hematol. 89 (2): 214–23. doi:10.1002/ajh.23644. PMID 24532337.
  5. ↑ Dispenzieri A (October 2015). "POEMS syndrome: update on diagnosis, risk-stratification, and management". Am. J. Hematol. 90 (10): 951–62. doi:10.1002/ajh.24171. PMID 26331353.
  6. ↑ 6.0 6.1 D'Souza A, Lacy M, Gertz M, Kumar S, Buadi F, Hayman S, Dingli D, Zeldenrust S, Kyle R, Ansell S, Inwards D, Johnston P, Micallef I, Porrata L, Litzow M, Gastineau D, Hogan W, Dispenzieri A (July 2012). "Long-term outcomes after autologous stem cell transplantation for patients with POEMS syndrome (osteosclerotic myeloma): a single-center experience". Blood. 120 (1): 56–62. doi:10.1182/blood-2012-04-423178. PMID 22611150.
  7. ↑ Cook G, Iacobelli S, van Biezen A, Ziagkos D, LeBlond V, Abraham J, McQuaker G, Schoenland S, Rambaldi A, Halaburda K, Rovira M, Sica S, Byrne J, Sanz RG, Nagler A, van de Donk NW, Sinisalo M, Cook M, Kröger N, De Witte T, Morris C, Garderet L (January 2017). "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". Haematologica. 102 (1): 160–167. doi:10.3324/haematol.2016.148460. PMC 5210246. PMID 27634201.
  8. ↑ Dispenzieri A, Lacy MQ, Hayman SR, Kumar SK, Buadi F, Dingli D, Litzow MR, Gastineau DA, Inwards DJ, Elliott MA, Micallef IN, Ansell SM, Hogan WJ, Porrata LF, Johnston PA, Afessa B, Bryce A, Kyle RA, Gertz MA (May 2008). "Peripheral blood stem cell transplant for POEMS syndrome is associated with high rates of engraftment syndrome". Eur. J. Haematol. 80 (5): 397–406. doi:10.1111/j.1600-0609.2008.01037.x. PMC 2327207. PMID 18221391.
  9. ↑ 9.0 9.1 Kanai K, Sawai S, Sogawa K, Mori M, Misawa S, Shibuya K, Isose S, Fujimaki Y, Noto Y, Sekiguchi Y, Nasu S, Nakaseko C, Takano S, Yoshitomi H, Miyazaki M, Nomura F, Kuwabara S (August 2012). "Markedly upregulated serum interleukin-12 as a novel biomarker in POEMS syndrome". Neurology. 79 (6): 575–82. doi:10.1212/WNL.0b013e318263c42b. PMID 22843279.
  10. ↑ Jimenez-Zepeda VH, Trudel S, Reece DE, Chen C, Rabea AM, Kukreti V (October 2011). "Cyclophosphamide and prednisone induction followed by cyclophosphamide mobilization effectively decreases the incidence of engraftment syndrome in patients with POEMS syndrome who undergo stem cell transplantation". Am. J. Hematol. 86 (10): 873–5. doi:10.1002/ajh.22115. PMID 21815185.
  11. ↑ Li J, Zhang W, Duan MH, Jiao L, Zhu TN, Han B, Zhang L, Gan J, Zhou DB (February 2013). "PBSC mobilization in newly diagnosed patients with POEMS syndrome: outcomes and prognostic factors". Bone Marrow Transplant. 48 (2): 233–7. doi:10.1038/bmt.2012.138. PMID 22825426.
  12. ↑ 12.0 12.1 Li J, Zhang W, Jiao L, Duan MH, Guan HZ, Zhu WG, Tian Z, Zhou DB (June 2011). "Combination of melphalan and dexamethasone for patients with newly diagnosed POEMS syndrome". Blood. 117 (24): 6445–9. doi:10.1182/blood-2010-12-328112. PMC 3123016. PMID 21393478.
  13. ↑ Misawa S, Sato Y, Katayama K, Nagashima K, Aoyagi R, Sekiguchi Y, Sobue G, Koike H, Yabe I, Sasaki H, Watanabe O, Takashima H, Nishizawa M, Kawachi I, Kusunoki S, Mitsui Y, Kikuchi S, Nakashima I, Ikeda S, Kohara N, Kanda T, Kira J, Hanaoka H, Kuwabara S (October 2016). "Safety and efficacy of thalidomide in patients with POEMS syndrome: a multicentre, randomised, double-blind, placebo-controlled trial". Lancet Neurol. 15 (11): 1129–37. doi:10.1016/S1474-4422(16)30157-0. PMID 27496680.
  14. ↑ 14.0 14.1 Kuwabara S, Misawa S, Kanai K, Sawai S, Hattori T, Nishimura M, Nakaseko C (November 2008). "Thalidomide reduces serum VEGF levels and improves peripheral neuropathy in POEMS syndrome". J. Neurol. Neurosurg. Psychiatry. 79 (11): 1255–7. doi:10.1136/jnnp.2008.150177. PMID 18469028.
  15. ↑ Sinisalo M, Hietaharju A, Sauranen J, Wirta O (May 2004). "Thalidomide in POEMS syndrome: case report". Am. J. Hematol. 76 (1): 66–8. doi:10.1002/ajh.20051. PMID 15114600.
  16. ↑ Dispenzieri A, Klein CJ, Mauermann ML (August 2007). "Lenalidomide therapy in a patient with POEMS syndrome". Blood. 110 (3): 1075–6. doi:10.1182/blood-2007-03-082354. PMID 17644745.
  17. ↑ Royer B, Merlusca L, Abraham J, Musset L, Haroche J, Choquet S, Leleu X, Sebban C, Decaux O, Galicier L, Roussel M, Recher C, Banos A, Guichard I, Brisseau JM, Godmer P, Hermine O, Deplanque G, Facon T, Asli B, Leblond V, Fermand JP, Marolleau JP, Jaccard A (March 2013). "Efficacy of lenalidomide in POEMS syndrome: a retrospective study of 20 patients". Am. J. Hematol. 88 (3): 207–12. doi:10.1002/ajh.23374. PMID 23335406.
  18. ↑ Vannata B, Laurenti L, Chiusolo P, Sorà F, Balducci M, Sabatelli M, Luigetti M, Giannotta C, De Stefano V, Leone G, Sica S (June 2012). "Efficacy of lenalidomide plus dexamethasone for POEMS syndrome relapsed after autologous peripheral stem-cell transplantation". Am. J. Hematol. 87 (6): 641–2. doi:10.1002/ajh.23195. PMID 22488443.
  19. ↑ Suyanı E, Yağcı M, Sucak GT (2011). "Complete remission with a combination of lenalidomide, cyclophosphamide and prednisolone in a patient with incomplete POEMS syndrome". Acta Haematol. 126 (4): 199–201. doi:10.1159/000329896. PMID 21849771.
  20. ↑ Cai QQ, Wang C, Cao XX, Cai H, Zhou DB, Li J (October 2015). "Efficacy and safety of low-dose lenalidomide plus dexamethasone in patients with relapsed or refractory POEMS syndrome". Eur. J. Haematol. 95 (4): 325–30. doi:10.1111/ejh.12492. PMID 25401269.
  21. ↑ Zagouri F, Kastritis E, Gavriatopoulou M, Sergentanis TN, Psaltopoulou T, Terpos E, Dimopoulos MA (September 2014). "Lenalidomide in patients with POEMS syndrome: a systematic review and pooled analysis". Leuk. Lymphoma. 55 (9): 2018–23. doi:10.3109/10428194.2013.869329. PMID 24295131.
  22. ↑ 22.0 22.1 Tang X, Shi X, Sun A, Qiu H, Gu B, Zhou H, Xue S, Liu Y, Ruan C, Wu D (December 2009). "Successful bortezomib-based treatment in POEMS syndrome". Eur. J. Haematol. 83 (6): 609–10. doi:10.1111/j.1600-0609.2009.01330.x. PMID 19674063.
  23. ↑ 23.0 23.1 Kaygusuz I, Tezcan H, Cetiner M, Kocakaya O, Uzay A, Bayik M (February 2010). "Bortezomib: a new therapeutic option for POEMS syndrome". Eur. J. Haematol. 84 (2): 175–7. doi:10.1111/j.1600-0609.2009.01341.x. PMID 19732138.
  24. ↑ 24.0 24.1 Warsame R, Kohut IE, Dispenzieri A (June 2012). "Successful use of cyclophosphamide, bortezomib, and dexamethasone to treat a case of relapsed POEMS". Eur. J. Haematol. 88 (6): 549–50. doi:10.1111/j.1600-0609.2012.01780.x. PMID 22416898.
  25. ↑ Ohguchi H, Ohba R, Onishi Y, Fukuhara N, Okitsu Y, Yamamoto J, Ishizawa K, Ichinohasama R, Harigae H (September 2011). "Successful treatment with bortezomib and thalidomide for POEMS syndrome". Ann. Hematol. 90 (9): 1113–4. doi:10.1007/s00277-010-1133-8. PMID 21153416.
  26. ↑ 26.0 26.1 Sekiguchi Y, Misawa S, Shibuya K, Nasu S, Mitsuma S, Iwai Y, Beppu M, Sawai S, Ito S, Hirano S, Nakaseko C, Kuwabara S (December 2013). "Ambiguous effects of anti-VEGF monoclonal antibody (bevacizumab) for POEMS syndrome". J. Neurol. Neurosurg. Psychiatry. 84 (12): 1346–8. doi:10.1136/jnnp-2012-304874. PMID 23463868.
  27. ↑ Straume O, Bergheim J, Ernst P (June 2006). "Bevacizumab therapy for POEMS syndrome". Blood. 107 (12): 4972–3, author reply 4973–4. doi:10.1182/blood-2005-12-5045. PMID 16754779.

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