POEMS syndrome natural history, complications and prognosis: Difference between revisions
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==Overview== | ==Overview== | ||
POEMS syndrome presents as a chronic progressive polyneuropathy. Patients may have coexisting multi-organ system disorders. The neuropathy is usually symmetrical | POEMS [[syndrome]] presents as a [[Chronic (medical)|chronic]] progressive [[polyneuropathy]]. [[Patient|Patients]] may have coexisting multi-[[Organ (anatomy)|organ]] system [[Disorder (medicine)|disorders]]. The [[neuropathy]] is usually symmetrical and ascending. [[Endocrinology|Endocrinopathy]], present in majority of cases, involves [[hypogonadism]], [[hypothyroidism]], and abnormalities of the [[Pituitary gland|pituitary]]-[[Adrenal gland|adrenal]] axis. If left untreated, [[patients]] suffering from POEMS [[syndrome]] may become wheel chair bound. [[Complication (medicine)|Complications]] of POEMS [[syndrome]] include [[Renal insufficiency|renal failure]], [[pulmonary hypertension]], [[Fracture|pathologic fractures]], [[ischemic stroke]], [[restrictive lung disease]], [[polycythemia]], [[thrombocytosis]], [[papilledema]], and [[myocardial infarction]]. 3, 5, and 10 year overall survival (OS) for [[Patient|patients]] newly [[Diagnosis|diagnosed]] with POEMS [[syndrome]] is 87.7%, 84.1%, and 77%. Age >50 years, [[pulmonary hypertension]], [[pleural effusion]], and estimated [[glomerular filtration rate]] <30 ml/min/1.73 m<sup>2</sup> were associated with inferior overall survival in the derivation cohort, with the use of multivariate Cox regression model based on randomized sample splitting of 362 patients. Low [[albumin]] (defined as<3.2 g/dl) at [[diagnosis]] and failure to achieve a complete [[Hematology|hematologic]] response to [[First-line treatment|first-line therapy]] can be independent [[risk factors]] for progression-free survival (PFS). Improvement of [[Plasma cell disorder|plasma cell dyscrasia]] leads to improvement or marked reduction in other abnormalities. [[Neuropathy]], [[stroke]], and [[myocardial infarction]] are the most common causes of death in POEMS [[syndrome]]. | ||
== Natural History == | == Natural History == | ||
* POEMS syndrome typically presents as a chronic progressive polyneuropathy with both sensory and motor disability, the motor component is usually present to a greater degree than the sensory component of the disease. | * POEMS [[syndrome]] typically presents as a [[Chronic (medical)|chronic]] progressive [[polyneuropathy]] with both [[Sensory system|sensory]] and motor disability, the motor component is usually present to a greater degree than the [[Sensory system|sensory]] component of the [[disease]]. | ||
* Many patients may have coexisting multi-organ system disorders such as organomegaly (liver, spleen and lymph nodes), edema, endocrinopathy (pituitary, thyroid, | * Many [[Patient|patients]] may have coexisting multi-[[Organ (anatomy)|organ]] system [[Disorder (medicine)|disorders]] such as [[organomegaly]] ([[liver]], [[spleen]] and [[Lymph node|lymph nodes]]), [[edema]], [[Endocrinology|endocrinopathy]] ([[Pituitary gland|pituitary]], [[thyroid]], [[Adrenal gland|adrenal glands]]), [[thrombocytosis]], [[polycythemia]], [[pulmonary hypertension]], an M-spike ([[M protein]] or para-[[protein]], may be [[Immunoglobulin A|IgA]] lambda component, [[Immunoglobulin G|IgG]] lambda component or [[light chain]] lambda component) and [[skin]] changes. The [[skin]] changes include [[Angiomatosis|angiomata]], [[hyperpigmentation]], [[hypertrichosis]]. | ||
* The neuropathy is usually symmetrical and ascending, with either insidious or rapidly progressing onset. Sensory neuropathy precedes motor dysfunction and pin-prick and vibratory sensations are typically affected. | * The [[neuropathy]] is usually symmetrical and ascending, with either insidious or rapidly progressing onset. [[Neuropathy|Sensory neuropathy]] precedes motor dysfunction and pin-prick and vibratory sensations are typically affected. | ||
* The | * The [[Endocrinology|endocrinopathy]] is present in a majority of cases of POEMS [[syndrome]]. Almost all [[Patient|patients]] exhibiting [[Endocrinology|endocrinopathy]] have [[Estrogen|hyperestrogenemia]]. The increased levels of [[estrogen]] may be the cause of [[hypogonadism]] and [[Erectile dysfunction|impotence]] seen in [[male]] [[Patient|patients]]. [[Hypogonadism]], [[hypothyroidism]], and abnormalities of the [[Pituitary gland|pituitary]]-[[Adrenal gland|adrenal]] axis are noted in order of descending frequency. | ||
* POEMS syndrome may also lead to glomerular disease (membranoproliferative glomerulonephritis) eventually resulting in renal failure. | * POEMS [[syndrome]] may also lead to [[glomerular disease]] ([[membranoproliferative glomerulonephritis]]) eventually resulting in [[Renal insufficiency|renal failure]]. | ||
* If left untreated, patients suffering from POEMS syndrome may become wheel chair bound. | * If left untreated, [[Patient|patients]] suffering from POEMS [[syndrome]] may become wheel chair bound. | ||
== Complications == | == Complications == | ||
* Patients suffering from POEMS syndrome may develop the following complications: | * [[Patient|Patients]] suffering from POEMS [[syndrome]] may develop the following [[Complication (medicine)|complications]]: | ||
** Renal failure | ** [[Renal insufficiency|Renal failure]] | ||
** Pulmonary hypertension | ** [[Pulmonary hypertension]] | ||
** Pathological fractures (due to | ** [[Fractures|Pathological fractures]] (due to lytic/[[Sclerosis|sclerotic]] [[bone]] [[Lesion|lesions]]) | ||
** Ischemic stroke | ** [[Ischemic stroke]] | ||
** Restrictive lung disease | ** [[Restrictive lung disease]] | ||
** Thrombocytosis | ** [[Thrombocytosis]] | ||
** Polycythemia | ** [[Polycythemia]] | ||
** Papilledema | ** [[Papilledema]] | ||
** Myocardial infarction | ** [[Myocardial infarction]] | ||
== Prognosis == | == Prognosis == | ||
* The median survival of patients with POEMS syndrome is 165 months. | * The [[median]] survival of [[Patient|patients]] with POEMS [[syndrome]] is 165 months. | ||
*3, 5, and 10 year overall survival (OS) for patients newly diagnosed with POEMS syndrome is 87.7%, 84.1%, and 77%.<ref name="pmid27338259">{{cite journal |vauthors=Wang C, Huang XF, Cai QQ, Cao XX, Duan MH, Cai H, Zhou DB, Li J |title=Prognostic study for overall survival in patients with newly diagnosed POEMS syndrome |journal=Leukemia |volume=31 |issue=1 |pages=100–106 |date=January 2017 |pmid=27338259 |doi=10.1038/leu.2016.168 |url=}}</ref> | *3, 5, and 10 year overall survival (OS) for [[Patient|patients]] newly [[Diagnosis|diagnosed]] with POEMS [[syndrome]] is 87.7%, 84.1%, and 77%.<ref name="pmid27338259">{{cite journal |vauthors=Wang C, Huang XF, Cai QQ, Cao XX, Duan MH, Cai H, Zhou DB, Li J |title=Prognostic study for overall survival in patients with newly diagnosed POEMS syndrome |journal=Leukemia |volume=31 |issue=1 |pages=100–106 |date=January 2017 |pmid=27338259 |doi=10.1038/leu.2016.168 |url=}}</ref> | ||
*Age >50 years, pulmonary hypertension, pleural effusion, and estimated glomerular filtration rate <30 ml/min/1.73 m<sup>2</sup> were associated with inferior overall survival in the derivation cohort, with the use of multivariate Cox regression model based on randomized sample splitting of 362 patients.<ref name="pmid27338259">{{cite journal |vauthors=Wang C, Huang XF, Cai QQ, Cao XX, Duan MH, Cai H, Zhou DB, Li J |title=Prognostic study for overall survival in patients with newly diagnosed POEMS syndrome |journal=Leukemia |volume=31 |issue=1 |pages=100–106 |date=January 2017 |pmid=27338259 |doi=10.1038/leu.2016.168 |url=}}</ref><ref name="pmid23395245">{{cite journal |vauthors=Clegg A, Young J, Iliffe S, Rikkert MO, Rockwood K |title=Frailty in elderly people |journal=Lancet |volume=381 |issue=9868 |pages=752–62 |date=March 2013 |pmid=23395245 |pmc=4098658 |doi=10.1016/S0140-6736(12)62167-9 |url=}}</ref><ref name="pmid9517610">{{cite journal |vauthors=Lesprit P, Godeau B, Authier FJ, Soubrier M, Zuber M, Larroche C, Viard JP, Wechsler B, Gherardi R |title=Pulmonary hypertension in POEMS syndrome: a new feature mediated by cytokines |journal=Am. J. Respir. Crit. Care Med. |volume=157 |issue=3 Pt 1 |pages=907–11 |date=March 1998 |pmid=9517610 |doi=10.1164/ajrccm.157.3.9707095 |url=}}</ref><ref name="pmid18198255">{{cite journal |vauthors=Allam JS, Kennedy CC, Aksamit TR, Dispenzieri A |title=Pulmonary manifestations in patients with POEMS syndrome: a retrospective review of 137 patients |journal=Chest |volume=133 |issue=4 |pages=969–74 |date=April 2008 |pmid=18198255 |doi=10.1378/chest.07-1800 |url=}}</ref><ref name="pmid22983590">{{cite journal |vauthors=Li J, Tian Z, Zheng HY, Zhang W, Duan MH, Liu YT, Cao XX, Zhou DB |title=Pulmonary hypertension in POEMS syndrome |journal=Haematologica |volume=98 |issue=3 |pages=393–8 |date=March 2013 |pmid=22983590 |pmc=3659947 |doi=10.3324/haematol.2012.073031 |url=}}</ref> | *Age >50 years, [[pulmonary hypertension]], [[pleural effusion]], and estimated [[glomerular filtration rate]] <30 ml/min/1.73 m<sup>2</sup> were associated with inferior overall survival in the derivation cohort, with the use of multivariate Cox regression model based on randomized sample splitting of 362 [[Patient|patients]].<ref name="pmid27338259">{{cite journal |vauthors=Wang C, Huang XF, Cai QQ, Cao XX, Duan MH, Cai H, Zhou DB, Li J |title=Prognostic study for overall survival in patients with newly diagnosed POEMS syndrome |journal=Leukemia |volume=31 |issue=1 |pages=100–106 |date=January 2017 |pmid=27338259 |doi=10.1038/leu.2016.168 |url=}}</ref><ref name="pmid23395245">{{cite journal |vauthors=Clegg A, Young J, Iliffe S, Rikkert MO, Rockwood K |title=Frailty in elderly people |journal=Lancet |volume=381 |issue=9868 |pages=752–62 |date=March 2013 |pmid=23395245 |pmc=4098658 |doi=10.1016/S0140-6736(12)62167-9 |url=}}</ref><ref name="pmid9517610">{{cite journal |vauthors=Lesprit P, Godeau B, Authier FJ, Soubrier M, Zuber M, Larroche C, Viard JP, Wechsler B, Gherardi R |title=Pulmonary hypertension in POEMS syndrome: a new feature mediated by cytokines |journal=Am. J. Respir. Crit. Care Med. |volume=157 |issue=3 Pt 1 |pages=907–11 |date=March 1998 |pmid=9517610 |doi=10.1164/ajrccm.157.3.9707095 |url=}}</ref><ref name="pmid18198255">{{cite journal |vauthors=Allam JS, Kennedy CC, Aksamit TR, Dispenzieri A |title=Pulmonary manifestations in patients with POEMS syndrome: a retrospective review of 137 patients |journal=Chest |volume=133 |issue=4 |pages=969–74 |date=April 2008 |pmid=18198255 |doi=10.1378/chest.07-1800 |url=}}</ref><ref name="pmid22983590">{{cite journal |vauthors=Li J, Tian Z, Zheng HY, Zhang W, Duan MH, Liu YT, Cao XX, Zhou DB |title=Pulmonary hypertension in POEMS syndrome |journal=Haematologica |volume=98 |issue=3 |pages=393–8 |date=March 2013 |pmid=22983590 |pmc=3659947 |doi=10.3324/haematol.2012.073031 |url=}}</ref> | ||
*Low albumin (defined as<3.2 g/dl) at diagnosis and failure to achieve a complete hematologic response to first-line therapy can be independent risk factors for progression-free survival (PFS).<ref name="pmid27338259">{{cite journal |vauthors=Wang C, Huang XF, Cai QQ, Cao XX, Duan MH, Cai H, Zhou DB, Li J |title=Prognostic study for overall survival in patients with newly diagnosed POEMS syndrome |journal=Leukemia |volume=31 |issue=1 |pages=100–106 |date=January 2017 |pmid=27338259 |doi=10.1038/leu.2016.168 |url=}}</ref><ref name="pmid26669974">{{cite journal |vauthors=Kourelis TV, Buadi FK, Gertz MA, Lacy MQ, Kumar SK, Kapoor P, Go RS, Lust JA, Hayman SR, Rajkumar V, Zeldenrust SR, Russell SJ, Dingli D, Lin Y, Leung N, Hwa YL, Gonsalves W, Kyle RA, Dispenzieri A |title=Risk factors for and outcomes of patients with POEMS syndrome who experience progression after first-line treatment |journal=Leukemia |volume=30 |issue=5 |pages=1079–85 |date=May 2016 |pmid=26669974 |doi=10.1038/leu.2015.344 |url=}}</ref> | *Low [[albumin]] (defined as<3.2 g/dl) at [[diagnosis]] and failure to achieve a complete [[Hematology|hematologic]] response to [[First-line treatment|first-line therapy]] can be independent [[Risk factor|risk factors]] for progression-free survival (PFS).<ref name="pmid27338259">{{cite journal |vauthors=Wang C, Huang XF, Cai QQ, Cao XX, Duan MH, Cai H, Zhou DB, Li J |title=Prognostic study for overall survival in patients with newly diagnosed POEMS syndrome |journal=Leukemia |volume=31 |issue=1 |pages=100–106 |date=January 2017 |pmid=27338259 |doi=10.1038/leu.2016.168 |url=}}</ref><ref name="pmid26669974">{{cite journal |vauthors=Kourelis TV, Buadi FK, Gertz MA, Lacy MQ, Kumar SK, Kapoor P, Go RS, Lust JA, Hayman SR, Rajkumar V, Zeldenrust SR, Russell SJ, Dingli D, Lin Y, Leung N, Hwa YL, Gonsalves W, Kyle RA, Dispenzieri A |title=Risk factors for and outcomes of patients with POEMS syndrome who experience progression after first-line treatment |journal=Leukemia |volume=30 |issue=5 |pages=1079–85 |date=May 2016 |pmid=26669974 |doi=10.1038/leu.2015.344 |url=}}</ref> | ||
* Improvement of plasma cell dyscrasia leads to improvement or marked reduction in other abnormalities. | * Improvement of [[Plasma cell disorder|plasma cell dyscrasia]] leads to improvement or marked reduction in other abnormalities. | ||
* Neuropathy, stroke and myocardial infarction are the most common causes of death in POEMS syndrome. | * [[Neuropathy]], [[stroke]] and [[myocardial infarction]] are the most common [[Causality|causes]] of death in POEMS [[syndrome]]. | ||
==References== | ==References== | ||
Revision as of 20:49, 5 February 2019
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]Associate Editor(s)-in-Chief:
Overview
POEMS syndrome presents as a chronic progressive polyneuropathy. Patients may have coexisting multi-organ system disorders. The neuropathy is usually symmetrical and ascending. Endocrinopathy, present in majority of cases, involves hypogonadism, hypothyroidism, and abnormalities of the pituitary-adrenal axis. If left untreated, patients suffering from POEMS syndrome may become wheel chair bound. Complications of POEMS syndrome include renal failure, pulmonary hypertension, pathologic fractures, ischemic stroke, restrictive lung disease, polycythemia, thrombocytosis, papilledema, and myocardial infarction. 3, 5, and 10 year overall survival (OS) for patients newly diagnosed with POEMS syndrome is 87.7%, 84.1%, and 77%. Age >50 years, pulmonary hypertension, pleural effusion, and estimated glomerular filtration rate <30 ml/min/1.73 m2 were associated with inferior overall survival in the derivation cohort, with the use of multivariate Cox regression model based on randomized sample splitting of 362 patients. Low albumin (defined as<3.2 g/dl) at diagnosis and failure to achieve a complete hematologic response to first-line therapy can be independent risk factors for progression-free survival (PFS). Improvement of plasma cell dyscrasia leads to improvement or marked reduction in other abnormalities. Neuropathy, stroke, and myocardial infarction are the most common causes of death in POEMS syndrome.
Natural History
- POEMS syndrome typically presents as a chronic progressive polyneuropathy with both sensory and motor disability, the motor component is usually present to a greater degree than the sensory component of the disease.
- Many patients may have coexisting multi-organ system disorders such as organomegaly (liver, spleen and lymph nodes), edema, endocrinopathy (pituitary, thyroid, adrenal glands), thrombocytosis, polycythemia, pulmonary hypertension, an M-spike (M protein or para-protein, may be IgA lambda component, IgG lambda component or light chain lambda component) and skin changes. The skin changes include angiomata, hyperpigmentation, hypertrichosis.
- The neuropathy is usually symmetrical and ascending, with either insidious or rapidly progressing onset. Sensory neuropathy precedes motor dysfunction and pin-prick and vibratory sensations are typically affected.
- The endocrinopathy is present in a majority of cases of POEMS syndrome. Almost all patients exhibiting endocrinopathy have hyperestrogenemia. The increased levels of estrogen may be the cause of hypogonadism and impotence seen in male patients. Hypogonadism, hypothyroidism, and abnormalities of the pituitary-adrenal axis are noted in order of descending frequency.
- POEMS syndrome may also lead to glomerular disease (membranoproliferative glomerulonephritis) eventually resulting in renal failure.
- If left untreated, patients suffering from POEMS syndrome may become wheel chair bound.
Complications
- Patients suffering from POEMS syndrome may develop the following complications:
Prognosis
- The median survival of patients with POEMS syndrome is 165 months.
- 3, 5, and 10 year overall survival (OS) for patients newly diagnosed with POEMS syndrome is 87.7%, 84.1%, and 77%.[1]
- Age >50 years, pulmonary hypertension, pleural effusion, and estimated glomerular filtration rate <30 ml/min/1.73 m2 were associated with inferior overall survival in the derivation cohort, with the use of multivariate Cox regression model based on randomized sample splitting of 362 patients.[1][2][3][4][5]
- Low albumin (defined as<3.2 g/dl) at diagnosis and failure to achieve a complete hematologic response to first-line therapy can be independent risk factors for progression-free survival (PFS).[1][6]
- Improvement of plasma cell dyscrasia leads to improvement or marked reduction in other abnormalities.
- Neuropathy, stroke and myocardial infarction are the most common causes of death in POEMS syndrome.
References
- ↑ 1.0 1.1 1.2 Wang C, Huang XF, Cai QQ, Cao XX, Duan MH, Cai H, Zhou DB, Li J (January 2017). "Prognostic study for overall survival in patients with newly diagnosed POEMS syndrome". Leukemia. 31 (1): 100–106. doi:10.1038/leu.2016.168. PMID 27338259.
- ↑ Clegg A, Young J, Iliffe S, Rikkert MO, Rockwood K (March 2013). "Frailty in elderly people". Lancet. 381 (9868): 752–62. doi:10.1016/S0140-6736(12)62167-9. PMC 4098658. PMID 23395245.
- ↑ Lesprit P, Godeau B, Authier FJ, Soubrier M, Zuber M, Larroche C, Viard JP, Wechsler B, Gherardi R (March 1998). "Pulmonary hypertension in POEMS syndrome: a new feature mediated by cytokines". Am. J. Respir. Crit. Care Med. 157 (3 Pt 1): 907–11. doi:10.1164/ajrccm.157.3.9707095. PMID 9517610.
- ↑ Allam JS, Kennedy CC, Aksamit TR, Dispenzieri A (April 2008). "Pulmonary manifestations in patients with POEMS syndrome: a retrospective review of 137 patients". Chest. 133 (4): 969–74. doi:10.1378/chest.07-1800. PMID 18198255.
- ↑ Li J, Tian Z, Zheng HY, Zhang W, Duan MH, Liu YT, Cao XX, Zhou DB (March 2013). "Pulmonary hypertension in POEMS syndrome". Haematologica. 98 (3): 393–8. doi:10.3324/haematol.2012.073031. PMC 3659947. PMID 22983590.
- ↑ Kourelis TV, Buadi FK, Gertz MA, Lacy MQ, Kumar SK, Kapoor P, Go RS, Lust JA, Hayman SR, Rajkumar V, Zeldenrust SR, Russell SJ, Dingli D, Lin Y, Leung N, Hwa YL, Gonsalves W, Kyle RA, Dispenzieri A (May 2016). "Risk factors for and outcomes of patients with POEMS syndrome who experience progression after first-line treatment". Leukemia. 30 (5): 1079–85. doi:10.1038/leu.2015.344. PMID 26669974.