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POEMS syndrome.

Stephen Keddie1, Michael P Lunn

  • 1Centre for Neuromuscular Disease, National Hospital for Neurology and Neurosurgery, Queen Square, London, United Kingdom.

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Summary
This summary is machine-generated.

Polyneuropathy organomegaly endocrinopathy M-protein and skin changes (POEMS) syndrome is a complex hematoneurological disorder. Early diagnosis and targeted treatments, guided by prognostic scores and biomarkers like VEGF, improve patient outcomes.

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Area of Science:

  • Hematology
  • Neurology
  • Endocrinology
  • Dermatology

Background:

  • POEMS syndrome is a multisystem hematoneurological disorder characterized by polyneuropathy, organomegaly, endocrinopathy, M-protein, and skin changes.
  • Early diagnosis is crucial to prevent significant disability from neuropathy.
  • POEMS syndrome shares similarities with CIDP and other monoclonal proliferative diseases, leading to common misdiagnosis.

Purpose of the Study:

  • To provide an overview of POEMS syndrome.
  • To detail new insights into its pathogenesis, prognostic factors, treatments, and outcome scores.
  • To highlight the evolution of POEMS syndrome into a well-characterized entity with developing treatment algorithms.

Main Methods:

  • Analysis of large multicentre national patient cohorts.
  • Identification of vascular endothelial growth factor (VEGF) as a key biomarker for diagnosis and disease activity monitoring.
  • Development of risk factors, prognostic scores, and treatment algorithms based on monoclonal proliferation extent and patient performance status.

Main Results:

  • Vascular endothelial growth factor (VEGF) is a validated biomarker for POEMS syndrome diagnosis and activity monitoring.
  • Therapeutics repurposed from myeloma treatment are now available.
  • Prognostic scores developed from phenotyped cohorts predict treatment response, progression-free survival, and overall survival.

Conclusions:

  • Understanding the link between monoclonal lambda plasma cell disorder and proinflammatory cytokines is key to POEMS pathophysiology.
  • Despite available treatments, further research into pathogenic mechanisms, treatment targets, and prognostic scores is needed for optimal treatment stratification.
  • Accurate diagnosis and risk stratification are essential for managing POEMS syndrome effectively.