Risk factors for and outcomes of patients with POEMS syndrome who experience progression after first-line treatment

Leukemia. 2016 May;30(5):1079-85. doi: 10.1038/leu.2015.344. Epub 2015 Dec 16.

Abstract

Although clinical improvement is almost universal with therapy in patients with POEMS (an acronym for polyneuropathy, organomegaly, endocrinopathies, monoclonal protein and a variety of skin changes) syndrome, outcomes and management of patients who relapse or progress (R/P) after first-line treatment have not been described. We retrospectively identified 262 patients with POEMS syndrome treated at the Mayo Clinic from 1974 to 2014 and who had follow-up information. The 5-year progression-free survival (PFS) and overall survival (OS) was 58% and 78%, respectively. Median time to R/P was 42 months. Seventy-nine patients (30%) had an R/P, with 52 (19%) experiencing a symptomatic R/P. Eighteen patients relapsed with symptoms or signs that were not documented at diagnosis. Median times to vascular endothelial growth factor, hematologic, radiographic and clinical R/P were 35 months (range, 4-327 months), 72 months (range, 4-327 months), 51 months (range, 4-327 months) and 48 months (range, 6-311 months), respectively. On multivariate analyses, low albumin at diagnosis and failure to achieve a complete hematologic response to first-line therapy were independent risk factors for PFS. Thirty patients had documentation of a second R/P at a median of 26 months from diagnosis of the first R/P. An early R/P was a risk factor for death, but most patients with an R/P had salvageable disease. A majority of patients are still without R/P at 5 years from diagnosis.

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Combined Modality Therapy
  • Disease Management
  • Disease Progression*
  • Disease-Free Survival
  • Female
  • Humans
  • Male
  • Middle Aged
  • POEMS Syndrome / diagnosis*
  • POEMS Syndrome / mortality
  • POEMS Syndrome / pathology
  • POEMS Syndrome / therapy*
  • Recurrence
  • Retrospective Studies
  • Risk Factors
  • Salvage Therapy / methods
  • Survival Rate
  • Treatment Outcome
  • Vascular Endothelial Growth Factor A / blood
  • Young Adult

Substances

  • Vascular Endothelial Growth Factor A