POEMS syndrome treated with melphalan high-dose therapy and autologous blood stem cell transplantation: a single-institution experience

Ann Hematol. 2012 Sep;91(9):1419-25. doi: 10.1007/s00277-012-1473-7. Epub 2012 Apr 29.

Abstract

The acronym POEMS syndrome stands for a rare multi-system disorder, comprised of polyneuropathy, organomegaly, endocrinopathy, M protein, and skin changes. Here, we present a single-center report of a series of five POEMS patients treated with melphalan high-dose therapy (HDT) with subsequent autologous blood stem cell transplantation (ABSCT). After a median follow-up of 52 months from time of diagnosis (range, 15-192) and a median follow-up of 18 months after ABSCT (range, 11-120), all patients were alive. Overall, no severe transplantation-associated complications such as engraftment syndrome or peri- or post-transplant death were noted. In two cases, HDT followed by ABSCT resulted in a complete hematologic response; in the additional three cases, partial responses (PR) were achieved including one very good hematologic PR. Only one patient with initial PR developed progressive disease nearly 2.5 years after transplantation. Consequently, a second HDT with ABSCT was successfully applied resulting in clinical improvement and hematologic PR. In line with previous single-center reports, melphalan HDT followed by ABSCT proved to be a first-line treatment option with tolerable side effects in severely affected POEMS patients with progressing symptoms.

Publication types

  • Evaluation Study

MeSH terms

  • Adult
  • Anthracyclines / therapeutic use
  • Combined Modality Therapy
  • Dexamethasone / therapeutic use
  • Female
  • Humans
  • Male
  • Melphalan / administration & dosage
  • Melphalan / therapeutic use*
  • Middle Aged
  • Myeloablative Agonists / administration & dosage
  • Myeloablative Agonists / therapeutic use*
  • Neurologic Examination
  • POEMS Syndrome / blood
  • POEMS Syndrome / drug therapy*
  • POEMS Syndrome / surgery
  • Peripheral Blood Stem Cell Transplantation*
  • Transplantation, Autologous
  • Treatment Outcome
  • Vascular Endothelial Growth Factor A / blood

Substances

  • Anthracyclines
  • Myeloablative Agonists
  • VEGFA protein, human
  • Vascular Endothelial Growth Factor A
  • Dexamethasone
  • Melphalan