Bortezomib plus dexamethasone as first-line therapy for patients with POEMS syndrome

Ann Hematol. 2021 Nov;100(11):2755-2761. doi: 10.1007/s00277-021-04609-6. Epub 2021 Jul 31.

Abstract

POEMS (polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes) syndrome is a rare plasma cell dyscrasia without standard front-line treatment. Merely, few studies have reported the responses and outcomes of bortezomib plus dexamethasone (BDex) in POEMS syndrome. In this study, a total of 69 patients (40 males) treated with front-line BDex were included. The median age at diagnosis was 50 years (range, 30-78 years). After a median of 9 cycles BDex (range 1-9), fifty-two (88.1%), thirty-two (46.4%), and forty-seven (71.2%) patients achieved the best neurologic response, hematological complete response, and serum vascular endothelial growth factor (VEGF) response, respectively. The extravascular overload, pulmonary hypertension, and renal impairment also substantially improved. No treatment-related death occurred. Two patients developed grade-1 bortezomib-induced peripheral neuropathy and were reversible after drug withdrawal. After a median follow-up of 22.5 months, the estimated 2-year overall survival and time to next treatment were 95.7% and 65.6%, respectively. In conclusion, the combination of bortezomib and dexamethasone is effective, with a high response rate and safety profile for patients with newly diagnosed POEMS syndrome.

Keywords: Front-line bortezomib; Overall survival; POEMS syndrome; Time to next treatment.

MeSH terms

  • Adult
  • Aged
  • Biomarkers
  • Bortezomib / adverse effects
  • Bortezomib / therapeutic use*
  • Chemical and Drug Induced Liver Injury / etiology
  • Dexamethasone / adverse effects
  • Dexamethasone / therapeutic use*
  • Diarrhea / chemically induced
  • Drug Evaluation
  • Drug Therapy, Combination
  • Female
  • Humans
  • Kaplan-Meier Estimate
  • Male
  • Middle Aged
  • POEMS Syndrome / blood
  • POEMS Syndrome / drug therapy*
  • Paraproteins / analysis
  • Paresthesia / chemically induced
  • Retrospective Studies
  • Vascular Endothelial Growth Factor A / blood

Substances

  • Biomarkers
  • Paraproteins
  • VEGFA protein, human
  • Vascular Endothelial Growth Factor A
  • Bortezomib
  • Dexamethasone