Retrospective study of long-term outcomes with first-line bortezomib-based therapies for newly diagnosed POEMS syndrome

POEMS syndrome is a rare hematologic condition with no established standard of therapy. A retrospective study at Shanghai Changzheng Hospital of the Navy Medical University (China) assessed long-term efficacy of first-line bortezomib-based regimens for newly diagnosed POEMS syndrome. The study enrolled 53 patients, who received a median of four treatment cycles, with a median follow-up of 92.4 months. Hematologic responses were evaluable in 42 patients, yielding an overall response rate of 85.7% including complete reponse in 64.3% of patients. VEGF responses were assessed in 44 patients, with an overall response rate of 88.6% and a complete response rate of 56.8%. Median VEGF levels decreased for 944.4 pg/mL to 137.8 pg/mL. Neurologic improvements were observed in 90.6% of patients, with 24.5% achieving a complete neurological response. Median progression-free survival and overall survival were not reached, with 5-year rates of 67.4% and 83.0%, respectively. FISH analysis identified 1q21 gain as the most frequent abnormality in this cohort and it was an independent prognostic factor for overall survival. The authors conclude that bortezomib-based regimens achieved favorable