Abstract Background POEMS syndrome is a rare hematologic condition with no established standard of therapy. Bortezomib, a proteasome inhibitor, has shown potential to offer significant clinical benefits for patients with newly diagnosed POEMS syndrome. However, comprehensive data on its long‐term efficacy remain scarce. This study aimed to assess the long‐term efficacy of bortezomib‐based regimens for newly diagnosed POEMS syndrome. Methods A total of 53 patients were enrolled in this study. Fluorescence in situ hybridization analysis was performed on CD138‐selected bone marrow samples. All patients received bortezomib‐based regimens as first‐line therapy. Hematologic responses, vascular endothelial growth factor (VEGF) levels, neurological response rates, and long‐term survival outcomes were evaluated. Results Patients received a median of four treatment cycles, with a median follow‐up time of 92.4 months. Hematologic responses were evaluable in 42 patients, yielding an overall response rate was 85.7%, including a complete response rate of 64.3%. 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 from 944.4 pg/mL to 137.8 pg/mL. Neurological improvements were observed in 90.6% of patients, with 24.5% achieving a complete neurological response. Median overall survival (OS) and progression‐free survival were not reached. The 5‐year OS and progression‐free survival rates were 83.0% and 67.4%, respectively. Fluorescence in situ hybridization analysis identified 1q21 gain as the most frequent abnormality in this cohort, and it was an independent prognostic factor for OS. Conclusions Bortezomib‐based regimens were effective and achieved favorable long‐term survival outcomes for patients with newly diagnosed POEMS syndrome.
He et al. (Sat,) studied this question.