Among patients with polyneuropathy, organomegaly, endocrinopathy, M-protein, and skin changes (POEMS) syndrome, bortezomib and autologous stem cell transplant (ASCT) may improve survival outcomes, according to research published in Clinical Lymphoma, Myeloma & Leukemia.
POEMS syndrome, a rare plasma cell disorder, is a paraneoplastic syndrome that is notably difficult to diagnose. Given its rarity, patient care may require multicenter cooperation.
Previous research noted that POEMS syndrome is associated with vascular endothelial growth factor (VEGF) levels, which decrease when patients initiate treatment. VEGF level decrease is not, however, perfectly correlated with clinical response.
Given a paucity of research into POEMS syndrome, furthermore, it is difficult to target treatment at the individual level. For this real-world multicenter analysis, researchers evaluated treatment strategies and outcomes in a cohort of patients with POEMS syndrome.
Data from 108 patients treated at 1 of 15 hematology centers were included. The median age in the cohort was 51 years (range, 24-90), 67% of patients were male sex, the median VEGF level was 741 pg/mL (in 47 evaluable patients), and 58% of patients had a present endocrinopathy.
Overall, 37% of patients received a proteasome inhibitor. Patients who received bortezomib monotherapy had the highest rates of complete or very good partial remission (69%). Among the 30% of patients who had a planned ASCT as part of frontline treatment, both progression-free survival and overall survival (OS) were improved (P =.003).
Patients older than 60 years, as well as those with anemia or thrombocytopenia, were more likely to have inferior outcomes.
The median follow-up was 2.6 years, at which point 9 deaths were noted, 4 of which were not related to POEMS syndrome. Median OS was not reached; the estimated 10-year OS rate was 80%.
“This is one of the largest analyses of front line treatment efficacy in POEMS and the first one with such a wide upfront use of proteasome inhibitors,” the authors wrote. “Proteasome inhibitors as single agents, the combination of a [proteasome inhibitor and immunomodulatory agent] and ASCT all demonstrate high responses and should be considered standard options for a newly diagnosed POEMS patient.”
Jurczyszyn A, Castillo JJ, Olszewska-Szopa M, et al. POEMS syndrome: real world experience in diagnosis and systemic therapy – 108 patients multicenter analysis. Clin Lymphoma Myeloma Leuk. Published online October 31, 2021. doi:10.1016/j.clml.2021.10.007