Ushering in a new era for POEMS.

Li and colleagues are to be congratulated for conducting the first prospective clinical trial for POEMS syndrome, which treated 31 patients with 12 cycles of melphalan and dexamethasone.1 Although the choice of regimen is an old standard, this study is novel and important on several levels. First, it demonstrates that accrual for a trial for this very rare disease is possible. Second, the authors tackle response assessment in a multisystem disease. Third, it challenges what many consider to be the standard for treatment for this population of patients, albeit in the context of a prospective phase 2 trial.