Successful Bortezomib-Based Therapy in POEMS Syndrome.

Abstract 4966

POEMS syndrome is a rare multisystem disease characterized by polyneuropathy (P), organomegaly (O), endocrinopathy (E), serum M-protein (M), and skin changes (S). Owing to the fairly little refined mechanisms underlying the pathogenesis of POEMS syndrome, standard and general-accepted regimens have not been established to date. Recently, we successfully treated a POEMS syndrome patient who was unresponsive to conventional therapies by using bortezomib-based regimen. This 37-year-old patient represented a number of typical manifestations including: 1) paresthesia, 2) increased level of IgA » type of M protein, 3) elevated concentration of serum VEGF (vascular endothelial growth factor), 4) splenomegaly, gynaecomastia, limbs edema, hydropericardium, hydrothorax, and plethora. According to the latest Mayo Clinic criteria, the diagnosis of POEMS syndrome could thus be comfirmed. After several courses of classical regimens [VAD (Vincristine, doxorubicin, dexamethasone), CMP (cyclophosphamide, melphalan and prednisone) and AD (doxorubicin and dexamethasone)], the patient poorly responded with only slight improvement and failed to achieve remission. Following the patient's informed consent and Ethics Committee approval, a tentative VDD regimen was carried out. The details were as follows: Bortezomib, at a dose of 1.3-1.6 mg/m² (initial cycle: 1.3 mg/m² in the first week, 1.6 mg/m²/w in following three weeks; next three cycles: 1.6 mg/m²/w×4 weeks in each cycle); 40 mg of Liposomal doxorubicin on the fourth day of the first week; and 10 mg of dexamethasone during the initial 4 days of first cycle. Each course was at 21 days interval. Following four cycles of VDD, the patient acquired remarkable improvement (concerning neurological disease, skin changes, clonal plasmacytosis, and organomegaly) and finally achieved complete remission. To our knowledge, It should be the first time in the world that the bortezomib-based new strategy could be applied effectively against the POEMS syndrome. Further, we could speculate that the VDD regimen would be a potent candidate in the treatment of POEMS syndrome, at least in the conventional therapy-resistant condition.