Background: Polyneuropathy, organomegaly, endocrinopathy, M-protein, and skin changes (POEMS) syndrome is a rare multi-system disorder associated with plasma cell dyscrasia. Several case series and reports have suggested high dose chemotherapy with autologous peripheral blood stem cell transplant is efficacious treatment, but this transplantation is not indicated for elderly patients and patients with renal failure.
Objective: To investigate the effects of thalidomide treatment for POEMS syndrome.
Methods: Nine patients, who were not indicated for high dose chemotherapy, were treated with thalidomide. Neurological disability scores, nerve conduction studies, and serum levels of vascular endothelial growth factor (VEGF) were prospectively examined. VEGF levels were measured by an enzyme-linked immunosorbent assay.
Results: During follow-up periods of 8 to 23 months (mean, 15 months), all patients showed substantial clinical improvement (n=6) or stabilization of symptoms (n=3). Serum VEGF levels decreased in all, and were normalized in five. Nerve conduction velocities in the median nerve increased in seven. There were no serious adverse effects including thalidomide neuropathy.
Conclusion: Thalidomide treatment should be further studied as a treatment of POEMS syndrome, particularly for patients who are not indicated for transplantation therapy.
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