Thalidomide reduces serum VEGF levels and improves peripheral neuropathy in POEMS syndrome.
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 that high-dose chemotherapy with autologous peripheral blood stem-cell transplantation 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 8-23 months (mean, 15 months), all patients showed substantial clinical improvement (n = 6) or stabilisation of symptoms (n = 3). Serum VEGF levels decreased in all patients and were normalised in five patients. Nerve conduction velocities in the median nerve increased in seven patients. There were no serious adverse effects, including thalidomide neuropathy.


CONCLUSIONS
Thalidomide treatment should be further studied as a treatment for POEMS syndrome, particularly for patients who are not indicated for transplantation therapy.
Kuwabara
S
S
Department of Neurology, Graduate School of Medicine, Chiba University, Chiba, Japan. kuwabara-s@faculty.chiba-u.jp
Misawa
S
S
Kanai
K
K
Sawai
S
S
Hattori
T
T
Nishimura
M
M
Nakaseko
C
C
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D020533:Angiogenesis Inhibitors / Q000494:pharmacology* / Q000627:therapeutic use*; D000893:Anti-Inflammatory Agents / Q000627:therapeutic use; D003907:Dexamethasone / Q000627:therapeutic use; D004334:Drug Administration Schedule; D004359:Drug Therapy, Combination; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D016878:POEMS Syndrome / Q000097:blood* / Q000188:drug therapy*; D010523:Peripheral Nervous System Diseases / Q000188:drug therapy* / Q000503:physiopathology*; D013792:Thalidomide / Q000494:pharmacology* / Q000627:therapeutic use*; D042461:Vascular Endothelial Growth Factor A / Q000037:antagonists & inhibitors* / Q000097:blood*
D020533:Angiogenesis Inhibitors; D000893:Anti-Inflammatory Agents; D042461:Vascular Endothelial Growth Factor A; D013792:Thalidomide; D003907:Dexamethasone