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Vascular endothelial growth factor as a predictive marker for POEMS syndrome
treatment response: retrospective cohort study
#MMPMID26560063
Misawa S
; Sato Y
; Katayama K
; Hanaoka H
; Sawai S
; Beppu M
; Nomura F
; Shibuya K
; Sekiguchi Y
; Iwai Y
; Watanabe K
; Amino H
; Ohwada C
; Takeuchi M
; Sakaida E
; Nakaseko C
; Kuwabara S
BMJ Open
2015[Nov]; 5
(11
): e009157
PMID26560063
show ga
OBJECTIVE: POEMS (polyneuropathy, organomegaly, endocrinopathy, M-protein and
skin changes) syndrome is a rare multisystem disease characterised by plasma cell
dyscrasia and overproduction of vascular endothelial growth factor (VEGF). VEGF
is assumed to be useful in monitoring disease activity, because VEGF levels
usually decrease after treatment. However, there is no study to investigate
whether the extent of decrease in VEGF correlates with clinical outcome. We
tested the predictive efficacy of serum VEGF levels in POEMS syndrome. METHOD:
This was an institutional review board approved retrospective observational
cohort study of 20 patients with POEMS monitored regularly for more than
12?months (median follow-up, 87?months) after treatment onset using our
prospectively accumulated database of POEMS from 1999 to 2015. Patients were
treated by autologous peripheral blood stem cell transplantation or thalidomide
administration. Serum VEGF was measured by ELISA. Outcome measures included
clinical and laboratory findings and relapse-free survival. RESULTS: Serum VEGF
levels decreased rapidly after treatment, and stabilised by 6?months post
treatment. Patients with normalised serum VEGF levels (<1040?pg/mL) at 6?months
showed prolonged relapse-free survival (HR=12.81, 95% CI 2.691 to 90.96;
p=0.0001) and greater later clinical improvement. The rate of serum VEGF
reduction over the first 6?months post treatment correlated with increased grip
strength, serum albumin levels, and compound muscle action potential amplitudes
at 12?months. CONCLUSIONS: Serum VEGF level at 6?months post treatment is a
predicative biomarker for disease activity and prognosis in POEMS syndrome. Serum
VEGF could be used as a surrogate endpoint for relapse-free survival or clinical
or laboratory improvement of POEMS syndrome for clinical trials.
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