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Clinical, electrophysiological and VEGF 2-year response after lenalidomide or stem cell transplantation in patients with POEMS syndrome
  1. Mariangela Bianco1,
  2. Fabrizia Terenghi1,
  3. Francesca Gallia1,
  4. Andrea Nozza2,
  5. Antonietta Scarale1,
  6. Mohammed Zhiad Fayoumi1,
  7. Claudia Giannotta1,
  8. Eduardo Nobile-Orazio1
  1. 1 Department of Medical Biotechnology and Translational Medicine, Milan University, Neuromuscular and Neuroimmunology Service, Humanitas Clinical and Research Center, Rozzano, Italy
  2. 2 Department of Medical Oncology and Haematology, Humanitas Clinical and Research Centre, Rozzano, Italy
  1. Correspondence to Professor Eduardo Nobile-Orazio, Department of Medical Biotechnology and Translational Medicine, Milan University, Neuromuscular and Neuroimmunology Service, Humanitas Clinical and Research Center, Milan, MI 20089, Italy; eduardo.nobile{at}

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POEMS syndrome is a rare multisystem disease characterised by polyneuropathy (P), organomegaly (O), endocrinopathy (E), monoclonal gammopathy (M) and skin changes (S).1 A usually severe demyelinating and axonal neuropathy is often the most prominent and disabling clinical manifestation.2 The pathogenesis of POEMS syndrome is unclear even if overproduction of vascular endothelial growth factor (VEGF) seems to play a major role.1 2 Autologous haematopoietic stem cell transplantation (HSCT) is now considered the most effective therapy in patients with disseminated disease.1 This therapy is however not feasible in all patients because of their advanced age or the presence of severe concomitant conditions. A number of immune or cytostatic agents have been also used with variable efficacy in POEMS syndrome,1 including lenalidomide whose efficacy we recently reported in 13 of the 18 treated patients.3 Little is known however on the degree of response to lenalidomide in comparison with HSCT.


We retrospectively reviewed the clinical, biological and electrophysiological data from 15 patients with POEMS syndrome diagnosed according the criteria proposed by Dispenzieri,1 who neurologically improved after therapy with lenalidomide or HSCT. All the patients had been treated and followed at our institution for at least 2 years. The study was approved by the institutional review board of our institute, and all the patients signed an informed consent for the review of their data.

Six patients (four males, two females; mean age at disease onset 48 years, range 41–53; …

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  • Contributors Study design: MB and EN-O. Experiments and procedures: FT, FG, AN, AS, MZF and CG. Data analysis and interpretation: MB and EN-O. Manuscript drafting: MB and EN-O. Manuscript editing: all authors. Approval of final draft: all authors.

  • Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.

  • Disclaimer The company had no role in this study. 

  • Competing interests EN-O: personal fees for lecturing or advisory/scientific/safety board membership not related to this study from Baxter, Italy; CSL Behring, Switzerland; Kedrion Biopharma, Italy; LFB, France; Novartis, Switzerland; UCB, UK; Astellas Biopharma, the Netherlands. The other authors have no conflicts of interests.

  • Patient consent Not required.

  • Ethics approval Ethic Committee of Humanitas Clinical Institute.

  • Provenance and peer review Not commissioned; externally peer reviewed.