title: Japanese POEMS syndrome with Thalidomide (J-POST) Trial: study protocol for a phase II/III multicentre, randomised, double-blind, placebo-controlled trial.
abstract: BACKGROUND
  Polyneuropathy, organomegaly, endocrinopathy, M-protein and skin changes (POEMS) syndrome is a fatal systemic disorder associated with plasma cell dyscrasia and the overproduction of the vascular endothelial growth factor (VEGF). Recently, the prognosis of POEMS was substantially improved by introduction of therapeutic intervention for myeloma. However, no randomised clinical trial has been performed because of the rarity and severity of the disease.
  
  
  METHODS
  The Japanese POEMS syndrome with Thalidomide (J-POST) Trial is a phase II/III multicentre, double-blinded, randomised, controlled trial that aims to evaluate the efficacy and safety of a 24-week treatment with thalidomide in POEMS syndrome, with an additional 48-week open-label safety study. Adults with POEMS syndrome who have no indication for transplantation are assessed for eligibility at 12 tertiary neurology centres in Japan. Patients who satisfy the eligibility criteria are randomised (1:1) to receive thalidomide (100-300 mg daily) plus dexamethasone (12 mg/m(2) on days 1-4 of a 28-day cycle) or placebo plus dexamethasone. Both treatments were administered for 24 weeks (six cycles; randomised comparative study period). Patients who complete the randomised study period or show subacute deterioration during the randomised period participate in the subsequent 48-week open-label safety study (long-term safety period). The primary end point of the study is the reduction rate of serum VEGF levels at 24 weeks.
  
  
  BACKGROUND
  The protocol was approved by the Institutional Review Board of each hospital. The trial was notified and registered at the Pharmaceutical and Medical Devices Agency, Japan (No. 22-1716). The J-POST Trial is currently ongoing and is due to finish in August 2015. The findings of this trial will be disseminated through peer-reviewed publications and conference presentations and will also be disseminated to participants.
  
  
  BACKGROUND
  UMIN000004179 and JMA-IIA00046.
authors.affiliation: Clinical Research Center, Chiba University Hospital, Chiba, Japan.
authors.forename: Kanako
authors.initials: K
authors.lastname: Katayama
authors.affiliation: Department of Neurology, Graduate School of Medicine, Chiba University, Chiba, Japan.
authors.forename: Sonoko
authors.initials: S
authors.lastname: Misawa
authors.affiliation: Clinical Research Center, Chiba University Hospital, Chiba, Japan.
authors.forename: Yasunori
authors.initials: Y
authors.lastname: Sato
authors.affiliation: Department of Neurology, Nagoya University Graduate School of Medicine, Nagoya, Japan.
authors.forename: Gen
authors.initials: G
authors.lastname: Sobue
authors.affiliation: Department of Neurology, Hokkaido University Graduate School of Medicine, Sapporo, Japan.
authors.forename: Ichiro
authors.initials: I
authors.lastname: Yabe
authors.affiliation: Department of Neurology and Geriatrics, Kagoshima University Graduate School of Medical and Dental Sciences, Kagoshima, Japan.
authors.forename: Osamu
authors.initials: O
authors.lastname: Watanabe
authors.affiliation: Department of Neurology, Brain Research Institute, Niigata University, Niigata, Japan.
authors.forename: Masatoyo
authors.initials: M
authors.lastname: Nishizawa
authors.affiliation: Department of Neurology, Faculty of Medicine, Kinki University, Osaka-Sayama, Japan.
authors.forename: Susumu
authors.initials: S
authors.lastname: Kusunoki
authors.affiliation: Department of Neurology, National Hospital Organization Hokkaido Medical Center, Sapporo, Japan.
authors.forename: Seiji
authors.initials: S
authors.lastname: Kikuchi
authors.affiliation: Department of Neurology, Tohoku University Graduate School of Medicine, Sendai, Japan.
authors.forename: Ichiro
authors.initials: I
authors.lastname: Nakashima
authors.affiliation: Department of Medicine (Neurology and Rheumatology), Shinshu University School of Medicine, Matsumoto, Japan.
authors.forename: Shu-Ichi
authors.initials: S
authors.lastname: Ikeda
authors.affiliation: Department of Neurology, Kobe City Medical Center General Hospital, Kobe, Japan.
authors.forename: Nobuo
authors.initials: N
authors.lastname: Kohara
authors.affiliation: Department of Neurology and Clinical Neuroscience, Graduate School of Medicine Yamaguchi University, Ube, Japan.
authors.forename: Takashi
authors.initials: T
authors.lastname: Kanda
authors.affiliation: Department of Neurology, Neurological Institute, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan.
authors.forename: Jun-Ichi
authors.initials: J
authors.lastname: Kira
authors.affiliation: Clinical Research Center, Chiba University Hospital, Chiba, Japan.
authors.forename: Hideki
authors.initials: H
authors.lastname: Hanaoka
authors.affiliation: Department of Neurology, Graduate School of Medicine, Chiba University, Chiba, Japan.
authors.forename: Satoshi
authors.initials: S
authors.lastname: Kuwabara
mesh_terms: D000328:Adult; D002985:Clinical Protocols; D004311:Double-Blind Method; D005260:Female; D006401:Hematologic Agents / Q000627:therapeutic use*; D006801:Humans; D007564:Japan; D008297:Male; D009101:Multiple Myeloma / Q000188:drug therapy; D017063:Outcome Assessment, Health Care; D016878:POEMS Syndrome / Q000097:blood / Q000188:drug therapy*; D010265:Paraproteinemias / Q000097:blood / Q000188:drug therapy*; D012107:Research Design; D013792:Thalidomide / Q000627:therapeutic use*; D042461:Vascular Endothelial Growth Factor A / Q000097:blood*
chemical_list: D006401:Hematologic Agents; D042461:Vascular Endothelial Growth Factor A; D013792:Thalidomide