title: Efficacy of allogeneic mesenchymal stem cell transplantation in patients with drug-resistant polymyositis and dermatomyositis.
abstract: OBJECTIVE
  To assess the safety and clinical efficacy of allogeneic mesenchymal stem cell transplantation (MSCT) in a small-scale pilot study with 10 patients with drug-resistant polymyositis (PM) or dermatomyositis (DM).
  
  
  METHODS
  A single-arm trial involving 10 patients with DM/PM who were either refractory to standard treatment, or had severe systemic involvement. All patients consented and underwent allogeneic MSCT. Clinical and laboratory manifestations were compared before and after MSCT.
  
  
  RESULTS
  Improvements were seen in serum creatine kinase (CK), CK-MB, patient global assessment by visual analogue scale and muscle strength by manual muscle test in all patients, as well as improvement in interstitial lung disease in selected patients. Improvement in chronic non-healing skin ulcers was noted in one patient. Clinical responses were also seen in patients undergoing a second MSCT for recurrence of disease.
  
  
  CONCLUSIONS
  MSCT appears safe and effective in drug-resistant patients with DM/PM. Larger-scale studies including a control group receiving standard treatment are needed to assess the long-term efficacy of allogeneic MSCT in refractory patients with DM/PM.
authors.affiliation: Department of Rheumatology and Immunology, The Affiliated Drum Tower Hospital of Nanjing University Medical School, China.
authors.forename: Dandan
authors.initials: D
authors.lastname: Wang
authors.forename: Huayong
authors.initials: H
authors.lastname: Zhang
authors.forename: Mengshu
authors.initials: M
authors.lastname: Cao
authors.forename: Yu
authors.initials: Y
authors.lastname: Tang
authors.forename: Jun
authors.initials: J
authors.lastname: Liang
authors.forename: Xuebing
authors.initials: X
authors.lastname: Feng
authors.forename: Hong
authors.initials: H
authors.lastname: Wang
authors.forename: Bingzhu
authors.initials: B
authors.lastname: Hua
authors.forename: Bujun
authors.initials: B
authors.lastname: Liu
authors.forename: Lingyun
authors.initials: L
authors.lastname: Sun
mesh_terms: D000328:Adult; D015415:Biomarkers / Q000097:blood; D003402:Creatine Kinase / Q000097:blood; D052279:Creatine Kinase, MB Form / Q000097:blood; D003882:Dermatomyositis / Q000188:drug therapy / Q000503:physiopathology / Q000628:therapy; D004351:Drug Resistance; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D045164:Mesenchymal Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods*; D053580:Muscle Strength; D010865:Pilot Projects; D017285:Polymyositis / Q000188:drug therapy / Q000503:physiopathology / Q000628:therapy*; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D015415:Biomarkers; D003402:Creatine Kinase; D052279:Creatine Kinase, MB Form