title: Safety, tolerability and potential efficacy of injection of autologous adipose-derived stromal vascular fraction in the fingers of patients with systemic sclerosis: an open-label phase I trial.
abstract: BACKGROUND
  In patients with systemic sclerosis (scleroderma, SSc), impaired hand function greatly contributes to disability and reduced quality of life, and is insufficiently relieved by currently available therapies. Adipose tissue-derived stromal vascular fraction (SVF) is increasingly recognised as an easily accessible source of regenerative cells with therapeutic potential in ischaemic or autoimmune diseases. We aimed to measure for the first time the safety, tolerability and potential efficacy of autologous SVF cells local injections in patients with SSc with hand disability.
  
  
  METHODS
  We did an open-label, single arm, at one study site with 6-month follow-up among 12 female SSc patients with Cochin Hand Function Scale score >20/90. Autologous SVF was obtained from lipoaspirates, using an automated processing system, and subsequently injected into the subcutaneous tissue of each finger in contact with neurovascular pedicles. Primary outcome was the number and the severity of adverse events related to SVF-based therapy. Secondary endpoints were changes in hand disability and fibrosis, vascular manifestations, pain and quality of life from baseline to 2 and 6 months after cell therapy.
  
  
  RESULTS
  All enrolled patients had surgery, and there were no dropouts or patients lost to follow-up. No severe adverse events occurred during the procedure and follow-up. Four minor adverse events were reported and resolved spontaneously. A significant improvement in hand disability and pain, Raynaud's phenomenon, finger oedema and quality of life was observed.
  
  
  CONCLUSIONS
  This study outlines the safety of the autologous SVF cells injection in the hands of patients with SSc. Preliminary assessments at 6 months suggest potential efficacy needing confirmation in a randomised placebo-controlled trial on a larger population.
  
  
  BACKGROUND
  GFRS (Groupe Francophone de Recherche sur la Sclérodermie).
  
  
  BACKGROUND
  NCT01813279.
authors.affiliation: Internal Medicine Department, Assistance Publique Hôpitaux de Marseilles (AP-HM), Aix-Marseilles University, Marseilles, France Vascular Research Center Marseille, INSERM UMRS-1076, Aix-Marseilles University, Marseilles, France.
authors.forename: Brigitte
authors.initials: B
authors.lastname: Granel
authors.affiliation: Internal Medicine Department, Assistance Publique Hôpitaux de Marseilles (AP-HM), Aix-Marseilles University, Marseilles, France.
authors.forename: Aurélie
authors.initials: A
authors.lastname: Daumas
authors.affiliation: CIC- CPCET, Assistance Publique Hôpitaux de Marseilles (AP-HM), Aix-Marseilles University, Marseilles, France.
authors.forename: Elisabeth
authors.initials: E
authors.lastname: Jouve
authors.affiliation: Internal Medicine Department, Assistance Publique Hôpitaux de Marseilles (AP-HM), Aix-Marseilles University, Marseilles, France.
authors.forename: Jean-Robert
authors.initials: JR
authors.lastname: Harlé
authors.affiliation: Plastic Surgery Department, Assistance Publique Hôpitaux de Marseilles (AP-HM), Aix-Marseilles University, Marseilles, France.
authors.forename: Pierre-Sébastien
authors.initials: PS
authors.lastname: Nguyen
authors.affiliation: Cell Therapy Facility & Inserm CBT 510, Institut Paoli Calmettes, Marseilles, France.
authors.forename: Christian
authors.initials: C
authors.lastname: Chabannon
authors.affiliation: Department of Radiology, Assistance Publique Hôpitaux de Marseille (AP-HM), Aix-Marseilles University, Marseilles, France.
authors.forename: Nathalie
authors.initials: N
authors.lastname: Colavolpe
authors.affiliation: Délégation de la Recherche et de l'Innovation, Assistance Publique Hôpitaux de Marseilles (AP-HM), Marseilles, France.
authors.forename: Jean-Charles
authors.initials: JC
authors.lastname: Reynier
authors.affiliation: CIC- CPCET, Assistance Publique Hôpitaux de Marseilles (AP-HM), Aix-Marseilles University, Marseilles, France.
authors.forename: Romain
authors.initials: R
authors.lastname: Truillet
authors.affiliation: Department of Dermatology, Assistance Publique Hôpitaux de Marseilles (AP-HM), Aix-Marseilles University, Marseilles, France.
authors.forename: Stéphanie
authors.initials: S
authors.lastname: Mallet
authors.affiliation: Plastic Surgery Department, Assistance Publique Hôpitaux de Marseilles (AP-HM), Aix-Marseilles University, Marseilles, France.
authors.forename: Antoine
authors.initials: A
authors.lastname: Baiada
authors.affiliation: Plastic Surgery Department, Assistance Publique Hôpitaux de Marseilles (AP-HM), Aix-Marseilles University, Marseilles, France.
authors.forename: Dominique
authors.initials: D
authors.lastname: Casanova
authors.affiliation: Culture and Cell Therapy Laboratory, INSERM CBT 510, Assistance Publique Hôpitaux de Marseilles (AP-HM), Aix-Marseilles University, Marseilles, France.
authors.forename: Laurent
authors.initials: L
authors.lastname: Giraudo
authors.affiliation: Hematology and Vascular Biology Laboratory, Assistance Publique Hôpitaux de Marseilles (AP-HM), Aix-Marseilles University, Marseilles, France.
authors.forename: Laurent
authors.initials: L
authors.lastname: Arnaud
authors.affiliation: Culture and Cell Therapy Laboratory, INSERM CBT 510, Assistance Publique Hôpitaux de Marseilles (AP-HM), Aix-Marseilles University, Marseilles, France.
authors.forename: Julie
authors.initials: J
authors.lastname: Veran
authors.affiliation: Vascular Research Center Marseille, INSERM UMRS-1076, Aix-Marseilles University, Marseilles, France Culture and Cell Therapy Laboratory, INSERM CBT 510, Assistance Publique Hôpitaux de Marseilles (AP-HM), Aix-Marseilles University, Marseilles, France Hematology and Vascular Biology Laboratory, Assistance Publique Hôpitaux de Marseilles (AP-HM), Aix-Marseilles University, Marseilles, France.
authors.forename: Florence
authors.initials: F
authors.lastname: Sabatier
authors.affiliation: Plastic Surgery Department, Assistance Publique Hôpitaux de Marseilles (AP-HM), Aix-Marseilles University, Marseilles, France Culture and Cell Therapy Laboratory, INSERM CBT 510, Assistance Publique Hôpitaux de Marseilles (AP-HM), Aix-Marseilles University, Marseilles, France.
authors.forename: Guy
authors.initials: G
authors.lastname: Magalon
mesh_terms: D000328:Adult; D000368:Aged; D004185:Disability Evaluation; D005260:Female; D005385:Fingers; D006801:Humans; D007267:Injections; D008297:Male; D045164:Mesenchymal Stem Cell Transplantation / Q000379:methods*; D008875:Middle Aged; D011788:Quality of Life; D012595:Scleroderma, Systemic / Q000534:rehabilitation / Q000628:therapy*; D014182:Transplantation, Autologous; D016896:Treatment Outcome
keywords: Autoimmune Diseases; Systemic Sclerosis; Treatment
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