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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
  1. Brigitte Granel1,2,
  2. Aurélie Daumas1,
  3. Elisabeth Jouve3,
  4. Jean-Robert Harlé1,
  5. Pierre-Sébastien Nguyen4,
  6. Christian Chabannon5,
  7. Nathalie Colavolpe6,
  8. Jean-Charles Reynier7,
  9. Romain Truillet3,
  10. Stéphanie Mallet8,
  11. Antoine Baiada4,
  12. Dominique Casanova4,
  13. Laurent Giraudo9,
  14. Laurent Arnaud10,
  15. Julie Veran9,
  16. Florence Sabatier2,9,10,
  17. Guy Magalon4,9
  1. 1Internal Medicine Department, Assistance Publique Hôpitaux de Marseilles (AP-HM), Aix-Marseilles University, Marseilles, France
  2. 2Vascular Research Center Marseille, INSERM UMRS-1076, Aix-Marseilles University, Marseilles, France
  3. 3CIC- CPCET, Assistance Publique Hôpitaux de Marseilles (AP-HM), Aix-Marseilles University, Marseilles, France
  4. 4Plastic Surgery Department, Assistance Publique Hôpitaux de Marseilles (AP-HM), Aix-Marseilles University, Marseilles, France
  5. 5Cell Therapy Facility & Inserm CBT 510, Institut Paoli Calmettes, Marseilles, France
  6. 6Department of Radiology, Assistance Publique Hôpitaux de Marseille (AP-HM), Aix-Marseilles University, Marseilles, France
  7. 7Délégation de la Recherche et de l'Innovation, Assistance Publique Hôpitaux de Marseilles (AP-HM), Marseilles, France
  8. 8Department of Dermatology, Assistance Publique Hôpitaux de Marseilles (AP-HM), Aix-Marseilles University, Marseilles, France
  9. 9Culture and Cell Therapy Laboratory, INSERM CBT 510, Assistance Publique Hôpitaux de Marseilles (AP-HM), Aix-Marseilles University, Marseilles, France
  10. 10Hematology and Vascular Biology Laboratory, Assistance Publique Hôpitaux de Marseilles (AP-HM), Aix-Marseilles University, Marseilles, France
  1. Correspondence to Professor Brigitte Granel, North Hospital, Assistance Publique Hôpitaux de Marseilles (AP-HM), Marseilles 139015, France; bgranel{at}ap-hm.fr

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.

Findings 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.

Interpretation 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.

Funding GFRS (Groupe Francophone de Recherche sur la Sclérodermie).

Clinical Trials number NCT01813279.

  • Autoimmune Diseases
  • Systemic Sclerosis
  • Treatment

This is an Open Access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/

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