A Daumas1, J Magalon2, E Jouve3, R Truillet3, D Casanova4, L Giraudo5, J Veran5, A Benyamine1, F Dignat-George6, G Magalon7, F Sabatier2, B Granel8. 1. Internal Medicine Department, Assistance publique-Hôpitaux de Marseille (AP-HM), CHU of Marseille, 13915 Marseille, France. 2. Culture and Cell Therapy Laboratory, INSERM CBT-1409, Assistance publique-Hôpitaux de Marseille (AP-HM), 13005 Aix Marseille University, France; Inserm, VRCM, UMR_S 1076, Aix Marseille University, 13005 Marseille, France. 3. Centre d'investigation clinique - centre de pharmacologie clinique et d'évaluations thérapeutiques (CIC-CPCET), AP-HM, CHU of Marseille, 13005 Marseille, France. 4. Plastic Surgery Department, AP-HM, CHU of Marseille, 13005 Marseille, France. 5. Culture and Cell Therapy Laboratory, INSERM CBT-1409, Assistance publique-Hôpitaux de Marseille (AP-HM), 13005 Aix Marseille University, France. 6. Hematology and Vascular Biology Laboratory, AP-HM, CHU of Marseille, 13005 Marseille, France; Inserm, VRCM, UMR_S 1076, Aix Marseille University, 13005 Marseille, France. 7. Centre d'investigation clinique - centre de pharmacologie clinique et d'évaluations thérapeutiques (CIC-CPCET), AP-HM, CHU of Marseille, 13005 Marseille, France; Plastic Surgery Department, AP-HM, CHU of Marseille, 13005 Marseille, France. 8. Internal Medicine Department, Assistance publique-Hôpitaux de Marseille (AP-HM), CHU of Marseille, 13915 Marseille, France; Inserm, VRCM, UMR_S 1076, Aix Marseille University, 13005 Marseille, France. Electronic address: bgranel@ap-hm.fr.
Abstract
INTRODUCTION: Hand involvement confers a substantial handicap in work and daily activities in patients with Systemic sclerosis (SSc). Autologous adipose-derived stromal vascular fraction is as an easily accessible source of cells with regenerative effects. We previously performed a phase I open-label clinical trial (NTC01813279) assessing the safety of subcutaneous injection of autologous adipose-derived stromal vascular fraction. Six and 12-month data have been reported. As patients were followed in our medical centre, we report their longer-term outcome beyond the end of the trial. PATIENTS AND METHOD: Twelve females, mean age 54.5±10.3 years, initially enrolled in the clinical trial were assessed during a scheduled medical care, which took place between 22 and 30months after treatment. RESULTS: Multiple patient-reported outcomes showed sustained improvement, in comparison with the assessment performed just before surgery: 62.5% in the Cochin Hand Function Scale, 51.1% in the Scleroderma Health Assessment Questionnaire, 33.1% in hand pain, and 88.3% in the Raynaud Condition Score. A decrease in the number of digital ulcers number was noted. Mobility, strength and fibrosis of the hand also showed improvement. None of the 8 patients who had previously received iloprost infusion required new infusion. CONCLUSION: Despite the limits of an open label study, the data are in favour of the long-term safety of the adipose-derived stromal vascular fraction injection. Two randomized double blind, placebo-controlled trials of this therapeutic agent are ongoing in the USA (NCT02396238) and in France (NCT02558543) and will help determine the place of this innovative therapy for SSc patients.
INTRODUCTION: Hand involvement confers a substantial handicap in work and daily activities in patients with Systemic sclerosis (SSc). Autologous adipose-derived stromal vascular fraction is as an easily accessible source of cells with regenerative effects. We previously performed a phase I open-label clinical trial (NTC01813279) assessing the safety of subcutaneous injection of autologous adipose-derived stromal vascular fraction. Six and 12-month data have been reported. As patients were followed in our medical centre, we report their longer-term outcome beyond the end of the trial. PATIENTS AND METHOD: Twelve females, mean age 54.5±10.3 years, initially enrolled in the clinical trial were assessed during a scheduled medical care, which took place between 22 and 30months after treatment. RESULTS: Multiple patient-reported outcomes showed sustained improvement, in comparison with the assessment performed just before surgery: 62.5% in the Cochin Hand Function Scale, 51.1% in the Scleroderma Health Assessment Questionnaire, 33.1% in hand pain, and 88.3% in the Raynaud Condition Score. A decrease in the number of digital ulcers number was noted. Mobility, strength and fibrosis of the hand also showed improvement. None of the 8 patients who had previously received iloprost infusion required new infusion. CONCLUSION: Despite the limits of an open label study, the data are in favour of the long-term safety of the adipose-derived stromal vascular fraction injection. Two randomized double blind, placebo-controlled trials of this therapeutic agent are ongoing in the USA (NCT02396238) and in France (NCT02558543) and will help determine the place of this innovative therapy for SSc patients.
Authors: Andrea L DiCarlo; Radia Tamarat; Carmen I Rios; Marc Benderitter; Christine W Czarniecki; Theresa C Allio; Francesca Macchiarini; Bert W Maidment; Jean-Rene Jourdain Journal: Radiat Res Date: 2017-06-12 Impact factor: 2.841