Adipose tissue-derived stromal vascular fraction for treating hands of patients with systemic sclerosis: a multicentre randomized trial Autologous AD-SVF versus placebo in systemic sclerosis
Aurélie Daumas, Jérémy Magalon, Élisabeth Jouve, Dominique Casanova, C. Philandrianos, Maxime Abellan Lopez, S. Mallet, Julie Véran, I. Auquit-Auckbur, Dominique Farge, H. Lévesque, Y. Benhamou, Laurent Arnaud, Laurent Giraudo, Chloé Dumoulin, Camille Giverne, Olivier Boyer, Alexandra Giuliani, Véronique Bourgarel‐Rey, Jean‐Robert Harlé, N. Schleinitz, Julie Brunet, Yves‐Marie Pers, Rosanna Ferreira, Audrey Cras, David Boccara, Jérôme Larghero, Joseph Château, A. Hot, Françoise Dignat‐George, G. Magalon, Florence Sabatier, B. Granel
Abstract
OBJECTIVE: To assess the superiority of adipose tissue-derived stromal vascular fraction (AD-SVF) injection into the fingers vs placebo in reducing hand disability in systemic sclerosis (SSc) patients. METHODS: We performed a double-blind, multicentre, phase II trial from October 2015 to January 2018 in France. SSc patients with a Cochin Hand Function Scale (CHFS) ≥20/90 were randomized 1:1 to receive injection of AD-SVF or placebo. AD-SVF was obtained using the automated processing Celution 800/CRS system. The placebo was lactated Ringer's solution. The primary efficacy end point was the change of the CHFS score from baseline to 3 months. Secondary efficacy endpoints included the CHFS score at 6 months, hand function, vasculopathy, hand pain, skin fibrosis, sensitivity of the finger pulps, Scleroderma Health Assessment Questionnaire, patients and physician satisfaction, and safety. RESULTS: Forty patients were randomized. The AD-SVF and placebo groups were comparable for age, sex ratio, disease duration, skin fibrosis of the hands and main cause of hand disability. After 3 months' follow-up, hand function significantly improved in both groups with no between-group difference of CHFS (mean change of -9.2 [12.2] in the AD-SVF group vs -7.6 [13.2] in the placebo group). At 6 months, hand function improved in both groups. CONCLUSION: This study showed an improvement of hand function in both groups over time, with no superiority of the AD-SVF. Considering the limits of this trial, studies on a larger population of patients with homogeneous phenotype and hand handicap should be encouraged to accurately assess the benefit of AD-SVF therapy. TRIAL REGISTRATION: ClinicalTrials.gov, https://clinicaltrials.gov, NCT02558543. Registered on September 24, 2015.