SNFGE SNFGE
 
Thématique :
- Colo-proctologie
Originalité :
Très original
Solidité :
Très solide
Doit faire évoluer notre pratique :
Pas encore
 
 
Nom du veilleur :
Docteur Vincent de PARADES
Coup de coeur :
 
 
Gastroenterology
  2018/04  
 
  2018 Apr;154(5):1334-1342.e4.  
  doi: 10.1053/j.gastro.2017.12.020.  
 
  Long-term Efficacy and Safety of Stem Cell Therapy (Cx601) for Complex Perianal Fistulas in Patients With Crohn's Disease.  
 
  Panés J, García-Olmo D, Van Assche G, Colombel JF, Reinisch W, Baumgart DC, Dignass A, Nachury M, Ferrante M, Kazemi-Shirazi L, Grimaud JC, de la Portilla F, Goldin E, Richard MP, Diez MC, Tagarro I, Leselbaum A, Danese S; ADMIRE CD Study Group Collaborators  
  https://www.ncbi.nlm.nih.gov/pubmed/29277560  
 
 

Abstract

BACKGROUND & AIMS:

Therapies for perianal fistulas in patients with Crohn's disease are often ineffective in producing long-term healing. We performed a randomized placebo-controlled trial to determine the long-term efficacy and safety of a single local administration of allogeneic expanded adipose-derived stem cells (Cx601) in patients with Crohn's disease and perianal fistulas.

METHODS:

We performed a double-blind study at 49 hospitals in Europe and Israel, comprising 212 patients with Crohn's disease and treatment-refractory, draining, complex perianal fistulas. Patients were randomly assigned (1:1) to groups given a single local injection of 120 million Cx601 cells or placebo (control), in addition to the standard of care. Efficacy endpoints evaluated in the modified intention-to-treat population (randomly assigned, treated, and with 1 or more post-baseline efficacy assessment) at week 52 included combined remission (closure of all treated external openings draining at baseline with absence of collections >2 cm, confirmed by magnetic resonance imaging) and clinical remission (absence of draining fistulas).

RESULTS:

The study's primary endpoint, at week 24, was previously reported (combined remission in 51.5% of patients given Cx601 vs 35.6% of controls, for a difference of 15.8 percentage points; 97.5% confidence interval [CI] 0.5-31.2; P = .021). At week 52, a significantly greater proportion of patients given Cx601 achieved combined remission (56.3%) vs controls (38.6%) (a difference of 17.7 percentage points; 95% CI 4.2-31.2; P = .010), and clinical remission (59.2% vs 41.6% of controls, for a difference of 17.6 percentage points; 95% CI 4.1-31.1; P = .013). Safety was maintained throughout week 52; adverse events occurred in 76.7% of patients in the Cx601 group and 72.5% of patients in the control group.

CONCLUSION:

In a phase 3 trial of patients with Crohn's disease and treatment-refractory complex perianal fistulas, we found Cx601 to be safe and effective in closing external openings, compared with placebo, after 1 year. ClinicalTrials.gov no: NCT01541579.

 

 
Question posée
 
Evaluer le taux de guérison à long terme de fistules ano-périnéales de Crohn traitées par injection de cellules souches mésenchymateuses allogéniques.
 
Question posée
 
A S52, le taux de guérison était de 56 % dans le groupe « cellules souches » versus 39 % dans le groupe placebo (p = 0,01).
 
Commentaires

La confirmation du maintien à long terme du taux de guérison obtenu à S24 dans l’essai du Lancet de 2016 par un traitement novateur qui devrait être commercialisé en France en 2019.

 
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