Thématique :
Originalité :
Très original
Solidité :
Doit faire évoluer notre pratique :
Nom du veilleur :
Professeur Philippe SEKSIK
Coup de coeur :
  2018 Jan;154(1):61-64.e6.  
  doi: 10.1053/j.gastro.2017.08.035.  
  Effects of Mongersen (GED-0301) on Endoscopic and Clinical Outcomes in Patients With Active Crohn's Disease  
  Feagan BG, Sands BE, Rossiter G, Li X, Usiskin K, Zhan X, Colombel JF  


GED-0301 is an antisense oligodeoxynucleotide with a sequence complementary to the Smad7 mRNA transcript. Smad7 is a negative regulator of transforming growth factor-β, which is increased in the intestinal mucosa of patients with active Crohn's disease (CD). We randomly assigned 63 CD patients to 4-, 8-, or 12-week treatment groups receiving oral GED-0301 (160 mg/day). The primary objective was to determine GED-0301's effect on endoscopic CD measures; secondary objectives included effects on clinical activity. Endoscopic improvement was observed in 37% of participants with evaluable endoscopy results at week 12. At week 12, 32% (4 weeks), 35% (8 weeks), and 48% (12 weeks) of patients receiving GED-0301 were in remission (CD activity index score <150); corresponding reductions from baseline in mean CD activity index scores were -124, -112, and -133 points. No new safety signals were observed. These findings support a GED-0301 benefit in active CD. no: NCT02367183.


Question posée
Quelle est l'efficacité " endoscopique " du Mongersen ?
Question posée
Le Mongersen induit une rémission endoscopique dans 37% des malades à 3 mois.

Des résultats intéressants même si le développeur a annoncé avoir interrompu deux essais (REVOLVE et SUSTAIN) de ce médicament dans la maladie de Crohn en raison de résultats décevants d’une analyse intermédiaire.