Long-term Efficacy and Safety of Stem Cell Therapy (Cx601) for Complex Perianal Fistulas in Patients With Crohn’s Disease Academic Article uri icon

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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.

authors

  • Panés, Julián
  • García-Olmo, Damián
  • Van Assche, Gert
  • Colombel, Jean Frederic
  • Reinisch, Walter
  • Baumgart, Daniel C
  • Dignass, Axel
  • Nachury, Maria
  • Ferrante, Marc
  • Kazemi-Shirazi, Lili
  • Grimaud, Jean C
  • de la Portilla, Fernando
  • Goldin, Eran
  • Richard, Marie Paule
  • Diez, Mary Carmen
  • Tagarro, Ignacio
  • Leselbaum, Anne
  • Danese, Silvio
  • Panés, Julián
  • Baumgart, Daniel C
  • Colombel, Jean F
  • Danese, Silvio
  • Van Assche, Gert
  • Reinisch, Walter
  • García-Olmo, Damián
  • Stift, Anton
  • Tschmelitsch, Jörg
  • Mrak, Karl
  • Tilg, Herbert
  • Kroberger, Irmgard
  • D’Hoore, André
  • De Looze, Danny
  • Baert, Filip
  • Pattyn, Paul
  • Zerbib, Philippe
  • Zerbib, Frank
  • Viennot, Stéphanie
  • Dupas, Jean-Louis
  • Orsoni, Pierre-Charles
  • Hebuterne, Xavier
  • Rahili, Amine
  • Allez, Matthieu
  • Panis, Yves
  • Reinshagen, Max
  • Scherer, Roland
  • Sturm, Andreas
  • Kruis, Wolfgang
  • Duek, Daniel-Simon
  • Waterman, Matti
  • Lahat-Zok, Adi
  • Zmora, Oded
  • Tulchinsky, Hagit
  • Edden, Yair
  • Spinelli, Antonino
  • Annese, Vito
  • Angriman, Imerio
  • Riegler, Gabriele
  • Selvaggi, Francesco
  • Oldenburg, Bas
  • Gilissen, Lennard
  • Van Montfort, Gust
  • Lowenberg, Mark
  • Bemelman, Adrianus Willem
  • Almenara, Raúl
  • Martín Arranz, María Dolores
  • García-Arranz, Mariano
  • Pérez Gisbert, Javier
  • Palasí, Rosana
  • Samsó, Carlos Taxonera
  • Herrera Justiniano, Jose Manuel
  • Rada, Ricardo
  • Butrón, Mª Teresa
  • López, Daniel Carpio
  • López-Sanromán, Antonio
  • Hinojosa de Val, Joaquín
  • Solana, Amparo
  • González Argenté, F Xavier
  • Pastor, Carlos
  • Guadalajara, Hector

publication date

  • April 2018

has subject area