Details

Autor(en) / Beteiligte

• 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
• Colombel, Jean F.
• 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

Titel

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

Ist Teil von

• Gastroenterology (New York, N.Y. 1943), 2018-04, Vol.154 (5), p.1334-1342.e4

Ort / Verlag

United States: Elsevier Inc

Erscheinungsjahr

2018

Quelle

MEDLINE

Beschreibungen/Notizen

• 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. 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). 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. 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. [Display omitted]