Autologous Myoblasts for the Treatment of Fecal Incontinence

Autor: Boyer, Olivier, Bridoux, Valérie, Giverne, Camille, Bisson, Aurélie, Koning, Edith, Leroi, Anne-Marie, Chambon, Pascal, Déhayes, Justine, Le Corre, Stéphanie, Jacquot, Serge, Bastit, Dominique, Martinet, Jérémie, Houivet, Estelle, Tuech, Jean-Jacques, Bénichou, Jacques, Michot, Francis
Přispěvatelé: Laboratoire d'immunologie et biothérapies [Rouen], Université de Rouen Normandie (UNIROUEN), Normandie Université (NU)-Normandie Université (NU)-CHU Rouen, Normandie Université (NU), Physiopathologie, Autoimmunité, maladies Neuromusculaires et THErapies Régénératrices (PANTHER), Normandie Université (NU)-Normandie Université (NU)-Institut National de la Santé et de la Recherche Médicale (INSERM), Service de chirurgie digestive [CHU Rouen], CHU Rouen, Normandie Université (NU)-Normandie Université (NU)-Université de Rouen Normandie (UNIROUEN), Nutrition, inflammation et dysfonctionnement de l'axe intestin-cerveau (ADEN), Institute for Research and Innovation in Biomedicine (IRIB), Normandie Université (NU)-Normandie Université (NU)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), UNIROUEN - UFR Santé (UNIROUEN UFR Santé), Normandie Université (NU)-Normandie Université (NU), Service de Radiologie [CHU Rouen], Service de physiologie digestive, urinaire, respiratoire et de l'exercice [CHU Rouen], Hôpital Charles Nicolle [Rouen]-CHU Rouen, Laboratoire de Cytologénétique CHU Rouen, Etablissement français du sang [Rouen], Unité de biostatistiques [CHU Rouen]
Jazyk: angličtina
Rok vydání: 2018
Předmět:
Zdroj: Annals of Surgery
Annals of Surgery, Lippincott, Williams & Wilkins, 2018, 267 (3), pp.443-450. ⟨10.1097/SLA.0000000000002268⟩
ISSN: 0003-4932
DOI: 10.1097/SLA.0000000000002268⟩
Popis: International audience; Objective: The aim of this study was to evaluate the efficacy of intrasphincteric injections of autologous myoblasts (AMs) in fecal incontinence (FI) in a controlled study.Summary of Background Data: Adult stem cell therapy is expected to definitively cure FI by regenerating damaged sphincter. Preclinical data and results of open-label trials suggest that myoblast therapy may represent a noninvasive treatment option.Methods: We conducted a phase 2 randomized, double-blind, placebo-controlled study of intrasphincteric injections of AM in 24 patients. The study compared outcome after AM (n = 12) or placebo (n = 12) injection using Cleveland Clinic Incontinence (CCI), score at 6 and 12 months. Patients in the placebo group were eligible to receive frozen AM after 1 year.Results: At 6 months, the median CCI score significantly decreased from baseline in both the AM (9 vs 15, P = 0.02) and placebo (10 vs 15, P = 0.01) groups. Hence, no significant difference was found between the 2 groups (primary endpoint) at 6 months. At 12 months, the median CCI score continued to ameliorate in the AM group (6.5 vs 15, P = 0.006), while effect was lost in the placebo group (14 vs 15, P = 0.35). Consequently, there was a higher response rate at 12 months in the treated than the placebo arm (58% vs 8%, P = 0.03). After delayed frozen AM injection in the placebo group, the response rate was 60% (6/10) at 12 months.Conclusions: Intrasphincteric AM injections in FI patients have shown tolerance, safety, and clinical benefit at 12 months despite a transient placebo effect at 6 months.
Databáze: OpenAIRE
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