2. Academic Validation
  3. A phase 3 randomized trial comparing inolimomab vs usual care in steroid-resistant acute GVHD

A phase 3 randomized trial comparing inolimomab vs usual care in steroid-resistant acute GVHD

  • Blood. 2017 Feb 2;129(5):643-649. doi: 10.1182/blood-2016-09-738625.
Gérard Socié 1 2 3,  Stéphane Vigouroux 4,  Ibrahim Yakoub-Agha 5,  Jacques-Olivier Bay 6,  Sabine Fürst 7,  Karin Bilger 8,  Felipe Suarez 9,  Mauricette Michallet 10,  Dominique Bron 11,  Philippe Gard 12,  Zakaria Medeghri 12,  Philippe Lehert 13,  Chinglin Lai 14,  Tim Corn 15,  Jean-Paul Vernant 16
Affiliations

Affiliations

  • 1 Service d'Hématologie-Greffe, Assistance Publique-Hôpitaux de Paris, Hôpital St-Louis Lariboisière, Paris, France.
  • 2 Department of Medicine, Université Paris 7 Denis Diderot, Paris, France.
  • 3 INSERM UMR 1160, Paris, France.
  • 4 Service d'hématologie clinique et thérapie cellulaire, Hôpital Haut-Lévêque, Pessac, France.
  • 5 CHU de Lille, LIRIC INSERM U995, Université Lille2, Lille, France.
  • 6 Service thérapie cellulaire et hématologie clinique, CHU, Clermont-Ferrand, France.
  • 7 Unité de Transplantation et de Thérapie Cellulaire, Institut Paoli Calmettes, Marseille, France.
  • 8 Service d'Onco-Hématologie, CHU Hautepierre, Strasbourg, France.
  • 9 Service d'hématologie adulte, Hôpital Necker, Paris, France.
  • 10 Service d'hématologie, Hôpital Lyon Sud, Lyon, France.
  • 11 Service d'hématologie, Institut Jules Bordet (ULB), Bruxelles, Belgium.
  • 12 EUSA Pharma (Europe) Limited (a Jazz Pharmaceuticals company), Lyon, France.
  • 13 Faculty of Medicine, University of Melbourne, Melbourne, VIC, Australia.
  • 14 Jazz Pharmaceuticals, Palo Alto, CA.
  • 15 Jazz Pharmaceuticals, Oxford, United Kingdom; and.
  • 16 Service d'hématologie clinique, Hôpital Pitié Salpêtrière, Paris, France.
PMID: 27899357 DOI: 10.1182/blood-2016-09-738625
Abstract

Treatment of steroid-resistant acute graft-versus-host disease (GVHD) remains an unmet clinical need. Inolimomab, a monoclonal antibody to CD25, has shown encouraging results in phase 2 trials. This phase 3 randomized, open-label, multicenter trial compared inolimomab vs usual care in adult patients with steroid-refractory acute GVHD. Patients were randomly selected to receive treatment with inolimomab or usual care (the control group was treated with antithymocyte globulin [ATG]). The primary objective was to evaluate overall survival at 1 year without changing baseline allocated therapy. A total of 100 patients were randomly placed: 49 patients in the inolimomab arm and 51 patients in the ATG arm. The primary criteria were reached by 14 patients (28.5%) in the inolimomab and 11 patients (21.5%) in the ATG arms, with a hazard ratio of 0.874 (P = .28). With a minimum follow-up of 1 year, 26 (53%) and 31 (60%) patients died in the inolimomab and ATG arms, respectively. Adverse events were similar in the 2 arms, with fewer viral infections in the inolimomab arm compared with the ATG arm. The primary end point of this randomized phase 3 trial was not achieved. The lack of a statistically significant effect confirms the need for development of more effective treatments for acute GVHD. This trial is registered to https://www.clinicaltrialsregister.eu/ctr-search/search as EUDRACT 2007-005009-24.

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