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Multicenter Study
. 2025 Jun;60(6):873-878.
doi: 10.1038/s41409-025-02586-2. Epub 2025 Apr 14.

Teduglutide for treatment-refractory severe intestinal acute graft-versus-host disease - a multicenter survey

Niklas Brehm  1 , Francesca Biavasco  1 , Johannes Clausen  2 , Johannes Jung  3 , Kristina Maas-Bauer  1 , Ralph Wäsch  1 , Mareike Verbeek  3 , Christoph Nuernbergk  3 , Gabriele Ihorst  4 , Stuart Seropian  5 , Jürgen Finke  1 , Lohith Gowda  5 , Rakefet Sidlik Muskatel  6 , Régis Peffault de Latour  7   8   9 , Gérard Socie  7   9 , Claudia Wehr  1 , David Michonneau  7   8   9 , Robert Zeiser  10
Affiliations
Multicenter Study

Teduglutide for treatment-refractory severe intestinal acute graft-versus-host disease - a multicenter survey

Niklas Brehm et al. Bone Marrow Transplant. 2025 Jun.

Abstract

Intestinal glucocorticoid-refractory (SR) acute (a) graft-versus-host disease (GVHD) causes high non-relapse mortality (NRM) in patients after allogeneic hematopoietic cell transplantation (allo-HCT). Recent preclinical data indicate that acute GVHD causes a loss of intestinal neuroendocrine L-cells leading to reduced levels of glucagon-like peptide-2 (GLP-2). GLP-2 substitution improved GVHD severity and increased Paneth cells and intestinal stem cells in mice. This motivated us to treat patients with refractory intestinal aGHVD using the GLP-2-analogon teduglutide. In this retrospective multicenter survey, 17 patients received teduglutide as salvage-therapy for SR-intestinal aGVHD. The best response (CR or PR) at any time point during and after treatment was 64.7% (11/17) including 41.2% (7/17) CR and 23.5% (4/17) PR. At a median follow-up of 28 weeks after teduglutide 10/17 patients are alive. Most patients experienced an increase of the albumin serum level within 2 months after the first teduglutide dose, including patients who clinically did not respond to teduglutide treatment. No specific teduglutide-related toxicity was observed. Our retrospective analysis suggests that teduglutide is safe and has activity in a fraction of patients with intestinal SR-aGVHD, which needs validation in a prospective trial.

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Conflict of interest statement

Competing interests: CW: received honoraria or travel support from Takeda, MSD, Jazz Pharmaceuticals and Grifols. DM: has received research grants from Novartis, CSL Behring and Sanofi, received consulting fees from Novartis, Incyte, Mallinckrodt and Sanofi, honoraria for lectures, presentations, speakers’ bureaus, manuscript writing, or educational events from Jazz Pharmaceuticals. He has received support for attending meetings and/or travel from Novartis and Sanofi. He has participated in a Data Safety Monitoring Board or Advisory Board for Incyte and Sanofi. GI: none for this work. JC: received speakers fees and research funding from Novartis and Takeda. JF: none for this work. JJ: none for this work. RPL: Expert consultant / speaker: Alexion, Amgen, Apellis, Jazz, Novartis, Pfizer, Roche & Samsung - Research grant: Alexion, Novartis and Pfizer. RW: received honoraria from Abbvie, Alexion Amgen, BMS, Janssen, Kite/Gilead, Pfizer Sanofi, Takeda. RZ: received speakers fees from Incyte, Novartis, Sanofi, Medac and Mallinckrodt/Therakos. The other authors have no conflict of interest with this manuscript to disclose.

Figures

Fig. 1
Fig. 1. Teduglutide in patients with intestinal SR-aGVHD.
a Bar graph showing the clinical or histological response after teduglutide treatment. CR complete response, PR partial response, NR non-responder. n indicates the number of patients per group. % indicates the relative number of patients per group. b Representative ileocolonoscopic-samples pre- and during teduglutide-treatment. c Bar graph showing the duration of treatment in days. Each dot represents one patient. The error bars indicate mean with SEM. d Bar graph indicating the time to response in days. Each dot represents one patient. The error bar indicates mean with SEM. e Graph describing the serum albumin levels in g/dl before teduglutide application and the best response within two months after first application. The connected dots indicate the two timepoints of one patient. The red dots indicate patients that were non-responders. A paired t-test was performed. P = 0.0001. f Kaplan Meier estimation of overall survival from start of teduglutide treatment.
See this image and copyright information in PMC

References

    1. Zeiser R, Blazar BR. Acute graft-versus-host disease — biologic process, prevention, and therapy. N Engl J Med. 2017;377:2167–79. - PMC - PubMed
    1. Holtan SG, Yu J, Choe HK, Paranagama D, Tang J, Naim A, et al. Disease progression, treatments, hospitalization, and clinical outcomes in acute GVHD: a multicenter chart review. Bone Marrow Transpl. 2022;57:1581–5. - PMC - PubMed
    1. Axt L, Naumann A, Toennies J, Haen SP, Vogel W, Schneidawind D, et al. Retrospective single center analysis of outcome, risk factors and therapy in steroid refractory graft-versus-host disease after allogeneic hematopoietic cell transplantation. Bone Marrow Transpl. 2019;54:1805–14. - PubMed
    1. Naymagon S, Naymagon L, Wong SY, Ko HM, Renteria A, Levine J, et al. Acute graft-versus-host disease of the gut: considerations for the gastroenterologist. Nat Rev Gastroenterol Hepatol. 2017;14:711–26. - PMC - PubMed
    1. Jamy O, Zeiser R, Chen YB. Novel developments in the prophylaxis and treatment of acute GVHD. Blood. 2023;142:1037–46. - PubMed

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