Abatacept as salvage therapy in chronic graft-versus-host disease—a retrospective analysis

Wertheimer T, Dohse M, Afram G, Weber D, Heidenreich M, Holler B, Kattner AS, Neubauer A, Mielke S, Ljungman P, Holler E, Herr W, Edinger M, Martinez AP, Fante M, Wolff D (2021)

Publication Type: Journal article

Publication year: 2021

Journal

Annals of Hematology Springer Verlag (Germany)

Book Volume: 100

Pages Range: 779-787

Journal Issue: 3

DOI: 10.1007/s00277-021-04434-x

Abstract

The immunomodulatory fusion protein abatacept has recently been investigated for the treatment of steroid-refractory chronic graft-versus-host disease (cGvHD) in a phase 1 clinical trial. We analyzed the safety and efficacy of abatacept for cGvHD therapy in a retrospective study with 15 patients who underwent allogeneic hematopoietic stem cell transplantation (allo-HSCT) and received abatacept for cGvHD with a median age of 49 years. Grading was performed as part of the clinical routine according to the National Institute of Health’s (NIH) consensus criteria at initiation of abatacept and 1, 3, 6, 9 and 12 months thereafter. The median time of follow-up was 191 days (range 55–393 days). Best overall response rate (ORR) was 40%. In particular, patients with bronchiolitis obliterans syndrome showed significant clinical improvement and durable responses following abatacept treatment with a response rate of 89% based on improvement in lung severity score (n = 6) or stabilized lung function (n = 4) or both (n = 3). Infectious complications CTCAE °III or higher were observed in 3/15 patients. None of the patients relapsed from the underlying malignancy. Thus, abatacept appears to be a promising treatment option for cGvHD, in particular for patients with lung involvement. However, further evaluation within a phase 2 clinical trial is required.

Involved external institutions

Universitätsklinikum Regensburg DE Germany (DE) Universitätsklinikum Gießen und Marburg (UKGM) DE Germany (DE) Karolinska University Hospital / Karolinska Universitetssjukhuset SE Sweden (SE) Universidad Autónoma de Madrid (UAM) ES Spain (ES)

How to cite

APA:

Wertheimer, T., Dohse, M., Afram, G., Weber, D., Heidenreich, M., Holler, B.,... Wolff, D. (2021). Abatacept as salvage therapy in chronic graft-versus-host disease—a retrospective analysis. Annals of Hematology, 100(3), 779-787. https://doi.org/10.1007/s00277-021-04434-x

MLA:

Wertheimer, Tobias, et al. "Abatacept as salvage therapy in chronic graft-versus-host disease—a retrospective analysis." Annals of Hematology 100.3 (2021): 779-787.

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