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

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Multicenter Study

. 2021 Mar;100(3):779-787.

doi: 10.1007/s00277-021-04434-x. Epub 2021 Jan 30.

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Multicenter Study

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

Tobias Wertheimer et al. Ann Hematol. 2021 Mar.

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.

Keywords: Abatacept; Bronchiolitis obliterans syndrome; Chronic graft-versus-host disease; Salvage therapy.

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Conflict of interest statement

D. W. received honoraria from Mallinckrodt, Novartis, Takeda, MACO, and Neovii.

The authors declare that they have no conflict of interest.

Figures

Fig. 1
Fig. 1

Clinical course during abatacept treatment

Fig. 2
Fig. 2

Forced expiratory volume % before and during abatacept therapy

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