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Abatacept treatment for autoimmune hemolytic anemia occurring post hematopoietic stem cell transplantation

Bone Marrow Transplantation volume 59, pages 288–290 (2024)Cite this article

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Autoimmune hemolytic anemia (AIHA) occurs in an estimated 2–20% of allogeneic hematopoietic stem cell transplant (HSCT) recipients, presenting unique challenges when compared to primary AIHA [1]. Corticosteroids represent the first-line treatment, rituximab and other immunosuppressants are used in case of failure or as steroid-sparing agents [1].

Abatacept is a CTLA4-analog that binds to CD80 and CD86, competing with CD28. As T-cell activation requires binding with CD28, by inhibiting this interaction, abatacept can modulate T-cell activation and contribute to improvement of autoimmune phenomena. This has been demonstrated for rheumatoid arthritis [2] but could possibly apply to other autoimmune conditions, such as AIHA [3,4,5]. Moreover, an increase in proportion of CD4+CD25+FOXP3+ regulatory T cells (Treg) was observed after abatacept treatment [3, 5], suggesting a restoration of Treg-cell function as FOXP3 is a Treg-cell function regulator. The pathogenesis of post-HSCT AIHA has not been completely elucidated, but a delayed immune reconstitution post HSCT, with lack of Treg and an imbalance between B and T-lymphocytes has been postulated [1]. Therefore, abatacept could ameliorate post-HSCT AIHA by inhibiting autoimmune T cell activation and increasing active Tregs. A small case series reported the efficacy of abatacept on post-HSCT AIHA in three patients [5]. Abatacept has been employed with success in the post-HSCT setting as a prophylactic and therapeutic agent against GVHD [6,7,8,9]. Due to the possible dual effect against AIHA and GVHD, abatacept is particularly attractive in the setting of post-HSCT AIHA.

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Data are available from the corresponding author upon request.

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Acknowledgements

The authors would like to thank their patients and their families.

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Authors and Affiliations

  1. Bone Marrow Transplantation, Great Ormond Street Hospital for Children, London, United Kingdom

    Maria Gabelli & Giovanna Lucchini

  2. Pediatric Hematology, Oncology and Stem Cell Transplantation, Università degli Studi di Padova, Padova, Italy

    Maria Gabelli

  3. Pediatric Hematology, Oncology and Stem Cell Transplantation, Hospital Universitario La Paz, Universidad Autónoma de Madrid, Instituto de Investigación La Paz, Madrid, Spain

    Antonio Pérez-Martínez & David Bueno Sánchez

  4. Bone Marrow Transplantation, CHU de Québec-Université Laval, Hotel-Dieu de Québec, Québec, Canada

    Louis-Philippe Grenier & Felix Couture

  5. Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute, Bethesda, MD, USA

    Emily Limerick

  6. Center for Cancer and Blood Disorders, Phoenix Children’s Hospital, Phoenix, AZ, USA

    Victoria Bernaud & Alexander Ngwube

  7. Pediatric Hematology, Oncology and Stem Cell Transplantation, Hospital Sant Joan de Déu, Barcelona, Spain

    Maria Trabazo del Castillo

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  1. Maria Gabelli
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  10. Alexander Ngwube
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Contributions

MG conceived the study, provided patient data, analyzed data and wrote the manuscript; AP-M, GL, L-PG and EL provided patient data; VB edited the manuscript, AN conceived the study and provided patient data. All authors critically reviewed the manuscript and approved the final version.

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Correspondence to Maria Gabelli.

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Gabelli, M., Pérez-Martínez, A., Bueno Sánchez, D. et al. Abatacept treatment for autoimmune hemolytic anemia occurring post hematopoietic stem cell transplantation. Bone Marrow Transplant 59, 288–290 (2024). https://doi.org/10.1038/s41409-023-02173-3

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