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Transplant-associated thrombotic microangiopathy: is the treatment more expensive than the disease?

Bone Marrow Transplantation volume 54, pages 913–916 (2019)Cite this article

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Hematopoietic cell transplant-associated thrombotic microangiopathy (TA-TMA) is a subtype of thrombotic microangiopathy that carries a high rate of mortality [1, 2]. The prognosis varies according to disease severity, but mortality rates greater than 90% have been reported for patients with multiorgan involvement [3]. In comparison, baseline nonrelapse mortality from allogeneic hematopoietic cell transplantation (HCT) is approximately 30% [4]. Although the pathophysiologic mechanism remains uncertain, recent data suggest that endothelial injury during HCT leads to complement activation and TA-TMA through neutrophil extracellular traps [5]. Risk factors for TA-TMA include older age, female sex, HLA-mismatched donors, unrelated donors, high-dose busulfan, total body irradiation, calcineurin inhibitors, and infections [6, 7].

The role of complement activation in the pathogenesis of TA-TMA has led to the utilization of complement blockade in TA-TMA. Eculizumab is a humanized monoclonal antibody that binds to C5 and prevents the formation of the membrane attack complex (C5b-9) of the terminal complement pathway. Eculizumab is expensive with a typical 8-week 20-vial course costing $156,560 based on an average wholesale price of $7828 per 30 mL vial [8]. At the Ohio State University Wexner Medical Center, eculizumab is the tenth most expensive medication out of 1562 medications on the hospital formulary. Despite the high cost of eculizumab, there are no published data regarding the financial cost associated with TA-TMA treated with eculizumab. Hence, we conducted this study, wherein we aimed to examine the clinical outcomes and financial cost of 20 patients with TA-TMA treated with eculizumab.

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Acknowledgements

Research reported in this publication was supported by the National Cancer Institute of the National Institutes of Health under Award Number P30CA016058.

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Authors and Affiliations

  1. Division of Hematology and Medical Oncology, Weill Cornell Medicine, New York, NY, USA

    John L. Vaughn

  2. Division of Hematology, The Ohio State University, Columbus, OH, USA

    Qiuhong Zhao, Narendranath Epperla, Patrick Elder, Samantha Jaglowski, Sam Penza, Basem William, Leslie Andritsos, Jonathan E. Brammer, Yvonne Efebera, Don Benson, Spero Cataland & Sumithira Vasu

  3. Department of Pharmacy, James Cancer Hospital, The Ohio State University, Columbus, OH, USA

    Marcin Puto & Julianna Roddy

  4. Department of Hematology and Medical Oncology, Emory University, Atlanta, GA, USA

    William Blum, Rebecca Klisovic & Craig Hofmeister

  5. National Marrow Donor Program, Minneapolis, MN, USA

    Steven Devine

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  1. John L. Vaughn
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Contributions

J.L.V. designed the study, collected data, and wrote the manuscript. Q.Z. analyzed data and revised the manuscript. N.E., M.P., J.R., P.E., W.B., R.K., S.J., S.P., B.W., L.A., J.E.B., C.H., Y.E., D.B., S.D. and S.C. interpreted the data and revised the manuscript. S.V. designed the study, interpreted data, and revised the manuscript.

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Correspondence to Sumithira Vasu.

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Conflict of interest

S.C. has received research funding and honoraria from Alexion, Inc., the manufacturer of eculizumab. The other authors declare that they have no conflict of interest.

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Vaughn, J.L., Zhao, Q., Epperla, N. et al. Transplant-associated thrombotic microangiopathy: is the treatment more expensive than the disease?. Bone Marrow Transplant 54, 913–916 (2019). https://doi.org/10.1038/s41409-018-0390-3

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  • DOI: https://doi.org/10.1038/s41409-018-0390-3

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