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Association between N-acetylcysteine treatment and in-hospital mortality in adult patients with acquired thrombotic thrombocytopenic purpura: a cohort study

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Abstract

Acquired thrombotic thrombocytopenic purpura (aTTP) is a fatal hematologic disease. Despite the currently high standards of care, some patients who develop refractory or recurrent disease still have a poor prognosis. Although N-acetylcysteine (NAC) is recommended for the treatment of aTTP, its use in aTTP treatment is still controversial. We aimed to evaluate the association of NAC with mortality in patients with aTTP. This was a retrospective cohort study of patients with aTTP with in-hospital mortality as the primary outcome and time to platelet recovery and neurological recovery as secondary outcomes. We used multifactorial COX regression analysis to check for an association of NAC with mortality. Moreover, we performed a sensitivity analysis check the stability of our results. Finally, 89 patients with aTTP were enrolled. After adjusting for potential confounders, we found NAC to be associated with 75% lower in-hospital mortality (HR = 0.25, 95% CI = 0.1–0.64). The results of sensitivity analyses performed remained stable as the risk of in-hospital mortality in patients reduced in patients with comorbid neurological symptoms (HR = 0.23, 95% CI = 0.06–0.89). However, NAC use did not affect the time to platelet recovery (HR = 1.19, 95% CI = 0.57–2.5) or neurological recovery (HR = 0.32, 95% CI = 0.08–1.25) in patients with aTTP. NAC treatment reduces in-hospital mortality in patients with aTTP but does not shorten the time to platelet recovery or neurological recovery.

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Data Availability

The datasets generated and analysed during the current study are available from the corresponding authors upon reasonable request.

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Acknowledgements

We thank Medjaden Inc. for scientific editing of this manuscript.

Funding

This work was supported by Gusu Health Talents Programme (No. GSWS2020006) and Pre-Research Fund Project of the Second Affiliated Hospital of Soochow University (SDFEYJC2105).

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Author notes

  1. Jing Li, You Zhang and Danqing Kong contributed equally to this work.

Authors and Affiliations

  1. Department of Intensive Care Medicine, The First Affiliated Hospital of Soochow University, No. 188 Shizi Street, Suzhou, 215006, China

    Jing Li, Yao Wei, Xueke Liu, Shiqi Lu, Jun Wang & Fang Huang

  2. Department of Cardiology, The Second Affiliated Hospital of Soochow University, Suzhou, China

    You Zhang

  3. National Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China

    Danqing Kong

  4. Department of Thrombosis and Haemostasis, JiangSu Institute of Hematology, Suzhou, China

    Jian Su

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  1. Jing Li
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Contributions

(I) Conception and design: J Li; (II) Administrative support: J Wang; (III) Provision of study materials or patients: J Li, Dq Kong, J Wang; (IV) Collection and assembly of data: J Li, Y Zhang; (V) Data analysis and interpretation: J Li, Y Zhang; (VI) Manuscript writing: All authors; (VII) Final approval of manuscript: All authors.

Corresponding authors

Correspondence to Jun Wang or Fang Huang.

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Ethical statement

All procedures performed in this study involving human participants were in accordance with the Declaration of Helsinki (as revised in 2013). The study protocol was reviewed and approved by the Ethics Committee of The First Affiliated Hospital of Soochow University (No. 2022–453).

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The requirement for consent was waived, because the study was a retrospective analysis and all data were anonymized prior to analysis.

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The authors declare that they have no conflict of interest.

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Li, J., Zhang, Y., Kong, D. et al. Association between N-acetylcysteine treatment and in-hospital mortality in adult patients with acquired thrombotic thrombocytopenic purpura: a cohort study. Ann Hematol 102, 2257–2265 (2023). https://doi.org/10.1007/s00277-023-05295-2

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