The long-term outcomes and safety of severe aplastic anemia treated with porcine antilymphocyte globulin plus cyclosporine, with or without thrombopoietin receptor agonists: a double-center retrospective study

ABSTRACT

Background

Porcine antilymphocyte globulin (p-ALG) combined with cyclosporine (CsA) has been commonly used for severe aplastic anemia (SAA) patients, but few studies on the combination of p-ALG and thrombopoietin receptor agonist (TPO-RA).

Research design and methods

We retrospectively analyzed the data of 85 people with diagnosed SAA who underwent p-ALG plus CsA, with or without TPO-RA from 2014 to 2023.

Results

The overall response rates were 55.3% and 65.9% at 3 and 6 months, and the TPO-RA group were 66.7% and 72.3% at 3 and 6 months, without TPO-RA group were 27.8% and 55.6%. In multivariate analysis, baseline platelet count of > 10 × 10⁹/L was a simple predictor of favorable response at 6 months (p = 0.015). The median follow-up time for all patients was 39 months (range 0.4 ~ 104), the 5-year overall survival (OS) rate was 90.6% [95% CI = 82.1–95.2%], and the failure-free survival (FFS) rate was 68.9% [95% CI = 56.6–78.4%]. Having hematologic responses in 6 months was an independent positive predictor for FFS (p = 0.000). Twelve patients (14.1%) suffered from serum sickness, and 9.5% of patients had mild hepatic impairment.

Conclusions

p-ALG along with CsA is an effective choice for patients with SAA. p-ALG combined with TPO-RA may contribute to the early restoration of hematopoiesis.

KEYWORDS:

• Severe aplastic anemia
• porcine antilymphocyte globulin
• long-term outcomes
• thrombopoietin receptor agonist
• predictor factor

Declaration of interests

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Author contributions

Conceptualization was performed by M Deng, S Xu*, and Y Xiao*. Investigation was performed by S Xu*, Y Xiao*, and Y Lu. Formal analysis was performed by S Xu*. Supervision was performed by M Deng. Raw data was provided by M Deng and X Liang. Writing, reviewing, and editing manuscript was performed by all authors. Approval of the final manuscript was performed by all authors.

*These authors contributed equally to this work and should be considered co-first authors.

Ethical approval

The ethical council of the Second Xiangya Hospital accepted this study. The Ethics Committee has approved the application for exemption from informed consent.

Acknowledgments

The authors wish to thank Huan Yuan for her contribution of re-collecting the data on the size of the paroxysmal nocturnal hemoglobinuria clone in all patients during the revisions of this article, and for performing the relevant statistical analyses.

Data availability statement

The data that support the findings of this study are available on request from the corresponding author, M Deng, upon reasonable request.

Supplementary material

Supplemental data for this article can be accessed online at https://doi.org/10.1080/17474086.2024.2350527

Additional information

Funding

This paper was not funded.