Adding cyclosporin A to eltrombopag for aplastic anemia secondary to chemotherapy for solid cancers - study efficacy and safety

Sep 24, 2026

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The article investigates whether adding cyclosporin A to eltrombopag improves outcomes for aplastic anemia occurring after chemotherapy in patients with solid tumors. A retrospective cohort from Peking Union Medical College Hospital evaluates eligibility, dosing strategies, and response definitions including complete and partial responses. Efficacy is reported as overall response rates across multiple timepoints, with treatment duration and subsequent regimen switching for non-responders. Safety and regimen details are referenced through supplementary materials.

LanguageEnglish

CategoryHealthcare

FormatPDF

Pages3

Reading Time8 min

AccessFree to download

Background and objective

Study design and eligibility

Treatment regimens and outcomes

Patient cohorts and response results

Response timing and follow-up

Adding cyclosporin A to eltrombopag for aplastic anemia secondary to chemotherapy for solid cancers Ziqi Wan1,2, Yang Chen1, Junling Zhuang1, Miao Chen1, Shuyang Zhang3, Bing Han1 1 Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Science, Beijing 100730, China; 2 Eight-Year Program of Clinical Medicine, M. D. Program, Department of Clinical Medicine, Peking Union Medical College, Beijing 100005, China; 3 Department of Cardiology, Peking Union Medical College Hospital, Chinese Academy of Medical Science, Beijing 100730, China. To the Editor: Myelosuppression is one of the most common side effects of chemotherapy. Some patients would develop long-lasting aplastic anemia (AA) even after stopping chemotherapy for over three months, due to persistent destruction of the bone marrow. As athrombopoietin-receptor agonist, eltrombopag (EPAG) has been widely used in patients with thrombocytopenia from various origins,[1] including those with AA secondary to chemotherapy. However, both EPAG and cyclosporin A (CsA) have been reported to possibly induce the expansion of abnormal clones or controversially increase the risk of neoplasms. [2] Thus, the challenge was to treat severe cytopenia while preventing cancer relapse. Our study reported the efﬁcacy and safety of EPAG with or without CsA for patients with AA secondary to chemotherapy, in an attempt to ﬁnd solutions to these difﬁculties. This retrospective study was approved by the Ethics Committee of Peking Union Medical College Hospital (PUMCH, No. S-K1976) . Patients were informed of this study and gave their written informed consent to participate in. We reviewed data from patients with solid tumors treated with chemotherapy at PUMCH, who developed AA from January 1, 2018, to November 20, 2021 . Participants were deemed potentially eligible if they had clear diagnosis of solid cancers without previous hematologic malignancies and persistent cytopenia for at least six months after the last chemotherapy (radiation) treatment. The detailed inclusion criteria are shown in the Supplementary materials [[http://links.lww.com/CM9/B453](http://links.lww.com/CM9/B453)]. Data were censored on May 21, 2022 . EPAG was started at25mg/day and increased by25mg/day to the maximum dosage of 150 mg/day. CsA was started at 3to5mg·kg􀀁1·day􀀁1 and adjusted monthly to achieve 100 | Access this article online | | | --- | --- | | Quick Response Code:
| Website:
[www.cmj.org](www.cmj.org) | | | DOI:
10.1097/CM9.0000000000002616 | to 200 ng/mL. The detailed methods of regimens, data collection, follow-ups, response criteria, statistical analysis and adverse events were presented in the Supplementary materials [[http://links.lww.com/CM9/B453](http://links.lww.com/CM9/B453)]. Responses were assessed as complete response (CR), partial response (PR), and no response (NR) .[3] Comparison between groups was conducted using Fisher’s exact test or the Kruskal–Wallis test as appropriate. Sixty-two patients were reviewed. We excluded 17 patients (reasons are shown in the Supplementary materials, [http://](http://)[ ](http://)[links.lww.com/CM9/B453](links.lww.com/CM9/B453) ), and 45 eligible patients were ﬁnally included. Group 1 included 32 patients who received EPAG and CsA simultaneously from the onset, namely EPAG + CsA group. Group 2 included 13 patients who received EPAG alone. The baseline characteristics of the two groups have been summarized in Supplementary Table 1 [[http://links.lww.com/CM9/B453](http://links.lww.com/CM9/B453)], includingthe baseline hemoglobin, neutrophils, and platelets. There were no statistically signiﬁcant differences between patients in two groups at baseline. Group 1 patients were administered the combination regimen for 15 (3–37) months. The overall response rates (ORRs) at 1, 2, 3, and 6 months were 28%(9/32), 56%(18/32), 72%(23/32), and 100%(26/26), corresponding to 9%(3/32), 9%(3/32), 13%(4/32), and 42%(11/

FAQs

Why was cyclosporin A added to eltrombopag in this study?

Because severe cytopenia needed effective treatment, while concerns existed that eltrombopag or cyclosporin A could potentially promote abnormal clones or increase neoplasm risk. The study aimed to balance efficacy and cancer relapse prevention.

How were patients selected for the retrospective analysis?

Patients with solid tumors receiving chemotherapy at PUMCH and developing aplastic anemia were reviewed. Eligibility required clear solid cancer diagnosis without prior hematologic malignancies and persistent cytopenia for at least six months after the last chemotherapy (or radiation) treatment.

What were the response outcomes for the combination versus eltrombopag alone?

The study reports overall response rates at multiple timepoints for the EPAG+CsA group and compares them with the EPAG-alone group. Group 1 (EPAG+CsA) showed increasing response over time, and a median follow-up described an overall response before switching regimens.

Sep 24, 2026

Uploaded by Aurelia