Sirolimus-based treatment regimens for antinuclear antibody (ANA)-positive immune thrombocytopenia: a retrospective single-center cohort study.

IF 2 4区医学 Q3 HEMATOLOGY

Hematology Pub Date : 2026-12-31 Epub Date: 2026-05-25 DOI:10.1080/16078454.2026.2678626

Aoxue Tan, Jia Liu, Xiaoli Chen, Yimei Feng, Xi Zhang

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引用次数: 0

Abstract

Objectives: To evaluate the efficacy and safety of sirolimus-based treatment regimens in patients with antinuclear antibody (ANA)-positive immune thrombocytopenia (ITP).

Methods: This retrospective single-center study enrolled patients with a clear clinical diagnosis of ANA-positive ITP. They were divided into two groups based on different treatment regimens: the sirolimus monotherapy group (SRL group) and the sirolimus plus dexamethasone combination group (combination group).

Results: During treatment, the combination group achieved the initial overall response (ORR) significantly faster than the SRL group (p < 0.05); additionally, in the early treatment phase (Week 1, Week 4), there were statistically significant differences in efficacy between the two groups, while no statistically significant difference in efficacy was observed during subsequent follow-up. Regarding treatment safety, all adverse events were Grade 1-2; no Grade 3 or higher adverse events were reported and no treatment-related deaths occurred. The most frequently observed adverse events were oral ulcers, gastrointestinal distress, and elevated liver enzymes. There was no statistically significant difference in the total incidence of adverse events between the two groups.

Conclusion: In this retrospective single-center study, sirolimus-based regimens showed favorable efficacy and acceptable safety in ANA-positive ITP. Adding dexamethasone to sirolimus led to faster initial responses versus sirolimus alone, though no significant difference was seen in follow-up. These findings support the feasibility and potential efficacy of sirolimus-based regimens in ANA-positive ITP, warranting prospective controlled studies.

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基于西罗莫司的抗核抗体（ANA）阳性免疫性血小板减少症治疗方案：一项回顾性单中心队列研究

目的：评价以西罗莫司为基础的治疗方案对抗核抗体（ANA）阳性免疫性血小板减少症（ITP）患者的疗效和安全性。方法：本回顾性单中心研究纳入临床明确诊断为ana阳性ITP的患者。根据治疗方案的不同分为西罗莫司单药组（SRL组）和西罗莫司加地塞米松联合组（联合组）。结果：在治疗过程中，联合组达到初始总反应（ORR）显著快于SRL组（p < 0.05）；另外，在治疗早期（第1周、第4周），两组疗效比较差异有统计学意义，而在后续随访中，两组疗效比较差异无统计学意义。在治疗安全性方面，所有不良事件均为1-2级；未报告3级或以上不良事件，也未发生与治疗相关的死亡。最常见的不良反应是口腔溃疡、胃肠不适和肝酶升高。两组患者不良事件总发生率无统计学差异。结论：在这项回顾性单中心研究中，西罗莫司为基础的方案在ana阳性ITP中显示出良好的疗效和可接受的安全性。与单独使用西罗莫司相比，在西罗莫司中加入地塞米松可以更快地产生初始反应，但在随访中没有发现显著差异。这些发现支持以西罗莫司为基础的方案治疗ana阳性ITP的可行性和潜在疗效，值得进行前瞻性对照研究。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Hematology 医学-血液学

CiteScore

2.60

自引率

5.30%

发文量

140

审稿时长

3 months

期刊介绍： Hematology is an international journal publishing original and review articles in the field of general hematology, including oncology, pathology, biology, clinical research and epidemiology. Of the fixed sections, annotations are accepted on any general or scientific field: technical annotations covering current laboratory practice in general hematology, blood transfusion and clinical trials, and current clinical practice reviews the consensus driven areas of care and management.