时间标签：数据日期 2026-10-31 · 生成时间 2026-11-01 05:30（Asia/Shanghai）｜ 检索窗口 PubMed edat 2026/10/28–2026/10/31 ｜ 情报发生日期 2026-10-31
研究问题
(, ) Other off-label approaches, including ruxolitinib, thalidomide, cyclosporine A, and anakinra, have also been used, but evidence remains sparse.；Real-world experience with sirolimus in TAFRO-enriched populations, particularly among Japanese patients, remains limited.
方法
Use of sirolimus was approved by the institutional off-label drug-use committee.；The study was approved by the institutional review board of Nagasaki University (approval number 22031401), and all participants provided written informed consent.；Castleman disease (CD) was diagnosed according to Japanese diagnostic criteria, () and TAFRO syndrome according to the updated Masaki 2019 criteria.；() All patients classified as iMCD-TAFRO or as TAFRO syndrome without iMCD fulfilled these diagnostic criteria at the time of diagnosis; the clinical data presented in this report describe the patients at sirolimus initiation, at which point individual features had in some patients already been modified by prior therapy.
核心结论
Among the five iMCD-TAFRO patients with active disease, 4 of 5 (80%; 95% CI 28–99; Cases 4, 6, 7, and 8) met the prespecified response criterion at 3 months.；At 12 months, 3 of 4 evaluable patients (75%; 95% CI 19–99; Cases 4, 6, and 7) remained responders.；In the broader TAFRO-phenotype group (n=6, including Case 3), response rates were 5 of 6 (83%; 95% CI 36–100) at 3 months and 4 of 5 evaluable patients (80%; 95% CI 28–99) at 12 months. ‖ 结论：In conclusion, in this small TAFRO-enriched series, add-on sirolimus was associated with clinical improvement in refractory iMCD-TAFRO, including in patients with severe TAFRO manifestations, and with treatment-sparing outcomes such as glucocorticoid withdrawal, extension of biologic dosing intervals, and discontinuation of hemodialysis, which were sustained after sirolimus was stopped.
局限
Because the present analysis was limited to a single patient without iMCD, and because the on-treatment sample was obtained while the patient was also receiving tocilizumab and prednisolone — so that the transcriptional differences reflect the combined regimen and clinical recovery rather than sirolimus alone — the findings remain preliminary and hypothesis-generating for the broader TAFRO-phenotype spectrum, including iMCD-TAFRO, and larger transcriptional studies are needed.；First, it was a small, single-center observational study with heterogeneous concomitant treatments, and with five and six patients in the efficacy analyses the confidence intervals around the response proportions are v
对本项目的价值
★☆☆☆☆ 方法学进展决定我们能否在有限样本下检出真实效应，属基础设施级收益。设计 single-cell。
全文来源
PMC JATS 全文（正文 47,162 字符，已过标题/DOI 逐字归属校验）
原文题录
- 英文标题：Add-on sirolimus in refractory iMCD-TAFRO: a TAFRO-enriched 8-patient case series with paired single-cell profiling.
- 期刊与标识：Front Immunol · PMID 42964130 · DOI 10.3389/fimmu.2026.1963440
A 级证据分级：A 级（同行评议人体研究）
本文属人体研究证据，但需注意：观察性或小样本干预的结论不等于已证实临床获益；效应量与长期安全性仍需更大规模、更长随访的试验验证。
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