血清中雷帕霉素的水平可预测巴塞杜氏眶病对西罗莫司的反应。

IF 5.4 2区 医学 Q1 Medicine
Simone Comi, Giada Cosentino, Elena Sabini, Dalì Antonia Ciampa, Francesca Menconi, Maria Novella Maglionico, Chiara Posarelli, Francesco Latrofa, Roberto Rocchi, Michele Figus, Ferruccio Santini, Michele Marinò
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引用次数: 0

摘要

目的:与甲基强的松龙相比,西罗莫司(雷帕霉素)治疗12周的患者在24周时对Graves眼病(GO)的反应更大。我们研究了第12周的血清西罗莫司水平是否能预测第24周的氧化石墨烯结局。方法:对30例患者进行回顾性调查[男4例;女性:26;年龄61.3(9.62)岁,患有中度至重度活性氧化GO,接受西罗莫司治疗(第一天2mg,随后每日0.5 mg,持续12周)。主要结局:第24周,第12周的血清西罗莫司反应者与无反应者,通过综合评价进行评估。次要结局:建立第12周西罗莫司浓度的临界值,预测治疗反应。结果:在第24周,总的氧化石墨烯反应者和无反应者分别为18人(60%)和12人(40%)。第24周应答者12周血清西罗莫司较高[4.6 (2.2)ng/mL比无应答者2.8 (1.6)ng/mL;平均差1.7;95% CI从0.1到3.1;p = 0.034)]。第12周血清西罗莫司的临界值为3.5 ng/mL;≥3.5 ng/mL的水平在第24周的氧化石墨烯应答者中比在无应答者中更常见[13/18 (72.2%)vs 3/12 (25%);OR: 7.8;95% CI从1.4到41.2;P = 0.015],阳性预测值为81.2% (95% CI为60.9 ~ 92.3),敏感性为72.2% (95% CI为46.5 ~ 90.3),特异性为75% (95% CI为42.8 ~ 94.5)。结论:第12周的血清西罗莫司水平可预测第24周的氧化石墨烯治疗反应,提示与氧化石墨烯结局存在剂量依赖关系。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Serum levels of rapamycin predict the response of Graves' orbitopathy to sirolimus.

Objective: Compared with methylprednisolone, a greater response of Graves' orbitopathy (GO) at 24 weeks was reported in patients treated with sirolimus (rapamycin) for 12 weeks. We investigated whether serum levels of sirolimus at week-12 predict the outcome of GO at week-24.

Methods: Retrospective investigation in 30 patients [males: 4; females: 26; age 61.3 (9.62) yr] with moderate-to-severe, active GO, treated with sirolimus (2 mg on day-one, followed by 0.5 mg QD for 12 weeks).

Primary outcome: serum sirolimus at week-12 in week-24 GO responders vs nonresponders, assessed by a composite evaluation.

Secondary outcome: establishment of a cut-off value of week-12 sirolimus concentrations predictive of response to treatment.

Results: At week-24, overall GO responders and nonresponders were 18 (60%) and 12 (40%), respectively. Week-12 serum sirolimus was higher in week-24 responders [4.6 (2.2) ng/mL vs 2.8 (1.6) ng/mL in nonresponders; mean difference 1.7; 95% CI from 0.1 to 3.1; P = 0.034)]. A cut-off value of week-12 serum sirolimus was established at 3.5 ng/mL; levels ≥ 3.5 ng/mL were more frequent in week-24 GO responders than in nonresponders [13/18 (72.2%) vs 3/12 (25%); OR: 7.8; 95% CI from 1.4 to 41.2; P = 0.015], with a positive predictive value of 81.2% (95% CI from 60.9 to 92.3), a sensitivity of 72.2% (95% CI from 46.5 to 90.3), and a specificity of 75% (95% CI from 42.8 to 94.5).

Conclusions: Serum levels of sirolimus at week-12 are predictive of GO response to treatment at week-24, suggesting a dose-dependent relationship with GO outcome.

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来源期刊
Journal of Endocrinological Investigation
Journal of Endocrinological Investigation ENDOCRINOLOGY & METABOLISM-
CiteScore
8.10
自引率
7.40%
发文量
242
期刊介绍: The Journal of Endocrinological Investigation is a well-established, e-only endocrine journal founded 36 years ago in 1978. It is the official journal of the Italian Society of Endocrinology (SIE), established in 1964. Other Italian societies in the endocrinology and metabolism field are affiliated to the journal: Italian Society of Andrology and Sexual Medicine, Italian Society of Obesity, Italian Society of Pediatric Endocrinology and Diabetology, Clinical Endocrinologists’ Association, Thyroid Association, Endocrine Surgical Units Association, Italian Society of Pharmacology.
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