Functional improvements in patients with lymphangioleiomyomatosis after sirolimus: an observational study.
Functional improvements in patients with lymphangioleiomyomatosis after sirolimus: an observational study.
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西罗莫司治疗后淋巴管平滑肌瘤病患者的功能改善：一项观察性研究
DOI：
10.1186/s13023-018-0775-9
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发表时间：
2018-02-20
影响因子：
3.7
通讯作者：
Xu KF
中科院分区：
文献类型：
--
作者：
Zhan Y;Shen L;Xu W;Wu X;Zhang W;Wang J;Li X;Yang Y;Tian X;Xu KF
西罗莫司已被证明对淋巴管平滑肌瘤病 (LAM) 患者有效。我们希望总结我们使用西罗莫司的经验及其对 LAM 患者的有效性。我们分析了北京协和医院 2007 年 1 月至 2015 年 6 月期间接受西罗莫司治疗的 98 例根据 2010 年欧洲呼吸学会 LAM 诊断标准诊断为确诊或疑似散发性 LAM 的患者的数据。开始西罗莫司治疗前后的数据包括肺功能检查、动脉血气分析、6 分钟步行距离（6MWD）、乳糜积液量和肾功能。血管平滑肌脂肪瘤 (AML)、圣乔治呼吸问卷 (SGRQ) 和血管内皮生长因子-D (VEGF-D) 水平。收集西罗莫司的血清水平和不良事件。中位随访时间为 2.5 年。大多数患者的 1 秒用力呼气量 (FEV1) 值低于预测值的 70% 或有症状的乳糜胸。开始服用西罗莫司之前和之后，FEV1 变化的平均变化分别为 - 31.12 ± 30.78 mL/月和 16.11± 36.00 mL/月 (n = 18，p = 0.002)，并且PaO2 变化为 − 0.55 ± 0.60 mmHg/月和 0.30 ± 1.19 mmHg/月 (n = 17，p = 0.018)。 6MWD从358.8 ± 114.4 m提高到415.6 ± 118.6 m（n = 46，p = 0.004），SGRQ总分从57.2 ± 21.0提高到47.5 ± 22.8 (n = 50，p < 0.001)。西罗莫司治疗后，中位 VEGF-D 浓度从 3075.6 pg/mL 降至 1609.4 pg/mL (n = 41，p < 0.001)。西罗莫司谷值水平为 5–9.9 ng/mL 的患者 FEV1 增加 (p<<0.05)。百分之六十五的患者 (13/20) 乳糜积液几乎完全消退。最常见的不良反应是口腔溃疡、月经失调、高脂血症和痤疮样皮疹，均为轻度。 LAM 患者长期使用西罗莫司是安全的。 FEV1 低于预测值 70% 且有症状的乳糜胸的 LAM 患者适合接受西罗莫司治疗。建议将西罗莫司的血清谷浓度维持在 5 至 9.99 ng/mL 之间。
Sirolimus has been shown to be effective in patients with lymphangioleiomyomatosis (LAM). We wish to summarize our experience using sirolimus and its effectiveness in LAM patients. We analyzed data from 98 patients who were diagnosed with definite or probable sporadic LAM based on the European Respiratory Society diagnosis criteria for LAM in 2010 at Peking Union Medical College Hospital and who had received sirolimus during January 2007 to June 2015. The data before and after the initiation of sirolimus therapy included pulmonary function tests, arterial blood gas analysis, 6-min walking distance (6MWD), size of chylous effusion and renal angiomyolipomas (AML), St. George’s Respiratory Questionnaires (SGRQ) and vascular endothelial growth factor-D (VEGF-D) levels. Serum levels of sirolimus and adverse events were collected. Median follow-up was 2.5 years. Most patients had forced expiratory volume in 1 s (FEV1) values less than 70% predicted or symptomatic chylothorax. The mean changes before and after the initiation of sirolimus were − 31.12 ± 30.78 mL/month and 16.11 ± 36.00 mL/month (n = 18,p = 0.002) for FEV1 change, and − 0.55 ± 0.60 mmHg/month and 0.30 ± 1.19 mmHg/month (n = 17, p = 0.018) for PaO2 change. 6MWD improved from 358.8 ± 114.4 m to 415.6 ± 118.6 m (n = 46, p = 0.004) and SGRQ total score from 57.2 ± 21.0 to 47.5 ± 22.8 (n = 50, p < 0.001). The median VEGF-D concentration decreased to 1609.4 pg/mL from 3075.6 pg/mL after sirolimus therapy (n = 41, p < 0.001). Patients with sirolimus trough levels of 5–9.9 ng/mL had an increase in FEV1 (p < 0.05). Sixty-five percent of patients (13/20) had almost complete resolution of chylous effusions. The most frequent adverse events were mouth ulcers, menstrual disorder, hyperlipidemia and acneiform rash, all were mild. Long-term use of sirolimus is safe in patients with LAM. LAM patients with FEV1 less than 70% predicted and symptomatic chylothorax are suitable for receiving sirolimus therapy. The maintaining serum trough levels of sirolimus are recommended between 5 to 9.99 ng/mL.
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DOI：
10.1590/s1806-37132015000004553
发表时间：
2015-05
期刊：
Jornal brasileiro de pneumologia : publicacao oficial da Sociedade Brasileira de Pneumologia e Tisilogia
影响因子：
--
作者：
Freitas CS;Baldi BG;Araújo MS;Heiden GI;Kairalla RA;Carvalho CR
通讯作者：
Carvalho CR
影响因子：
158.5
作者：
Vestbo, Jorgen;Edwards, Lisa D.;Rennard, Stephen I.
通讯作者：
Rennard, Stephen I.
影响因子：
9.6
作者：
Taveira-DaSilva, AM;Stylianou, MP;Moss, J
通讯作者：
Moss, J
DOI：
10.1056/nejmoa1100391
发表时间：
2011-04-28
期刊：
The New England journal of medicine
影响因子：
--
作者：
McCormack FX;Inoue Y;Moss J;Singer LG;Strange C;Nakata K;Barker AF;Chapman JT;Brantly ML;Stocks JM;Brown KK;Lynch JP 3rd;Goldberg HJ;Young LR;Kinder BW;Downey GP;Sullivan EJ;Colby TV;McKay RT;Cohen MM;Korbee L;Taveira-DaSilva AM;Lee HS;Krischer JP;Trapnell BC;National Institutes of Health Rare Lung Diseases Consortium;MILES Trial Group
通讯作者：
MILES Trial Group
影响因子：
0.8
作者：
Ellender, Claire M;Williams, Trevor J;Gooi, Julian;Snell, Gregory I;Whitford, Helen M
通讯作者：
Whitford, Helen M