Use of sirolimus in the treatment of lymphangioleiomyomatosis: favorable responses in patients with different extrapulmonary manifestations.
Use of sirolimus in the treatment of lymphangioleiomyomatosis: favorable responses in patients with different extrapulmonary manifestations.
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DOI：
10.1590/s1806-37132015000004553
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发表时间：
2015-05
期刊：
影响因子：
--
通讯作者：
Carvalho CR
中科院分区：
文献类型：
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作者：
Freitas CS;Baldi BG;Araújo MS;Heiden GI;Kairalla RA;Carvalho CR
淋巴管平滑肌瘤病（LAM）是一种罕见的疾病，目前被认为是一种具有转移潜力和可变进展的低级别肿瘤。哺乳动物靶向雷帕霉素（mTOR）抑制剂，如西罗莫司和依维莫司，最近已成为LAM患者的治疗选择，特别是那些有肺外表现的患者。本研究的目的是描述巴西4例LAM患者的病例系列，这些患者在接受西罗莫司（1-4 mg/天）治疗后表现出显著改善，特别是肺外表现。我们描述了4例具有不同肺外表现的LAM患者使用西罗莫司治疗。西罗莫司治疗12个月后，1例患者出现严重乳糜胸消退；一组肾血管平滑肌脂肪瘤体积显著减少；一例腹膜后淋巴管平滑肌瘤明显消退，腹部淋巴结肿大。在西罗莫司治疗6个月后，剩下的患者腹膜后巨大淋巴管平滑肌瘤的体积显著减少。我们的研究结果证实，mTOR抑制剂对LAM患者是有益的，特别是那些有肺外表现的患者，如肾血管平滑肌脂肪瘤、淋巴管平滑肌瘤和乳糜积液。然而，某些方面，如最佳剂量、治疗持续时间和长期不良反应，尚未得到充分澄清，因此mTOR抑制剂尚未纳入LAM管理方案。
Lymphangioleiomyomatosis (LAM) is a rare disease that is currently considered a low-grade neoplasm with metastatic potential and variable progression. Mammalian target of rapamycin (mTOR) inhibitors, such as sirolimus and everolimus, have recently become a treatment option for LAM patients, especially those with extrapulmonary manifestations. The objective of the present study was to describe a case series of four patients with LAM in Brazil who showed significant improvement, particularly in their extrapulmonary manifestations, after treatment with sirolimus (at 1-4 mg/day). We describe four cases of LAM patients with different extrapulmonary manifestations who were treated with sirolimus. After treatment with sirolimus for 12 months, one patient presented resolution of severe chylothorax; one had a significant reduction in renal angiomyolipoma volume; and one showed significant regression of retroperitoneal lymphangioleiomyomas and abdominal lymph node enlargement. After treatment with sirolimus for 6 months, the remaining patient had a significant reduction in the volume of a massive retroperitoneal lymphangioleiomyoma. Our findings confirm that mTOR inhibitors are beneficial for patients with LAM, especially those with extrapulmonary manifestations, such as renal angiomyolipoma, lymphangioleiomyomas, and chylous effusions. However, certain aspects, such as the optimal dose, duration of treatment, and long-term adverse effects, have yet to be sufficiently clarified for mTOR inhibitors to be incorporated into LAM management protocols.