Use of sirolimus in the treatment of lymphangioleiomyomatosis: favorable responses in patients with different extrapulmonary manifestations
Autor(a) principal: | |
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Data de Publicação: | 2015 |
Outros Autores: | , , , , |
Tipo de documento: | Relatório |
Idioma: | eng |
Título da fonte: | Jornal Brasileiro de Pneumologia (Online) |
Texto Completo: | http://old.scielo.br/scielo.php?script=sci_arttext&pid=S1806-37132015000300275 |
Resumo: | OBJECTIVE: 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). METHODS: We describe four cases of LAM patients with different extrapulmonary manifestations who were treated with sirolimus. RESULTS: 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. CONCLUSIONS: 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. |
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Jornal Brasileiro de Pneumologia (Online) |
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Use of sirolimus in the treatment of lymphangioleiomyomatosis: favorable responses in patients with different extrapulmonary manifestationsNeoplasmsLymphangioleiomyomatosis/therapyTOR serine-threonine kinasesSirolimus OBJECTIVE: 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). METHODS: We describe four cases of LAM patients with different extrapulmonary manifestations who were treated with sirolimus. RESULTS: 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. CONCLUSIONS: 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. Sociedade Brasileira de Pneumologia e Tisiologia2015-06-01info:eu-repo/semantics/reportinfo:eu-repo/semantics/publishedVersiontext/htmlhttp://old.scielo.br/scielo.php?script=sci_arttext&pid=S1806-37132015000300275Jornal Brasileiro de Pneumologia v.41 n.3 2015reponame:Jornal Brasileiro de Pneumologia (Online)instname:Sociedade Brasileira de Pneumologia e Tisiologia (SBPT)instacron:SBPT10.1590/S1806-37132015000004553info:eu-repo/semantics/openAccessFreitas,Carolina Salim GonçalvesBaldi,Bruno GuedesAraújo,Mariana SponholzHeiden,Glaucia ItamaroKairalla,Ronaldo AdibCarvalho,Carlos Roberto Ribeiroeng2015-07-06T00:00:00Zoai:scielo:S1806-37132015000300275Revistahttp://www.jornaldepneumologia.com.br/default.aspONGhttps://old.scielo.br/oai/scielo-oai.php||jbp@jbp.org.br|| jpneumo@jornaldepneumologia.com.br1806-37561806-3713opendoar:2015-07-06T00:00Jornal Brasileiro de Pneumologia (Online) - Sociedade Brasileira de Pneumologia e Tisiologia (SBPT)false |
dc.title.none.fl_str_mv |
Use of sirolimus in the treatment of lymphangioleiomyomatosis: favorable responses in patients with different extrapulmonary manifestations |
title |
Use of sirolimus in the treatment of lymphangioleiomyomatosis: favorable responses in patients with different extrapulmonary manifestations |
spellingShingle |
Use of sirolimus in the treatment of lymphangioleiomyomatosis: favorable responses in patients with different extrapulmonary manifestations Freitas,Carolina Salim Gonçalves Neoplasms Lymphangioleiomyomatosis/therapy TOR serine-threonine kinases Sirolimus |
title_short |
Use of sirolimus in the treatment of lymphangioleiomyomatosis: favorable responses in patients with different extrapulmonary manifestations |
title_full |
Use of sirolimus in the treatment of lymphangioleiomyomatosis: favorable responses in patients with different extrapulmonary manifestations |
title_fullStr |
Use of sirolimus in the treatment of lymphangioleiomyomatosis: favorable responses in patients with different extrapulmonary manifestations |
title_full_unstemmed |
Use of sirolimus in the treatment of lymphangioleiomyomatosis: favorable responses in patients with different extrapulmonary manifestations |
title_sort |
Use of sirolimus in the treatment of lymphangioleiomyomatosis: favorable responses in patients with different extrapulmonary manifestations |
author |
Freitas,Carolina Salim Gonçalves |
author_facet |
Freitas,Carolina Salim Gonçalves Baldi,Bruno Guedes Araújo,Mariana Sponholz Heiden,Glaucia Itamaro Kairalla,Ronaldo Adib Carvalho,Carlos Roberto Ribeiro |
author_role |
author |
author2 |
Baldi,Bruno Guedes Araújo,Mariana Sponholz Heiden,Glaucia Itamaro Kairalla,Ronaldo Adib Carvalho,Carlos Roberto Ribeiro |
author2_role |
author author author author author |
dc.contributor.author.fl_str_mv |
Freitas,Carolina Salim Gonçalves Baldi,Bruno Guedes Araújo,Mariana Sponholz Heiden,Glaucia Itamaro Kairalla,Ronaldo Adib Carvalho,Carlos Roberto Ribeiro |
dc.subject.por.fl_str_mv |
Neoplasms Lymphangioleiomyomatosis/therapy TOR serine-threonine kinases Sirolimus |
topic |
Neoplasms Lymphangioleiomyomatosis/therapy TOR serine-threonine kinases Sirolimus |
description |
OBJECTIVE: 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). METHODS: We describe four cases of LAM patients with different extrapulmonary manifestations who were treated with sirolimus. RESULTS: 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. CONCLUSIONS: 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. |
publishDate |
2015 |
dc.date.none.fl_str_mv |
2015-06-01 |
dc.type.driver.fl_str_mv |
info:eu-repo/semantics/report |
dc.type.status.fl_str_mv |
info:eu-repo/semantics/publishedVersion |
format |
report |
status_str |
publishedVersion |
dc.identifier.uri.fl_str_mv |
http://old.scielo.br/scielo.php?script=sci_arttext&pid=S1806-37132015000300275 |
url |
http://old.scielo.br/scielo.php?script=sci_arttext&pid=S1806-37132015000300275 |
dc.language.iso.fl_str_mv |
eng |
language |
eng |
dc.relation.none.fl_str_mv |
10.1590/S1806-37132015000004553 |
dc.rights.driver.fl_str_mv |
info:eu-repo/semantics/openAccess |
eu_rights_str_mv |
openAccess |
dc.format.none.fl_str_mv |
text/html |
dc.publisher.none.fl_str_mv |
Sociedade Brasileira de Pneumologia e Tisiologia |
publisher.none.fl_str_mv |
Sociedade Brasileira de Pneumologia e Tisiologia |
dc.source.none.fl_str_mv |
Jornal Brasileiro de Pneumologia v.41 n.3 2015 reponame:Jornal Brasileiro de Pneumologia (Online) instname:Sociedade Brasileira de Pneumologia e Tisiologia (SBPT) instacron:SBPT |
instname_str |
Sociedade Brasileira de Pneumologia e Tisiologia (SBPT) |
instacron_str |
SBPT |
institution |
SBPT |
reponame_str |
Jornal Brasileiro de Pneumologia (Online) |
collection |
Jornal Brasileiro de Pneumologia (Online) |
repository.name.fl_str_mv |
Jornal Brasileiro de Pneumologia (Online) - Sociedade Brasileira de Pneumologia e Tisiologia (SBPT) |
repository.mail.fl_str_mv |
||jbp@jbp.org.br|| jpneumo@jornaldepneumologia.com.br |
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1750318346307371008 |