Addition of Antiestrogen Treatment in Patients with Malignant PEComa Progressing to mTOR Inhibitors.
Aged
Estrogen Antagonists
/ administration & dosage
Everolimus
/ administration & dosage
Female
Humans
Kaplan-Meier Estimate
Middle Aged
Perivascular Epithelioid Cell Neoplasms
/ drug therapy
Progression-Free Survival
Sarcoma
/ drug therapy
Signal Transduction
/ drug effects
Sirolimus
/ administration & dosage
TOR Serine-Threonine Kinases
/ antagonists & inhibitors
Triptorelin Pamoate
/ administration & dosage
Journal
Clinical cancer research : an official journal of the American Association for Cancer Research
ISSN: 1557-3265
Titre abrégé: Clin Cancer Res
Pays: United States
ID NLM: 9502500
Informations de publication
Date de publication:
15 10 2020
15 10 2020
Historique:
received:
30
03
2020
revised:
30
04
2020
accepted:
24
06
2020
pubmed:
2
7
2020
medline:
24
11
2021
entrez:
2
7
2020
Statut:
ppublish
Résumé
Perivascular epithelioid cell tumors (PEComa) are rare mesenchymal neoplasms. mTOR inhibitors are the most active agents in PEComa and in patients progressing to mTOR inhibitors, other available therapies have limited benefit. Preclinical evidences showed a cross-talk between the mTOR pathway and estrogen receptor signaling. This provided a rationale for adding an antiestrogen treatment in female patients becoming resistant to mTOR inhibitors. Since April 2018, female patients with advanced/metastatic PEComa progressing to mTOR inhibitors were treated with a combination of sirolimus and exemestane with or without LHRH analogue (based on menopausal status). This case series was retrospectively reviewed. Survival analyses were performed using the Kaplan-Meier method. A total of seven consecutive patients treated with the combination of sirolimus and antiestrogen treatment were retrospectively reviewed. Six (86%) received a combination of sirolimus and exemestane, whereas one patient (14%) received a combination of sirolimus, exemestane, and triptorelin since in premenopausal status. After a median follow-up of 13.1 months, three patients (43%) experienced a partial response, three patients (43%) experienced a stabilization of disease, and one patient (14%) had disease progression with an overall response rate of 43% and a disease control rate of 86%. In this small retrospective case series, the addition of antiestrogen treatment in female patients with advanced PEComa progressing to mTOR inhibitors resulted in a remarkable clinical benefit in a setting where no other options are available.
Identifiants
pubmed: 32605908
pii: 1078-0432.CCR-20-1191
doi: 10.1158/1078-0432.CCR-20-1191
doi:
Substances chimiques
Estrogen Antagonists
0
Triptorelin Pamoate
08AN7WA2G0
Everolimus
9HW64Q8G6G
MTOR protein, human
EC 2.7.1.1
TOR Serine-Threonine Kinases
EC 2.7.11.1
Sirolimus
W36ZG6FT64
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
5534-5538Informations de copyright
©2020 American Association for Cancer Research.
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