Activity of sirolimus in patients with progressive epithelioid hemangioendothelioma: A case‐series analysis within the Italian Rare Cancer Network

Autor: Francesca Greco, Carlo Morosi, Anna Maria Frezza, Antonella Brunello, Elena Palassini, Salvatore Lo Vullo, Paolo G. Casali, Luigi Mariani, Silvia Stacchiotti, Bruno Vincenzi, Angelo Paolo Dei Tos, Giacomo Giulio Baldi, Marta Sbaraglia, Gianpaolo Dagrada, Paola Collini, Noemi Simeone
Rok vydání: 2020
Předmět:
Male
epitheliod hamangioendothelioma
Cancer Research
sarcoma
medicine.medical_treatment
Kaplan-Meier Estimate
chemotherapy
Gastroenterology
Metastasis
0302 clinical medicine
Interquartile range
hemic and lymphatic diseases
Antineoplastic Combined Chemotherapy Protocols
030212 general & internal medicine
Child
integumentary system
Intracellular Signaling Peptides and Proteins
Middle Aged
Prognosis
epithelioid hemangioendothelioma
Editorial
Italy
Oncology
Response Evaluation Criteria in Solid Tumors
030220 oncology & carcinogenesis
Disease Progression
Hemangioendothelioma
Epithelioid
Female
Sarcoma
medicine.symptom
medicine.drug
Adult
medicine.medical_specialty
03 medical and health sciences
Internal medicine
medicine
metastasis
Humans
Epithelioid hemangioendothelioma
Aged
Sirolimus
Chemotherapy
business.industry
Editorials
medicine.disease
Irregular menstruation
prognosis
serosal effusion
sirolimus
Transcriptional Coactivator with PDZ-Binding Motif Proteins
business
EHE
Zdroj: Cancer
ISSN: 1097-0142
0008-543X
DOI: 10.1002/cncr.33247
Popis: Background The objective of this study was to report on a retrospective series of patients with epithelioid hemangioendothelioma (EHE) who received treatment with sirolimus within the Italian Rare Cancer Network. Methods From January 2005, 38 adult patients with advanced EHE received continuous-dosing sirolimus, 5 mg daily, until they developed either toxicity or disease progression. Disease progression in the 6 months before the start of treatment was required. Each pathologic diagnosis was reviewed. The daily dose of sirolimus was adjusted based on plasma levels. Response was retrospectively assessed by local investigators using Response Evaluation Criteria in Solid Tumors, version 1.1 (RECIST). Survival was estimated using the Kaplan-Meier method. Results All 38 patients (WW Domain Containing Transcription Regulator 1 [WWTR1]-positive, n = 37; transcription factor E3 [TFE3]-positive, n = 1) had disease progression before starting sirolimus (at baseline, 13 of 38 patients had the presence of serosal effusions and systemic symptoms). Thirty-seven patients were evaluable for response (there was 1 early interruption). The best RECIST responses were a partial response in 4 patients (10.8%), stable disease in 28 patients (75.7%), and disease progression in 5 patients (13.5%). At a 41.5-month median follow-up (interquartile range [IQR], 23.9-56.8 months), the median PFS was 13 months (95% CI, 3.7 months to not estimated [NE]), and the median OS was 18.8 months (95% CI, 10.6 months to NE). In patients who had serosal effusions at baseline, the median PFS was 4.8 months (IQR, 3.5-11.7 months), and the median OS was 10.6 months (IQR, 5.1-13.0 months), compared with 47.8 months (IQR, 11.4 months to NE) and 47.8 months (IQR, 15.7 months to NE), respectively, in patients without serosal effusions. Overall, sirolimus was fairly well tolerated, with 10 patients reporting irregular menstruation/ovary disfunction. Conclusions The current results confirm that sirolimus is active in EHE, leading to prolonged stabilization in most patients who present without serosal effusions. Serosal effusions are confirmed as an unfavorable prognostic sign associated with short survival, and sirolimus displays limited activity in this subgroup.
Databáze: OpenAIRE
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