Clinical Activity of Single-Agent Cabozantinib (XL184), a Multi-receptor Tyrosine Kinase Inhibitor, in Patients with Refractory Soft-Tissue Sarcomas.


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 01 2022
Historique:
received: 06 07 2021
revised: 17 09 2021
accepted: 26 10 2021
pubmed: 31 10 2021
medline: 19 2 2022
entrez: 30 10 2021
Statut: ppublish

Résumé

Soft-tissue sarcomas (STS) are a rare, heterogeneous group of mesenchymal tumors. For decades the mainstay of treatment for advanced, unresectable STS has been palliative chemotherapy. High levels of activated MET receptor have been reported in various sarcoma cell lines, together with elevated vascular endothelial growth factor (VEGF) levels in patients with STS, suggesting that dual targeting of the VEGF and MET pathways with the multi-receptor tyrosine kinase inhibitor cabozantinib would result in clinical benefit in this population. We performed an open-label, multi-institution, single-arm phase II trial of single-agent cabozantinib in adult patients with advanced STS and progressive disease after at least 1 standard line of systemic therapy. Patients received 60 mg oral cabozantinib once daily in 28-day cycles, and dual primary endpoints of overall response rate and 6-month progression-free survival (PFS) were assessed. Changes in several circulating biomarkers were assessed as secondary endpoints. Six (11.1%; 95% CI, 4.2%-22.6%) of the 54 evaluable patients enrolled experienced objective responses (all partial responses). Six-month PFS was 49.3% (95% CI, 36.2%-67.3%), with a median time on study of 4 cycles (range, 1-99). The most common grade 3/4 adverse events were hypertension (7.4%) and neutropenia (16.7%). Patients' levels of circulating hepatocyte growth factor (HGF), soluble MET, and VEGF-A generally increased after a cycle of therapy, while soluble VEGFR2 levels decreased, regardless of clinical outcome. Cabozantinib single-agent antitumor activity was observed in patients with selected STS histologic subtypes (alveolar soft-part sarcoma, undifferentiated pleomorphic sarcoma, extraskeletal myxoid chondrosarcoma, and leiomyosarcoma) highlighting the biomolecular diversity of STS.

Identifiants

pubmed: 34716194
pii: 1078-0432.CCR-21-2480
doi: 10.1158/1078-0432.CCR-21-2480
pmc: PMC8776602
mid: NIHMS1754275
doi:

Substances chimiques

Anilides 0
Protein Kinase Inhibitors 0
Pyridines 0
Vascular Endothelial Growth Factor A 0
cabozantinib 1C39JW444G

Types de publication

Journal Article Research Support, U.S. Gov't, P.H.S. Comment

Langues

eng

Sous-ensembles de citation

IM

Pagination

279-288

Subventions

Organisme : NCI NIH HHS
ID : HHSN261201500003C
Pays : United States
Organisme : NCI NIH HHS
ID : HHSN261201500003I
Pays : United States
Organisme : NCI NIH HHS
ID : UM1 CA186717
Pays : United States
Organisme : Intramural NIH HHS
ID : Z99 CA999999
Pays : United States

Commentaires et corrections

Type : CommentOn

Informations de copyright

©2021 The Authors; Published by the American Association for Cancer Research.

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Auteurs

Geraldine O'Sullivan Coyne (G)

Early Clinical Trials Development Program, Division of Cancer Treatment and Diagnosis, NCI, NIH, Bethesda, Maryland.

Shivaani Kummar (S)

Early Clinical Trials Development Program, Division of Cancer Treatment and Diagnosis, NCI, NIH, Bethesda, Maryland.

James Hu (J)

University of Southern California, Los Angeles, California.

Kristen Ganjoo (K)

Stanford Cancer Center, Stanford University, Palo Alto, California.

Warren A Chow (WA)

City of Hope Medical Center, Duarte, California.

Khanh T Do (KT)

Early Clinical Trials Development Program, Division of Cancer Treatment and Diagnosis, NCI, NIH, Bethesda, Maryland.

Jennifer Zlott (J)

Early Clinical Trials Development Program, Division of Cancer Treatment and Diagnosis, NCI, NIH, Bethesda, Maryland.

Ashley Bruns (A)

Early Clinical Trials Development Program, Division of Cancer Treatment and Diagnosis, NCI, NIH, Bethesda, Maryland.

Lawrence Rubinstein (L)

Biometric Research Program, Division of Cancer Treatment and Diagnosis, NCI, NIH, Bethesda, Maryland.

Jared C Foster (JC)

Biometric Research Program, Division of Cancer Treatment and Diagnosis, NCI, NIH, Bethesda, Maryland.

Lamin Juwara (L)

Clinical Monitoring Research Program Directorate, Frederick National Laboratory for Cancer Research, Frederick, Maryland.

Robert Meehan (R)

Early Clinical Trials Development Program, Division of Cancer Treatment and Diagnosis, NCI, NIH, Bethesda, Maryland.

Richard Piekarz (R)

Cancer Therapy Evaluation Program, Division of Cancer Treatment and Diagnosis, NCI, NIH, Bethesda, Maryland.

Howard Streicher (H)

Cancer Therapy Evaluation Program, Division of Cancer Treatment and Diagnosis, NCI, NIH, Bethesda, Maryland.

Elad Sharon (E)

Cancer Therapy Evaluation Program, Division of Cancer Treatment and Diagnosis, NCI, NIH, Bethesda, Maryland.

Naoko Takebe (N)

Early Clinical Trials Development Program, Division of Cancer Treatment and Diagnosis, NCI, NIH, Bethesda, Maryland.

Andrea Regier Voth (AR)

Applied/Developmental Research Directorate, Frederick National Laboratory for Cancer Research, Frederick, Maryland.

Donald Bottaro (D)

Center for Cancer Research, NCI, NIH, Bethesda, Maryland.

Rene Costello (R)

Center for Cancer Research, NCI, NIH, Bethesda, Maryland.

John J Wright (JJ)

Cancer Therapy Evaluation Program, Division of Cancer Treatment and Diagnosis, NCI, NIH, Bethesda, Maryland.

James H Doroshow (JH)

Center for Cancer Research, NCI, NIH, Bethesda, Maryland.
Division of Cancer Treatment and Diagnosis, NCI, NIH, Bethesda, Maryland.

Alice P Chen (AP)

Early Clinical Trials Development Program, Division of Cancer Treatment and Diagnosis, NCI, NIH, Bethesda, Maryland. chenali@mail.nih.gov.

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