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Open AccessJournal ArticleDOI

Phase II Trial of the CDK4 Inhibitor PD0332991 in Patients With Advanced CDK4-Amplified Well-Differentiated or Dedifferentiated Liposarcoma

TLDR
Treatment with theCDK4 inhibitor PD0332991 was associated with a favorable progression-free rate in patients with CDK4-amplified and RB-expressing WDLS/DDLS who had progressive disease despite systemic therapy.
Abstract
Purpose CDK4 is amplified in 90% of well-differentiated (WDLS) and dedifferentiated liposarcomas (DDLS). The selective cyclin-dependent kinase 4 (CDK4)/CDK6 inhibitor PD0332991 inhibits growth and induces senescence in cell lines and xenografts. In a phase I trial of PD0332991, several patients with WDLS or DDLS experienced prolonged stable disease. We performed an open-label phase II study to determine the safety and efficacy of PD0332991 in patients with advanced WDLS/DDLS.

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Soft Tissue Sarcomas

Journal ArticleDOI

Systemic Treatment of Liposarcomas: Light at the End of the Tunnel?

TL;DR: The only curative option for soft tissue sarcomas is surgical excision with clear margins, and the quality of resection is an important prognostic factor for survival, especially in retroperitoneal liposarcoma.
Journal ArticleDOI

The Next Frontier in Sarcoma: Molecular Pathways and Associated Targeted Therapies

Ted T. Kim, +1 more
- 01 Mar 2023 - 
TL;DR: A comprehensive review of targeted therapies for soft tissue sarcoma can be found in this article , which discusses the current understanding of tumorigenesis of specific STS subtypes, including oncogenic pathway alterations involved in cell cycle regulation, angiogenesis, NOTCH signaling, and aberrant genetic rearrangements.
Journal ArticleDOI

Less Can Be More for Gene Dose and Drug Sensitivity

TL;DR: In this paper, CDK4 was preclinically validated as a therapeutic target in PAX3-FOXO1 fusion gene-positive rhabdomyosarcomas.
Journal ArticleDOI

Personalised pharmacotherapy options for soft tissue sarcomas

TL;DR: In this paper , the authors report updated data on precision therapy in patients with soft tissue sarcomas, particularly those of immediate utility in clinical practice, and provide a high unmet need for effective systemic therapy for patients with STS.
References
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Book

Statistical methods for rates and proportions

TL;DR: In this paper, the basic theory of Maximum Likelihood Estimation (MLE) is used to detect a difference between two different proportions of a given proportion in a single proportion.
Journal ArticleDOI

Statistical Methods for Rates and Proportions.

B. S. Everitt, +1 more
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Journal Article

[New response evaluation criteria in solid tumours-revised RECIST guideline (version 1.1)].

TL;DR: This paper is an overview of the new response evaluation criteria in solid tumours: revised RECIST guideline (version 1. 1), with a focus on updated contents.
Journal Article

Specific inhibition of cyclin-dependent kinase 4/6 by PD 0332991 and associated antitumor activity in human tumor xenografts

TL;DR: Results indicate that inhibition of Cdk4/6 alone is sufficient to cause tumor regression and a net reduction in tumor burden in some tumors.
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