Treatment patterns and clinical outcomes with pazopanib in patients with advanced soft tissue sarcomas in a compassionate use setting: results of the SPIRE study

Gelderblom, Hans; Judson, Ian; Benson, Charlotte; Merimsky, Ofer; Grignani, Giovanni; Katz, Daniela; Freivogel, Klaus; Stein, Dara; Jobanputra, Minesh; Mungul, Arron; Manson, Stephanie; Sanfilippo, Roberta

doi:10.1080/0284186x.2017.1332779

Cited by 34 publications

(30 citation statements)

References 25 publications

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“…However, as mainstream care for patients with non-adipocytic sarcomas, the use of pazopanib had induced several grade ≥3 toxicities including neutropenia, thrombocytopenia, pulmonary embolism, and nausea. 26 , 27 Rapid and fatal acute heart failure had been even reported which induced by pazopanib. 35 The quality of life was significantly decreased in SS patients who administrated pazopanib because of AEs (ie, diarrhea, anorexia, nausea, vomiting, and asthenia).…”

Section: Discussionmentioning

confidence: 99%

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<p>The Efficacy and Safety of Apatinib in Advanced Synovial Sarcoma: A Case Series of Twenty-One Patients in One Single Institution</p>

Wang

Zhou

et al. 2020

CMAR

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Background: Synovial sarcoma (SS) is a highly aggressive soft-tissue sarcoma (STS) with poor prognosis. Tyrosine kinase inhibitor (TKI) has shown a promising impact on advanced STS patients. This study aimed to evaluate the efficacy and safety of apatinib, an oral multi-TKI, which especially inhibited vascular endothelial growth factor receptor, as second-line therapy for patients with advanced SS. Patients and Methods: This retrospective analysis included 21 advanced SS patients, who had a poor response to anthracycline-based chemotherapy alone or combined with ifosfamide at least one cycle. All the patients received an apatinib containing regimen between May 2016 and October 2019 in our institution. Apatinib 500-750 mg (250 mg for patients younger than 10) was given daily. Tumor responses were assessed by response evaluation criteria in solid tumors. Survival analysis was performed by the Kaplan-Meier test, and a safety profile was recorded. Results: The median follow-up was 15.2 months (95% CI, 12.2-NE). Nine (42.9%) patients had partial response (PR), and eight (38.1%) had stable disease. The median progression-free survival (PFS) was 13.1 months (95% CI, 6.7-NE). The 6-and 12-month PFS rates were 76.2% (95% CI, 60.0-96.8) and 55.4% (95% CI, 37.3-82.3), respectively. Additionally, the median overall survival (OS) was 15.5 months (95% CI, 10.7-NE). The 6-and 12-month OS rates were 81.0% (95% CI, 65.8, 99.6) and 64.9% (95% CI, 46.9-90.0), respectively. Moreover, the objective response rate was 42.9% (9/21) for advanced SS patients. The disease control rate was 81.0% (17/21). For the nine patients with the best response of PR, the median duration of response was 7.7 months. Conclusion: Apatinib was proved to be a potential second-line treatment option for advanced SS patients with chemo-resistance. Apatinib showed promising efficacy and acceptable safety profile in advanced SS, with considerable OS and particularly PFS. Indeed, further multicenter studies with a longer follow-up time are needed to fully determine the clinical application of apatinib in advanced SS.

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Section: Discussionmentioning

confidence: 99%

“…Median PFS=161d Van der Graaf, W. T. et al [15] 2012 H. et al [26] 2017 Jee Hung Kim et al [27] 2019 [3] 2017 13 Yes Regorafenib vs Placebo -Median PFS=4.0m;…”

Section: Dovepressmentioning

confidence: 99%

<p>The Efficacy and Safety of Apatinib in Advanced Synovial Sarcoma: A Case Series of Twenty-One Patients in One Single Institution</p>

Wang

Zhou

et al. 2020

CMAR

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“…Beyond that, pazopanib, a tyrosine kinase inhibitor, has been used to treat PEComa, and exhibits certain effects with PFS from 0.9 to 9.6 months. [ 1 , 17 , 18 ] In terms of the anti-tumor outcomes of nano-rapamycin, a multicenter phase II clinical trial on the use of ABI-009 (Nab-rapamycin) for PEComa (NO. NCT02494570) is ongoing and the results are highly anticipated.…”

Section: Discussionmentioning

confidence: 99%

Clinical features of 18 perivascular epithelioid cell tumor cases

Jia

Jiang

Zhou³

et al. 2020

Medicine

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To investigate the biological behavior and clinical characteristics of perivascular epithelioid cell tumor (PEComa). Eighteen PEComa patients admitted to Zhongshan Hospital and the Central Hospital of Xuhui District in China from January 2006 to October 2018 were included. All patients were diagnosed based on pathological findings and treated with surgical resection or medication. Among the 18 patients, 1 underwent lymph node biopsy for multiple enlarged lymph nodes and 17 underwent mass resection. The median disease-free survival was 22 months after the first resection and over 12 months following a second resection. Treatment with mechanistic target of rapamycin (mTOR) inhibitors was effective for patients with unresectable or metastatic lesions. The median progression-free survival was approximately 13 months. Surgery is the predominant treatment approach for PEComa and patients can benefit from multiple operations. mTOR inhibitors are considered for patients with multiple lesions or intolerance to surgery. Anti-angiogenetic drugs can be selected when mTOR inhibitors fail to control the illness.

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“…In line with this, a case was reported recently when treatment with an ID of one patient was approved by the FDA based merely on data from preclinical studies; the treatment was performed at a leading medical center and the results were published by a top-tier medical journal [ 30 ]. However, large programs involving hundreds or thousands of patients have been begun mostly after the completion of Phase 3 trials [ 31 , 32 ].…”

Section: Main Textmentioning

confidence: 99%

Ethics framework for treatment use of investigational drugs

Borysowski

Górski

2020

BMC Med Ethics

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Background Expanded access is the use of investigational drugs (IDs) outside of clinical trials. Generally it is performed in patients with serious and life-threatening diseases who cannot be treated satisfactorily with authorized drugs. Legal regulations of expanded access to IDs have been introduced among others in the USA, the European Union (EU), Canada and Australia. In addition, in the USA an alternative to expanded access is treatment under the Right-to-Try law. However, the treatment use of IDs is inherently associated with a number of ethically relevant problems. Main text The objective of this article is to present a coherent framework made up of eight requirements which have to be met for any treatment use of an ID to be ethical. These include a justified need for the use of an ID, no threat to clinical development of the ID, adequate scientific evidence to support the treatment, patient’s benefit as the primary goal of the use of an ID, informed decision of a patient, fair access of patients to IDs, independent review, as well as the dissemination of treatment results. Conclusions While this framework is essentially consistent with the legal regulations of expanded access of the USA, the EU, Canada and Australia, it is substantially wider in scope because it addresses some important issues that are not covered by the regulations. Overall, the framework that we developed minimizes the risks and threats, and maximizes potential benefits to each of the four key stakeholders involved in the treatment use of IDs including patients, doctors, drug manufacturers, and society at large.

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Treatment patterns and clinical outcomes with pazopanib in patients with advanced soft tissue sarcomas in a compassionate use setting: results of the SPIRE study

Abstract: The SPIRE study demonstrated activity of pazopanib in heavily pretreated aSTS patients in a compassionate use setting. No new safety concerns were noted. Reassuringly, the relative dose intensity of pazopanib was 92%.

Cited by 34 publications

References 25 publications

<p>The Efficacy and Safety of Apatinib in Advanced Synovial Sarcoma: A Case Series of Twenty-One Patients in One Single Institution</p>

<p>The Efficacy and Safety of Apatinib in Advanced Synovial Sarcoma: A Case Series of Twenty-One Patients in One Single Institution</p>

Clinical features of 18 perivascular epithelioid cell tumor cases

Ethics framework for treatment use of investigational drugs

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