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Targeting Sporadic and Neurofibromatosis Type 1 (NF1) Related Refractory Malignant Peripheral Nerve Sheath Tumors (MPNST) in a Phase II Study of Everolimus in Combination with Bevacizumab (SARC016)
Joint Authors
Okuno, Scott H.
Milhem, Mohammed
Hirbe, Angela C.
Kim, AeRang
Widemann, Brigitte C.
Lu, Yao
Reinke, Denise
Perentesis, John
Cichowski, Karen
Chugh, Rashmi
Meyer, Christian F.
Cote, Gregory M.
Turpin, Brian
Pressey, Joseph G.
Dombi, Eva
Jayaprakash, Nalini
Helman, Lee J.
Onwudiwe, Ndidi
Source
Issue
Vol. 2019, Issue 2019 (31 Dec. 2019), pp.1-8, 8 p.
Publisher
Hindawi Publishing Corporation
Publication Date
2019-07-24
Country of Publication
Egypt
No. of Pages
8
Main Subjects
Abstract EN
Purpose.
There are no known effective medical treatments for refractory MPNST.
Inactivation of the NF1 tumor suppressor in MPNST results in upregulation of mTOR (mammalian target of rapamycin) signaling and angiogenesis, which contributes to disease progression.
We conducted a phase II study for patients (pts) with refractory MPNST combining everolimus (10 mg PO once daily) with bevacizumab (10 mg/kg IV every 2 weeks) to determine the clinical benefit rate (CBR) (complete response, partial response (PR), or stable disease (SD) ≥ 4 months).
Patients and Methods.
Patients ≥18 years old with chemotherapy refractory sporadic or NF1 MPNST were eligible.
Tumor response was assessed after every 2 cycles (the WHO criteria).
A two-stage design targeting a 25% CBR was used: if ≥ 1/15 pts in stage 1 responded, enrollment would be expanded by 10 pts, and if ≥ 4/25 patients had clinical benefit, the combination would be considered active.
Results.
Twenty-five pts, 17 with NF1 and 8 with sporadic MPNST, enrolled.
One of 15 pts in stage 1 had clinical benefit.
Of 10 additional pts enrolled, 2 had clinical benefit.
The median number of completed cycles was 3 (range 1–16).
Adverse events were similar to those known for this combination.
Conclusion.
With a CBR of 12% (3/25), the combination of everolimus and bevacizumab did not reach the study’s target response rate and is not considered active in refractory MPNST.
American Psychological Association (APA)
Widemann, Brigitte C.& Lu, Yao& Reinke, Denise& Okuno, Scott H.& Meyer, Christian F.& Cote, Gregory M.…[et al.]. 2019. Targeting Sporadic and Neurofibromatosis Type 1 (NF1) Related Refractory Malignant Peripheral Nerve Sheath Tumors (MPNST) in a Phase II Study of Everolimus in Combination with Bevacizumab (SARC016). Complexity،Vol. 2019, no. 2019, pp.1-8.
https://search.emarefa.net/detail/BIM-1207731
Modern Language Association (MLA)
Widemann, Brigitte C.…[et al.]. Targeting Sporadic and Neurofibromatosis Type 1 (NF1) Related Refractory Malignant Peripheral Nerve Sheath Tumors (MPNST) in a Phase II Study of Everolimus in Combination with Bevacizumab (SARC016). Complexity No. 2019 (2019), pp.1-8.
https://search.emarefa.net/detail/BIM-1207731
American Medical Association (AMA)
Widemann, Brigitte C.& Lu, Yao& Reinke, Denise& Okuno, Scott H.& Meyer, Christian F.& Cote, Gregory M.…[et al.]. Targeting Sporadic and Neurofibromatosis Type 1 (NF1) Related Refractory Malignant Peripheral Nerve Sheath Tumors (MPNST) in a Phase II Study of Everolimus in Combination with Bevacizumab (SARC016). Complexity. 2019. Vol. 2019, no. 2019, pp.1-8.
https://search.emarefa.net/detail/BIM-1207731
Data Type
Journal Articles
Language
English
Notes
Includes bibliographical references
Record ID
BIM-1207731