Activity of axitinib in progressive advanced solitary fibrous tumour: Results from an exploratory, investigator-driven phase 2 clinical study

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Abstract

Background: To explore the activity of axitinib in advanced solitary fibrous tumour (SFT). Patients and methods: In this investigator-driven phase II study on axitinib in advanced and progressive SFT, patients received axitinib, 5 mg bis in day (BID), until progression or limiting toxicity. Pathologic diagnosis was centrally reviewed, distinguishing malignant SFT (M-SFT) and high-grade/dedifferentiated SFT (HG/D-SFT) subtypes. The primary end-point was the overall response rate (ORR) by Choi criteria (Choi). Secondary end-points were response by Response Evaluation Criteria in Solid Tumours (RECIST), progression-free survival (PFS) and overall survival (OS). Results: From April 2015 and October 2017, 17 eligible patients entered the study (metastatic: 17; SFT subtype: 13 M-SFT, 4 HG/D-SFT; prior treatment: 9 antiangiogenics, 5 cytotoxics). All patients were evaluable for response. The best Choi response was seven partial response (PR) (ORR, 41.2%), six stable disease (SD) and four progressions. Choi-ORR was 54% (7/13) when only M-SFTs were considered. Four of seven responsive patients were pretreated with pazopanib. No responses were detected in HG/D-SFT. Best RECIST response was one PR (5.9%), 14 SD and two progressions. Toxicity was as expected. Median Choi-PFS was 5.1 (interquartile range [IQR]: 2.5–14.8) months. Median Choi-PFS was 14.8 (IQR: 5.1–18.0) and 2.8 (IQR: 2.0–5.9) months for patients responsive and non-responsive by Choi, respectively (p = 0.0416). At a 14.4-month median follow-up, median OS was 25.3 months. Conclusion: This study showed that axitinib is active in progressive advanced SFT. One-half of patients carrying the malignant variant of the disease responded, with a >12-month median progression arrest. Responses were better detected with Choi and seen even in patients resistant to other antiangiogenics. Tolerability was good.

Original languageEnglish
Pages (from-to)225-233
Number of pages9
JournalEuropean Journal of Cancer
Volume106
DOIs
Publication statusPublished - Jan 2019

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Solitary Fibrous Tumors
Research Personnel
Disease-Free Survival
Disease Progression
Survival
axitinib
Clinical Studies

Keywords

  • Antiangiogenic
  • Axitinib
  • Hemangiopericytoma
  • Sarcoma
  • Solitary fibrous tumour
  • Target therapy

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

Cite this

@article{399d1df6169a4ecc8718c2043712daa3,
title = "Activity of axitinib in progressive advanced solitary fibrous tumour: Results from an exploratory, investigator-driven phase 2 clinical study",
abstract = "Background: To explore the activity of axitinib in advanced solitary fibrous tumour (SFT). Patients and methods: In this investigator-driven phase II study on axitinib in advanced and progressive SFT, patients received axitinib, 5 mg bis in day (BID), until progression or limiting toxicity. Pathologic diagnosis was centrally reviewed, distinguishing malignant SFT (M-SFT) and high-grade/dedifferentiated SFT (HG/D-SFT) subtypes. The primary end-point was the overall response rate (ORR) by Choi criteria (Choi). Secondary end-points were response by Response Evaluation Criteria in Solid Tumours (RECIST), progression-free survival (PFS) and overall survival (OS). Results: From April 2015 and October 2017, 17 eligible patients entered the study (metastatic: 17; SFT subtype: 13 M-SFT, 4 HG/D-SFT; prior treatment: 9 antiangiogenics, 5 cytotoxics). All patients were evaluable for response. The best Choi response was seven partial response (PR) (ORR, 41.2{\%}), six stable disease (SD) and four progressions. Choi-ORR was 54{\%} (7/13) when only M-SFTs were considered. Four of seven responsive patients were pretreated with pazopanib. No responses were detected in HG/D-SFT. Best RECIST response was one PR (5.9{\%}), 14 SD and two progressions. Toxicity was as expected. Median Choi-PFS was 5.1 (interquartile range [IQR]: 2.5–14.8) months. Median Choi-PFS was 14.8 (IQR: 5.1–18.0) and 2.8 (IQR: 2.0–5.9) months for patients responsive and non-responsive by Choi, respectively (p = 0.0416). At a 14.4-month median follow-up, median OS was 25.3 months. Conclusion: This study showed that axitinib is active in progressive advanced SFT. One-half of patients carrying the malignant variant of the disease responded, with a >12-month median progression arrest. Responses were better detected with Choi and seen even in patients resistant to other antiangiogenics. Tolerability was good.",
keywords = "Antiangiogenic, Axitinib, Hemangiopericytoma, Sarcoma, Solitary fibrous tumour, Target therapy",
author = "S. Stacchiotti and N. Simeone and {Lo Vullo}, S. and C. Morosi and Greco, {F. G.} and A. Gronchi and M. Barisella and P. Collini and N. Zaffaroni and Dagrada, {G. P.} and Frezza, {A. M.} and L. Mariani and Casali, {P. G.}",
year = "2019",
month = "1",
doi = "10.1016/j.ejca.2018.10.024",
language = "English",
volume = "106",
pages = "225--233",
journal = "European Journal of Cancer",
issn = "0959-8049",
publisher = "Elsevier Ltd",

}

TY - JOUR

T1 - Activity of axitinib in progressive advanced solitary fibrous tumour

T2 - Results from an exploratory, investigator-driven phase 2 clinical study

AU - Stacchiotti, S.

AU - Simeone, N.

AU - Lo Vullo, S.

AU - Morosi, C.

AU - Greco, F. G.

AU - Gronchi, A.

AU - Barisella, M.

AU - Collini, P.

AU - Zaffaroni, N.

AU - Dagrada, G. P.

AU - Frezza, A. M.

AU - Mariani, L.

AU - Casali, P. G.

PY - 2019/1

Y1 - 2019/1

N2 - Background: To explore the activity of axitinib in advanced solitary fibrous tumour (SFT). Patients and methods: In this investigator-driven phase II study on axitinib in advanced and progressive SFT, patients received axitinib, 5 mg bis in day (BID), until progression or limiting toxicity. Pathologic diagnosis was centrally reviewed, distinguishing malignant SFT (M-SFT) and high-grade/dedifferentiated SFT (HG/D-SFT) subtypes. The primary end-point was the overall response rate (ORR) by Choi criteria (Choi). Secondary end-points were response by Response Evaluation Criteria in Solid Tumours (RECIST), progression-free survival (PFS) and overall survival (OS). Results: From April 2015 and October 2017, 17 eligible patients entered the study (metastatic: 17; SFT subtype: 13 M-SFT, 4 HG/D-SFT; prior treatment: 9 antiangiogenics, 5 cytotoxics). All patients were evaluable for response. The best Choi response was seven partial response (PR) (ORR, 41.2%), six stable disease (SD) and four progressions. Choi-ORR was 54% (7/13) when only M-SFTs were considered. Four of seven responsive patients were pretreated with pazopanib. No responses were detected in HG/D-SFT. Best RECIST response was one PR (5.9%), 14 SD and two progressions. Toxicity was as expected. Median Choi-PFS was 5.1 (interquartile range [IQR]: 2.5–14.8) months. Median Choi-PFS was 14.8 (IQR: 5.1–18.0) and 2.8 (IQR: 2.0–5.9) months for patients responsive and non-responsive by Choi, respectively (p = 0.0416). At a 14.4-month median follow-up, median OS was 25.3 months. Conclusion: This study showed that axitinib is active in progressive advanced SFT. One-half of patients carrying the malignant variant of the disease responded, with a >12-month median progression arrest. Responses were better detected with Choi and seen even in patients resistant to other antiangiogenics. Tolerability was good.

AB - Background: To explore the activity of axitinib in advanced solitary fibrous tumour (SFT). Patients and methods: In this investigator-driven phase II study on axitinib in advanced and progressive SFT, patients received axitinib, 5 mg bis in day (BID), until progression or limiting toxicity. Pathologic diagnosis was centrally reviewed, distinguishing malignant SFT (M-SFT) and high-grade/dedifferentiated SFT (HG/D-SFT) subtypes. The primary end-point was the overall response rate (ORR) by Choi criteria (Choi). Secondary end-points were response by Response Evaluation Criteria in Solid Tumours (RECIST), progression-free survival (PFS) and overall survival (OS). Results: From April 2015 and October 2017, 17 eligible patients entered the study (metastatic: 17; SFT subtype: 13 M-SFT, 4 HG/D-SFT; prior treatment: 9 antiangiogenics, 5 cytotoxics). All patients were evaluable for response. The best Choi response was seven partial response (PR) (ORR, 41.2%), six stable disease (SD) and four progressions. Choi-ORR was 54% (7/13) when only M-SFTs were considered. Four of seven responsive patients were pretreated with pazopanib. No responses were detected in HG/D-SFT. Best RECIST response was one PR (5.9%), 14 SD and two progressions. Toxicity was as expected. Median Choi-PFS was 5.1 (interquartile range [IQR]: 2.5–14.8) months. Median Choi-PFS was 14.8 (IQR: 5.1–18.0) and 2.8 (IQR: 2.0–5.9) months for patients responsive and non-responsive by Choi, respectively (p = 0.0416). At a 14.4-month median follow-up, median OS was 25.3 months. Conclusion: This study showed that axitinib is active in progressive advanced SFT. One-half of patients carrying the malignant variant of the disease responded, with a >12-month median progression arrest. Responses were better detected with Choi and seen even in patients resistant to other antiangiogenics. Tolerability was good.

KW - Antiangiogenic

KW - Axitinib

KW - Hemangiopericytoma

KW - Sarcoma

KW - Solitary fibrous tumour

KW - Target therapy

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U2 - 10.1016/j.ejca.2018.10.024

DO - 10.1016/j.ejca.2018.10.024

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C2 - 30528807

AN - SCOPUS:85057880637

VL - 106

SP - 225

EP - 233

JO - European Journal of Cancer

JF - European Journal of Cancer

SN - 0959-8049

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