Anthracycline, Gemcitabine, and Pazopanib in Epithelioid Sarcoma: A Multi-institutional Case Series.

Anna Maria Frezza, Robin L Jones, Salvatore Lo Vullo, Naofumi Asano, Francesca Lucibello, Eytan Ben-Ami, Ravin Ratan, Pawel Teterycz, Kjetil Boye, Mehdi Brahmi, Emanuela Palmerini, Alexander Fedenko, Bruno Vincenzi, Antonella Brunello, Ingrid M E Desar, Robert S Benjamin, Jean Yves Blay, Javier Martin Broto, Paolo G Casali, Hans GelderblomGiovanni Grignani, Alessandro Gronchi, Kirsten Sundby Hall, Olivier Mir, Piotr Rutkowski, Andrew J Wagner, Olga Anurova, Paola Collini, Angelo P Dei Tos, Uta Flucke, Jason L Hornick, Ingvild Lobmaier, Terrier Philippe, Piero Picci, Dominique Ranchere, Salvatore L Renne, Marta Sbaraglia, Khin Thway, Michal Wagrodzki, Wei-Lien Wang, Akihiko Yoshida, Luigi Mariani, Akira Kawai, Silvia Stacchiotti

Research output: Contribution to journalArticle

Abstract

Epithelioid sarcoma (ES) is an exceedingly rare malignant neoplasm with distinctive pathologic, molecular, and clinical features as well as the potential to respond to new targeted drugs. Little is known on the activity of anthracycline-based regimens, gemcitabine-based regimens, and pazopanib in this disease. To report on the activity of anthracycline-based regimens, gemcitabine-based regimens, and pazopanib in patients with advanced ES. Seventeen sarcoma reference centers in Europe, the United States, and Japan contributed data to this retrospective analysis of patients with locally advanced/metastatic ES diagnosed between 1990 and 2016. Local pathological review was performed in all cases to confirm diagnosis according to most recent criteria. All patients included in the study received anthracycline-based regimens, gemcitabine-based regimens, or pazopanib. Response was assessed by RECIST. Progression-free survival (PFS) and overall survival (OS) were computed by Kaplan-Meier method. Classic and proximal subtypes were defined based on morphology (according to 2013 World Health Organization guidelines). Overall, 115 patients were included, 80 (70%) were men and 35 (30%) were women, with a median age of 32 years (range, 15-77 years). Of the 115 patients with ES, 85 were treated with anthracycline-based regimens, 41 with gemcitabine-based regimens, and 18 with pazopanib. Twenty-four received more than 1 treatment. Median follow-up was 34 months. Response rate for anthracycline-based regimens was 22%, with a median PFS of 6 months. One complete response (CR) was reported. A trend toward a higher response rate was noticed in morphological proximal type (26%) vs classic type (19%) and in proximal vs distal primary site (26% vs 18%). The response rate for gemcitabine-based regimens was 27%, with 2 CR and a median PFS of 4 months. In this group, a trend toward a higher response rate was reported in classic vs proximal morphological type (30% vs 22%) and in distal vs proximal primary site (40% vs 14%). In the pazopanib group, no objective responses were seen, and median PFS was 3 months. This is the largest retrospective series of systemic therapy in ES. We confirm a moderate activity of anthracycline-based and gemcitabine-based regimens in ES, with a similar response rate and PFS in both groups. The value of pazopanib was low. These data may serve as a benchmark for trials of novel agents in ES.
Original languageUndefined/Unknown
Pages (from-to)e180219
JournalJAMA oncology
Volume4
DOIs
Publication statusPublished - Sep 1 2018

Cite this

Frezza, A. M., Jones, R. L., Lo Vullo, S., Asano, N., Lucibello, F., Ben-Ami, E., ... Stacchiotti, S. (2018). Anthracycline, Gemcitabine, and Pazopanib in Epithelioid Sarcoma: A Multi-institutional Case Series. JAMA oncology, 4, e180219. https://doi.org/10.1001/jamaoncol.2018.0219

Anthracycline, Gemcitabine, and Pazopanib in Epithelioid Sarcoma: A Multi-institutional Case Series. / Frezza, Anna Maria; Jones, Robin L; Lo Vullo, Salvatore; Asano, Naofumi; Lucibello, Francesca; Ben-Ami, Eytan; Ratan, Ravin; Teterycz, Pawel; Boye, Kjetil; Brahmi, Mehdi; Palmerini, Emanuela; Fedenko, Alexander; Vincenzi, Bruno; Brunello, Antonella; Desar, Ingrid M E; Benjamin, Robert S; Blay, Jean Yves; Broto, Javier Martin; Casali, Paolo G; Gelderblom, Hans; Grignani, Giovanni; Gronchi, Alessandro; Hall, Kirsten Sundby; Mir, Olivier; Rutkowski, Piotr; Wagner, Andrew J; Anurova, Olga; Collini, Paola; Dei Tos, Angelo P; Flucke, Uta; Hornick, Jason L; Lobmaier, Ingvild; Philippe, Terrier; Picci, Piero; Ranchere, Dominique; Renne, Salvatore L; Sbaraglia, Marta; Thway, Khin; Wagrodzki, Michal; Wang, Wei-Lien; Yoshida, Akihiko; Mariani, Luigi; Kawai, Akira; Stacchiotti, Silvia.

In: JAMA oncology, Vol. 4, 01.09.2018, p. e180219.

Research output: Contribution to journalArticle

Frezza, AM, Jones, RL, Lo Vullo, S, Asano, N, Lucibello, F, Ben-Ami, E, Ratan, R, Teterycz, P, Boye, K, Brahmi, M, Palmerini, E, Fedenko, A, Vincenzi, B, Brunello, A, Desar, IME, Benjamin, RS, Blay, JY, Broto, JM, Casali, PG, Gelderblom, H, Grignani, G, Gronchi, A, Hall, KS, Mir, O, Rutkowski, P, Wagner, AJ, Anurova, O, Collini, P, Dei Tos, AP, Flucke, U, Hornick, JL, Lobmaier, I, Philippe, T, Picci, P, Ranchere, D, Renne, SL, Sbaraglia, M, Thway, K, Wagrodzki, M, Wang, W-L, Yoshida, A, Mariani, L, Kawai, A & Stacchiotti, S 2018, 'Anthracycline, Gemcitabine, and Pazopanib in Epithelioid Sarcoma: A Multi-institutional Case Series.', JAMA oncology, vol. 4, pp. e180219. https://doi.org/10.1001/jamaoncol.2018.0219
Frezza, Anna Maria ; Jones, Robin L ; Lo Vullo, Salvatore ; Asano, Naofumi ; Lucibello, Francesca ; Ben-Ami, Eytan ; Ratan, Ravin ; Teterycz, Pawel ; Boye, Kjetil ; Brahmi, Mehdi ; Palmerini, Emanuela ; Fedenko, Alexander ; Vincenzi, Bruno ; Brunello, Antonella ; Desar, Ingrid M E ; Benjamin, Robert S ; Blay, Jean Yves ; Broto, Javier Martin ; Casali, Paolo G ; Gelderblom, Hans ; Grignani, Giovanni ; Gronchi, Alessandro ; Hall, Kirsten Sundby ; Mir, Olivier ; Rutkowski, Piotr ; Wagner, Andrew J ; Anurova, Olga ; Collini, Paola ; Dei Tos, Angelo P ; Flucke, Uta ; Hornick, Jason L ; Lobmaier, Ingvild ; Philippe, Terrier ; Picci, Piero ; Ranchere, Dominique ; Renne, Salvatore L ; Sbaraglia, Marta ; Thway, Khin ; Wagrodzki, Michal ; Wang, Wei-Lien ; Yoshida, Akihiko ; Mariani, Luigi ; Kawai, Akira ; Stacchiotti, Silvia. / Anthracycline, Gemcitabine, and Pazopanib in Epithelioid Sarcoma: A Multi-institutional Case Series. In: JAMA oncology. 2018 ; Vol. 4. pp. e180219.
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abstract = "Epithelioid sarcoma (ES) is an exceedingly rare malignant neoplasm with distinctive pathologic, molecular, and clinical features as well as the potential to respond to new targeted drugs. Little is known on the activity of anthracycline-based regimens, gemcitabine-based regimens, and pazopanib in this disease. To report on the activity of anthracycline-based regimens, gemcitabine-based regimens, and pazopanib in patients with advanced ES. Seventeen sarcoma reference centers in Europe, the United States, and Japan contributed data to this retrospective analysis of patients with locally advanced/metastatic ES diagnosed between 1990 and 2016. Local pathological review was performed in all cases to confirm diagnosis according to most recent criteria. All patients included in the study received anthracycline-based regimens, gemcitabine-based regimens, or pazopanib. Response was assessed by RECIST. Progression-free survival (PFS) and overall survival (OS) were computed by Kaplan-Meier method. Classic and proximal subtypes were defined based on morphology (according to 2013 World Health Organization guidelines). Overall, 115 patients were included, 80 (70{\%}) were men and 35 (30{\%}) were women, with a median age of 32 years (range, 15-77 years). Of the 115 patients with ES, 85 were treated with anthracycline-based regimens, 41 with gemcitabine-based regimens, and 18 with pazopanib. Twenty-four received more than 1 treatment. Median follow-up was 34 months. Response rate for anthracycline-based regimens was 22{\%}, with a median PFS of 6 months. One complete response (CR) was reported. A trend toward a higher response rate was noticed in morphological proximal type (26{\%}) vs classic type (19{\%}) and in proximal vs distal primary site (26{\%} vs 18{\%}). The response rate for gemcitabine-based regimens was 27{\%}, with 2 CR and a median PFS of 4 months. In this group, a trend toward a higher response rate was reported in classic vs proximal morphological type (30{\%} vs 22{\%}) and in distal vs proximal primary site (40{\%} vs 14{\%}). In the pazopanib group, no objective responses were seen, and median PFS was 3 months. This is the largest retrospective series of systemic therapy in ES. We confirm a moderate activity of anthracycline-based and gemcitabine-based regimens in ES, with a similar response rate and PFS in both groups. The value of pazopanib was low. These data may serve as a benchmark for trials of novel agents in ES.",
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T1 - Anthracycline, Gemcitabine, and Pazopanib in Epithelioid Sarcoma: A Multi-institutional Case Series.

AU - Frezza, Anna Maria

AU - Jones, Robin L

AU - Lo Vullo, Salvatore

AU - Asano, Naofumi

AU - Lucibello, Francesca

AU - Ben-Ami, Eytan

AU - Ratan, Ravin

AU - Teterycz, Pawel

AU - Boye, Kjetil

AU - Brahmi, Mehdi

AU - Palmerini, Emanuela

AU - Fedenko, Alexander

AU - Vincenzi, Bruno

AU - Brunello, Antonella

AU - Desar, Ingrid M E

AU - Benjamin, Robert S

AU - Blay, Jean Yves

AU - Broto, Javier Martin

AU - Casali, Paolo G

AU - Gelderblom, Hans

AU - Grignani, Giovanni

AU - Gronchi, Alessandro

AU - Hall, Kirsten Sundby

AU - Mir, Olivier

AU - Rutkowski, Piotr

AU - Wagner, Andrew J

AU - Anurova, Olga

AU - Collini, Paola

AU - Dei Tos, Angelo P

AU - Flucke, Uta

AU - Hornick, Jason L

AU - Lobmaier, Ingvild

AU - Philippe, Terrier

AU - Picci, Piero

AU - Ranchere, Dominique

AU - Renne, Salvatore L

AU - Sbaraglia, Marta

AU - Thway, Khin

AU - Wagrodzki, Michal

AU - Wang, Wei-Lien

AU - Yoshida, Akihiko

AU - Mariani, Luigi

AU - Kawai, Akira

AU - Stacchiotti, Silvia

PY - 2018/9/1

Y1 - 2018/9/1

N2 - Epithelioid sarcoma (ES) is an exceedingly rare malignant neoplasm with distinctive pathologic, molecular, and clinical features as well as the potential to respond to new targeted drugs. Little is known on the activity of anthracycline-based regimens, gemcitabine-based regimens, and pazopanib in this disease. To report on the activity of anthracycline-based regimens, gemcitabine-based regimens, and pazopanib in patients with advanced ES. Seventeen sarcoma reference centers in Europe, the United States, and Japan contributed data to this retrospective analysis of patients with locally advanced/metastatic ES diagnosed between 1990 and 2016. Local pathological review was performed in all cases to confirm diagnosis according to most recent criteria. All patients included in the study received anthracycline-based regimens, gemcitabine-based regimens, or pazopanib. Response was assessed by RECIST. Progression-free survival (PFS) and overall survival (OS) were computed by Kaplan-Meier method. Classic and proximal subtypes were defined based on morphology (according to 2013 World Health Organization guidelines). Overall, 115 patients were included, 80 (70%) were men and 35 (30%) were women, with a median age of 32 years (range, 15-77 years). Of the 115 patients with ES, 85 were treated with anthracycline-based regimens, 41 with gemcitabine-based regimens, and 18 with pazopanib. Twenty-four received more than 1 treatment. Median follow-up was 34 months. Response rate for anthracycline-based regimens was 22%, with a median PFS of 6 months. One complete response (CR) was reported. A trend toward a higher response rate was noticed in morphological proximal type (26%) vs classic type (19%) and in proximal vs distal primary site (26% vs 18%). The response rate for gemcitabine-based regimens was 27%, with 2 CR and a median PFS of 4 months. In this group, a trend toward a higher response rate was reported in classic vs proximal morphological type (30% vs 22%) and in distal vs proximal primary site (40% vs 14%). In the pazopanib group, no objective responses were seen, and median PFS was 3 months. This is the largest retrospective series of systemic therapy in ES. We confirm a moderate activity of anthracycline-based and gemcitabine-based regimens in ES, with a similar response rate and PFS in both groups. The value of pazopanib was low. These data may serve as a benchmark for trials of novel agents in ES.

AB - Epithelioid sarcoma (ES) is an exceedingly rare malignant neoplasm with distinctive pathologic, molecular, and clinical features as well as the potential to respond to new targeted drugs. Little is known on the activity of anthracycline-based regimens, gemcitabine-based regimens, and pazopanib in this disease. To report on the activity of anthracycline-based regimens, gemcitabine-based regimens, and pazopanib in patients with advanced ES. Seventeen sarcoma reference centers in Europe, the United States, and Japan contributed data to this retrospective analysis of patients with locally advanced/metastatic ES diagnosed between 1990 and 2016. Local pathological review was performed in all cases to confirm diagnosis according to most recent criteria. All patients included in the study received anthracycline-based regimens, gemcitabine-based regimens, or pazopanib. Response was assessed by RECIST. Progression-free survival (PFS) and overall survival (OS) were computed by Kaplan-Meier method. Classic and proximal subtypes were defined based on morphology (according to 2013 World Health Organization guidelines). Overall, 115 patients were included, 80 (70%) were men and 35 (30%) were women, with a median age of 32 years (range, 15-77 years). Of the 115 patients with ES, 85 were treated with anthracycline-based regimens, 41 with gemcitabine-based regimens, and 18 with pazopanib. Twenty-four received more than 1 treatment. Median follow-up was 34 months. Response rate for anthracycline-based regimens was 22%, with a median PFS of 6 months. One complete response (CR) was reported. A trend toward a higher response rate was noticed in morphological proximal type (26%) vs classic type (19%) and in proximal vs distal primary site (26% vs 18%). The response rate for gemcitabine-based regimens was 27%, with 2 CR and a median PFS of 4 months. In this group, a trend toward a higher response rate was reported in classic vs proximal morphological type (30% vs 22%) and in distal vs proximal primary site (40% vs 14%). In the pazopanib group, no objective responses were seen, and median PFS was 3 months. This is the largest retrospective series of systemic therapy in ES. We confirm a moderate activity of anthracycline-based and gemcitabine-based regimens in ES, with a similar response rate and PFS in both groups. The value of pazopanib was low. These data may serve as a benchmark for trials of novel agents in ES.

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DO - 10.1001/jamaoncol.2018.0219

M3 - Articolo

VL - 4

SP - e180219

JO - JAMA oncology

JF - JAMA oncology

SN - 2374-2437

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