Efficacy of immune checkpoint inhibitors in alveolar soft-part sarcoma : results from a retrospective worldwide registry
(2023) In ESMO Open 8(6).- Abstract
Background: Conventional cytotoxic drugs are not effective in alveolar soft-part sarcoma (ASPS). Immune checkpoint (programmed cell death protein 1/programmed death-ligand 1) inhibitors (ICIs) are promising drugs in ASPS. A worldwide registry explored the efficacy of ICI in ASPS. Materials and methods: Data from adult patients diagnosed with ASPS and treated with ICI for advanced disease in expert sarcoma centers from Europe, Australia and North America were retrospectively collected, including demographics and data related to treatments and outcome. Results: Seventy-six ASPS patients, with a median age at diagnosis of 25 years (range 3-61 years), were registered. All patients received ICI for metastatic disease. Immunotherapy regimens... (More)
Background: Conventional cytotoxic drugs are not effective in alveolar soft-part sarcoma (ASPS). Immune checkpoint (programmed cell death protein 1/programmed death-ligand 1) inhibitors (ICIs) are promising drugs in ASPS. A worldwide registry explored the efficacy of ICI in ASPS. Materials and methods: Data from adult patients diagnosed with ASPS and treated with ICI for advanced disease in expert sarcoma centers from Europe, Australia and North America were retrospectively collected, including demographics and data related to treatments and outcome. Results: Seventy-six ASPS patients, with a median age at diagnosis of 25 years (range 3-61 years), were registered. All patients received ICI for metastatic disease. Immunotherapy regimens consisted of monotherapy in 38 patients (50%) and combination in 38 (50%) (23 with a tyrosine kinase inhibitor). Among the 68 assessable patients, there were 3 complete responses and 34 partial responses, translating into an overall response rate of 54.4%. After a median follow-up of 36 months [95% confidence interval (CI) 32-40 months] since the start of immunotherapy, 45 (59%) patients have progressed on ICI, with a median progression-free survival (PFS) of 16.3 months (95% CI 8-25 months). Receiving ICI in first line (P = 0.042) and achieving an objective response (P = 0.043) correlated with a better PFS. Median estimated overall survival (OS) from ICI initiation has not been reached. The 12-month and 24-month OS rates were 94% and 81%, respectively. Conclusions: This registry constitutes the largest available series of ASPS treated with ICI. Our results suggest that the ICI treatment provides long-lasting disease control and prolonged OS in patients with advanced ASPS, an ultra-rare entity with limited active therapeutic options.
(Less)
- author
- organization
- publishing date
- 2023-12
- type
- Contribution to journal
- publication status
- published
- subject
- keywords
- alveolar soft-part sarcoma, immune checkpoint, immunotherapy, progression-free survival
- in
- ESMO Open
- volume
- 8
- issue
- 6
- article number
- 102045
- publisher
- BMJ Publishing Group
- external identifiers
-
- scopus:85178386399
- pmid:38016251
- ISSN
- 2059-7029
- DOI
- 10.1016/j.esmoop.2023.102045
- language
- English
- LU publication?
- yes
- additional info
- Funding Information: DSM is a recipient of a Sara Borrell postdoctoral fellowship funded by the National Institute of Health Carlos III (ISCIII) (CD20/00155). The authors thank SELNET project. SELNET has received funding from the European Union’s Horizon 2020 research and innovation program under grant agreement No. 825806. Publisher Copyright: © 2023 The Authors
- id
- 1ede7563-37b1-4de7-adae-2096dcfb68ba
- date added to LUP
- 2023-12-21 10:23:55
- date last changed
- 2024-12-15 20:52:25
@article{1ede7563-37b1-4de7-adae-2096dcfb68ba,
abstract = {{<p>Background: Conventional cytotoxic drugs are not effective in alveolar soft-part sarcoma (ASPS). Immune checkpoint (programmed cell death protein 1/programmed death-ligand 1) inhibitors (ICIs) are promising drugs in ASPS. A worldwide registry explored the efficacy of ICI in ASPS. Materials and methods: Data from adult patients diagnosed with ASPS and treated with ICI for advanced disease in expert sarcoma centers from Europe, Australia and North America were retrospectively collected, including demographics and data related to treatments and outcome. Results: Seventy-six ASPS patients, with a median age at diagnosis of 25 years (range 3-61 years), were registered. All patients received ICI for metastatic disease. Immunotherapy regimens consisted of monotherapy in 38 patients (50%) and combination in 38 (50%) (23 with a tyrosine kinase inhibitor). Among the 68 assessable patients, there were 3 complete responses and 34 partial responses, translating into an overall response rate of 54.4%. After a median follow-up of 36 months [95% confidence interval (CI) 32-40 months] since the start of immunotherapy, 45 (59%) patients have progressed on ICI, with a median progression-free survival (PFS) of 16.3 months (95% CI 8-25 months). Receiving ICI in first line (P = 0.042) and achieving an objective response (P = 0.043) correlated with a better PFS. Median estimated overall survival (OS) from ICI initiation has not been reached. The 12-month and 24-month OS rates were 94% and 81%, respectively. Conclusions: This registry constitutes the largest available series of ASPS treated with ICI. Our results suggest that the ICI treatment provides long-lasting disease control and prolonged OS in patients with advanced ASPS, an ultra-rare entity with limited active therapeutic options.</p>}},
author = {{Hindi, N. and Razak, A. and Rosembaum, E. and Jonczak, E. and Hamacher, R. and Rutkowski, P. and Bhadri, V. A. and Skryd, A. and Brahmi, M. and Alshibany, A. and Jagodzinska-Mucha, P. and Bauer, S. and Connolly, Emma and Gelderblom, H. and Boye, K. and Henon, C. and Bae, S. and Bogefors, K. and Vincenzi, B. and Martinez-Trufero, J. and Lopez-Martin, J. A. and Redondo, A. and Valverde, C. and Blay, J. Y. and Moura, D. S. and Gutierrez, A. and Tap, W. and Martin-Broto, Javier}},
issn = {{2059-7029}},
keywords = {{alveolar soft-part sarcoma; immune checkpoint; immunotherapy; progression-free survival}},
language = {{eng}},
number = {{6}},
publisher = {{BMJ Publishing Group}},
series = {{ESMO Open}},
title = {{Efficacy of immune checkpoint inhibitors in alveolar soft-part sarcoma : results from a retrospective worldwide registry}},
url = {{http://dx.doi.org/10.1016/j.esmoop.2023.102045}},
doi = {{10.1016/j.esmoop.2023.102045}},
volume = {{8}},
year = {{2023}},
}