Efficacy of immune checkpoint inhibitors in alveolar soft-part sarcoma: Results from a retrospective worldwide registry Journal Article
| Authors: | Hindi, N.; Razak, A.; Rosembaum, E.; Jonczak, E.; Hamacher, R.; Rutkowski, P.; Bhadri, V. A.; Skryd, A.; Brahmi, M.; Alshibany, A.; Jagodzinska-Mucha, P.; Bauer, S.; Connolly, E.; Gelderblom, H.; Boye, K.; Henon, C.; Bae, S.; Bogefors, K.; Vincenzi, B.; Martinez-Trufero, J.; Lopez-Martin, J. A.; Redondo, A.; Valverde, C.; Blay, J. Y.; Moura, D. S.; Gutierrez, A.; Tap, W.; Martin-Broto, J. |
| Article Title: | Efficacy of immune checkpoint inhibitors in alveolar soft-part sarcoma: Results from a retrospective worldwide registry |
| 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 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. © 2023 The Authors |
| Keywords: | adolescent; adult; cancer chemotherapy; child; treatment response; unclassified drug; major clinical study; overall survival; clinical feature; drug activity; angiogenesis inhibitor; sunitinib; drug efficacy; monotherapy; gemcitabine; follow up; ipilimumab; ticilimumab; metastasis; progression free survival; bortezomib; etoposide; clinical assessment; cohort analysis; retrospective study; information processing; ifosfamide; protein tyrosine kinase inhibitor; immunotherapy; patient coding; pazopanib; trabectedin; axitinib; anthracycline; disease exacerbation; belinostat; alveolar soft part sarcoma; progression-free survival; clinical outcome; demographics; overall response rate; crizotinib; first-line treatment; immune checkpoint inhibitor; nivolumab; immune checkpoint; human; male; female; article; pembrolizumab; durvalumab; atezolizumab; vinorelbine tartrate; programmed death 1 receptor inhibitor; toripalimab; patient registry; catequentinib; alveolar soft-part sarcoma; geptanolimab; tqb 2450; worldwide registry |
| Journal Title: | ESMO Open |
| Volume: | 8 |
| Issue: | 6 |
| ISSN: | 2059-7029 |
| Publisher: | European Society for Medical Oncology |
| Date Published: | 2023-12-01 |
| Start Page: | 102045 |
| Language: | English |
| DOI: | 10.1016/j.esmoop.2023.102045 |
| PUBMED: | 38016251 |
| PROVIDER: | scopus |
| PMCID: | PMC10698259 |
| DOI/URL: | |
| Notes: | Article -- Source: Scopus |
