{"entity":{"id":"paper-florence-duffaud-lancet-oncol-2020","kind":"paper","name":"Cabozantinib in patients with advanced Ewing sarcoma or osteosarcoma (CABONE): a multicentre, single-arm, phase 2 trial","aka":[],"tldr":"Paper by Florence Duffaud indexed on Europe PMC as PubMed record 32078813, in The Lancet Oncology (2020), one of the most cited records naming an author with this name at Hôpital de la Timone, Assistance Publique-Hôpitaux de Marseille.","summary":"Background: Patients with Ewing sarcoma or osteosarcoma have a median overall survival of less than 12 months after diagnosis, and a standard treatment strategy has not yet been established. Pharmacological inhibition of MET signalling and aberrant angiogenesis has shown promising results in several preclinical models of Ewing sarcoma and osteosarcoma. We aimed to investigate the activity of cabozantinib, an inhibitor of MET and VEGFR2, in patients with advanced Ewing sarcoma and osteosarcoma.\n\nMethods: We did a multicentre, single-arm, two-stage, phase 2 trial in patients with advanced Ewing sarcoma or osteosarcoma recruited from ten centres in the French Sarcoma Group. Key eligibility criteria were aged 12 years or older, Eastern Cooperative Oncology Group performance status of 0-1, and documented disease progression (according to Response Evaluation Criteria in Solid Tumors version 1.1) before study entry. The number of previous lines of treatment was not limited. Patients received cabozantinib (adults 60 mg, children [<16 years] 40 mg/m 2) orally once daily in 28-day cycles until disease progression, unacceptable toxicity, the investigator's decision to discontinue, or participant withdrawal. The primary endpoint for Ewing sarcoma was best objective response within 6 months of treatment onset; for osteosarcoma, a dual primary endpoint of 6-month objective response and 6-month non-progression was assessed. All enrolled patients who received at least one dose of cabozantinib were included in the safety analysis, and all participants who received at least one complete or two incomplete treatment cycles were included in the efficacy population. This study was registered with ClinicalTrials.gov, number NCT02243605.\n\nFindings: Between April 16, 2015, and July 12, 2018, 90 patients (45 with Ewing sarcoma 45 with osteosarcoma) were recruited to the study. Median follow-up was 31·3 months (95% CI 12·4-35·4) for patients with Ewing sarcoma and 31·1 months (24·4-31·7) for patients with osteosarcoma. 39 (87%) patients with Ewing sarcoma and 42 (93%) patients with osteosarcoma were assessable for efficacy after histological and radiological review. In patients with Ewing sarcoma, ten (26%; 95% CI 13-42) of 39 patients had an objective response (all partial responses) by 6 months; in patients with osteosarcoma, five (12%; 4-26) of 42 patients had an objective response (all partial responses) and 14 (33%; 20-50) had 6-month non-progression. The most common grade 3 or 4 adverse events were hypophosphataemia (five [11%] for Ewing sarcoma, three [7%] for osteosarcoma), aspartate aminotransferase increase (two [4%] for Ewing sarcoma, three [7%] for osteosarcoma), palmar-plantar syndrome (three [7%] for Ewing sarcoma, two [4%] for osteosarcoma), pneumothorax (one [2%] for Ewing sarcoma, four [9%] for osteosarcoma), and neutropenia (two [4%] for Ewing sarcoma, four [9%] for osteosarcoma). At least one serious adverse event was reported in 61 (68%) of 90 patients. No patients died from drug-related toxic effects.\n\nInterpretation: Cabozantinib has antitumor activity in patients with advanced Ewing sarcoma and osteosarcoma and was generally well tolerated. Cabozantinib could represent a new therapeutic option in this setting, and deserves further investigation.\n\nFunding: Institut Bergonié; French National Cancer Institute; Association pour la Recherche contre le Cancer.\n\nIndexed on Europe PMC as PubMed record 32078813 (DOI 10.1016/s1470-2045(19)30825-3). Its author list gives \"Duffaud F\" with the affiliation \"Department of Medical Oncology, Assistance Publique des Hôpitaux de Marseille, Hôpital La Timone, Marseille, France\", which names Hôpital de la Timone, Assistance Publique-Hôpitaux de Marseille; that is how the record was matched to Florence Duffaud, and no figure has been checked by an editor.","asOf":"2026-09-22","links":[{"label":"Lancet Oncol 2020","url":"https://doi.org/10.1016/s1470-2045(19)30825-3"},{"label":"PubMed","url":"https://pubmed.ncbi.nlm.nih.gov/32078813/"},{"label":"Europe PMC","url":"https://europepmc.org/article/MED/32078813"}],"tags":["europepmc-ingest"],"related":[],"cancers":[],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":["florence-duffaud"],"bottlenecks":[],"keyPapers":[],"journals":["lancet-oncology"],"dependsOn":[],"notes":[],"journal":"The Lancet Oncology","year":2020,"doi":"10.1016/s1470-2045(19)30825-3","pmid":"32078813","authors":"Italiano A, Mir O, Mathoulin-Pelissier S, et al.","paperType":"observational","findings":[],"whatItMeans":"One of the most cited papers Europe PMC returns for Florence Duffaud at Hôpital de la Timone, Assistance Publique-Hôpitaux de Marseille, so it is a natural starting point for reading their work. The record was linked automatically from the author list and affiliation; read the abstract above and the paper itself before relying on any figure.","caveats":["Matched to the person by surname, initials and an affiliation string naming the institution on the Europe PMC record; the summary reproduces the record's abstract and no figure has been verified against the full paper.","Two authors sharing a surname, initials and institution cannot be told apart by this method; the person's profile links are the place to confirm authorship."]},"route":"/key-papers/paper-florence-duffaud-lancet-oncol-2020/","neighbours":{"person":[{"id":"florence-duffaud","kind":"person","name":"Florence Duffaud","route":"/people/florence-duffaud/"}],"journal":[{"id":"lancet-oncology","kind":"journal","name":"The Lancet Oncology","route":"/journals/lancet-oncology/"}]}}