Trabectedin for patients with advanced soft tissue sarcoma: A non-interventional, retrospective, multicenter study of the italian sarcoma group

The Italian Sarcoma Group performed this retrospective analysis of patients with advanced soft tissue sarcoma, pretreated with ≥1 anthracycline-based treatment, and treated with trabectedin every three weeks. Primary endpoint was to describe real-life use of trabectedin across Italy. Secondary endpoints included objective response rate (ORR) and safety. Overall, 512 patients from 20 Italian centers were evaluated. Leiomyosarcoma (37.7%)/liposarcoma (30.3%) were the most prevalent histological types (abbreviated as L-sarcoma). Patients received a median of four trabectedin cycles (range: 1–40), mostly as a second-line treatment (~60% of patients). The ORR was 13.7% superior (p < 0.0001) in patients with L-sarcoma compared with patients with non-L-sarcoma (16.6% vs. 9.0%). Median progression-free survival (PFS) was 5.1 months, whereas median overall survival (OS) was 21.6 months. Significantly better PFS and OS were observed in patients with L-sarcoma, those with objective responses and/or disease stabilization, treated in an early line and treated with reduced dose. Bone marrow toxicity (61.4%) and transaminase increases (21.9%) were the most common grade 3/4 adverse events. The results of this real-life study suggest that trabectedin is an active treatment, which is mostly given as a second-line treatment to patients with a good performance status and high-grade, metastatic L-sarcoma (clinical trial information: NCT02793050)

Patolologia neoplastica e libertà di cura: aspetti medico legali della prescrizione off label in oncologia

RIASSUNTO Gli Autori, dopo aver preso in rassegna le varie forme attraverso le quali può articolarsi la prescrizione off label e dopo aver esaminato l’iter autorizzativo che precede l’immissione in commercio di un farmaco, analizzano l’epidemiologia del fenomeno, con particolare riguardo all’ambito dell’Oncologia, e le ragioni che in ambito clinico possono portare alla prescrizione di un farmaco al di fuoi delle indicazioni per le quali vi è stata l’autorizzazione da parte delle Autorità Regolatorie. Vengono quindi presi in considerazione gli aspetti medico legali dell’utilizzo secondo modalità off label di un farmaco, in particolare per ciò che concerne la libertà di cura del medico e i relativi profili di responsabilità professionale, alla luce del quadro normativo italiano e delle indicazioni giurisprudenziali in materia