NICE is recommending trabectedin as a treatment option for advanced soft tissue sarcoma if:
- Treatment with anthracyclines and ifosfamide has failed or,
- The patient is intolerant of or has contraindications for treatment with anthracyclines and ifosfamide.
As part of the appraisal process, NICE considered evidence, including a phase 2 randomised trial (STS-201) evaluating the efficacy of trabectedin in participants with locally advanced or metastatic soft tissue sarcoma in whom the disease had relapsed or become refractory after treatment with at least 1 anthracycline and ifosfamide, given either in combination or in sequence. All participants had liposarcomas or leiomyosarcomas (L-sarcomas) and an ECOG performance status of 0 or 1.
Participants were randomised to one of two dosing regimens of trabectedin. One group received the dosage of trabectedin specified in the marketing authorisation (1.5 mg/m2 every three weeks as a 24-hour intravenous infusion; n=136) and the other group (n=134) received trabectedin 0.58 mg/m2 every week as a three-hour intravenous infusion.
Median time to progression from intention-to-treat analyses was 3.7 months with the recommended dose, compared with 2.3 months for the comparator dosage.
Median overall survival was 13.9 months for the licensed dosage. Three-month and six-month median progression-free survival was 51.5% and 35.5%. Findings from a pre-planned subgroup analysis suggest efficacy is more favourable in patients with liposarcomas than in those with leiomyosarcomas, regardless of the study arm.
