Abstract 83P
Background
DeFi trial demonstrated clinical benefit of Ni. DeNi study describes the behavior of pts treated with Ni within the compassionate use program.
Methods
DeNi is an exhaustive real-world retrospective study base on NETSARC data. The objectives were objective response (RECIST criteria), pain improvement, toxicity and 1-year progression free survival (1-y PFS).
Results
From 02/2024 to 09/2024, 55 pts were identified (37 women). The median age was 38.6 years (range: 18 - 66.7). The most common sites were the limbs (49%) and trunk (27%). CTNNB1 mutations were present in 46 pts. The median number of previous treatments (TTT), including local TTT but excluding active surveillance, was 2 [1-5]. Previous treatments were: active surveillance in 11 pts (20%), local TTT in 21 pts (38%) (8 (15%) surgery, 6 (11%) radiotherapy, 11 (20%) cryotherapy), and systemic TTT for all pts (18 (33%) NSAID, 9 (16%) hormonotherapy (HT), 41 (75%) chemotherapy (CT), 35 (64%) tyrosine kinase inhibitors (TKI)). The median duration of previous TTT was 5.1 months (1.0-48.0) for NSAID, 9.8 (1.0-85.4) for HT, 5.9 (0.2 - 20.1) for CT, and 9.0 (1.0 - 21.6) for TKI. Eight pts (15%) had fertility preservation. Median follow-up was 9.6 months [95%CI: 6.6;11.9]. Partial response and stable disease (SD) were observed in 28 (52%) and 21 pts (39%). Five pts (9%) had progressive disease (PD), with majority of extra-abdominal tumors and CTNNB1 mutations (4/5). 38 pts (73%) had pain relief and 33 pts (77%) were able to reduce or interrupt analgesic TTT. Among pts with SD, 50% were less painful. The 1-y PFS rate was 77.5% [64.1%;93.8%] and 73.4% of pts [59.0%;91.4%] were without TTT change after 1 year. The most common side effects were diarrhea (51%), fatigue (45%) and cutaneous rash (27%). Ovarian dysfunction was reported for 9 pts. 13 pts had dose reduction (24%). At the time of analysis, 34 pts (62%) are continuing Ni, 21 (38%) discontinued Ni because of PD (N=5), toxicity (N=8) or personal decision (N=8).
Conclusions
DeNi study confirms the clinical benefit of Ni with pain improvement even without tumor shrinkage. Questions remain such as TTT duration and first-line use.
Clinical trial identification
Editorial acknowledgement
Legal entity responsible for the study
The authors.
Funding
Has not received any funding.
Disclosure
All authors have declared no conflicts of interest.