DT are rare tumors arising from musculoaponeurotic tissues. Although benign, they may be locally aggressive, leading to pain and disability, and exceptionally death. ESMO guidelines recommend frontline watchful waiting. Medical treatment includes NSAID’s, anti-estrogens, chemotherapy, antiangiogenics or radiation therapy in progressing tumors. Some pts do not respond for whom there is an unmet need for new treatments. Cryoablation (CA) is an interventional radiology technique that is suitable for DT pts. The procedure is based on repeated cycles of freezing/passive thawing of the tumor, leading to cell death.
CRYODESMO-01 was the first prospective, open-label, non-randomized, non- comparative, multicentric pilot study assessing CA in non-abdominopelvic progressive DT pts. The primary endpoint was the rate of non-progression at 12 months; secondary endpoints included safety, quality of life (QoL), assessment of pain and functional status. Inclusion criteria were: pts 18 y.o., DT deemed accessible by the operator, measurable lesion (RECIST), progressive disease after at least two lines of adequate medical therapy or with functional symptoms/pain, adequate biological parameters, informed consent and affiliation to social security.
50 pts were enrolled (78% female). Mean age was 41 y.o (range 19-73). The median number of prior treatments was 2.00 [1-4]. Tumor location were limbs (36%); trunk (60%) and cervical area (4%). The median tumor volume was 111cm3 (0.6-1 068). The rate of non-progressing disease +12 months was 86% (CI95% 73%-94%). Grade 1 and 2 toxicity occured in 32.8 and 44.5% of cases, whereas 11 pts (22%) had grade 3 and 4 AEs, all with a favorable outcome. 63% and 83% of pts had better functional status and pain scores, respectively. Female sex (p = 0.046) was correlated to response at + 12 months (90.38% vs: 62.82%).
The study met is primary endpoint with 86% of non-progressive disease at + 12 months, reduced pain and better functional status. CA in the hands of expert interventional radiologists, should be considered in guidelines as a reasonable treatment option for these pts. CA will soon challenge medical therapy as front-line therapy in the forthcoming randomized trial CRYODESMO-02.
Clinical trial identification
Legal entity responsible for the study
Hôpitaux Universitaires de Strasbourg.
French National Cancer Institute (INCa).
J. Kurtz: Travel / Accommodation / Expenses: PharmaMar; Travel / Accommodation / Expenses: Roche; Advisory / Consultancy, Travel / Accommodation / Expenses: Tesaro; Advisory / Consultancy, Travel / Accommodation / Expenses: AstraZeneca. X. Buy: Honoraria (self): Galil BTG. F. Deschamps: Honoraria (self): BTG; Honoraria (self): Medtronic; Honoraria (self): Covidien. A. gangi: Speaker Bureau / Expert testimony, Travel / Accommodation / Expenses: Canon medical; Licensing / Royalties: Apriomed; Non-remunerated activity/ies: BTG. All other authors have declared no conflicts of interest.