Refractory and relapsed paediatric ACC in the MET studies – A challenging situation necessitating novel diagnostic and therapeutic concepts

BackgroundPaediatric adrenocortical carcinomas (ACC) are highly aggressive malignancies with a dismal prognosis in advanced and metastatic diseases. Little is known about outcome of patients refractory relapsed (r/r) disease.ProcedureNational retrospective multicentre study including r/r ACC diagnosed aged< 18 years registered the MET studies between January 1997 December 2021ResultsA total 16 (5 male; median age 12.9 years) disease were included. Median time to progression was 0.6 [0.0–1.3]. Site locoregional (n = 1), distant 3), combined 12). 3-year overall (OS) progression-free (PFS) survival both 0%. Thirty relapse (11 7.3 identified. 0.7 [0.1–3.2]. 8), 15), 7). At last follow-up, 20 had died or complications alive disease, 10 second complete remission (median follow-up: 6.8 [0–10.5]). OS PFS following 39.1% 31.9%. Survival superior (59.6%) compared (28.6%) (14.3%) (p 0.028) surgical resection all sites recurrence (70.0%) incomplete (21.4%) no surgery (0%) 0.003).ConclusionsFor nonresponsive first-line therapy who experience relapse, options scarce. resectability define prognosis. Novel therapeutic concepts needed improve paediatric ACC.