Outcomes for children with recurrent/refractory atypical teratoid rhabdoid tumor: A single-institution study with molecular correlation.

Abstract

Survival data for recurrent pediatric atypical teratoid rhabdoid tumor (ATRT) and its association to molecular groups are extremely limited. Single-institution retrospective study of 64 children less than 21years old with recurrent or treatment-refractory (progressive disease [PD]) ATRT treated at St. Jude Hospital from January 2000 to December 2020. Demographic, clinicopathologic, treatment, molecular grouping (SHH, TYR, and MYC) and germline data were collected. Progression-free survival (PFS2: time from PD to subsequent first progression) and overall survival (OSpostPD: time from PD to death/last follow-up) were estimated by Kaplan-Meier analysis. Median age at and time from initial diagnosis to PD were 2.1years (range: 0.5-17.9years) and 5.4months (range: 0.5-125.6months), respectively. Only five of 64 children (7.8%) are alive at median follow-up of 10.9 (range: 4.2-18.1) years from PD. The 2/5-year PFS2 and OSpostPD were 3.1% (±1.8%)/1.6% (±1.1%) and 20.3% (±4.8%)/7.3% (±3.5%), respectively. Children with TYR group (n=10) had a better OSpostPD compared to those with MYC (n=11) (2-year survival estimates: 60.0%±14.3% vs. 18.2%±9.5%; p=.019), or those with SHH (n=21; 4.8%±3.3%; p=.014). In univariate analyses, OSpostPD was better with older age at diagnosis (p=.037), female gender (p=.008), and metastatic site of PD compared to local or combined sites of PD (p<.001). Two-year OSpostPD for patients receiving any salvage therapy (n=39) post PD was 33.3%±7.3%. Children with recurrent/refractory ATRT have dismal outcomes. Older age at diagnosis, female gender, TYR group, and metastatic site of PD were associated with relatively longer survival in our study.