{"doi":"10.1002/pbc.31208","title":"Outcomes for children with recurrent/refractory atypical teratoid rhabdoid tumor: A single‐institution study with molecular correlation","abstract":"BACKGROUND: Survival data for recurrent pediatric atypical teratoid rhabdoid tumor (ATRT) and its association to molecular groups are extremely limited. METHODS: Single-institution retrospective study of 64 children less than 21 years 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. RESULTS: Median age at and time from initial diagnosis to PD were 2.1 years (range: 0.5-17.9 years) and 5.4 months (range: 0.5-125.6 months), 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%. CONCLUSIONS: 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.","journal":"Pediatric Blood & Cancer","year":2024,"id":447152,"datarank":0.0,"base_score":0.0,"endowment":0.0,"self_citation_contribution":0.0,"citation_network_contribution":0.0,"self_endowment_contribution":0.0,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":6,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.6014,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2024-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":16862,"name":"Jie Huang","orcid":"0000-0003-2782-2453","position":1,"is_corresponding":false},{"id":225914,"name":"Jason R. 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Boop","orcid":"0000-0002-2381-3392","position":9,"is_corresponding":false},{"id":364413,"name":"Congyu Lu","orcid":"0000-0003-2651-6251","position":10,"is_corresponding":false},{"id":686182,"name":"Edgar Sioson","orcid":"0000-0002-7540-8516","position":11,"is_corresponding":false},{"id":252141,"name":"Xin Zhou","orcid":"0000-0003-3979-8200","position":12,"is_corresponding":false},{"id":255622,"name":"Kim E. Nichols","orcid":"0000-0002-5581-6555","position":13,"is_corresponding":false},{"id":302803,"name":"Thomas E. Merchant","orcid":"0000-0002-0412-6255","position":14,"is_corresponding":false},{"id":271073,"name":"David W. Ellison","orcid":"0000-0003-1239-7757","position":15,"is_corresponding":false},{"id":5549,"name":"Giles Robinson","orcid":"0000-0001-7441-9486","position":16,"is_corresponding":false},{"id":288898,"name":"Arzu Onar‐Thomas","orcid":"0000-0003-1005-2413","position":17,"is_corresponding":false},{"id":5546,"name":"Amar Gajjar","orcid":"0000-0001-5019-0699","position":18,"is_corresponding":false},{"id":558756,"name":"Santhosh A. Upadhyaya","orcid":"0000-0001-6101-149X","position":19,"is_corresponding":false},{"id":1264780,"name":"Steven S. 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