Table 2.
Major Registries of Patients With Atypical Teratoid/Rhabdoid Tumors (AT/RT) Treated With Conventional Chemotherapy and Radiotherapy
| Reference | Demographics | Treatment | Outcomes |
|---|---|---|---|
| Hilden et al, 200415 | N = 42; median age 24 months (range, 3-62 months) | Surgery + chemotherapy (all patients; numerous regimens, IT chemotherapy in 16 patients) + HDSCT (13 patients) + radiation (13 patients; CSI N = 4, tumor bed only N = 9) | Median OS 16.75 months; median EFS 10 months |
| Tekautz et al, 20057 | N = 31; <3 years of age N = 22, ≥ 3 years of age N = 9 | Surgery + chemotherapy ± radiation ≥ 3 years: one of numerous regimens, radiation (N = 3, local in 2, CSI in 1). <3 years: ICE, SJMB96 or none; CSI (N = 7) |
≥ 3 years: 2-year EFS 78%, OS 89% <3 years: 2-year EFS 11%; OS 17% |
| Gardner et al, 2008112 | N = 13 HS-I: N = 6, median age 36 months HS-II: N = 7, median age 28 months |
Surgery + chemotherapy + HDSCT (HS-I/II protocol: induction with cisplatin, vincristine, cyclophosphamide, etoposide ± methotrexate; consolidation with carboplatin, thiotepa, etoposide) | HS-I: median OS 10.25 months; median EFS 4.25 months HS-II median OS > 36 months months; median EFS > 10.5 months (3 patients alive at time of publication) |
| Chi et al, 2009108 | N = 20; median age 26 months (range, 2.4 months- 19.5 years) | Surgery + modified IRS-III regimen: chemotherapy (vincristine, dactinomycin, cyclophosphamide, cisplatin, doxorubicin, imidazole, temozolomide ± IT methotrexate, cytarabine, hydrocortisone) + radiation (focal ± CSI depending on age) | 2-year EFS 53%; 2-year OS 70% |
| Von Hoff et al, 2011109 | N = 56; median age 1.2 years (range, 0.1-4.0 years) | Surgery + chemotherapy (HIT 2000 protocol, N = 18; HIT-SKK-92, N = 9; HIT-91, N = 6, and HIT-SKK-87, N = 6; CWS-96, SIOP-93-01 and others, N = 17) + radiation (N = 29) | 3-year EFS 13%; 3-year OS 22% |
| Dufour et al, 2012111 | N = 58; median age 1.4 years (range, 14 days-8.5 years) | Surgery + chemotherapy (N = 47; BB-SFOP, N = 9; PNET-HR; N = 11; AT/RT-04, N = 24; other, N = 3) + HDSCT (N = 11) + radiation (N = 16) | Median OS 9 months |
| Lafay- Cousin et al, 2012105 | N = 50; median age 16.7 months | Surgery + chemotherapy (IRS-III-like; ICE; HS; or carboplatin, thiotepa ± methotrexate) + HDSCT (N = 18) + radiation (N = 21) | Median OS 13.5 months |
| Slavc et al, 2014113 | N = 22 Cohort A (N = 9), median age 24 months (range, 9 months-17 years) Cohort B (N = 13), median age 30 months (range, 2 months-22 years) |
Surgery + chemotherapy + radiotherapy depending on timing Cohort A: chemotherapy (MUV-AT/RT: doxorubicin, cyclophosphamide, vincristine, ifosfamide, cisplatin, etoposide, methotrexate + IT chemotherapy) + HDSCT + focal radiation Cohort B: chemotherapy (HIT-SKK-92, HIT-91, PEI, HIT-2000 or MUV-ATRT) + radiation (N = 7) |
Cohort A: 5-year OS 100% and EFS of 88.9% Cohort B: 5-year OS and EFS 28.8% |
| Zaky et al, 2014114 | N = 19, median age 14 months (range, 0 months-32 months) | Surgery + chemotherapy (HS-III (cisplatin, vincristine, etoposide, cyclophosphamide, methotrexate, temozolomide, thiotepa, etoposide, carboplatin) + HDSCT + radiation (N = 5, all patients at progression) | 3-year EFS 21%; 3-year OS 26% |
| Bartelheim et al, 2016110 | N = 31; median age 20 months | Surgery + chemotherapy (Rhabdoid 2007: vincristine, cyclophosphamide, doxorubicin, ifosfamide, carboplatin, etoposide ± IT methotrexate, cytarabine, hydrocortisone) + HDSCT (N = 8) + radiotherapy (N = 23); also see Benesch et al, 2014.115 | 6-year OS 46% (±0.10) 6-year EFS 45% (±0.09) |
| Reddy et al, 2016116; (abstract only) | N = 65 | Surgery + chemotherapy (ACNS0333) + HDSCT + radiation | 24-month EFS 42%; 24-month OS 53% |
Abbreviations: BB-SFOP, Baby Brain-French Society of Pediatric Oncology; CSI, craniospinal radiation; CWS, Cooperative Soft Tissue Sarcoma trial; EFS, event-free survival; HDSCT, hematopoietic stem cell rescue; HIT-SKK-92, Therapieprotokoll für Säuglinge und Kleinkinder mit Hirntumoren; HS, Head Start; ICE, ifosfamide, platinum, etoposide; IRS-III, Intergroup Rhabdomyosarcoma Study; IT, intrathecal; MUV, Medical University of Vienna; N, number; OS, overall survival; PEI, cisplatin, etoposide, ifosfamide; PFS, progression-free survival; PNET, primitive neuroectodermal tumor; SIOP, International Society of Pediatric Oncology; SJMB96, St. Jude Children’s Research Hospital Medulloblastoma protocol 96.