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ACNS0333 was the first trial built only for atypical teratoid/rhabdoid tumour, a brain cancer of very young children that used to be almost always fatal; its intensive plan of chemotherapy, high-dose chemotherapy with stem cell rescue and focused radiotherapy more than halved the risk of relapse or death compared with earlier treatment and became the template for care.
ACNS0333 was the Children's Oncology Group's first trial specific to atypical teratoid/rhabdoid tumour. After surgery, 70 patients received two courses of multi-agent induction chemotherapy including high-dose methotrexate, then three courses of high-dose carboplatin and thiotepa with peripheral blood stem cell rescue, with involved-field three-dimensional conformal radiotherapy given before or after consolidation according to age and disease location. SMARCB1 testing of tumour and blood was mandatory. The primary analysis compared event-free survival in patients under 36 months with a historical cooperative group cohort.
The regimen cut the hazard of an event-free survival event by more than half in children under 36 months (hazard rate 0.43), and four-year event-free and overall survival for the whole cohort were 37 and 43 percent, far above historical figures; 91 percent of relapses occurred within two years and four patients died of treatment. The corpus's atypical teratoid/rhabdoid tumour page describes ACNS0333-style induction, high-dose chemotherapy with autologous rescue and focal radiotherapy on this evidence.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
70 enrolled.
Numbers not yet public.
Source95% CI 25 to 49
Source95% CI 31 to 55
Source| Endpoint | Arm | n | Value | HR (95% CI) | p | Source |
|---|---|---|---|---|---|---|
| Event-free survival, patients under 36 months against a historical cooperative group cohortprimary | ACNS0333 regimen (induction, high-dose chemotherapy with stem cell rescue, conformal radiotherapy) | 54 | - | 0.43 (0.28 to 0.66) | <0.0005 | link |
| Historical cohort | - | - | ||||
| Event-free survival at 4 years, entire cohort | ACNS0333 regimen | 65 | 37% | - | - | link |
| Overall survival at 4 years, entire cohort | ACNS0333 regimen | 65 | 43% | - | - | link |
| Treatment-related deaths | ACNS0333 regimen | 65 | 4 participants | - | - | link |
Shares Brain and spinal cord tumours (all types), Childhood cancers (all types), Children's Oncology Group (COG), Vincristine and the tag soc-trials.
Shares Thiotepa, Autologous stem cell transplant (high-dose therapy), Brain and spinal cord tumours (all types), Childhood cancers (all types) and the tag soc-trials.
Shares Brain and spinal cord tumours (all types), Childhood cancers (all types), Children's Oncology Group (COG), Vincristine and the tag soc-trials.
Shares Autologous stem cell transplant (high-dose therapy), Childhood cancers (all types), Children's Oncology Group (COG), Vincristine and the tag soc-trials.
Shares Brain and spinal cord tumours (all types), Childhood cancers (all types), Children's Oncology Group (COG), Etoposide and the tag soc-trials.
Shares Childhood cancers (all types), Vincristine, Etoposide, Cyclophosphamide and the tag soc-trials.
Shares Childhood cancers (all types), Children's Oncology Group (COG), Etoposide, Cisplatin and the tag soc-trials.
Shares Childhood cancers (all types), Children's Oncology Group (COG), Vincristine, Etoposide and the tag soc-trials.