ACNS0333
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.
Overview
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.
- The treated group had about 57 percent lower chance of the event at any given time (hazard ratio 0.43).
- The absolute difference, how many more people out of 100 were helped, is not reported here.
- This is a surrogate endpoint: it measures the cancer being controlled or absent on scans and tests, which often, but not always, translates into living longer.
- 37 out of 100 people free of a major event at 4 years with ACNS0333 regimen.
- There was no comparison group, so this number cannot tell you how much of the effect is due to the treatment.
- This is a surrogate endpoint: it measures the cancer being controlled or absent on scans and tests, which often, but not always, translates into living longer.
- 43 out of 100 people alive at 4 years with ACNS0333 regimen.
- There was no comparison group, so this number cannot tell you how much of the effect is due to the treatment.
- Overall survival counts deaths from any cause, so it is the most direct measure of whether a treatment helps people live longer.
- ACNS0333 regimen: 4 participants.
- This endpoint is not one of the standard survival or response measures; read it alongside the trial's primary result.
- These results apply to the people the trial enrolled: Newly diagnosed atypical teratoid/rhabdoid tumour of the central nervous system from birth to 22 years: surgery, two courses of induction chemotherapy, three courses of high-dose chemotherapy with peripheral blood stem cell rescue and involved-field conformal radiotherapy timed by age and disease extent. People in a different situation may not see the same effect.
- Only 70 people took part, so the numbers are less certain than in a large trial.
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 |
Similar pages
not linked directly; found by shared links- TrialACNS0121
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- TrialACNS1123
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- TrialMATRix/IELSG43
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