A3961
Treatment for Infants and Children With Intermediate Risk Neuroblastoma: A Phase III Intergroup CCG/POG Study
Phase 3N = 479NCT00003093
- Population
- Newly diagnosed intermediate-risk neuroblastoma without MYCN amplification: infants (younger than 365 days) with stage 3 or 4 disease, children aged 365 days or older with stage 3 tumours with favorable histopathological features, and infants with stage 4S disease with a diploid DNA index or unfavorable histopathological features.
- Design
- Prospective, phase 3, nonrandomised Children's Oncology Group intergroup trial, 1997 to 2005, of reduced duration and dose of chemotherapy assigned by tumour biology. Patients with favorable histopathological features and hyperdiploidy were assigned to four cycles; those with an incomplete response or either unfavorable feature were assigned to eight cycles. The aim was to determine whether 3-year estimated overall survival above 90% could be maintained. 479 eligible patients are reported in the paper (3-year survival estimates for the 476 with follow-up data; registry record NCT00003093: enrolment 573, actual, a difference the paper does not explain).
- Primary endpoint
- OS
- Arms
- Biology-based reduced chemotherapy (experimental, n = 479): Four or eight cycles of chemotherapy at reduced duration and dose, assigned by tumour biology (histopathology, DNA index) and response
- People
- Baker DL, Children's Oncology Group
Show the numbers
| Endpoint | Analysis | Biology-based reduced chemotherapy | Control | Effect |
|---|---|---|---|---|
| OSEligible patients with follow-up data (476 of 479 enrolled) | primaryBaker 2010 | 96% at 3 y (94–97%) | — | Reported as 96 ± 1% (± is the standard error, stated in the abstract and full text) with 95% CI 94 to 97 (full text, Results). The abstract says 'entire group'; the full text specifies 476 of 479 eligible patients with follow-up data. |
| EFSEligible patients with follow-up data (476 of 479 enrolled) | finalBaker 2010 | 88% at 3 y (84–90%) | — | Reported as 88 ± 2% (± is the standard error) with 95% CI 84 to 90 in the full text; the abstract does not give event-free survival. Median follow-up of patients who survived without an event was 5.2 years. The protocol monitored whether a 3-year EFS of 90% was maintained. |
| OSPatients with tumours with favorable biologic features (323 patients) | finalBaker 2010 | 98% at 3 y | — | Reported in the abstract as 98 ± 1% (± is the standard error, stated in the abstract); no confidence interval is given. The abstract reports 323 and 141 patients with favorable and unfavorable features; the full text counts 464 patients with fully known biologic features (15 more had incomplete assessment) and 461 with known biologic type and follow-up data. |
| OSPatients with tumours with unfavorable biologic features (141 patients) | finalBaker 2010 | 93% at 3 y | — | Reported in the abstract as 93 ± 2% (± is the standard error, stated in the abstract); no confidence interval is given. The abstract reports 323 and 141 patients with favorable and unfavorable features; the full text counts 464 patients with fully known biologic features (15 more had incomplete assessment) and 461 with known biologic type and follow-up data. |
| Endpoint | Analysis | Biology-based reduced chemotherapy | Control | Effect |
|---|---|---|---|---|
| OSEligible patients with follow-up data (476 of 479 enrolled) | primaryBaker 2010 | 96% at 3 y (94–97%) | — | Reported as 96 ± 1% (± is the standard error, stated in the abstract and full text) with 95% CI 94 to 97 (full text, Results). The abstract says 'entire group'; the full text specifies 476 of 479 eligible patients with follow-up data. |
| EFSEligible patients with follow-up data (476 of 479 enrolled) | finalBaker 2010 | 88% at 3 y (84–90%) | — | Reported as 88 ± 2% (± is the standard error) with 95% CI 84 to 90 in the full text; the abstract does not give event-free survival. Median follow-up of patients who survived without an event was 5.2 years. The protocol monitored whether a 3-year EFS of 90% was maintained. |
| OSPatients with tumours with favorable biologic features (323 patients) | finalBaker 2010 | 98% at 3 y | — | Reported in the abstract as 98 ± 1% (± is the standard error, stated in the abstract); no confidence interval is given. The abstract reports 323 and 141 patients with favorable and unfavorable features; the full text counts 464 patients with fully known biologic features (15 more had incomplete assessment) and 461 with known biologic type and follow-up data. |
| OSPatients with tumours with unfavorable biologic features (141 patients) | finalBaker 2010 | 93% at 3 y | — | Reported in the abstract as 93 ± 2% (± is the standard error, stated in the abstract); no confidence interval is given. The abstract reports 323 and 141 patients with favorable and unfavorable features; the full text counts 464 patients with fully known biologic features (15 more had incomplete assessment) and 461 with known biologic type and follow-up data. |