ANBL00P2
Needs reviewPerinatal Neuroblastoma: Expectant Observation A Children's Oncology Group Pilot Study
N = 87NCT00445718
- Population
- Infants aged 6 months or younger (the abstract says younger than 6 months; the full text eligibility is 6 months or younger) with small adrenal masses and no evidence of spread beyond the primary tumour
- Design
- Prospective Children's Oncology Group study (open to accrual July 2001 to January 2010; registry: observational natural-history study with no phase) of expectant observation for infants aged 6 months or younger with small adrenal masses and no evidence of spread beyond the primary tumour. Parents could choose observation or immediate surgical resection. Serial abdominal sonograms and urinary vanillylmandelic acid and homovanillic acid were measured over 90 weeks; infants with a 50% increase in mass volume, an increase in urine catecholamine values, or a homovanillic acid to vanillylmandelic acid ratio above 2 were referred for resection. 87 eligible patients in the paper: 83 chose observation, 4 immediate surgery (registry record NCT00445718: enrolment 97, actual).
- Arms
- Eligible infants: expectant observation or parent-chosen resection (experimental, n = 87): Expectant observation with serial sonograms and urinary catecholamine measurements, with resection if protocol criteria were met; 4 of 87 chose immediate resection
- People
- Nuchtern JG, Children's Oncology Group
Show the numbers
| Endpoint | Analysis | Eligible infants: expectant observation or parent-chosen resection | Control | Effect |
|---|---|---|---|---|
| EFSEntire cohort of eligible patients (n = 87), including 4 who chose immediate surgery; event = neuroblastoma event | finalmedian follow-up 3.2 yearsNuchtern 2012 | 97.7% at 3 y | — | Reported as 97.7% ± 2.2%; the full-text Methods state that 3-year estimates are presented ± standard error. A 'neuroblastoma event' is progression or recurrence to INSS stage 2 or higher after resection (full text). The objective was stated as showing excellent event-free and overall survival; the paper names no primary endpoint. The median follow-up of 3.2 years is that of patients without an event (full text). No confidence interval is given. |
| OSEntire cohort of eligible patients (n = 87) | finalmedian follow-up 3.2 yearsNuchtern 2012 | 100% at 3 y | — | Reported as 100%. The median follow-up of 3.2 years is that of patients without an event (full text). |
| EFSEligible patients (87) in the long-term report | long term-updateDholaria 2025 | 93.7% at 10 y | — | Reported in the abstract without an interval. Event-free survival in this report counts any neuroblastoma event, any malignant event or death from any cause, a different definition from the 3-year figure (neuroblastoma event). Unreconciled: the full-text Results give 10-year EFS 93.3% (± 8.1%) and OS 97.9% (± 4.5%) for the 83 infants on the observation arm, not 93.7% and 98.0% for 87; the full text does not show how the abstract values were derived. |
| OSEligible patients (87) in the long-term report | long term-updateDholaria 2025 | 98% at 10 y | — | Reported in the abstract without an interval; see the note on the 10-year EFS for the difference from the full-text Results (97.9% for the 83 infants on the observation arm). |
| Endpoint | Analysis | Eligible infants: expectant observation or parent-chosen resection | Control | Effect |
|---|---|---|---|---|
| EFSEntire cohort of eligible patients (n = 87), including 4 who chose immediate surgery; event = neuroblastoma event | finalmedian follow-up 3.2 yearsNuchtern 2012 | 97.7% at 3 y | — | Reported as 97.7% ± 2.2%; the full-text Methods state that 3-year estimates are presented ± standard error. A 'neuroblastoma event' is progression or recurrence to INSS stage 2 or higher after resection (full text). The objective was stated as showing excellent event-free and overall survival; the paper names no primary endpoint. The median follow-up of 3.2 years is that of patients without an event (full text). No confidence interval is given. |
| OSEntire cohort of eligible patients (n = 87) | finalmedian follow-up 3.2 yearsNuchtern 2012 | 100% at 3 y | — | Reported as 100%. The median follow-up of 3.2 years is that of patients without an event (full text). |
| EFSEligible patients (87) in the long-term report | long term-updateDholaria 2025 | 93.7% at 10 y | — | Reported in the abstract without an interval. Event-free survival in this report counts any neuroblastoma event, any malignant event or death from any cause, a different definition from the 3-year figure (neuroblastoma event). Unreconciled: the full-text Results give 10-year EFS 93.3% (± 8.1%) and OS 97.9% (± 4.5%) for the 83 infants on the observation arm, not 93.7% and 98.0% for 87; the full text does not show how the abstract values were derived. |
| OSEligible patients (87) in the long-term report | long term-updateDholaria 2025 | 98% at 10 y | — | Reported in the abstract without an interval; see the note on the 10-year EFS for the difference from the full-text Results (97.9% for the 83 infants on the observation arm). |
Appears in
Sources
Nuchtern JG, London WB, Barnewolt CE, Naranjo A, McGrady PW, Geiger JD, et al. A prospective study of expectant observation as primary therapy for neuroblastoma in young infants: a Children's Oncology Group study. Ann Surg. 2012;256(4):573-80.
Dholaria H, Naranjo A, Zhang FF, London WB, Diller L, Shamberger RC, et al. Long-Term Outcome of Young Infants With Suspected Neuroblastoma following Observation as Primary Therapy: A Report From the Children's Oncology Group. Pediatr Blood Cancer. 2025;72(8):e31829.