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SIOP WT 2001

Omission of doxorubicin from the treatment of stage II-III, intermediate-risk Wilms' tumour (SIOP WT 2001): an open-label, non-inferiority, randomised controlled trial

Phase 3N = 583NCT00047138

Population
Children with stage II-III, histological intermediate-risk Wilms tumour after 4 weeks of preoperative vincristine and actinomycin D and delayed nephrectomy
Design
International, multicentre, open-label, non-inferiority, phase 3 randomised trial: children aged 6 months to 18 years from 251 hospitals in 26 countries who had received 4 weeks of preoperative vincristine and actinomycin D; 583 children with stage II (341) or stage III (242) intermediate-risk Wilms tumour assessed after delayed nephrectomy were randomly assigned 1:1 by minimisation, between Nov 1, 2001 and Dec 16, 2009, to vincristine and actinomycin D with five doses of doxorubicin 50 mg/m2 (standard) or without doxorubicin (experimental). A newly defined high-risk blastemal subtype was excluded from randomisation. Primary endpoint: non-inferiority of 2-year event-free survival, intention-to-treat analysis, margin 10%. The registry record NCT00047138 lists 350 (estimated) and status unknown.
Primary endpoint
EFS
Arms
  • Vincristine and actinomycin D with doxorubicin (control, n = 291): Vincristine 1.5 mg/m2 weeks 1-8, 11, 12, 14, 15, 17, 18, 20, 21, 23, 24, 26 and 27, plus actinomycin D 45 μg/kg every 3 weeks from week 2, with five doses of doxorubicin 50 mg/m2 every 6 weeks from week 2
  • Vincristine and actinomycin D without doxorubicin (experimental, n = 292): The same vincristine and actinomycin D schedule without doxorubicin
People
Pritchard-Jones K
Δ 24-month EFS−4.4 pointsΔ 60-month OS−0.7 points
Show the numbers
Results reported for SIOP WT 2001
EndpointAnalysisVincristine and actinomycin D without doxorubicinVincristine and actinomycin D with doxorubicinEffect
EFSprimarymedian follow-up 60.8 monthsPritchard-Jones 201588.2% at 2 y (84.5–92.1%)92.6% at 2 y (89.6–95.7%)Non-inferiority endpoint. Difference 4.4% (95% CI 0.4 to 9.3), which did not exceed the predefined 10% margin. Median follow-up 60.8 months (IQR 40.8 to 79.8).
OSRandomised children; the abstract does not state the analysis population for overall survivalprimarymedian follow-up 60.8 monthsPritchard-Jones 201595.8% at 5 y (93.3–98.4%)96.5% at 5 y (94.3–98.8%)Secondary to the non-inferiority endpoint; no test is printed in the abstract.
Results reported for SIOP WT 2001
EndpointAnalysisVincristine and actinomycin D without doxorubicinVincristine and actinomycin D with doxorubicinEffect
EFSprimarymedian follow-up 60.8 monthsPritchard-Jones 201588.2% at 2 y (84.5–92.1%)92.6% at 2 y (89.6–95.7%)Non-inferiority endpoint. Difference 4.4% (95% CI 0.4 to 9.3), which did not exceed the predefined 10% margin. Median follow-up 60.8 months (IQR 40.8 to 79.8).
OSRandomised children; the abstract does not state the analysis population for overall survivalprimarymedian follow-up 60.8 monthsPritchard-Jones 201595.8% at 5 y (93.3–98.4%)96.5% at 5 y (94.3–98.8%)Secondary to the non-inferiority endpoint; no test is printed in the abstract.
Primary publication: Pritchard-Jones K, Bergeron C, de Camargo B, van den Heuvel-Eibrink MM, Acha T, Godzinski J, et al. Omission of doxorubicin from the treatment of stage II-III, intermediate-risk Wilms' tumour (SIOP WT 2001): an open-label, non-inferiority, randomised controlled trial. Lancet. 2015;386(9999):1156-64. PubMedDOI

Appears in

Sources

  1. Pritchard-Jones K, Bergeron C, de Camargo B, van den Heuvel-Eibrink MM, Acha T, Godzinski J, et al. Omission of doxorubicin from the treatment of stage II-III, intermediate-risk Wilms' tumour (SIOP WT 2001): an open-label, non-inferiority, randomised controlled trial. Lancet. 2015;386(9999):1156-64.

    PubMedDOI