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HR-NBL1/SIOPEN

HR-NBL1/SIOPEN: busulfan and melphalan versus carboplatin, etoposide and melphalan as high-dose chemotherapy

Phase 3N = 598NCT01704716

Population
Patients aged 1 to 20 years with high-risk neuroblastoma; 1,347 enrolled between June 2002 and October 2010, 676 eligible for random allocation, 598 randomised.
Design
International, randomised, multi-arm, open-label phase 3 (128 institutions in 18 countries); 1:1 randomisation by minimisation to busulfan-melphalan or carboplatin-etoposide-melphalan after induction and adequate response, then local radiotherapy and maintenance. Primary endpoint 3-year EFS, intention to treat.
Primary endpoint
EFS
Arms
  • Carboplatin, etoposide, melphalan (control, n = 302): Carboplatin, etoposide and melphalan high-dose chemotherapy with stem-cell rescue
  • Busulfan and melphalan (experimental, n = 296): Busulfan (oral, then intravenous after 8 November 2007) and melphalan 140 mg/m2 high-dose chemotherapy with stem-cell rescue
People
Ladenstein R, senior author Valteau-Couanet D, SIOP Europe Neuroblastoma Group (SIOPEN)
Δ 36-month EFS+12 points
Show the numbers
Results reported for HR-NBL1/SIOPEN
EndpointAnalysisBusulfan and melphalanCarboplatin, etoposide, melphalanEffect
EFSITT (598 randomised patients)primarymedian follow-up 7.2 yearsLadenstein 201750% at 3 y (45–56%)38% at 3 y (32–43%)P=0.0005Median follow-up 7.2 years (IQR 5.3 to 9.2). 146 of 296 events with busulfan-melphalan and 188 of 302 with carboplatin-etoposide-melphalan at 3 years.
Results reported for HR-NBL1/SIOPEN
EndpointAnalysisBusulfan and melphalanCarboplatin, etoposide, melphalanEffect
EFSITT (598 randomised patients)primarymedian follow-up 7.2 yearsLadenstein 201750% at 3 y (45–56%)38% at 3 y (32–43%)P=0.0005Median follow-up 7.2 years (IQR 5.3 to 9.2). 146 of 296 events with busulfan-melphalan and 188 of 302 with carboplatin-etoposide-melphalan at 3 years.
Primary publication: Ladenstein R, Pötschger U, Pearson ADJ, Brock P, Luksch R, Castel V, et al. Busulfan and melphalan versus carboplatin, etoposide, and melphalan as high-dose chemotherapy for high-risk neuroblastoma (HR-NBL1/SIOPEN): an international, randomised, multi-arm, open-label, phase 3 trial. Lancet Oncol. 2017;18(4):500-514. PubMedDOI

Appears in

Sources

  1. Ladenstein R, Pötschger U, Pearson ADJ, Brock P, Luksch R, Castel V, et al. Busulfan and melphalan versus carboplatin, etoposide, and melphalan as high-dose chemotherapy for high-risk neuroblastoma (HR-NBL1/SIOPEN): an international, randomised, multi-arm, open-label, phase 3 trial. Lancet Oncol. 2017;18(4):500-514.

    PubMedDOI