{"entity":{"id":"paper-hr-nbl1-busulfan-melphalan-ladenstein-lancet-oncol-2017","kind":"paper","name":"HR-NBL1/SIOPEN: busulfan-melphalan versus carboplatin-etoposide-melphalan high-dose chemotherapy for high-risk neuroblastoma","aka":[],"tldr":"Busulfan and melphalan as the high-dose conditioning before stem cell rescue gave better event-free survival and less severe toxicity than the carboplatin-etoposide-melphalan regimen in high-risk neuroblastoma, becoming Europe's standard.","summary":"Phase 3 trial within the European HR-NBL1 study randomising 598 children with high-risk neuroblastoma after rapid COJEC induction to busulfan-melphalan or carboplatin-etoposide-melphalan (CEM) high-dose chemotherapy with autologous stem cell rescue.\n\nThree-year event-free survival was 50 versus 38 percent (hazard ratio about 0.7), overall survival 60 versus 48 percent, and severe toxicity and toxic deaths were fewer with busulfan-melphalan.","asOf":"2026-09-17","links":[{"label":"Lancet Oncol 2017","url":"https://doi.org/10.1016/S1470-2045(17)30070-0"},{"label":"PubMed","url":"https://pubmed.ncbi.nlm.nih.gov/28259608/"}],"tags":[],"related":[],"cancers":["neuroblastoma-high-risk"],"sections":[],"technologies":[],"targets":[],"drugs":["busulfan","carboplatin","etoposide"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["hr-nbl1"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":["lancet-oncology"],"dependsOn":[],"notes":[],"journal":"The Lancet Oncology","year":2017,"doi":"10.1016/S1470-2045(17)30070-0","pmid":"28259608","authors":"Ladenstein R, Pötschger U, Pearson ADJ, et al.","paperType":"rct","findings":["Three-year event-free survival 50 percent vs 38 percent.","Severe acute toxicity 4 percent vs 10 percent."],"whatItMeans":"Busulfan-melphalan is the standard single high-dose regimen for high-risk neuroblastoma in Europe and many other regions; North America uses tandem transplant.","caveats":["Randomisation occurred after induction, so early failures are excluded.","Veno-occlusive disease is the characteristic busulfan toxicity."],"changedPractice":true,"participants":598},"route":"/key-papers/paper-hr-nbl1-busulfan-melphalan-ladenstein-lancet-oncol-2017/","neighbours":{"cancer":[{"id":"neuroblastoma-high-risk","kind":"cancer","name":"High-risk neuroblastoma","route":"/cancers/neuroblastoma-high-risk/"}],"drug":[{"id":"busulfan","kind":"drug","name":"Busulfan","route":"/drugs/busulfan/"},{"id":"carboplatin","kind":"drug","name":"Carboplatin","route":"/drugs/carboplatin/"},{"id":"etoposide","kind":"drug","name":"Etoposide","route":"/drugs/etoposide/"}],"trial":[{"id":"hr-nbl1","kind":"trial","name":"SIOPEN HR-NBL1","route":"/trials/hr-nbl1/"}],"journal":[{"id":"lancet-oncology","kind":"journal","name":"The Lancet Oncology","route":"/journals/lancet-oncology/"}]}}