{"entity":{"id":"busulfan","kind":"drug","name":"Busulfan","aka":["Busilvex"],"tldr":"Busulfan is one of the oldest cancer drugs. As a tablet it controlled chronic myeloid leukaemia before imatinib; as an infusion it is used to clear the bone marrow before a stem cell transplant.","summary":"Oral busulfan (Myleran) was approved in 1954 for palliative treatment of chronic myelogenous leukaemia and was the standard until hydroxyurea, interferon and then imatinib displaced it. Intravenous busulfan (Busulfex, approved 1999) is indicated with cyclophosphamide as a conditioning regimen before allogeneic haematopoietic progenitor cell transplantation for CML, and pharmacokinetically targeted dosing is routine in transplant units. The EU authorised Busilvex in 2003 for conditioning before transplantation in adults and children. Hepatic sinusoidal obstruction syndrome, seizures (anticonvulsant prophylaxis is given) and prolonged myelosuppression are the main risks; pulmonary fibrosis (busulfan lung) follows chronic oral use.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Busulfan","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=busulfan"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/busulfan"},{"label":"EPAR (Busilvex)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/busilvex"}],"tags":["nci-list"],"related":[],"cancers":["cml"],"sections":[],"technologies":["cytotoxic-chemotherapy","allogeneic-hsct"],"targets":[],"drugs":[],"companies":["otsuka"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Myleran / Busulfex","modality":"Alkylating agent (alkyl sulfonate)","mechanism":"Bifunctional alkylating agent that cross-links DNA; highly lipophilic and myeloablative at transplant doses.","approvals":[{"region":"US","year":1954,"indication":"Palliative treatment of chronic myelogenous leukaemia (Myleran tablets)"},{"region":"US","year":1999,"indication":"Conditioning with cyclophosphamide before allogeneic HPC transplantation for CML (Busulfex)"},{"region":"EU","year":2003,"indication":"Conditioning before haematopoietic progenitor cell transplantation (Busilvex)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},"route":"/drugs/busulfan/","neighbours":{"cancer":[{"id":"cml","kind":"cancer","name":"Chronic myeloid leukaemia (CML)","route":"/cancers/cml/"},{"id":"neuroblastoma-high-risk","kind":"cancer","name":"High-risk neuroblastoma","route":"/cancers/neuroblastoma-high-risk/"},{"id":"primary-cns-lymphoma","kind":"cancer","name":"Primary CNS lymphoma","route":"/cancers/primary-cns-lymphoma/"}],"technology":[{"id":"allogeneic-hsct","kind":"technology","name":"Allogeneic stem cell transplantation","route":"/technologies/allogeneic-hsct/"},{"id":"cytotoxic-chemotherapy","kind":"technology","name":"Cytotoxic chemotherapy","route":"/technologies/cytotoxic-chemotherapy/"},{"id":"rejuv-paed-fertility-male","kind":"technology","name":"Fertility in boys and young men treated for cancer, including testicular tissue banking","route":"/technologies/rejuv-paed-fertility-male/"},{"id":"rejuv-paed-fertility-female","kind":"technology","name":"Fertility in girls and young women treated for cancer, including ovarian tissue freezing","route":"/technologies/rejuv-paed-fertility-female/"},{"id":"pkpd-modelling","kind":"technology","name":"Pharmacokinetic and pharmacodynamic modelling","route":"/technologies/pkpd-modelling/"}],"company":[{"id":"otsuka","kind":"company","name":"Otsuka Pharmaceutical","route":"/companies/otsuka/"},{"id":"pierre-fabre","kind":"company","name":"Pierre Fabre","route":"/companies/pierre-fabre/"}],"trial":[{"id":"nct00822393","kind":"trial","name":"Clinical Phase III Trial Treosulfan-based Conditioning Versus Reduced-intensity Conditioning (RIC)","route":"/trials/nct00822393/"},{"id":"nct04221035","kind":"trial","name":"High-Risk Neuroblastoma Study 2 of SIOP-Europa-Neuroblastoma (SIOPEN)","route":"/trials/nct04221035/"},{"id":"nct02506959","kind":"trial","name":"Panobinostat, Gemcitabine Hydrochloride, Busulfan, and Melphalan Before Stem Cell Transplant in Treating Patients With Refractory or Relapsed Multiple Myeloma","route":"/trials/nct02506959/"},{"id":"hr-nbl1","kind":"trial","name":"SIOPEN HR-NBL1","route":"/trials/hr-nbl1/"},{"id":"nct05917405","kind":"trial","name":"Study Comparing the Efficacy of 2 RIC Regimens (Clofarabine vs Fludarabine) in Adults With AML Eligible to Allo-SCT","route":"/trials/nct05917405/"}],"term":[{"id":"lymphoma-tx-fertility-preservation","kind":"term","name":"Fertility before lymphoma treatment: what to ask for, and when","route":"/terms/lymphoma-tx-fertility-preservation/"},{"id":"alopecia-persistent-chemotherapy","kind":"term","name":"Persistent chemotherapy-induced alopecia","route":"/terms/alopecia-persistent-chemotherapy/"},{"id":"lymphoma-tx-transplant-role","kind":"term","name":"Stem cell transplant in lymphoma: what it is still for","route":"/terms/lymphoma-tx-transplant-role/"}],"drug":[{"id":"mitobronitol","kind":"drug","name":"Mitobronitol","route":"/drugs/mitobronitol/"}],"paper":[{"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","route":"/key-papers/paper-hr-nbl1-busulfan-melphalan-ladenstein-lancet-oncol-2017/"}]}}