Substance / Medication

Busulphan

Overview

Active Ingredient

busulfan

RxNorm CUI

1828

Indications

BUSULFEX is indicated for use in combination with cyclophosphamide as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous leukemia.

Labeler: Otsuka America Pharmaceutical, Inc.Updated: 2025-10-20T00:00:00.000ZFull label on DailyMed

Boxed Warning

FDA Black Box Warning

Warnings and Precautions (5.1) [see]. BUSULFEX (busulfan) Injection causes severe and prolonged myelosuppression at the recommended dosage. Hematopoietic progenitor cell transplantation is required to prevent potentially fatal complications of the prolonged myelosuppression

Contraindications

When this intervention should not be used

BUSULFEX is contraindicated in patients with a history of hypersensitivity to any of its components.

Indications & Related Conditions

Conditions associated via SNOMED clinical relationships

Myelofibrosis

Myelosclerosis with myeloid metaplasia

Administration & Protocol

Dosing, route, and treatment protocol

Detailed dosage and administration information is available in the full FDA drug label.

View full prescribing information on DailyMed

Monitoring & Follow-Up

Biomarkers relevant to this intervention via related conditions

No monitoring biomarkers have been mapped yet. Biomarker-intervention linkages are derived through related conditions and will expand as the knowledge graph grows.

Research Evidence

Published studies and systematic reviews

Sort:

Total body irradiation plus cyclophosphamide versus busulphan with cyclophosphamide as conditioning regimen for patients with leukemia undergoing allogeneic stem cell transplantation: a meta-analysis.

Shi-Xia Xu, Xian-Hua Tang, Hai-Qin Xu et al. · Leuk Lymphoma · 2010

PMID: 20055658Meta-Analysis

[Comparison of total body irradiation-cyclophosphamide versus busulphan-cyclophosphamide as conditioning regimens for myelogenous leukemia: a meta-analysis].

Xu Shi-Xia, Tang Xian-Hua, Chen Hai-Qing et al. · Zhongguo Shi Yan Xue Ye Xue Za Zhi · 2008

PMID: 19099643Meta-Analysis

Continuous infusion idarubicin and intravenous busulphan as conditioning regimen to autologous stem cell transplantation for patients with acute myeloid leukaemia.

Ferrara Felicetto, Mele Giuseppina, Palmieri Salvatore et al. · Hematol Oncol · 2009

PMID: 19475701Trial

Busulphan is safe and efficient treatment in elderly patients with essential thrombocythemia.

Shvidel L, Sigler E, Haran M et al. · Leukemia · 2007

PMID: 17611572Trial

High-dose idarubicin and busulphan as conditioning to autologous stem cell transplantation in adult patients with acute myeloid leukaemia.

Ferrara Felicetto, Palmieri Salvatore, De Simone Mariacarla et al. · Br J Haematol · 2005

PMID: 15638859Trial

Busulphan given as four single daily doses of 150 mg/m2 is safe and effective in children of all ages.

Shaw P J, Nath C, Berry A et al. · Bone Marrow Transplant · 2004

PMID: 15195074Trial

Comparison of busulphan, hydroxyurea and allogeneic bone marrow transplantation (BMT) in chronic myeloid leukaemia: BMT prolongs survival.

Olsson-Strömberg Ulla, Simonsson Bengt, Ahlgren Tomas et al. · Hematol J · 2004

PMID: 15570285Trial

Reduced-intensity allogeneic hematopoietic stem cell transplantation for myelodysplastic syndrome and acute myeloid leukemia with multilineage dysplasia using fludarabine, busulphan, and alemtuzumab (FBC) conditioning.

Ho Aloysius Y L, Pagliuca Antonio, Kenyon Michelle et al. · Blood · 2004

PMID: 15059843Trial

Fractionated TBI correlates with less T cell mixed chimerism but increased risk of relapse compared to busulphan in patients with haematological malignancies after allogeneic stem cell transplantation.

Mattsson J, Uzunel M, Remberger M et al. · Bone Marrow Transplant · 2003

PMID: 12942093Trial

A phase II trial of liposomal busulphan as an intravenous myeloablative agent prior to stem cell transplantation: 500 mg/m(2) as a optimal total dose for conditioning.

Hassan M, Nilsson C, Hassan Z et al. · Bone Marrow Transplant · 2002

PMID: 12476274Trial

Research data from MEDLINE/PubMed

Benefits & Expected Outcomes

Benefits, expected outcomes, efficacy data, and NNT (Number Needed to Treat) are pending physician authorship and evidence review.

Risks & Side Effects

Adverse reaction and safety data for this drug is sourced from the FDA-approved label.

View adverse reactions & drug interactions on DailyMed

Related Symptoms

Symptoms associated with conditions this intervention addresses

No related symptoms have been mapped yet. Symptom linkages are derived through associated conditions.

Alternatives & Comparisons

Alternative treatments, comparison data, and clinical decision support are pending physician authorship.

Medical Disclaimer

This information is for educational purposes only and does not constitute medical advice, diagnosis, or treatment. Always consult with a qualified healthcare provider regarding any medical condition or treatment plan.

Do not start, stop, or change any treatment without consulting your healthcare provider.

Quick Facts

Type: Substance / Medication
Fully Specified Name: Busulfan (substance)
SNOMED CT: 387138002
UMLS CUI: C0006463
RxNorm CUI: 1828
Labeler: Otsuka America Pharmaceutical, Inc.

Clinical Data

This intervention maps to 2 entities in the Ltrl knowledge graph.

Conditions

Biomarkers

Specialists

Symptoms

Data is sourced from SNOMED CT, UMLS, and the Ltrl clinical knowledge graph. Content sections marked as pending require physician authorship. Consult a healthcare provider before starting any treatment.