What is Busulfan used to treat?

From the Guidelines

Busulfan is used to treat certain types of cancer, specifically chronic myeloid leukemia (CML) and other hematologic disorders, as part of conditioning regimens before hematopoietic stem cell transplantation. It works as an alkylating agent that interferes with DNA replication in rapidly dividing cells, thereby inhibiting cancer cell growth. The medication effectively destroys the patient's existing bone marrow cells to make room for new, healthy donor cells. Typical dosing varies based on the specific indication, patient characteristics, and whether it's being used alone or as part of a combination therapy. Common side effects include bone marrow suppression, nausea, vomiting, and potential long-term effects on fertility. Due to its potent effects and narrow therapeutic window, busulfan treatment requires careful monitoring of blood counts and is typically administered under close medical supervision by oncologists or hematologists 1.

Key Points

• Busulfan is used in the treatment of certain cancers, including CML and as part of conditioning regimens for hematopoietic stem cell transplantation.
• It is an alkylating agent that interferes with DNA replication in rapidly dividing cells.
• The medication is used to destroy existing bone marrow cells to make room for new, healthy donor cells.
• Common side effects include bone marrow suppression, nausea, vomiting, and potential long-term effects on fertility.
• Busulfan treatment requires careful monitoring of blood counts and is typically administered under close medical supervision by oncologists or hematologists.

Treatment Considerations

• The choice of conditioning regimen may influence outcome, with myeloablative total body irradiation (TBI) showing a clear benefit in pediatric ALL, but busulfan-based regimens being an alternative for patients who cannot tolerate TBI 1.
• Reduced-intensity conditioning (RIC) regimens may be considered for older patients or those with comorbidities, but the role of RIC in these patients remains uncertain 1.
• Busulfan is commonly used in combination with other medications, such as fludarabine or cyclophosphamide, as part of conditioning regimens for hematopoietic stem cell transplantation 1.

Recent Evidence

• A recent study found that busulfan-based conditioning regimens were associated with similar outcomes to TBI-based regimens in patients with acute myeloid leukemia (AML) 1.
• Another study found that busulfan/melphalan was a preferred conditioning regimen in Europe, with superior EFS compared to cyclophosphamide/etoposide/melphalan (CEM) 1.

From the FDA Drug Label

BUSULFAN is an alkylating drug indicated for: Use in combination with cyclophosphamide as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous leukemia (CML) (1) BUSULFAN is indicated for use in combination with cyclophosphamide as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous leukemia. BUSULFAN is an alkylating drug indicated for: Use in combination with cyclophosphamide as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous leukemia (CML) (1)

Busulfan is used to treat chronic myelogenous leukemia (CML), specifically as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation in combination with cyclophosphamide 2, 2, 2.

From the Research

Busulfan Treatment

• Busulfan is used to treat chronic myelogenous leukemia (CML) 3, 4, 5, 6, 7
• It is also used in conditioning regimens for autologous or allogeneic bone marrow transplantation for acute or chronic leukemia and other nonmalignant disorders 3, 5, 6
• Busulfan has been used at conventional dosages (1-12 mg/d) for the treatment of CML 3
• High-dose busulfan (usually 16 mg/kg) is used in combination with other cytotoxic drugs, such as cyclophosphamide, as a preparative regimen for bone marrow transplantation 3, 5, 6

Efficacy and Safety

• Busulfan is a useful and sufficiently safe drug in the treatment of patients with CML 3
• The pharmacokinetics and metabolism of busulfan are age-dependent, with lower systemic exposure and tissue and tumor exposure in children than in adults 3
• Relationships between toxicity (principally neutropenia, hepatic veno-occlusive disease, incidence of seizures) and drug exposure have been found for busulfan 3
• Busulfan has been compared to other treatments, such as hydroxyurea and interferon-alpha, in terms of efficacy and safety 4, 7