A hematopoietic stem cell transplant (HSCT) is the only curative treatment for patients with chronic myelogenous leukemia (CML).^1,2 According to the Center for International Blood and Marrow Transplant Research (CIBMTR), there are approximately 17,700 HSCTs conducted in North America each year. Approximately 41% of the total HSCTs are allogeneic, in which the stem cells are derived from a donor. About 70% of the allogeneic transplants are for leukemia and myeloproliferative diseases.

Conditioning Regimens for HSCT

Prior to stem cell transplantation, a conditioning or preparative regimen is administered to reduce the number of malignant cells and limit the patient's immune response.¹ IV BUSULFEX is a bifunctional alkylating agent commonly used as a component of a myeloablative conditioning regimen.

Chemotherapeutics and total body irradiation are used in conditioning regimens for HSCT. Transplant conditioning regimens can be either myeloablative or nonmyeloablative, depending on the objective of therapy:

• Myeloablative therapy uses high-dose regimens that provide maximum tumor cytoreduction while avoiding organ toxicities.³
• Nonmyeloablative therapy uses lower-dose regimens that provide myelosuppression and sufficient immune suppression to prevent graft rejection, while relying on an immune graft-versus-leukemia effect for transplant efficacy.⁴

More than 200 combinations of conditioning regimens have been reported to the Center for International Blood and Marrow Transplant Research (CIBMTR). The choice of conditioning regimen depends on multiple factors, with the patient’s disease and disease status at the time of transplant being important considerations. Delivery of the intended conditioning regimen is a key contributing factor in determining the overall success of the transplant.

1. Savage DG, Goldman JM. Allogeneic hematopoietic stem cell transplantation for chronic myeloid leukemia. In: Atkinson K, Chaplin R, Ritz J, Fibbe WE, Ljungman P, Brenner MK, eds. Clinical Bone Marrow and Blood Stem Cell Transplantation. 3rd ed. Cambridge, England: Cambridge University Press; 2004:814-831.
2. National Comprehensive Cancer Network. Clinical Practice Guidelines in Oncology–v.1.2007: Chronic Myelogenous Leukemia. Available at: http://www.nccn.org/professionals/physician_gls/f_guidelines.asp?button=I+Agree#site. Accessed October 6, 2006.
3. Bensinger WI. High-dose chemotherapy and chemoradiotherapy preparative regimens. In: Atkinson K, Chaplin R, Ritz J, Fibbe WE, Ljungman P, Brenner MK, eds. Clinical Bone Marrow and Blood Stem Cell Transplantation. 3rd ed. Cambridge, England: Cambridge University Press; 2004:337-356.
4. Giralt S, Khouri I, Champlin R. Non-myeloablative conditioning: induction of graft-versus-disease effect as a therapeutic modality. In: Atkinson K, Chaplin R, Ritz J, Fibbe WE, Ljungman P, Brenner MK, eds. Clinical Bone Marrow and Blood Stem Cell Transplantation. 3rd ed. Cambridge, England: Cambridge University Press; 2004:357-368.

The only FDA-approved agent for use in combination with cyclophosphamide as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous leukemia (CML)

Important Safety Information At the recommended dosage, IV BUSULFEX^® (busulfan) produced profound myelosuppression in all patients (ie, severe granulocytopenia, thrombocytopenia, anemia, or a combination thereof). Frequent complete blood counts should be monitored during treatment and until recovery. Hepatic veno-occlusive disease was diagnosed in 5/61 patients and was fatal in 2/5 cases. Anticonvulsant prophylactic therapy should be administered prior to treatment. Caution should be exercised in patients with a history of seizure disorder or head trauma or who are receiving other potentially epileptogenic drugs. Bronchopulmonary dysplasia with pulmonary fibrosis is a rare but serious condition following chronic busulfan therapy. Women of childbearing potential should be advised to avoid becoming pregnant as busulfan may cause fetal harm.

The most common nonhematologic adverse events were nausea (92% mild or moderate, 7% severe), stomatitis (71% grade 1-2, 26% grade 3-4), and vomiting (95% mild or moderate), anorexia (64% mild or moderate, 21% severe), diarrhea (75% mild or moderate, 5% grade 3-4), insomnia (83% mild or moderate, 1% severe), and fever (78% mild or moderate, 3% life-threatening).

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