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

In a phase II study, BUSULFEX was used in combination with IV cyclophosphamide as a conditioning regimen in 61 patients prior to allogeneic stem cell transplantation from an HLA-matched sibling donor. The clinical end points of the study were regimen-related toxicity pattern, engraftment, overall survival, disease-free survival, and relapse.

Patients in the phase II trial had various hematologic malignancies¹

• CML (n=17; 27%)²
• Acute myelogenous leukemia (n=26; 43%)²
• Lymphoma (n=9; 15%)²
• Myelodysplastic syndrome (n=9; 15%)²

All evaluable patients (60/60) engrafted at a median of 13 days post-transplant (range, 9 to 29 days)¹

There were no reports of graft failure¹

Prospective, single-arm, open-label trial of 61 patients with hematologic cancers—CML (17; 27%), acute myelogenous leukemia (26; 43%), lymphoma (9; 15%), and myelodysplastic syndrome (9; 15%)—who received IV BUSULFEX doses of 0.8 mg/kg every 6 hours as a 2-hour infusion for 4 days, for a total of 16 doses, followed by cyclophosphamide 60 mg/kg once per day for 2 days (BuCy2 regimen). Patients (median age, 37 years; range, 20-63 years) had an allogeneic stem cell transplant from an HLA*-matched sibling donor. Forty-eight percent of patients (29/61) were heavily pretreated, and 75% (46/61) were transplanted with active disease.

38% (23/61) of patients relapsed at a median of 183 days post-transplant (range, 36 to 406 days)¹

70% (43/61) of patients were alive at a median follow-up of 288 days post-transplant (range, 51 to 583 days)¹

62% (38/61) of patients were disease-free at a median time to follow-up of 269 days (range, 20 to 583 days)¹

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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