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.
100% (61/61) completed the 16-dose regimen1
18% incidence of acute graft-versus-host disease (GVHD) (10% grades 3-4)1
- 15% mild or moderate; 3% severe1
- No deaths prior to day 100 due to GVHD or its secondary complications2
- 3 deaths (5%) after day 100 due to GVHD1,2
The most common adverse events were nausea, stomatitis, and vomiting1
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)
WARNING: BUSULFEX® (busulfan) Injection is a potent cytotoxic drug that causes profound myelosuppression at the recommended dosage. It should be administered under the supervision of a qualified physician who is experienced in allogeneic hematopoietic stem cell transplantation, the use of cancer chemotherapeutic drugs, and the management of patients with severe pancytopenia. Appropriate management of therapy and complications is only possible when adequate diagnostic and treatment facilities are readily available. SEE "WARNINGS" SECTION OF FULL PRESCRIBING INFORMATION FOR INFORMATION REGARDING BUSULFAN-INDUCED PANCYTOPENIA IN HUMANS.
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).
Please see Full Prescribing Information.
