Mitoxantrone as a single agent and in combination chemotherapy in patients with refractory acute leukemia.

A total of 47 patients with relapsed or primarily refractory leukemia were treated with mitoxantrone alone or in combination with vincristine sulfate and prednisone or cytarabine. Eligible patients included those with adequate renal and hepatic function, normal left ventricular ejection fraction, and those who had received previous treatment. When mitoxantrone was given alone in a once daily times five schedule, 5 of 12 acute lymphoblastic leukemia patients achieved complete remission; 4 of these patients had been refractory to reinduction and 1 to induction chemotherapy with anthracycline-containing treatments. Four of these patients had progressive disease, and three died during induction. Of 12 patients with acute myeloid leukemia, 1 had a complete remission, 1 had a partial remission, 8 had progressive disease, and 2 died during induction. Mitoxantrone was also found to be active in two patients in the blastic transformation of chronic myeloid leukemia with a response in one patient lasting 17 weeks. Combinations of mitoxantrone with vincristine sulfate and prednisone resulted in complete remission in four of nine acute lymphoblastic leukemia patients and one of four patients with Tdt-positive chronic myeloid leukemia in blast crisis. Three of these patients had not experienced a prior remission following anthracycline-containing treatments. Partial remission occurred in two of the acute lymphoblastic leukemia patients and one of the Tdt-positive chronic myeloid leukemia patients. Two of this latter group of patients died in induction. Treatment with mitoxantrone and cytarabine resulted in two acute myeloid leukemia patients achieving complete remission and one a partial remission; two patients had progressive disease, and one died in induction. No response was seen in a patient with Tdt-negative chronic myeloid leukemia after two courses of treatment. One patient with acute leukemia in the course of myelofibrosis died in induction. All the patients achieving complete remission are alive and have been in complete remission from 2 to 12 months. Side effects included mild nausea and vomiting in 9 of 13 patients treated with the mitoxantrone-vincristine sulfate-prednisone combination, and in 3 of 8 patients treated with the mitoxantrone-cytarabine combination. Other side effects of the combination treatments include drug-induced oral mucositis (of a lesser degree than with mitoxantrone alone), transient hepatic abnormalities, and infectious complications, such as sepsis, Candida sp colonization of the upper digestive tract, and soft tissue cellulitis, in a few patients.(ABSTRACT TRUNCATED AT 400 WORDS)