Review of clinical and haematological response to low‐dose cytosine arabinoside in acute myeloid leukaemia

15 patients with acute myeloid leukaemia (AML) were treated with low-dose cytosine arabinoside (LD ARA-C). 2 patients had complete remissions, which lasted for 8 and 3 months, and 5 patients had a partial remission. 46% of the patients thus responded to LD ARA-C. This included 1 responding patient who had not previously responded to therapy with 6-mercaptopurine, thioguanine, or vinblastine. The 2 patients with complete remission did not show LD ARA-C-induced hypoplasia of bone marrow, although 1 had hypoplastic AML before therapy. Leukaemic cells from 1 patient showed in vivo maturation from M1 to M3 after LD ARA-C treatment. The present results, together with the published data, indicate that: a. LD ARA-C treatment, although it may have some toxic effects, is an effective treatment for some patients with AML, especially those with hypoplastic AML; b. Response to LD ARA-C can be obtained after one or several courses of treatment; c. LD ARA-C-induced remissions are sometimes obtained even in patients who fail in more conventional treatments; d. LD ARA-C-induced remissions can be achieved without bone marrow hypoplasia, and induction of hypoplasia by itself does not always result in complete remission; e. LD ARA-C can induce in vivo maturation of leukaemic cells. It is suggested that induction of remission in AML patients by LD ARA-C may result from either differentiation of leukaemic blast cells, cytotoxicity to leukaemic blasts, or both mechanisms acting together.