Background: Carboplatin is an analogue of cisplatin with less nonhematologic toxicity and a similar spectrum of antineoplastic activity. Although cisplatin has not been found to be an active agent against leukemia, carboplatin-induced complete remissions have been observed in adults with acute myelogenous leukemia (AML), and antileukemic activity has been observed in a Phase I trial involving children with acute lymphoblastic leukemia (ALL) and AML. Therefore, a pediatric Phase II study was undertaken to determine the degree of activity of carboplatin in childhood ALL and AML.
Methods: Between October 1991 and November 1994, the Children's Cancer Group conducted a Phase II study of carboplatin given by 5-day continuous intravenous infusion to children with acute leukemia recurring in bone marrow.
Results: Minimal antileukemic activity was demonstrated in patients with ALL and AML. One of 21 eligible patients with ALL achieved a partial response. Of 23 eligible patients with AML, including 1 patient with chronic myelogenous leukemia in blast crisis, 1 had hypocellular M1 bone marrow with a platelet count of 15,000/mm3, and 2 achieved partial responses. Nonhematologic toxicities, which were infrequent, included mild hepatic and renal dysfunction.
Conclusions: In this pediatric Phase II trial of carboplatin as a treatment for acute leukemia, minimal activity was demonstrated in patients with ALL and AML recurring in bone marrow. Further evaluation of carboplatin as a treatment for childhood leukemia, using the dose schedule of 216 mg/m2/day given by 5-day continuous intravenous infusion, does not appear warranted.