Abstract
Background
Homoharringtonine (HHT) is a plant alkaloid that is derived from a Chinese evergreen tree. Its mechanism of action is thought to be by inhibition of protein, DNA, and RNA synthesis by inhibition of chain initiation. The treatment of acute myelogenous leukemia (AML) in children, has been hampered by few new effective agents developed in the past 30 years. Significant numbers of children still die of this disease. The purpose of this study is to evaluate the efficacy and toxicity of HHT for the therapy of refractory AML in children.
Procedure
Patients entered the study and were treated with HHT 7mg/m^2^/day for 10 days. The cycles could be repeated every 21 days depending on recovery from myelosuppression.
Results
Thirtyβseven patients entered the study, with twentyβeight evaluable for response. Complete response was obtained in four and a partial response in one (response rate 5/28β=β18%). Significant toxicities included prolonged severe myelosuppression in all responsive patients and neuropathogenic pain in two patients. The median duration of response was 62 days with a range of 28β126 days.
Conclusions
HHT has activity against chemotherapy resistant AML in children, with tolerable toxicity. This agent warrants further clinical evaluation in combination with other agents or perhaps biologic response modifiers which will hopefully lead to useful therapeutic options. Med Pediatr Oncol 2001;37:103β107. Β© 2001 WileyβLiss, Inc.