Clofarabine is a second-generation nucleoside analogue which has been rationally developed with the aim to combine the therapeutic qualities and avoid the toxic limitations of fludarabine and cladribine. Clofarabine has been reimbursed in Belgium for the treatment of paediatric patients up to the age of 21 years with relapsed or refractory Acute Lymphatic Leukaemia (ALL) after two or three preceding regimens respectively. Clinical efficacy has also been demonstrated in newly diagnosed and advanced Acute Myoblastic Leukaemia (AML). The drug is currently being investigated in several randomised trials in AML and ALL and as part of the conditioning regimen prior to stem cell transplantation. This article focuses on the pharmacology, toxicity and clinical efficacy of clofarabine.

(BELG J HEMATOL 2012;3:17–22)