Intravenous
fludarabine is a well-established
therapy for the first-line treatment of
chronic lymphocytic leukemia and the standard of care for second-line treatment. More recently, an oral formulation of
fludarabine has been developed, with equivalent efficacy and tolerability to the intravenous formulation, but with improved convenience of administration and potentially greater cost effectiveness. In previously treated patients receiving oral
fludarabine monotherapy, overall response rates of 46-51% were achieved, depending on the response criteria used. Oral
fludarabine is also an effective first-line treatment, both as monotherapy (overall response 72-80%) and in combination with
cyclophosphamide (overall response 80%). Infusion-related adverse effects are eliminated with
oral administration. Importantly, WHO performance status is maintained or improved in more than 50% of patients. As oral
fludarabine can be taken at home, administration costs are greatly reduced due to fewer physician and nursing interventions and less time spent in hospital. Oral
fludarabine was approved first in the UK as second-line
therapy for
chronic lymphocytic leukemia and, based on its ease of administration and potentially greater cost effectiveness, is recommended in preference to the intravenous formulation by the UK National Institute for Clinical Excellence. The oral formulation is also now available in the majority of European countries. Therefore, with equivalent efficacy and tolerability to the intravenous preparation, oral
fludarabine gives the hematologist an important new option in the management of chronic
lymphocytic leukemia.