Oral chemotherapy agents have been an important component of the treatment
of leukaemia for many years. Obstacles such as poor or erratic bioavailabil
ity and noncompliance have often limited the utility of oral agents in the
treatment of leukaemia. However, recent evaluations of new or existing oral
agents have expanded the clinician's options and understanding of the use
of these drugs in the treatment of leukaemia. One major advance is the use
of tretinoin (all-trans retinoic acid) in the treatment of acute promyelocy
tic leukaemia (APL). Tretinoin, an oral vitamin A derivative that reverses
abnormal differentiation in APL is now an essential component of first-line
therapy for APL, replacing standard intravenous chemotherapy induction reg
imens. Other advances include an increased understanding of the pharmacokin
etic and pharmacodynamic profile of oral chemotherapy agents such as etopos
ide and high dose busulfan, allowing for modifications or individualisation
of administration regimens to enhance efficacy or minimise toxicity. Evalu
ations of noncompliance with oral agents in the treatment of leukaemia have
also provided the clinician with important information on how this obstacl
e to oral therapy may be overcome or minimised.