Drug-induced aplastic anemia is one of the few life-threatening reactions to drugs. Although the majority of reported cases have been associated with chloramphenicol, many drugs have the potential to be toxic to the bone marrow. There are two distinct types of toxicity with differing pathogenic mechanisms--a dose-related reversible marrow aplasia and a dose-independent idiosyncratic aplasia with a high mortality. These two forms of marrow suppression may be difficult to distinguish. The pathogenesis of idiosyncratic marrow aplasia is not well understood. Various studies have demonstrated biochemical, immune, pharmacokinetic, and genetic defects that could affect hematopoietic stem cells. The clinical significance of the reported experimental findings is not established. The prognosis of drug-induced aplastic anemia is similar to that of idiopathic aplastic anemia. Patients with this condition respond to bone marrow transplantation or immunosuppressive therapy in a manner similar to patients with idiopathic marrow aplasia. Many questions regarding drug-induced aplastic anemia remain to be answered; little progress has been made in the last decade.