Hairy cell leukemia (HCL) was once considered an untreatable form of chronic lymphoid
malignancy. Based upon the recognition of the importance of
adenosine deaminase to the normal B cell survival and proliferation, a hypothesis was developed that temporary inhibition of this
enzyme might be therapeutically successful in treating
chronic B cell leukemias.
Pentostatin was initially explored in patients with refractory
chronic lymphocytic leukemia (CLL). Both
pentostatin and
cladribine,
purine nucleoside analogs, have been utilized to successfully treat HCL. The high degree of complete and durable remission observed with either agent resulted in many believing that the treatment of this
rare disease had been fully optimized. However, a considerable number of patients will relapse. While tremendous progress has been made in initial management, the issues related to optimal
therapy, timing of initiation of treatment, and discovery of novel agents that may be effective in those who have relapsed are important. Investigational agents currently being explored in
chronic lymphocytic leukemia may also have benefit for those patients who have relapsed or are resistant to
therapy of
hairy cell leukemia. Many important questions remain (e.g. importance of
minimal residual disease) and will require international collaboration to fully address these unanswered questions. The
Hairy Cell Leukemia Consortium was established to address these unanswered questions.