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Abstract
Hairy cell leukemia (HCL) is a rare and incurable disease that represents approximately 2% of lymphoid leukemias. Treatment is indicated for patients with symptomatic disease manifested by splenomegaly, cytopenias or serious infections. Purine nucleoside analogs (PNA), such as cladribine and pentostatin, are the preferred drugs for first line treatment in most cases. With PNA high rates of complete response and long disease-free periods are obtained, however, relapses are frequent. Second line treatment includes PNA combined with rituximab.
Even active, recurring PNA are associated with a significant accumulated toxicity. Hence, the need for investigate new therapeutic alternatives as moxetumomab pasudotox (CAT-8015). Two multicenter studies, one in phase I and one in phase III, had positive results, since moxetumomab pasudotox (CAT-8015) achieved high rates of complete responses, lasting responses and negative minimal residual disease, also proving safe for patients with relapsed or refractory HCL.
References
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