The role of rituximab in combination with pentostatin or cladribine for the treatment of recurrent/refractory hairy cell leukemia

BACKGROUND. The purine analogs pentostatin and cladribine have revolutionized the treatment of hairy cell leukemia (HCL) with overall responses in greater than 85% of patients and a median progression‐free survival of up to 15 years. They continue to be effective at second‐ and even third‐line therapy; however, alternative treatments are needed for patients who are or have become refractory to these agents or whose remissions are shorter with each course of therapy. METHODS. The authors conducted a retrospective review of 8 patients who received pentostatin or cladribine combined concurrently (n = 6 patients) or sequentially (n = 2 patients) with rituximab at second‐line therapy (n = 3 patients) and at subsequent lines of therapy (n = 5 patients). Results from a previously reported database of 219 patients with HCL (73 patients who received second‐line therapy and 20 patients who received third‐line therapy) were used as a historic control group against which to measure benefit. RESULTS. All 8 patients responded to therapy, with 7 complete responses (CRs) (87.5%) and minimal toxicity. All patients who had CRs were negative for minimal residual disease (MRD). At a median follow‐up of 29 months (range, 5–39 months) 1 patient developed recurrent disease, and the estimated 2‐year recurrence rate was 20% (0% after second‐line therapy and 25% after subsequent lines of therapy). In the historic control group, the CR rates were 70% after second‐line therapy and 45% after third‐line therapy, and the recurrence rates at 2 years were 15% and 33%, respectively. CONCLUSIONS. The combination of purine analogs with rituximab was safe and effective for patients with recurrent and/or refractory HCL, and the current results suggested an added benefit compared with standard treatment. Cancer 2007 © 2007 American Cancer Society.