Background and objectives: To investigate the effectiveness of recombinant human erythropoietin (r-HuEPO) on disease-related anemia in patients with B-chronic lymphocytic leukemia (B-CLL) and to explore whether improvement of anemia could delay the initiation of cytotoxic therapy.
Design and methods: Twenty five B-CLL patients (12 males and 13 females; median age 70 years) with disease-related anemia were treated with r-HuEPO. Patients were either on no treatment or on a standard regimen, and had at least Rai stage III disease, with a hematocrit (Hct) <32%. Eleven were newly diagnosed, whereas 14 developed anemia during follow-up. Treatment induction lasted for a maximum of 3 months, during which patients were receiving 150 IU/kg of r-HuEPO s.c. t.i.w. with an escalation to 300 IU/kg t.i.w. if response was slow after one month. Responding patients were placed on maintenance r-HuEPO with 150 IU/kg s.c. once weekly, continuously. Complete response (CR) was defined as an increase of Hct to 38% or more and partial response (PR) as an increase of >6% from pretreatment level.
Results: CR was observed in 18/25 (72%) and PR in 2/25 (8%) of the patients. Six patients were downstaged to stage Rai 0, 9 to Rai I and 4 to Rai II. Response was sustained with maintenance therapy. At a median follow-up of 32 months only 4 of the responders required antileukemic treatment. The median survival of responders has not been reached, and 3-year survival is 84%.
Interpretation and conclusions: r-HuEPO was efficient in downstaging Rai stage III B-CLL patients, and delayed the initiation of antileukemic therapy. Whether this effect can be translated into better survival rates remains to be clarified in randomized trials.