Effect of subcutaneous recombinant human erythropoetin in cancer patients receiving radiotherapy: final report of a randomized, open-labelled, phase II trial

The purpose of this study was to determine the safety, efficacy and impact on quality of life of recombinant human erythropoietin (r-HuEPO) for cancer patients undergoing radiotherapy (RT). An open-labelled randomized design was used, with patients randomized to either treatment or control arms. Pat ients in the treatment arm received r-HuEPO given by subcutaneous injection at a dose of 200 units kg-l day(-1) plus oral iron supplements (ferrous su lphate 325 mg p.o. t.i.d.). Entry was restricted to patients with carcinoma of the lung, uterine cervix, prostate or breast who presented for RT with anaemia parameters reflective of the anaemia of chronic disease', Radiother apy policies (portals, doses, fraction size, etc.) were determined by the s ite and stage of disease. Complete blood counts (CBCs) were obtained weekly . The target level of haemoglobin was 15 g dl(-1) for men and 149 dl(-1) fo r women, Quality of life (QOL) was assessed weekly by using an analogue sca le to judge energy, activities of daily living and overall quality of life. Forty-eight patients were entered in the study, 24 in the treatment arm an d 24 in the control arm. The prerandomization demographic characteristics a nd mean laboratory values were comparable in both arms. The mean haemoglobi n at completion was 13.6 g dl(-1) for r-HuEPO-treated patients compared wit h 11.0 g dl(-1) for control subjects (P = 0.0012). Patients who received r- HuEPO demonstrated a mean weekly haemoglobin increase of 0.41 g dl(-1) comp ared with a decrease in mean haemoglobin level in controls for 6 of the 7 w eeks of the study (mean weekly decrease of 0.073 g dl(-1)), Target levels o f haemoglobin were achieved by 41.6% of r-HuEPO-treated patients compared w ith none of the control subjects. The mean platelet count declined in both arms of the study with RT but the decline from pretreatment was less rapid in r-HuEPO-treated patients (11.2% decrease) compared with controls (26.3% decrease) and was statistically significant during weeks 4-6. Toxicity was minor with only mild irritation at the injection site. Mean quality of life end points were superior in the treatment arm but not statistically signif icant. r-HuEPO had a beneficial effect on weekly haemoglobin levels in pati ents undergoing RT with response rates similar to other studies. There was also a less rapid decline in weekly platelet counts in r-HuEPO-treated pati ents compared with control subjects. Further studies are needed to address the optimum dose and scheduling as well as the impact of r-HuEPO on clinica l outcomes.