Children with cancer who develop anemia during chemotherapy can benefit from a weekly dose of erythropoietin (EPO), according to researchers at St. Jude Children’s Research Hospital. The drug reduces the need for red blood cell transfusions and improves quality of life in children whose anemia is corrected by this treatment, according to results of a Phase III clinical trial at St. Jude.

Anemia is an abnormally low level of hemoglobin (Hb), the oxygen-carrying protein in red blood cells; EPO is a natural hormone that stimulates production of the Hb-containing red blood cells.

This is the first large-scale study of anemic children with cancer that randomly assigned patients to receive either EPO or a placebo (inactive “drug”) intravenously, and the first to measure the effect of EPO on quality of life in children, according to Bassem Razzouk, M.D., an associate member of the Department of Oncology at St. Jude. Razzouk is the lead author of a report on this study that appears in the August 1 issue of the Journal of Clinical Oncology.

“EPO was already known to benefit adults with cancer by increasing their hemoglobin level and improving their quality of life,” Razzouk said. “But even though many children with cancer are anemic, there has been little evidence to support the use of EPO in such children who are receiving chemotherapy.” Chemotherapy can suppress the production of red blood cells and cause anemia. “Our study showed that EPO not only improves the child’s condition, but is also well tolerated, which makes it more acceptable to the patient,” he added.

The study was also significant because a smaller clinical trial at another institution included three subcutaneous injections per week of EPO, while children in the current study received EPO only once a week intravenously. “Our use of intravenous administration of EPO instead of subcutaneous injections reduced the suffering of children and allowed us to complete a major clinical trial that demonstrated the effectiveness of this treatment,” Razzouk said.

The study, which was led by St. Jude, was conducted at 26 sites in the United States and included anemic patients 5 to 18 years of age who were receiving chemotherapy for solid tumors (excluding brain tumors), lymphoma and leukemia. A total of 111 patients received EPO and 111 received a placebo.

The researchers studied the effect of EPO treatment on quality of life using a test called the Pediatric Quality of Life Inventory™ 4.0 Generic Core Scales (PedsQL™-GCS), a survey that includes questions on physical, emotional and social functioning as well as school performance.

The team concluded that EPO increases Hb levels in children with anemia, reduces their need for transfusions and improves the quality of life in those who have an increase in Hb.

The other authors of this study include Jeffrey Hord (Children’s Hospital Medical Center of Akron, Ohio); Marilyn Hockenberry (Texas Children’s Cancer Center, Houston); Pamela Hinds (Nursing Research, St. Jude); James Feusner (Children’s Hospital Oakland, CA); and Denise Williams and Wayne Rackoff (Johnson & Johnson Pharmaceutical Research & Development, L.L.C., Raritan, NJ).

This study was supported in part by a National Cancer Institute Cancer Center Support Grant and ALSAC. The EPO and additional research support was provided by Johnson & Johnson.

St. Jude Children’s Research Hospital