Treatment > Blood Diseases and Disorders

Abstract

Double-Blind, Placebo-Controlled Study of Quality of Life, Hematologic End Points, and Safety of Weekly Epoetin Alfa in Children With Cancer Receiving Myelosuppressive Chemotherapy

Bassem I. Razzouk, Jeffrey D. Hord, Marilyn Hockenberry, Pamela S. Hinds, James Feusner, Denise Williams, Wayne R. Rackoff

From the St Jude Children's Research Hospital, Memphis, TN; Children's Hospital Medical Center of Akron, Akron, OH; Texas Children's Cancer Center, Houston, TX; Children's Hospital Oakland, Oakland, CA; and Johnson & Johnson Pharmaceutical Research & Development LLC, Raritan, NJ. -- Address reprint requests to Bassem I. Razzouk, MD, 332 N Lauderdale St, Mail Stop 721, Memphis, TN 38105; e-mail: bassem.razzouk@stjude.org.

Methods. Anemic patients 5 years to 18 years of age receiving myelosuppressive chemotherapy for nonmyeloid malignancies, excluding brain tumors, received intravenous EPO 600 units/kg to 900 units/kg or placebo once-weekly for 16 weeks. 
Patients and parents completed the pediatric health-related quality-of-life generic scales (GS) and cancer-specific scales (CS).

Results. One hundred eleven patients received EPO and 111 patients received placebo. 
Mean final values for GS total score (P = .763 among patients; P = .219 among parents) and CS domain scores (P ≥ .238; P ≥ .081, respectively) were not significantly different between treatment groups. 
EPO-treated patients had greater increases in Hb overall (P = .002) and were more likely to be transfusion free after 4 weeks (38.7% v 22.5%; P = .010). 
Change in Hb was correlated with change in PedsQL-GCS total score in the EPO group (r = 0.242; P = .018), but was not in the placebo group (r = 0.086; P = .430). 
Adverse events were comparable between treatment groups.

Conclusion. This study confirmed the tolerability and hematologic benefits of once-weekly EPO in children with cancer. 
No significant difference in HRQOL was detected between treatment groups, but a significant positive correlation was observed between Hb changes and HRQOL changes in the EPO group. 
Additional studies are warranted to assess HRQOL when anemia is managed optimally in children with cancer.

This protocol is identified on the ClinicalTrials.gov Web site as NCT00261677. -- Presented in part at the 40th Annual Meeting of the American Society of Clinical Oncology, New Orleans, LA, June 5-8, 2004 (abstract 8527); the 46th Annual Meeting of the American Society of Hematology, San Diego, CA, December 4-7, 2004 (abstract 2218); and the 30th Annual Congress of the Oncology Nursing Society, Orlando, FL, April 28-May 1, 2005 (abstract 200).