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, and Wayne R. Rackoff A B S T R A C T Purpose To evaluate the effects of once-weekly epoetin alfa (EPO) on health-related quality of life (HRQOL), hemoglobin (Hb), transfusions, and tolerability in children with cancer. 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. J Clin Oncol 24:3583-3589. © 2006 by American Society of Clinical Oncology INTRODUCTION In adults with cancer, improving hemoglobin (Hb) has been shown to affect health-related quality of life (HRQOL). 1 Many children with cancer are anemic, but little evidence supports the use of epoetin alfa (EPO) in anemic children with cancer who are re- ceiving chemotherapy. Several studies assessed the effect of EPO in anemic pediatric patients with can- cer and reported that Hb increased while blood transfusion requirements decreased. 2-10 However, a randomized, controlled study of 323 anemic chil- dren with cancer reported hematologic benefits of once-weekly EPO only among children with acute lymphoblastic leukemia (ALL). 11 None of the stud- ies evaluated how HRQOL was affected by or how it correlated with hematologic end points. The Pediat- ric Quality of Life Inventory Generic Core Scales 4.0 (PedsQL-GCS; J.W. Varni, PhD, College Station, TX) have been shown to discriminate between HRQOL in healthy children and those with can- cer. 12,13 The objective of this clinical study was to test the tolerability and effects of once-weekly EPO on patient-reported HRQOL, Hb levels, and blood transfusion requirements in anemic chil- dren with malignancies who were receiving my- elosuppressive chemotherapy. METHODS This randomized, double-blind, placebo-controlled study was conducted at 27 sites. It began as two studies with 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 Pharmaceuti- cal Research & Development LLC, Raritan, NJ. Submitted July 28, 2005; accepted June 12, 2006. Sponsored by Ortho Biotech Clinical Affairs LLC, Bridgewater, NJ; B.I.R. and P.S.H. supported in part by National Cancer Institute Cancer Center support Grant No. P30 CA 21765 and by the American Lebanese Syrian Associated Charities (ALSAC). This protocol is iden- tified 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). B.I.R. and J.D.H. contributed equally to this article. Authors’ disclosures of potential con- flicts of interest and author contribu- tions are found at the end of this article. Address reprint requests to Bassem I. Razzouk, MD, 332 N Lauderdale St, Mail Stop 721, Memphis, TN 38105; e-mail: bassem.razzouk@stjude.org. © 2006 by American Society of Clinical Oncology 0732-183X/06/2422-3583/$20.00 DOI: 10.1200/JCO.2005.03.4371 JOURNAL OF CLINICAL ONCOLOGY O R I G I N A L R E P O R T VOLUME 24  NUMBER 22  AUGUST 1 2006 3583 Downloaded from jco.ascopubs.org on March 6, 2016. For personal use only. No other uses without permission. Copyright © 2006 American Society of Clinical Oncology. All rights reserved.