In a double-blind, placebo (PBO)-controlled study of children age 5–18 years (y) with cancer and anemia who received myelosuppressive chemotherapy (CT), the use of epoetin alfa (EPO) increased hemoglobin (Hb) levels (Hb increase ≥1 g/dL from baseline after 4 weeks [wks], independent of RBC transfusion in the previous 28 days; 65.0% for EPO vs 50.0% for PBO; P=.034) and reduced transfusion requirements (transfusion after wk 4 = 51.4% for EPO vs 69.4% for PBO; P=.008). However, no difference in the primary end point, self-reported quality of life (QOL) using the Pediatric Quality of Life Inventory (PedsQL-I™), was observed overall between the 2 treatment arms (ProcASCO 23:abstract 8527, 2004). In adults, increases in Hb during EPO therapy are associated with improved QOL (

Cancer 95:888, 2002
). Given these findings, this post-hoc analysis of the pediatric study was undertaken to determine if a difference in QOL outcomes is observed when the Hb response of children is considered. The study included patients (pts) who were receiving myelosuppressive CT for malignant solid tumors (ST), Hodgkin’s disease (HD), acute lymphocytic leukemia (ALL), or non-Hodgkin’s lymphoma (NHL) and who were anemic (Hb <12 g/dL for boys >12 y, Hb <11 g/dL for girls >12 y, and Hb <10.5 g/dL for children 5–12 y) at study entry. Pts were stratified by tumor type (ST/HD or ALL/NHL) and randomized 1:1 to receive intravenous EPO 600 IU/kg or PBO weekly for 16 wks. The dose was increased to 900 IU/kg weekly after 3–4 wks if Hb increased by <1 g/dL. A total of 222 pts (111 EPO, 111 PBO) were included in primary analysis (98 ST, 75 ALL, 27 HD, and 22 NHL). For this analysis, Hb and PedsQL-I data beyond week 4 were available for 97 EPO pts and 89 PBO pts. PedsQL-I scores improved significantly from baseline among Hb responders but not among Hb nonresponders in both treatment arms (TABLE). The results of this analysis demonstrate that among pediatric cancer pts receiving myelosuppressive CT, Hb response is associated with improvement in QOL. The fact the Hb nonresponders reported higher QOL at baseline in the PBO group than the EPO group may explain, in part, why no difference in final QOL was observed overall in the primary analysis. The significant improvement in QOL among Hb responders suggests that additional studies are warranted to predict and optimize Hb response, QOL, and long-term outcomes with EPO treatment in anemic children with cancer.

Hb Response and PedsQL-I Score

GroupsHb ResponderBaselineFinalChangeP-Value
Data presented as mean ± SD (no. of patients) 
EPO Yes 69.9±17.15 (65) 79.1±15.53 (66) 9.2±20.20 (65) <.001 
 No 67.1±19.15 (29) 70.0±15.79 (31) 2.6±16.52 (29) .407 
PBO Yes 70.0±15.68 (46) 78.0±17.38 (49) 8.5±13.10 (46) <.001 
 No 72.9±15.76 (40) 75.6±16.51 (40) 2.7±16.96 (40) .315 
GroupsHb ResponderBaselineFinalChangeP-Value
Data presented as mean ± SD (no. of patients) 
EPO Yes 69.9±17.15 (65) 79.1±15.53 (66) 9.2±20.20 (65) <.001 
 No 67.1±19.15 (29) 70.0±15.79 (31) 2.6±16.52 (29) .407 
PBO Yes 70.0±15.68 (46) 78.0±17.38 (49) 8.5±13.10 (46) <.001 
 No 72.9±15.76 (40) 75.6±16.51 (40) 2.7±16.96 (40) .315 
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Topics:
anemia, chemotherapy regimen, childhood cancer, epoetin alfa, hemoglobin, quality of life, brachial plexus neuritis, cancer, transfusion, acute lymphocytic leukemia

Author notes

Corresponding author

2005, The American Society of Hematology
2004
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