Bierer et al. (J Pediatr Surg 2009;44:1540-5) completed a small randomized double-blind clinical trial in which 20 neonates were recruited when they were about a week old. The randomization process allocated 10 patients (birth weight: 2034 308 g) in a group where participants received erythropoietin during 14 days (200 units/kg/day); 10 other patients (birth weight: 2400 184 g) received a placebo. Absolute reticulocyte count increased in the first group only. The hematocrit also increased from 34.4 to 37.3% in the erythropoietin group while it dropped from 35.9 to 33.2% in the control group, but the difference was not statistically significant. No adverse event was observed.
The representativeness of the sample collected in this pilot trial may be suboptimal since only 20 patients were randomized while 61 infants met study criteria; no description of non-included patients is provided. Moreover, there is some imbalance in the two groups (gestation at birth: 34.1 1.7 vs. 36.6 0.7 weeks), and the sample size is too small to draw definitive conclusions. In spite of these limitations, this study showed that erythropoietin is safe when used in neonates and that it can activate erythropoiesis even during the first month of life. A large randomized clinical trial is required in order to demonstrate the efficacy of erythropoietin to prevent anemia and/or to decrease the number of transfusions given to neonates requiring an aggressive surgery while they are less than a few weeks old.
– Jacques Lacroix