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Published on: July 16, 2011
Recombinant human erythropoietin therapy in children maintained by haemodialysis
S P Rigden1, G Montini, M Morris
1Department of Paediatrics, Evelina Children's Hospital, London, UK.
Insights
Recombinant human erythropoietin (rHuEPO) effectively treats anemia in children with end-stage renal failure undergoing hemodialysis. This therapy increased hemoglobin levels, reduced transfusion needs, and showed manageable risks.
Area of Science:
- Pediatric Nephrology
- Hematology
- Renal Replacement Therapy
Background:
- Children with end-stage renal failure (ESRF) often suffer from anemia, impacting their quality of life and requiring frequent blood transfusions.
- Anemia in ESRF is typically associated with decreased erythropoietin production and can be exacerbated by dialysis.
- Blood transfusions, while necessary, carry risks such as infection, iron overload, and alloimmunization.
Purpose of the Study:
- To evaluate the efficacy and safety of recombinant human erythropoietin (rHuEPO) in treating anemia in pediatric patients with ESRF on hemodialysis.
- To assess the impact of rHuEPO therapy on hemoglobin levels, transfusion requirements, and iron status.
- To monitor for adverse events associated with rHuEPO treatment in this vulnerable population.
Main Methods:
- Six pediatric patients (3y 11m to 15y 9m) with ESRF and anemia (mean Hb 7.1 g/dl) received intravenous rHuEPO post-dialysis.
- An escalating dose regimen was employed, targeting hemoglobin levels within the lower half of age- and sex-appropriate normal ranges.
- Patients were monitored for hemoglobin response, reticulocyte count, iron status (serum ferritin), HLA antibodies, and adverse events.
Main Results:
- All patients responded to rHuEPO, showing increased reticulocyte counts and hemoglobin levels within a mean of 11 weeks at doses of 100-150 units/kg thrice weekly.
- Mean hemoglobin levels increased to 10.9 g/dl at 12 weeks and 10.5 g/dl at 24 weeks.
- Four children no longer required blood transfusions; serum ferritin decreased in those with iron overload, and HLA antibodies reduced in all patients. One patient experienced vascular access thrombosis.
Conclusions:
- Recombinant human erythropoietin therapy is effective in improving hemoglobin levels and reducing transfusion dependency in anemic children with ESRF on hemodialysis.
- The treatment demonstrated a favorable safety profile, with manageable risks when carefully monitored.
- rHuEPO offers significant potential benefits for pediatric ESRF patients, improving hematological parameters and mitigating transfusion-related complications.
Abstract:
Six children (aged 3 years 11 months to 15 years 9 months) with end-stage renal failure and anaemia (mean haemoglobin 7.1 g/dl, range 6.3-7.7 g/dl) on thrice-weekly haemodialysis were treated with recombinant human erythropoietin (rHuEPO), given as an intravenous bolus in an escalating dose regime after dialysis. All responded with an increase in reticulocyte count and haemoglobin concentration in a mean time of 11 weeks (range 9-13 weeks) and at a dose of 100 or 150 units/kg thrice weekly. The dose of rHuEPO was then adjusted to maintain the haemoglobin concentration within the lower half of the normal range for the child's age and sex. The mean haemoglobin after 12 weeks treatment was 10.9 g/dl (range 8.5-12.1 g/dl) and after 24 weeks, 10.5 g/dl (range 7.9-13.3 g/dl). Four children had no further need for blood transfusion and are thus no longer at risk of blood-borne infection, iron overload and sensitisation to HLA histocompatibility antigens. Serum ferritin fell in the three patients with evidence of iron overload; the three with low or normal iron stores at the onset of treatment maintained erythropoiesis with oral iron supplementation. HLA antibodies decreased in all patients. The only serious complication encountered was thrombosis of vascular access in one child. No child became seriously hypertensive or developed cerebral symptoms. The benefits of rHuEPO therapy for children with end-stage renal failure are potentially considerable and with careful monitoring, the risks low.
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