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Erythropoietin in children with hemolytic uremic syndrome: a pilot randomized controlled trial

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Abstract

Background

The efficacy of recombinant human erythropoietin (rHuEPO) in sparing red blood cell (RBC) transfusions in children with hemolytic uremic syndrome related to Shiga toxin–producing Escherichia coli (STEC-HUS) is uncertain.

Methods

We conducted a pilot randomized controlled open trial between December 2018 and January 2021. Children were randomized to the intervention (subcutaneous rHuEPO 50 U/kg three times weekly until discharge + RBC transfusion if hemoglobin ≤ 7 g/dL and/or hemodynamic instability) or to the control arm (RBC transfusion if hemoglobin ≤ 7 g/dL and/or hemodynamic instability). Primary outcome was the number of RBC transfusions received during hospitalization. Secondary outcomes were to explore whether baseline EPO levels were adequate to the degree of anemia, to correlate selected acute phase parameters with the number of RBC transfusions, and to assess possible adverse events.

Results

Twelve patients per arm were included; they were comparable at recruitment and throughout the disease course. Median number of RBC transfusions was similar between groups (1.5, p = 0.76). Most patients had baseline EPO levels adequate to the degree of anemia, which did not correlate with the number of transfusions (r = 0.19, p = 0.44). Conversely, baseline (r = 0.73, p = 0.032) and maximum lactic dehydrogenase levels (r = 0.78, p = 0.003), creatinine peak (r = 0.71, p = 0.03) and dialysis duration (r = 0.7, p = 0.04) correlated significantly with RBC requirements. No side effects were recorded.

Conclusion

In children with STEC-HUS, the administration of rHuEPO did not reduce the number of RBC transfusions. Larger studies addressing higher doses and similar severity of kidney failure at rHuEPO initiation (e.g. at start of dialysis) are warranted.

Trial registration

ClinicalTrials.gov identifier: NCT03776851.

Graphical abstract

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Funding

The study is funded by research grant from the Consejo de Investigaciones de la Ciudad Autónoma de Buenos Aires to MAC (2609/MSGC).

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Authors and Affiliations

  1. Nephrology Unit, Hospital General de Niños Pedro de Elizalde, Montes de Oca 40, 1270, Ciudad Autónoma de Buenos Aires, Argentina

    Alejandro Balestracci, Marina Andrea Capone, Luciana Meni Battaglia, Ismael Toledo, Sandra Mariel Martin & Laura Beaudoin

  2. Division of Inmunology, Hospital General de Niños Pedro de Elizalde, Hospital General de Niños Pedro de Elizalde, Montes de Oca 40, 1270, Ciudad Autónoma de Buenos Aires, Argentina

    Jeanette Balbaryski

  3. Laboratory of Endocrinology, Hospital Donación Francisco Santojanni, Pilar 950, 1408, Ciudad Autónoma de Buenos Aires, Argentina

    Lorena Gómez

Authors
  1. Alejandro Balestracci
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  2. Marina Andrea Capone
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  3. Luciana Meni Battaglia
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  4. Ismael Toledo
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Correspondence to Alejandro Balestracci.

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The study was approved by the ethics committee of our institution.

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Balestracci, A., Capone, M.A., Meni Battaglia, L. et al. Erythropoietin in children with hemolytic uremic syndrome: a pilot randomized controlled trial. Pediatr Nephrol 37, 2383–2392 (2022). https://doi.org/10.1007/s00467-022-05474-9

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  • DOI: https://doi.org/10.1007/s00467-022-05474-9

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