Compassionate use of roxadustat for treatment of refractory renal anemia in an infant.
Anemia
Chronic kidney disease
Erythropoiesis-stimulating agent
Hypoxia-inducible factor prolyl hydroxylase inhibitor
Infant
Roxadustat
Journal
Pediatric nephrology (Berlin, Germany)
ISSN: 1432-198X
Titre abrégé: Pediatr Nephrol
Pays: Germany
ID NLM: 8708728
Informations de publication
Date de publication:
13 Dec 2023
13 Dec 2023
Historique:
received:
31
05
2023
accepted:
20
11
2023
revised:
18
11
2023
medline:
13
12
2023
pubmed:
13
12
2023
entrez:
12
12
2023
Statut:
aheadofprint
Résumé
Erythropoiesis-stimulating agents (ESAs) have played an important role in the treatment of renal anemia in children, but cannot improve hemoglobin to target level in some cases. Roxadustat, a hypoxia-inducible factor prolyl hydroxylase inhibitor, can stimulate endogenous erythropoietin production and regulate iron metabolism even in patients with kidney failure. However, roxadustat has not yet been approved for use in children. We report a case of refractory renal anemia in an 80-day-old boy, who was hyporesponsive to ESAs even in combination with iron supplementation and transfusion. Compassionate use of roxadustat successfully corrected the intractable anemia. Hyperkalemia is a manageable adverse event of concern during follow-up. The successful experience in this case may inform the clinical utility of roxadustat for refractory renal anemia in children, which should be further confirmed by well-designed prospective clinical trials.
Sections du résumé
BACKGROUND
BACKGROUND
Erythropoiesis-stimulating agents (ESAs) have played an important role in the treatment of renal anemia in children, but cannot improve hemoglobin to target level in some cases. Roxadustat, a hypoxia-inducible factor prolyl hydroxylase inhibitor, can stimulate endogenous erythropoietin production and regulate iron metabolism even in patients with kidney failure. However, roxadustat has not yet been approved for use in children.
CASE-DIAGNOSIS/TREATMENT
METHODS
We report a case of refractory renal anemia in an 80-day-old boy, who was hyporesponsive to ESAs even in combination with iron supplementation and transfusion. Compassionate use of roxadustat successfully corrected the intractable anemia. Hyperkalemia is a manageable adverse event of concern during follow-up.
CONCLUSION
CONCLUSIONS
The successful experience in this case may inform the clinical utility of roxadustat for refractory renal anemia in children, which should be further confirmed by well-designed prospective clinical trials.
Identifiants
pubmed: 38086983
doi: 10.1007/s00467-023-06240-1
pii: 10.1007/s00467-023-06240-1
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Informations de copyright
© 2023. The Author(s).
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