Effects of deferasirox dose and decreasing serum ferritin concentrations on kidney function in paediatric patients: an analysis of clinical laboratory data from pooled clinical studies.


Journal

The Lancet. Child & adolescent health
ISSN: 2352-4650
Titre abrégé: Lancet Child Adolesc Health
Pays: England
ID NLM: 101712925

Informations de publication

Date de publication:
01 2019
Historique:
received: 07 09 2018
revised: 02 10 2018
accepted: 04 10 2018
pubmed: 21 11 2018
medline: 7 5 2020
entrez: 21 11 2018
Statut: ppublish

Résumé

Serious and fatal deferasirox-induced kidney injury has been reported in paediatric patients. This study aimed to investigate the effects of deferasirox dose and serum ferritin concentrations on kidney function and the effect of impaired kidney function on dose-normalised deferasirox minimum plasma concentration (C We did a case-control analysis using pooled data from ten clinical studies. We identified transfusion-dependent patients with thalassaemia, aged 2-15 years, who were receiving deferasirox and had available baseline and follow-up serum creatinine and ferritin measurements. Cases of acute kidney injury (AKI) were defined according to an estimated glomerular filtration rate (eGFR) threshold of 90 mL/min per 1·73 m Among 1213 deferasirox-treated paediatric patients, 162 cases of AKI and 621 matched control visits were identified. Patients with AKI had a mean 50·2% (SD 15·5) decrease in eGFR from baseline, compared with a 6·9% (29·8) decrease in controls. A significantly increased risk for AKI (rate ratio 1·26, 95% CI 1·08-1·48, p=0·00418) was observed per 5 mg/kg per day increase in deferasirox dispersible tablet dose (equivalent to a 3·5 mg/kg per day dose of film-coated tablets or granules), above the typical starting dose (20 mg/kg per day). An increased risk (1·25, 1·01-1·56, p=0·0400) for AKI was also observed per 250 μg/L decrease in serum ferritin, starting from 1250 μg/L. High-dose deferasirox (dispersible tablet dose >30 mg/kg per day) resulted in an increased risk (4·47, 1·25-15·95, p=0·0209) for AKI when serum ferritin was less than 1000 μg/L. Decreases in eGFR were associated with increased C Deferasirox can cause AKI in a dose-dependent manner. The increased AKI risk with high-dose deferasirox and lower serum ferritin concentration is consistent with overchelation as a causative factor. Small decreases in eGFR correlate with increased deferasirox C None.

Sections du résumé

BACKGROUND
Serious and fatal deferasirox-induced kidney injury has been reported in paediatric patients. This study aimed to investigate the effects of deferasirox dose and serum ferritin concentrations on kidney function and the effect of impaired kidney function on dose-normalised deferasirox minimum plasma concentration (C
METHODS
We did a case-control analysis using pooled data from ten clinical studies. We identified transfusion-dependent patients with thalassaemia, aged 2-15 years, who were receiving deferasirox and had available baseline and follow-up serum creatinine and ferritin measurements. Cases of acute kidney injury (AKI) were defined according to an estimated glomerular filtration rate (eGFR) threshold of 90 mL/min per 1·73 m
FINDINGS
Among 1213 deferasirox-treated paediatric patients, 162 cases of AKI and 621 matched control visits were identified. Patients with AKI had a mean 50·2% (SD 15·5) decrease in eGFR from baseline, compared with a 6·9% (29·8) decrease in controls. A significantly increased risk for AKI (rate ratio 1·26, 95% CI 1·08-1·48, p=0·00418) was observed per 5 mg/kg per day increase in deferasirox dispersible tablet dose (equivalent to a 3·5 mg/kg per day dose of film-coated tablets or granules), above the typical starting dose (20 mg/kg per day). An increased risk (1·25, 1·01-1·56, p=0·0400) for AKI was also observed per 250 μg/L decrease in serum ferritin, starting from 1250 μg/L. High-dose deferasirox (dispersible tablet dose >30 mg/kg per day) resulted in an increased risk (4·47, 1·25-15·95, p=0·0209) for AKI when serum ferritin was less than 1000 μg/L. Decreases in eGFR were associated with increased C
INTERPRETATION
Deferasirox can cause AKI in a dose-dependent manner. The increased AKI risk with high-dose deferasirox and lower serum ferritin concentration is consistent with overchelation as a causative factor. Small decreases in eGFR correlate with increased deferasirox C
FUNDING
None.

Identifiants

pubmed: 30455109
pii: S2352-4642(18)30335-3
doi: 10.1016/S2352-4642(18)30335-3
pii:
doi:

Substances chimiques

Iron Chelating Agents 0
Ferritins 9007-73-2
Deferasirox V8G4MOF2V9

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

15-22

Commentaires et corrections

Type : CommentIn

Informations de copyright

Copyright © 2019 Elsevier Ltd. All rights reserved.

Auteurs

Steven T Bird (ST)

Division of Epidemiology, Food and Drug Administration, Silver Spring, MD, USA. Electronic address: steven.bird@fda.hhs.gov.

Richard S Swain (RS)

Division of Epidemiology, Food and Drug Administration, Silver Spring, MD, USA.

Fang Tian (F)

Division of Epidemiology, Food and Drug Administration, Silver Spring, MD, USA.

Olanrewaju O Okusanya (OO)

Office of Clinical Pharmacology, Food and Drug Administration, Silver Spring, MD, USA.

Peter Waldron (P)

Division of Pharmacovigilance, Food and Drug Administration, Silver Spring, MD, USA.

Mona Khurana (M)

Division of Pediatric and Maternal Health, Food and Drug Administration, Silver Spring, MD, USA.

Elizabeth L Durmowicz (EL)

Division of Pediatric and Maternal Health, Food and Drug Administration, Silver Spring, MD, USA.

Yong Ma (Y)

Division of Biometrics, Office of Biostatistics, Food and Drug Administration, Silver Spring, MD, USA.

Jacqueline M Major (JM)

Division of Epidemiology, Food and Drug Administration, Silver Spring, MD, USA.

Kate Gelperin (K)

Division of Epidemiology, Food and Drug Administration, Silver Spring, MD, USA.

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Classifications MeSH