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A retrospective review of outcomes in the treatment of hyperammonemia with renal replacement therapy due to inborn errors of metabolism

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Abstract

Background

Outcomes for severe hyperammonemia treated with renal replacement therapy (RRT) reported in the literature vary widely. This has created differing recommendations regarding when RRT is beneficial for hyperammonemic patients.

Methods

To evaluate our institution’s experience with RRT in pediatric patients with inborn errors of metabolism (IEMs) and potential prognostic indicators of a better or worse outcome, we performed a retrospective chart review of patients who received RRT for hyperammonemia. Our cohort included 19 patients with confirmed IEMs who received RRT between 2000 and 2017. Descriptive statistics are presented as medians with interquartile ranges with appropriate statistical testing assuming unequal variance.

Results

There were 16 males (84%) and 3 females (16%) identified for inclusion in this study. There were 9 survivors (47%) and 10 non-survivors (53%). The average age of survivors was 67 months (age range from 3 days to 15.6 years). The average age of non-survivors was 1.8 months (age range from 2 days to 18.7 months). Peak ammonia, ammonia on admission, and at RRT initiation were higher in non-survivors compared with survivors. Higher ammonia levels and no change in ammonia between admission and RRT initiation were associated with an increased risk of mortality.

Conclusions

Hyperammonemia affects two distinct patient populations; neonates with markedly elevated ammonia levels on presentation and older children who often have established IEM diagnoses and require RRT after failing nitrogen-scavenging therapy. Our experience demonstrates no significant change in mortality associated with neonatal hyperammonemia, which remains high despite improvements in RRT and intensive care.

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Data availability

The data presented in this submission is available upon request after de-identification beginning 3 months and ending 5 years following article publication. Proposals should be directed to corresponding author. To gain access, data requestors will need to sign a data access agreement and provide a methodologically sound proposal.

Abbreviations

RRT:

Renal replacement therapy

PD:

Peritoneal dialysis

HD:

Hemodialysis

CRRT:

Continuous renal replacement therapy

IEM:

Inborn error of metabolism

UCD:

Urea cycle disorder

OA:

Organic acidemia

GFR:

Glomerular filtration rate

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Authors and Affiliations

  1. Division of Pediatric Genetics, Metabolism, and Genomic Medicine, Department of Pediatrics, University of Michigan, D5240 Medical Professional Building, 1500 E. Medical Center Dr, Ann Arbor, MI, 48109, USA

    Elizabeth G. Ames & Ayesha Ahmad

  2. Division of Pediatric Nephrology, Department of Pediatrics, University of Michigan, Ann Arbor, MI, USA

    Kera E. Luckritz

Authors
  1. Elizabeth G. Ames
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  2. Kera E. Luckritz
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  3. Ayesha Ahmad
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Correspondence to Elizabeth G. Ames.

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There are no conflicts of interest for the authors.

Prior presentation of data

Subsets of the data reported in this manuscript have been presented at annual meetings of the Pediatric Academic Society in 2015 and the American College of Medical Genetics in 2018.

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Ames, E.G., Luckritz, K.E. & Ahmad, A. A retrospective review of outcomes in the treatment of hyperammonemia with renal replacement therapy due to inborn errors of metabolism. Pediatr Nephrol 35, 1761–1769 (2020). https://doi.org/10.1007/s00467-020-04533-3

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  • DOI: https://doi.org/10.1007/s00467-020-04533-3

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