Aims & Objectives:
Accumulation of toxic metabolites can have devastating effects on the growing brains in children with inborn errors of metabolism (IEM), hence reduction in the level of circulating toxins is a matter of urgency. Both scavenger therapy and haemofiltration (CVVH) have been shown to be useful therapies. However no conclusive data exists on optimal time of starting therapy or safe levels of toxic metabolites. We hypothesised that outcomes are better with earlier treatment.
This is a retrospective single centre study from the pediatric intensive care unit (PICU) in a tertiary teaching hospital in London, UK. Children who were referred to our PICU via the metabolic or Children’s Acute Transport teams from 2009–2017 with suspected IEM were identified electronically, and their records reviewed. Patients with metabolic acidosis from septic shock or heart disease were excluded.
36 children with IEM were identified. Mortality was 25%. There was no difference between survivors and non-survivors with regards to age, sex, length of PICU stay, or CVVH duration. In non-survivors compared to survivors, the delay from the time of referral to starting scavengers (Mean±SD 2.22 ± 1.30 vs 4.42 ± 5.10 hours) and CVVH (mean±SD 24.07 ± 9.13 vs 16.98 ± 10.15 hours) was not significantly different. The time delay did not influence the final outcome although initiation of CVVH was associated with improved survival (25% vs 83%, p value 0.02). However CVVH was not started in some due to an expected poor prognosis.
CVVH improves survival in IEM but delay in starting scavenger drugs or CVVH did not influence the final outcome.