Lipid profile in pediatric and adult patients with lysosomal acid lipase deficiency treated with sebelipase alfa: longitudinal evidence from the International LAL-D Registry.

Authors

Don P Wilson, Lorenzo D'Antiga, Manisha Balwani, Annalisa Madeo, Olaf Bodamer, Florian Abel, Jennifer Auchterlonie, William Balistreri, Emilio Ros

Published in

Atherosclerosis. Volume 421. Pages 121867. Aug 05, 2026. Epub Aug 05, 2026.

Abstract

Dyslipidemia, including elevated low-density lipoprotein cholesterol (LDL-C) and decreased high-density lipoprotein cholesterol (HDL-C), is common among patients with lysosomal acid lipase deficiency (LAL-D). The aim of this analysis was to assess the lipid profile of patients with LAL-D treated with sebelipase alfa enzyme replacement therapy.
Children and adults enrolled in the International LAL-D Registry who had a confirmed diagnosis of LAL-D were treated with sebelipase alfa, and had not received lipid-modifying medications were included. The proportions of patients with lipid parameters outside the limits of normal or clinically relevant cutoffs were analyzed before first treatment (baseline) and for up to 3 years of follow-up.
Data from 144 patients (median age at diagnosis, 9.5 years; 92% White; 54% male) were analyzed. From baseline to after 1 year of sebelipase alfa treatment, decreases were observed in the proportion of patients with LDL-C >160 mg/dL (78% to 46%), non-HDL-C >190 mg/dL (70% to 47%), and triglycerides above the upper limit of normal (63% to 32%); the proportion of patients with HDL-C at or above the lower limit of normal increased (21% to 35%). Results were sustained through 3 years of follow-up and were similar in a cohort of patients who had results for all lipid components over 3 consecutive years of treatment.
In patients with LAL-D not receiving lipid-modifying medication, sebelipase alfa treatment improved lipid abnormalities, with sustained reductions of LDL-C and triglycerides and elevations of HDL-C.

PMID:
42617290
Bibliographic data and abstract were imported from PubMed on 20 Aug 2026.

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