ESPEYB21 12. Type 2 Diabetes, Metabolic Syndrome and Lipids Lipid Metabolism (5 abstracts)
12.15. Cardiovascular outcomes in patients with homozygous familial hypercholesterolaemia on lipoprotein apheresis initiated during childhood: long-term follow-up of an international cohort from two registries
Reijman MD , Tromp TR , Hutten BA , Hovingh GK , Blom DJ , Catapano AL , Cuchel M , Dann EJ , Gallo A , Hudgins LC , Raal FJ , Ray KK , Sadiq F , Soran H , Groothoff JW , Wiegman A & Kusters DM
Homozygous Familial Hypercholesterolaemia International Clinical Collaborators (HICC); Children with Homozygous Hypercholesterolemia on Lipoprotein Apheresis: an International Registry (CHAIN) consortia.
Lancet Child Adolesc Health. 2024 Jul;8(7):491-499. doi:10.1016/S2352-4642(24)00073-7.
Brief Summary: Data from two large registries of children with homozygous familial hypercholesterolemia (HoFH) revealed that initiating lipoprotein apheresis in childhood, compared to pharmacotherapy, improves plasma LDL-C level and reduces cardiovascular death.
Comment: Homozygous familial hypercholesterolaemia (HoFH) is a rare inherited disorder, which results in extremely elevated low-density lipoprotein cholesterol (LDL-C) levels and premature atherosclerotic cardiovascular disease (ASCVD).
Lipoprotein apheresis is an extracorporeal lipid-lowering treatment that has been well established for 3 decades; it reduces serum LDL-C levels by > 70% per session. The current study assessed long-term cardiovascular outcomes associated with lipoprotein apheresis initiated in childhood or adolescence. It analysed the largest cohort to date with 250 children and adolescents: 125 treated with medication and 125 with apheresis.
Reductions in LDL-C concentrations were greater in the lipoprotein apheresis group (–55%) than medication group (–31%). However, given the very high initial LDL-C of 688 mg/dL, even this marked decrease could not prevent severe outcomes. Cardiovascular death was more common on medication-only than lipoprotein apheresis [8% vs 1%]; p=0 010). However, the median age at coronary artery bypass grafting was lower in the lipoprotein apheresis group than medication-only group (15 0 or ·0 vs 30 5 years p=0 037), and the prevalence and age of onset of other atherosclerotic cardiovascular disease were similar.
These findings are unsurprising given the low frequency of lipoprotein apheresis, which varied from twice per week to once per month, with biweekly sessions being the most common. Fewer than 10% of patients in each cohort reached target LDL-C concentrations, indicating that both therapies were generally sub-optimal.
A consensus statement on lipoprotein apheresis in children was recently published, based on current available evidence and expert opinions from around the world.1 It covers indications, methods, vascular access, treatment targets, monitoring of clinical efficacy, and side effects. By following this consensus and collecting data internationally, we will be able to learn valuable lessons to better understand the benefits and limitations of this treatment. In the meantime, you should review the effects of Evinacumab for treating HoFH.
Reference: 1. Reijman MD, et al. Clinical practice recommendations on lipoprotein apheresis for children with homozygous familial hypercholesterolemia: an expert consensus statement from ERKNet and ESPN. Atherosclerosis. 2024 May;392:117525. doi:10.1016/j.atherosclerosis.2024.117525.
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ESPE Yearbook of Paediatric Endocrinology
ISSN 1662-4009 (Online)
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