Evidence map›Paper›PMID 39751968›Full record

ReviewCurrent atherosclerosis reports2025

Homozygous Familial Hypercholesterolemia Treatment: New Developments.

Dirk J Blom, A David Marais, Frederick J Raal

Abstract readReview
In one paragraph

Review in Current atherosclerosis reports, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed, 1 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

9 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. AlirocumabCurrent cardiology reviews · 2026
    Pooled it
  2. Article
  3. Article
  4. mRNA medicine for cardiovascular disease.Nature cardiovascular research · 2026
    Review
  5. Review
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  7. Article
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

3 authors.

Dirk J BlomDivision of Lipidology and Cape Heart Institute, Department of Medicine, University of Cape Town, Cape Town, South Africa. dirk.blom@uct.ac.za.
A David MaraisDivision of Chemical Pathology, Department of Pathology, University of Cape Town, Cape Town, South Africa.
Frederick J RaalCarbohydrate and Lipid Metabolism Research Unit, Department of Medicine, University of the Witwatersrand, Johannesburg, South Africa.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

purpose of reviewHomozygous familial hypercholesterolaemia (HoFH) is characterized by marked elevation of low-density lipoprotein cholesterol (LDLC) and premature atherosclerotic cardiovascular disease. This is a review of novel pharmacological therapies to lower LDLC in patients with HoFH. RECENT

findingsNovel therapies can be broadly divided by whether their efficacy is dependent or independent of residual low-density lipoprotein receptor (LDLR) function. Novel LDLR dependent therapies that reduce proprotein subtilisin kexin type 9 levels include monoclonal antibodies (alirocumab and evolocumab) and a small inhibitory RNA (inclisiran). LDLC reductions are highly variable and depend on residual LDLR function. Microsomal triglyceride inhibitors (lomitapide) and therapies that reduce angiopoietin like factor 3 (evinacumab and zodasiran) both reduce LDLC by approximately 50%, irrespective of residual LDLR function. Most patients with HoFH require multiple therapies to achieve LDLC targets. Better LDLC control with LDLR independent therapies is likely to improve the outlook for patients with HoFH while at the same time reducing the need for other therapies such as apheresis or hepatic transplantation.

Indexed as

Receptors, LDLAntibodies, MonoclonalAnticholesteremic AgentsCholesterol, LDLHomozygous Familial HypercholesterolemiaHumansHyperlipoproteinemia Type IIAntibodies, MonoclonalAnticholesteremic AgentsCholesterol, LDLReceptors, LDLAngiopoietin like factor 3Gene editingHomozygous familial hypercholesterolemiaMicrosomalProprotein convertase subtilisin kexin type 3Triglyceride transfer protein

Identifiers

PMID39751968
PMCPMC11698773

What OpenQuestion holds

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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.