Evidence map›Paper›PMID 25897792›Full record

Trial reportAtherosclerosis2015

The lipid-lowering effects of lomitapide are unaffected by adjunctive apheresis in patients with homozygous familial hypercholesterolaemia - a post-hoc analysis of a Phase 3, single-arm, open-label trial.

C Stefanutti, D J Blom, M R Averna, E A Meagher, H dT Theron, A D Marais, R A Hegele, C R Sirtori, P K Shah, D Gaudet and 9 more

Open access · bronzeAbstract readClinical Trial, Phase IIIMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Atherosclerosis, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
15citing papers in PubMed, 2 pooled it
6.8field-weighted citation impact, top 3% of its field
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

15 citing papers in PubMed, 2 syntheses or guidelines pooled it, 45 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Trial
  4. Review
  5. Review
  6. Current Role of Lipoprotein Apheresis.Current atherosclerosis reports · 2019
    Review
  7. Review
  8. Article
  9. The 1Blood transfusion = Trasfusione del sangue · 2017
    Review
  10. Novel Therapies for Familial Hypercholesterolemia.Current treatment options in cardiovascular medicine · 2016
    Review
  11. Review
  12. Review
  13. Review
  14. Review
  15. Article
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

19 authors at 11 institutions in 4 countries.

C Stefanutti'Sapienza' University of Rome, Rome, Italy.
D J BlomUniversity of Cape Town, Cape Town, South Africa.
M R AvernaUniversità di Palermo, Palermo, Italy.
E A MeagherUniversity of Pennsylvania, Philadelphia, PA, USA.
H dT TheronNetcare Private Hospital, Bloemfontein, South Africa.
A D MaraisUniversity of Cape Town, Cape Town, South Africa.
R A HegeleRobarts Research Institute and University of Western Ontario, London, ON, Canada.
C R SirtoriOspedale Niguarda, Milano, Italy.
P K ShahCedars-Sinai Heart Institute, Los Angeles, CA, USA.
D GaudetUniversité de Montreal, Chicoutimi, Canada.
G B VignaUniversità di Ferrara, Ferrara, Italy.
B S SachaisUniversity of Pennsylvania, Philadelphia, PA, USA.
S Di Giacomo'Sapienza' University of Rome, Rome, Italy.
A M E du PlessisUniversity of Pretoria, South Africa.
L T BloedonAegerion Pharmaceuticals, Cambridge, MA, USA.
J BalserVeristat, Holliston, MA, USA.
D J RaderUniversity of Pennsylvania, Philadelphia, PA, USA.
M CuchelUniversity of Pennsylvania, Philadelphia, PA, USA. Electronic address: mcuchel@mail.med.upenn.edu.
Phase 3 HoFH Lomitapide Study Investigators
University of Pennsylvania · USSapienza University of Rome · ITUniversity of Cape Town · ZAAegerion Pharmaceuticals (United States) · USAzienda Socio Sanitaria Territoriale Grande Ospedale Metropolitano Niguarda · ITNetcare Unitas Hospital · ZAUniversité du Québec à Chicoutimi · CAUniversity of Ferrara · ITUniversity of Palermo · ITUniversity of Pretoria · ZAWestern University · CA

Funding

INSTITUTIONAL CLINICAL AND TRANSLATIONAL SCIENCE AWARDUL1RR024134 · NCRR · UNIVERSITY OF PENNSYLVANIA · PI FITZGERALD, GARRET A · 2006 to 2011
$70.4M
NCRR NIH HHS UL1 RR024134
6 · The paper itself

Abstract

objectiveLomitapide (a microsomal triglyceride transfer protein inhibitor) is an adjunctive treatment for homozygous familial hypercholesterolaemia (HoFH), a rare genetic condition characterised by elevated low-density lipoprotein-cholesterol (LDL-C), and premature, severe, accelerated atherosclerosis. Standard of care for HoFH includes lipid-lowering drugs and lipoprotein apheresis. We conducted a post-hoc analysis using data from a Phase 3 study to assess whether concomitant apheresis affected the lipid-lowering efficacy of lomitapide.

methodsExisting lipid-lowering therapy, including apheresis, was to remain stable from Week -6 to Week 26. Lomitapide dose was escalated on the basis of individual safety/tolerability from 5 mg to 60 mg a day (maximum). The primary endpoint was mean percent change in LDL-C from baseline to Week 26 (efficacy phase), after which patients remained on lomitapide through Week 78 for safety assessment and further evaluation of efficacy. During this latter period, apheresis could be adjusted. We analysed the impact of apheresis on LDL-C reductions in patients receiving lomitapide.

resultsOf the 29 patients that entered the efficacy phase, 18 (62%) were receiving apheresis at baseline. Twenty-three patients (13 receiving apheresis) completed the Week 26 evaluation. Of the six patients who discontinued in the first 26 weeks, five were receiving apheresis. There were no significant differences in percent change from baseline of LDL-C at Week 26 in patients treated (-48%) and not treated (-55%) with apheresis (p = 0.545). Changes in Lp(a) levels were modest and not different between groups (p = 0.436).

conclusionThe LDL-C lowering efficacy of lomitapide is unaffected by lipoprotein apheresis.

Indexed as

HomozygoteAdultAnticholesteremic AgentsBenzimidazolesBiomarkersBlood Component RemovalCholesterol, LDLCombined Modality TherapyFemaleGenetic Predisposition to DiseaseHumansHyperlipoproteinemia Type IILipoprotein(a)MalePhenotypeTime FactorsAnticholesteremic AgentsBenzimidazolesBiomarkersBMS201038Cholesterol, LDLLipoprotein(a)Homozygous familial hypercholesterolaemiaLipoprotein apheresisLomitapide

Identifiers

PMID25897792
PMCPMC4573555
OpenAlexW2064508165

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

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.