LDL Apheresis: An Effective and Safe Treatment for Refractory Hypercholesterolemia

Hudgins, Lisa C.; Gordon, Bruce R.; Parker, Thomas S.; Saal, Stuart D.; Levine, Daniel M.; Rubin, Albert L.

doi:10.1111/j.1527-3466.2002.tb00097.x

Cited by 18 publications

(6 citation statements)

References 22 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…We underline the fact that our patient already had a right coronary artery stenosis at the time of liver transplantation. A LDL apheresis procedure can transiently reduce LDL cholesterol levels by more than 50% (17) and may delay the onset of atherosclerosis (18). Nevertheless, it must be frequently repeated (every one to two wk) and is both costly and not widely available.…”

Section: Discussionmentioning

confidence: 99%

Domino liver transplantation using a graft from a donor with familial hypercholesterolemia: seven‐yr follow‐up

Popescu

Habib

Dima

et al. 2009

Clinical Transplantation

View full text Add to dashboard Cite

A 46-yr-old female with hepatocellular carcinoma and severe hepatitis B-related liver cirrhosis received a domino liver graft from a 25-yr-old female with homozygous familial hypercholesterolemia (HFHC) in September 2001. Hypercholesterolemia occurred in the graft recipient within one yr after transplantation and was partially controlled by atorvastatin. Three yr after transplantation, an autologous CD34(+) cell transplantation was performed in order to better control the hypercholesterolemia. Only preliminary results of this domino liver transplantation (DLT) were published in 2003, without a long-term analysis of the hypercholesterolemic effects in recipient. Subsequent to DLT, the average plasma cholesterol level in the domino donor rapidly normalized and seven yr after had a value of 182 mg/dL. After seven-yr follow-up, the domino recipient has no hepatocarcinoma recurrence. Moreover, no signs of cardiovascular or atherosclerotic lesions were noted despite an elevated plasma cholesterol level (339 mg/dL after seven yr of follow-up) resistant to drug therapy and stem cell autotransplantation. In conclusion, DLT using a liver graft from a patient with HFHC provides a viable option for marginal recipients.

show abstract

Section: Discussionmentioning

confidence: 99%

Domino liver transplantation using a graft from a donor with familial hypercholesterolemia: seven‐yr follow‐up

Popescu

Habib

Dima

et al. 2009

Clinical Transplantation

View full text Add to dashboard Cite

show abstract

“…Addition of ezetimibe to statin treatment may result in an additional 20% LDL-C reduction 7 . Apheresis treatment may acutely lower LDL-C level by 70–80% and result in a time-average reduction by 40–50% when performed regularly 24 . Recently a phase 3 randomized placebo control study assessing the efficacy of an anti-apoB ASO, mipomersen, showed a reduction LDL-C of approximately 25% in HoFH treated with maximal tolerated lipid-lowering drug therapy 13 .…”

Section: Panel - Research In Contextmentioning

confidence: 99%

Efficacy and safety of a microsomal triglyceride transfer protein inhibitor in patients with homozygous familial hypercholesterolaemia: a single-arm, open-label, phase 3 study

et al. 2013

View full text Add to dashboard Cite

Background Patients with homozygous familial hypercholesterolemia (HoFH) respond inadequately to existing drugs. We conducted a phase 3 study to assess the efficacy and safety of the microsomal triglyceride transfer protein inhibitor lomitapide in adults with HoFH. Methods Twenty-nine subjects enrolled into a single-arm, open-label study and maintained current lipid lowering therapy from six weeks before baseline through at least week 26. Lomitapide dose was escalated based on safety and tolerability from 5 mg to a maximum of 60 mg/day. The primary endpoint was mean percent change from baseline in LDL-C at week 26, after which patients remained on lomitapide through week 78 for safety assessment. Findings Twenty-three subjects completed weeks 26 and 78. The median dose of lomitapide was 40 mg/day. LDL-C was reduced by 50% from baseline at week 26 (4·3 ± 2·5 mmol/L vs. 8·7 ± 2·9 mmol/L, p<0.0001). Eight subjects achieved LDL-C <2·6 mmol/L at this time point. LDL-C was reduced by 44% at week 56 and 38% at week 78 (p<0.0001 for both). Gastrointestinal symptoms were the most common adverse event. Four patients had aminotransaminase > 5× ULN that resolved after dose reduction or temporary interruption of lomitapide. No subject permanently discontinued treatment due to liver abnormalities. Liver fat content assessed by nuclear magnetic resonance spectroscopy (NMRS; n=20) was 1·0 ± 1·3 % at baseline, 8·6 ± 8·1% at week 26 and remained stable up to week 78 (8·3± 5·3%). Interpretation These data demonstrate that lomitapide had a robust and durable efficacy in lowering LDL-C in patients with HoFH with an acceptable safety and tolerability profile.

show abstract

“…However, lipoprotein apheresis is invasive, time consuming, and not readily available for many patients. 4, 5 …”

Section: Introductionmentioning

confidence: 99%

Long-term clinical results of microsomal triglyceride transfer protein inhibitor use in a patient with homozygous familial hypercholesterolemia

Raper

Kolansky

Sachais

et al. 2015

Journal of Clinical Lipidology

View full text Add to dashboard Cite

LDL Apheresis: An Effective and Safe Treatment for Refractory Hypercholesterolemia

Cited by 18 publications

References 22 publications

Domino liver transplantation using a graft from a donor with familial hypercholesterolemia: seven‐yr follow‐up

Domino liver transplantation using a graft from a donor with familial hypercholesterolemia: seven‐yr follow‐up

Efficacy and safety of a microsomal triglyceride transfer protein inhibitor in patients with homozygous familial hypercholesterolaemia: a single-arm, open-label, phase 3 study

Long-term clinical results of microsomal triglyceride transfer protein inhibitor use in a patient with homozygous familial hypercholesterolemia

Contact Info

Product

Resources

About