{"title":"Association between uric acid to high-density lipoprotein cholesterol ratio and abdominal aortic calcification: A cross-sectional study.","authors":"Yuanming Li, Lishan Bai","doi":"10.1016/j.jacl.2025.05.017","DOIUrl":"https://doi.org/10.1016/j.jacl.2025.05.017","url":null,"abstract":"<p><strong>Background: </strong>The association between uric acid to high-density lipoprotein cholesterol ratio (UHR) and abdominal aortic calcification (AAC) is not fully understood. This study aimed to explore the potential association between UHR and AAC in adults older than 40 years.</p><p><strong>Methods: </strong>In this cross-sectional study, data were collected from the 2013 to 2014 National Health and Nutrition Examination Survey (NHANES). UHR equals uric acid (mg/dL) divided by high-density lipoprotein cholesterol (mg/dL). We used weighted multiple logistic regression or linear regression analysis to investigate the association between UHR and AAC. We conducted subgroup and interaction analyses to investigate whether these associations varied by different confounders. Receiver operating characteristic curve studies were used to compare the diagnostic power of the Framingham scoring model with the Framingham and UHR models for AAC and severe abdominal aortic calcification (SAAC). Sensitivity analyses to determine the robustness of the UHR and AAC association results.</p><p><strong>Results: </strong>The final evaluation covered 2294 participants older than 40 years (mean age: 59.01 years; 52.57% female). A 1-unit rise in the log-UHR led to a 0.80 increase in the AAC scores (β [95% CI]: 0.80 [0.46, 1.13]) and a 90% higher risk of AAC (odds ratio [OR] [95% CI]: 1.90 [1.42, 2.54]), and the risk of SAAC increased by 95% (OR [95% CI]:1.95 [1.37, 2.77]). Subgroup analyses showed that the association between log-UHR and AAC scores with SAAC varied by sex.</p><p><strong>Conclusions: </strong>This study identified a positive association between UHR and AAC risk, whereas the association of UHR with AAC scores and SAAC was significant only in the female subgroup.</p>","PeriodicalId":15392,"journal":{"name":"Journal of clinical lipidology","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-06-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144512031","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Mary Katherine Cheeley, Carol F Kirkpatrick, Emily E Brown, Dave L Dixon, Heather Gunter, Diane S Osborn, Michael J Wilkinson
{"title":"Multidisciplinary teams in clinical lipidology and cardiometabolic care: A National Lipid Association Expert Clinical Review.","authors":"Mary Katherine Cheeley, Carol F Kirkpatrick, Emily E Brown, Dave L Dixon, Heather Gunter, Diane S Osborn, Michael J Wilkinson","doi":"10.1016/j.jacl.2025.05.002","DOIUrl":"https://doi.org/10.1016/j.jacl.2025.05.002","url":null,"abstract":"<p><p>This National Lipid Association Expert Clinical Review (ECR) describes the roles of multidisciplinary care team members in optimizing the management of lipid disorders and promoting cardiometabolic health, including the contributions of physicians, advanced practice providers, pharmacists, registered dietitian nutritionists, nurses, and genetic counselors. Patients benefit from a collaborative approach to the management of dyslipidemias and cardiometabolic risk through improved diagnosis and treatment, access to comprehensive and evidence-based lifestyle interventions, optimization of pharmacotherapy, and patient education and empowerment. Certain multidisciplinary team members may not be readily accessible in all practice settings. Therefore, this ECR provides suggestions for accessing community resources to expand the reach of multidisciplinary care to a greater number of patients.</p>","PeriodicalId":15392,"journal":{"name":"Journal of clinical lipidology","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-05-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144248078","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Silvia de la Cruz-Ares, María Del Pilar Coronado-Carvajal, Oriol Alberto Rangel-Zúñiga, José David Torres-Peña, Antonio Pablo Arenas-de Larriva, Alejandro López-Moreno, Niki Katsiki, José María Ordovás, Javier Delgado-Lista, Pablo Pérez-Martínez, Francisco Miguel Gutiérrez-Mariscal, José López-Miranda
{"title":"Corrigendum to Lipoprotein particle profile in the presence of peripheral artery disease among patients with coronary heart disease: Data from the CORDIOPREV study, Journal of Clinical Lipidology 19 (2025) 256-266.","authors":"Silvia de la Cruz-Ares, María Del Pilar Coronado-Carvajal, Oriol Alberto Rangel-Zúñiga, José David Torres-Peña, Antonio Pablo Arenas-de Larriva, Alejandro López-Moreno, Niki Katsiki, José María Ordovás, Javier Delgado-Lista, Pablo Pérez-Martínez, Francisco Miguel Gutiérrez-Mariscal, José López-Miranda","doi":"10.1016/j.jacl.2025.05.001","DOIUrl":"https://doi.org/10.1016/j.jacl.2025.05.001","url":null,"abstract":"","PeriodicalId":15392,"journal":{"name":"Journal of clinical lipidology","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144142621","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Veronica J Alexander, Thomas A Prohaska, Ewa Karwatowska-Prokopczuk, Shuting Xia, Sotirios Tsimikas
{"title":"Effect of olezarsen on routinely measured lipase and amylase levels in familial chylomicronemia syndrome.","authors":"Veronica J Alexander, Thomas A Prohaska, Ewa Karwatowska-Prokopczuk, Shuting Xia, Sotirios Tsimikas","doi":"10.1016/j.jacl.2025.05.007","DOIUrl":"https://doi.org/10.1016/j.jacl.2025.05.007","url":null,"abstract":"<p><strong>Background: </strong>Olezarsen reduces acute pancreatitis events in patients with familial chylomicronemia syndrome (FCS). Lipase and amylase are biomarkers of acute pancreatitis released by the pancreas.</p><p><strong>Objective: </strong>To assess whether routinely measured plasma levels of lipase and amylase are modified by treatment with olezarsen in patients with FCS.</p><p><strong>Methods: </strong>Lipase and amylase were measured at baseline, and days 85, 169, 253, and 365 in patients randomly assigned to placebo, olezarsen 50 mg and 80 mg monthly for 365 days in the Balance trial.</p><p><strong>Results: </strong>Compared to placebo, significant differences were present in lipase levels in olezarsen 80 mg at days 85 (P = .026), 169 (P = .036) and 253 (P = .036) with a trend at day 365 (P = .096). Average percent change from baseline from day 1 to 365 revealed a mean (SD) percent increase in lipase of 12.6% (32.4) in placebo, a 6.7% (45.4) increase in olezarsen 50 mg (P = .26 vs placebo) and a reduction of -10.4% (22.7) in olezarsen 80 mg (P = .035 vs placebo) groups. In patients without a reported adverse event of abdominal pain, the placebo group showed a mean percent increase in lipase ranging from 7.91% to 39.1% from day 1 to day 365, olezarsen 50 mg -8.47% to 24.0%, and olezarsen 80 mg -15.4% to -29.6%, leading to significant differences at day 85 (P = .014), day 169 (P = .028), day 253 (P = .008), and day 365 (P = .045) (P-values vs placebo). Changes in amylase mirrored changes in lipase but did not reach statistical significance.</p><p><strong>Conclusion: </strong>Changes in routinely measured lipase levels may reflect subclinical pancreatic injury. Olezarsen favorably modifies changes in lipase in patients with FCS.</p>","PeriodicalId":15392,"journal":{"name":"Journal of clinical lipidology","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-05-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144608473","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Beyond the cataract: Comprehensive ophthalmologic and retinal imaging analysis in cerebrotendinous xanthomatosis.","authors":"Semra Tiryaki Demir, Tuğçe Dursun Yılmazşamlı, Kağan Çalışgan, Fatih Kerem Dedeli, Hanım Babazade, Mehmet Dedeler, Ertuğrul Kıykım, Çiğdem Aktuğlu-Zeybek, Tanyel Zubarıoglu","doi":"10.1016/j.jacl.2025.05.008","DOIUrl":"https://doi.org/10.1016/j.jacl.2025.05.008","url":null,"abstract":"<p><strong>Background: </strong>Cerebrotendinous xanthomatosis (CTX) is a rare metabolic disorder characterized by cholesterol and cholestanol accumulation, leading to neurological and ocular complications. While cataracts and optic neuropathy are common, retinal structural and vascular alterations remain poorly understood.</p><p><strong>Objective: </strong>To comprehensively evaluate ophthalmologic findings and retinal imaging characteristics in CTX patients.</p><p><strong>Methods: </strong>This prospective, cross-sectional study included detailed ophthalmologic examinations of CTX patients, including best corrected visual acuity (BCVA), intraocular pressure (IOP), biomicroscopy, and fundus examination. Retinal imaging was performed using optical coherence tomography (OCT) and OCT-angiography (OCTA). Central foveal thickness (CFT), parafoveal retinal thickness (RT), ganglion cell layer thickness (GCLT), and vessel densities (VDs) of the superficial (SCP) and deep capillary plexus (DCP) were analyzed and compared with age-matched healthy controls.</p><p><strong>Results: </strong>A total of 18 eyes from 9 CTX patients and 18 eyes from 9 controls were included. Five patients (55.5%) had prior cataract surgery. All eyes that underwent cataract surgery exhibited myopic/astigmatic refractive errors, and 3 (30%) had elevated IOP. Fundoscopy revealed optic disc hypoplasia, pallor, blurred disc margins, and increased vascular tortuosity in some cases (44.4%). BCVA, parafoveal RTs, and parafoveal SCP-VDs were significantly lower in CTX patients (P < .05). CFT, foveal SCP-VD, and GCLT were lower but not statistically significant (P > .05).</p><p><strong>Conclusion: </strong>Cataracts and optic nerve abnormalities are the most common ocular findings in CTX. Eyes undergoing cataract surgery should be closely monitored for refractive errors and IOP. Retinal neurovascular pathologies can be detected noninvasively using OCT and OCTA imaging.</p>","PeriodicalId":15392,"journal":{"name":"Journal of clinical lipidology","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-05-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144608472","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Chen Gurevitz, Irina Shalaurova, Margery A Connelly, Robert S Rosenson
{"title":"LpX and LpZ associated hypercholesterolemia in a patient with primary sclerosing cholangitis - A case report.","authors":"Chen Gurevitz, Irina Shalaurova, Margery A Connelly, Robert S Rosenson","doi":"10.1016/j.jacl.2025.05.009","DOIUrl":"https://doi.org/10.1016/j.jacl.2025.05.009","url":null,"abstract":"<p><p>This case report highlights the complex interplay between cholestatic liver disease and lipoproteins in a 45-year-old male with primary sclerosing cholangitis who presents with severe hypercholesterolemia. Nuclear magnetic resonance lipoprofile revealed marked elevations in lipoprotein X and lipoprotein Z (LpZ), with LpZ being the predominant abnormal lipoprotein. Treatment with evolocumab, a proprotein convertase subtilisin/kexin type 9 (PCSK9) monoclonal antibody, effectively reduced LpZ levels, with a consequent decrease in low-density lipoprotein particle concentration. Subsequent treatment with rosuvastatin 5 mg daily further lowered LpZ levels without exacerbating liver dysfunction. This case emphasizes the importance of distinguishing secondary lipoprotein abnormalities from primary hypercholesterolemia in patients with liver disease, in order to guide personalized therapeutic strategies.</p>","PeriodicalId":15392,"journal":{"name":"Journal of clinical lipidology","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-05-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144325893","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Kleisson P Maia, Márcio H Miname, Flávia P Maia, Marcio S Bittencourt, Marjorie H Mizuta, Viviane Z Rocha, Ana Paula Marte, Cinthia E Jannes, Alexandre C Pereira, José E Krieger, Raul D Santos
{"title":"Cardiovascular disease and cholesterol lowering therapy in women and men with molecularly defined heterozygous familial hypercholesterolemia from Brazil.","authors":"Kleisson P Maia, Márcio H Miname, Flávia P Maia, Marcio S Bittencourt, Marjorie H Mizuta, Viviane Z Rocha, Ana Paula Marte, Cinthia E Jannes, Alexandre C Pereira, José E Krieger, Raul D Santos","doi":"10.1016/j.jacl.2025.05.006","DOIUrl":"https://doi.org/10.1016/j.jacl.2025.05.006","url":null,"abstract":"<p><strong>Background: </strong>Data on the epidemiology of familial hypercholesterolemia (FH) in developing regions based on contemporary, molecularly defined FH cohorts categorized by sex is scarce.</p><p><strong>Objective: </strong>Evaluate the differences in cardiovascular disease (CVD) outcomes and lipid-lowering therapy (LLT) between men and women with molecularly defined heterozygous FH participating in a cascade screening program.</p><p><strong>Methods: </strong>We included 794 adult FH patients (age 47 ± 15 years, 56.8% women). The median follow-up was 59.0 (IQR 32.5-86.0) months.</p><p><strong>Results: </strong>At baseline, there were no sex differences regarding genetic defects, low-density lipoprotein cholesterol (LDL-C) years score (12,687± 6047 and 13,011 ± 6576 in men and women, P = .477), and intensive LLT use (74.7% and 75.1% in men and women; P = .915). Men had a higher frequency of prior CVD, 30.4% vs 13.8% (P < .001). During follow-up, men and women were treated similarly with intensive LLT (88.6% and 87.8%; P = .983); however, most participants remained with elevated LDL-C concentrations. The rate of events (1000 patient-years) was 34.40 (95% CI: 26.21- 45.15) and 17.69 (95% CI: 13.03-24.03) for men and women, respectively (P = .001). Current smoking (hazard ratio [HR]: 3.058, 95% CI: 1.597-5.885, P < .001), corneal arcus (HR: 1.763, 95% CI: 1.092-2.847, P = .02), prior CVD (HR: 1.704, 95% CI: 1.006-2.887, P = .048), triglycerides (HR: 1.002, 95% CI 1.000-1.003, P = .008) and high-density lipoprotein cholesterol (HR= 0.975, 95% CI: 0.953-0.998, P = .033) were independently associated with incident events.</p><p><strong>Conclusions: </strong>Men with FH were at a higher and earlier CVD risk than women; there was no difference in treatment intensity, with most patients remaining with high LDL-C.</p>","PeriodicalId":15392,"journal":{"name":"Journal of clinical lipidology","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-05-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144659356","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Triglyceride-glucose index and 28-day all-cause mortality in critically ill obese patients: A MIMIC-IV database analysis.","authors":"Wen-Qiang Wang, Mei-Zhu Chen, Yan-Hui Yang","doi":"10.1016/j.jacl.2025.05.005","DOIUrl":"https://doi.org/10.1016/j.jacl.2025.05.005","url":null,"abstract":"<p><strong>Background: </strong>The triglyceride-glucose (TyG) index, a surrogate marker of insulin resistance, has been linked to various metabolic disorders. This study aimed to investigate the association between the TyG index and 28-day all-cause mortality in obese critically ill patients.</p><p><strong>Methods: </strong>This study utilized the Medical Information Mart for Intensive Care IV (MIMIC-IV) database and included adult patients with body mass index ≥30 kg/m² admitted to the intensive care unit (ICU) for the first time. The TyG index was calculated as ln[fasting triglycerides (mg/dL) × fasting glucose (mg/dL)/2]. The relationship between the TyG index and 28-day all-cause mortality was evaluated using Cox proportional hazards models, and restricted cubic splines (RCS) were employed to explore the dose-response relationship. Subgroup analyses were used to confirm the robustness of the results.</p><p><strong>Results: </strong>During a mean ICU stay of 7.22 days, 291 patients (22.79%) experienced 28-day all-cause mortality. Kaplan-Meier analysis revealed a significantly increased mortality risk with higher TyG index quartiles (log-rank P < .001). Multivariable Cox regression showed that each 1-unit increase in the TyG index was associated with a 41% higher mortality risk (hazard ratio [HR] = 1.41, 95% CI: 1.21-1.63). Patients in quartile 4 had a 98% higher risk compared to quartile 1 (HR = 1.98, 95% CI: 1.30-3.02). RCS analysis showed that higher levels of TyG index (>9.25) were associated with an increased risk of 28-day all-cause mortality. Subgroup analyses confirmed consistent associations across age, sex, and comorbidity subgroups.</p><p><strong>Conclusion: </strong>The TyG index is significantly associated with 28-day all-cause mortality in obese critically ill patients. A higher TyG index serves as an independent predictor of short-term mortality risk in this population.</p>","PeriodicalId":15392,"journal":{"name":"Journal of clinical lipidology","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-05-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144317043","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Daniele Tramontano, Michele di Martino, Francesco Baratta, Alessia Di Costanzo, Nicholas Cocomello, Daniela Commodari, Simone Bini, Ilenia Minicocci, Marcello Arca, Laura D'Erasmo
{"title":"Lomitapide-induced fatty liver is a reversible condition: Evidence from a case of familial chylomicronemia syndrome.","authors":"Daniele Tramontano, Michele di Martino, Francesco Baratta, Alessia Di Costanzo, Nicholas Cocomello, Daniela Commodari, Simone Bini, Ilenia Minicocci, Marcello Arca, Laura D'Erasmo","doi":"10.1016/j.jacl.2025.05.004","DOIUrl":"https://doi.org/10.1016/j.jacl.2025.05.004","url":null,"abstract":"<p><p>Familial chylomicronemia syndrome (FCS) is a rare autosomal recessive disorder characterized by severe hypertriglyceridemia. It is caused by loss-of-function variants in the genes encoding the lipoprotein lipase (LPL) enzyme and its cofactors, which severely impair the hydrolysis of triglycerides (TG). Its main complication is represented by acute pancreatitis (AP), a potentially life-threatening condition. Conventional TG-lowering therapies are poorly effective in FCS, thus requiring the search of novel treatments. Lomitapide, an inhibitor of microsomal triglyceride transfer protein (MTP), has demonstrated efficacy in reducing TG levels in FCS. However, it is associated with hepatic side effects, namely liver fat accumulation. Here we present a case study of a 71-year-old female patient with genetically confirmed FCS, baseline TG level of 2300 mg/dL (25.97 mmol/L) and a history of AP, who was treated with lomitapide for almost 5 years. The treatment allowed a marked reduction of TG (about 90%) and no recurrence of AP. However, hepatic monitoring during treatment revealed a progressive worsening of liver fat accumulation as detected by magnetic resonance imaging (MRI), which was associated with pronounced increases in liver transaminases and liver stiffness (up to 15 kPa). Due to these hepatic adverse events, it was decided to discontinue therapy with lomitapide. An MRI scan repeated after 70 days of drug withdrawal revealed complete resolution of fatty liver disease associated with normalization of liver stiffness (4.1 kPa) and liver transaminases. This case demonstrates the reversibility of lomitapide-induced fatty liver and underscores the importance of regular monitoring of the liver safety during lomitapide to guide timely interventions.</p>","PeriodicalId":15392,"journal":{"name":"Journal of clinical lipidology","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-05-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144275006","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Alejandro Román-González, Alejandro Castellanos, Diego Perdomo, Christian Colón, Juan Jose Vargas, Carlos O Mendivil, Johnayro Gutierrez
{"title":"Real life evidence of volanesorsen for familial chylomicronemia syndrome in Colombia.","authors":"Alejandro Román-González, Alejandro Castellanos, Diego Perdomo, Christian Colón, Juan Jose Vargas, Carlos O Mendivil, Johnayro Gutierrez","doi":"10.1016/j.jacl.2025.04.203","DOIUrl":"https://doi.org/10.1016/j.jacl.2025.04.203","url":null,"abstract":"<p><strong>Background: </strong>Familial chylomicronemia syndrome (FCS) is an ultra-rare disorder associated with pathogenic variants in genes implicated in chylomicron metabolism such as LPL, APOA5, APOC2, GPIHBP1, and LMF1. Patients with FCS have severe hypertriglyceridemia complicated with recurrent episodes of pancreatitis. Volanesorsen is a treatment option for such patients. However, this treatment is not approved or available in all countries.</p><p><strong>Objective: </strong>To present the real-life evidence of clinical response to volanesorsen in patients with FCS in Colombia.</p><p><strong>Methods: </strong>All patients treated with volanesorsen in Colombia as of June 25, 2024, were included. After informed consent, relevant clinical and laboratory data were obtained through review of clinical charts and records from the volanesorsen patient support program.</p><p><strong>Results: </strong>Ten patients with FCS and treated with volanesorsen were included. Most cases were caused by variants in LPL. A total of 90% of cases had at least 1 episode of pancreatis, the mean number of pancreatitis episodes was 5. Median follow-up was 56.5 weeks (IQR 38.3-82.3). The median highest plasma triglyceride (TG) before treatment was 3111 mg/dL (IQR 1738-3810), while the median lowest TG level after treatment was 493 mg/dL (IQR 147-812). The mean percent decreases in plasma TG at months 1, 3, 6, and 12 were 53.6%, 59.7%, 51.5%, and 40.5%, respectively. There were no new pancreatitis episodes after initiation of volanesorsen treatment. Side effects were consistent with those reported in clinical trials.</p><p><strong>Conclusion: </strong>Real-life data of volanesorsen treatment for FCS in Colombia demonstrate efficacy and safety similar to pivotal clinical trials.</p>","PeriodicalId":15392,"journal":{"name":"Journal of clinical lipidology","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-05-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144248079","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}