Characterizing familial chylomicronemia syndrome: Baseline data of the APPROACH study Dirk J. Blom, MD, PhD, Louis O'Dea, MD, Andres Digenio, MD, PhD,

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Presentation transcript:

Characterizing familial chylomicronemia syndrome: Baseline data of the APPROACH study Dirk J. Blom, MD, PhD, Louis O'Dea, MD, Andres Digenio, MD, PhD, Veronica J. Alexander, PhD, Ewa Karwatowska-Prokopczuk, MD, PhD, Karren R. Williams, PhD, Linda Hemphill, MD, Ovidio Muñiz-Grijalvo, MD, PhD, Raul D. Santos, MD, Seth Baum, MD, Joseph L. Witztum, MD Journal of Clinical Lipidology Volume 12, Issue 5, Pages 1234-1243.e5 (September 2018) DOI: 10.1016/j.jacl.2018.05.013 Copyright © 2018 National Lipid Association Terms and Conditions

Figure 1 Schematic of APPROACH study design. f/u, follow up; OLE, open-label extension; R, randomization; SC, subcutaneous. Journal of Clinical Lipidology 2018 12, 1234-1243.e5DOI: (10.1016/j.jacl.2018.05.013) Copyright © 2018 National Lipid Association Terms and Conditions

Figure 2 Patient recruitment into the APPROACH study. Journal of Clinical Lipidology 2018 12, 1234-1243.e5DOI: (10.1016/j.jacl.2018.05.013) Copyright © 2018 National Lipid Association Terms and Conditions

Figure 3 Frequency distribution of age at FCS diagnosis and age at first pancreatitis. FCS diagnosis: n = 46; First pancreatitis event: n = 50. FCS, familial chylomicronemia syndrome. Journal of Clinical Lipidology 2018 12, 1234-1243.e5DOI: (10.1016/j.jacl.2018.05.013) Copyright © 2018 National Lipid Association Terms and Conditions

Figure 4 Spearman correlation for baseline lipoprotein lipase activity and baseline triglycerides in patients with biallelic lipoprotein lipase mutations. Journal of Clinical Lipidology 2018 12, 1234-1243.e5DOI: (10.1016/j.jacl.2018.05.013) Copyright © 2018 National Lipid Association Terms and Conditions

Figure 5 Postprandial triglycerides following meal test (randomized patients n = 67). SEM, standard error of the mean postprandial TG excursions in FCS patients following standardized meal. Following an overnight fast (>10 hours), patients consumed a liquid meal at 8 AM consisting of 3 bottles of Boost Original (720 cal, 12 g fat, 123 g carbohydrates, 30 g protein) over 30–45 minutes. Blood samples were drawn before the liquid meal (time 0) and at 1-hour intervals for up to 9 hours. Patients were only permitted to drink water, herbal tea, or decaffeinated coffee during the assessment. After the 9-hour time point, patients consumed a nonstandardized low-fat meal (containing <10 g fat) and then returned to the clinic the next morning (fasted) for a 24-hour blood draw. This figure includes data from one additional patient who was randomized but never received treatment. Journal of Clinical Lipidology 2018 12, 1234-1243.e5DOI: (10.1016/j.jacl.2018.05.013) Copyright © 2018 National Lipid Association Terms and Conditions

Supplementary Figure 1 (A) Correlation of hepatic fat and BMI at baseline. BMI, body mass index. (B) Correlation of hepatic fat and fasting glucose at baseline. (C) Correlation of hepatic fat and hsCRP at baseline. hsCRP, high-sensitivity C-reactive protein. (D) Correlation of hepatic fat and fasting triglycerides at baseline. (E) Correlation of hepatic fat and ALT at baseline. ALT, alanine aminotransferase. Journal of Clinical Lipidology 2018 12, 1234-1243.e5DOI: (10.1016/j.jacl.2018.05.013) Copyright © 2018 National Lipid Association Terms and Conditions

Supplementary Figure 2 (A) Correlation of hepatic volume with BMI at baseline. BMI, body mass index. (B) Correlation of hepatic volume with baseline fasting triglycerides. (C) Correlation of hepatic volume with baseline hepatic fat content. Journal of Clinical Lipidology 2018 12, 1234-1243.e5DOI: (10.1016/j.jacl.2018.05.013) Copyright © 2018 National Lipid Association Terms and Conditions

Supplementary Figure 3 (A) Correlation of splenic volume and platelets at baseline. (B) Correlation of splenic volume and white cell count at baseline. (C) Correlation of splenic volume and hemoglobin at baseline. (D) Correlation of splenic volume and fasting triglycerides at baseline. Journal of Clinical Lipidology 2018 12, 1234-1243.e5DOI: (10.1016/j.jacl.2018.05.013) Copyright © 2018 National Lipid Association Terms and Conditions