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Expert Review of Cardiovascular Therapy Volume 16, 2018 - Issue 7
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Original Research

The effect of volanesorsen treatment on the burden associated with familial chylomicronemia syndrome: the results of the ReFOCUS study

Marcello ArcaDepartment of Lipid and Atherosclerosis, La Sapienza University of Rome, Rome, ItalyView further author information
,
Andrew HsiehAkcea Therapeutics, Cambridge, MA, USAView further author information
,
Handrean SoranDepartment of Medicine, Manchester University Hospital NHS Foundation Trust, Manchester, UKView further author information
,
Paul RosenblitDivision of Endocrinology, University of California, Irvine, Irvine, CA and Diabetes/Lipid Management & Research Center, Huntington Beach, CA, USAView further author information
,
Louis O’DeaAkcea Therapeutics, Cambridge, MA, USAView further author information
&
Michael StevensonAkcea Therapeutics, Cambridge, MA, USACorrespondence[email protected]
View further author information
Pages 537-546 | Received 27 Mar 2018, Accepted 07 Jun 2018, Published online: 22 Jun 2018

References

  • Stroes E, Moulin P, Parhofer KG, et al. Diagnostic algorithm for familial chylomicronemia syndrome. Atheroscler Suppl. 2017;23:1–7.
  • Brahm AJ, Hegele RA. Chylomicronaemia—current diagnosis and future therapies. Nat Rev Endocrinol. 2015;11:352–362
  • Chait A, Brunzell JD. Chylomicronemia syndrome. Adv Intern Med. 1992;37:249–273.
  • Davidson M, Stevenson M, Hsieh A, et al. The burden of familial chylomicronemia syndrome: interim results from the IN-FOCUS study. Expert Rev Cardiovasc Ther. 2017;15(5):415–423.
  • Das SLM, Kennedy JIC, Murphy R, et al. Relationship between the exocrine and endocrine pancreas after acute pancreatitis. World Journal of Gastroenterology: WJG. 2014;20(45):17196–17205.
  • Valdivielso P, Ramirez-Bueno A, Ewald N,J. Current knowledge of hypertriglyceridemic pancreatitis. Eur Med. 2014;25:689–694.
  • Nawaz H, Koutroumpakis E, Easler J, et al. Elevated serum triglycerides are independently associated with persistent organ failure in acute pancreatitis. Am J Gastroenterol. 2015;110(10):1497–1503.
  • Murphy MJ, Sheng X, MacDonald TM, et al. Hypertriglyceridemia and acute pancreatitis. JAMA Intern Med. 2013;173(2):162–164.
  • Gupta N, Ahmed S, Shaffer L, et al. Severe hypertriglyceridemia induced pancreatitis in pregnancy. Case Rep Obstet Gynecol. 2014;2014:485493. doi: 10.1155/2014/485493.
  • Kayataş SE, Eser M, Cam C, et al. Acute pancreatitis associated with hypertriglyceridemia: a life-threatening complication. Arch Gynecol Obstet. 2010;281(3):427–429.
  • Watts G, Morton K, Jackson P, et al. Management of patients with severe hypertriglyceridaemia during pregnancy: report of two cases with familial lipoprotein lipase deficiency. Br J Obstet Gynaecol. 1992;99(2):163–166.
  • Williams L, Wilson DP. Editorial commentary: dietary management of familial chylomicronemia syndrome. J Clin Lipidol. 2016;10(3):462–465.
  • Gaudet D, De Wal J, Tremblay K, et al. Review of the clinical development of alipogene tiparvovec gene therapy for lipoprotein lipase deficiency. Atherosclerosis Supplements. 2010;11(1):55–60.
  • Gangabadage CS, Zdunek J, Tessari M, et al. Structure and dynamics of human apolipoprotein CIII. J Biol Chem. 2008;283(25):17416–17427.
  • Gaudet D, Brisson D, Tremblay K, et al. Targeting APOC3 in the familial chylomicronemia syndrome. N Engl J Med. 2014;371(23):2200–2206.
  • Graham MJ, Lee RG, Bell TA, et al. Antisense oligonucleotide inhibition of apolipoprotein C-III reduces plasma triglycerides in rodents, nonhuman primates, and humans: novelty and significance. Circ Res. 2013;112(11):1479.
  • Gaudet D, Digenio A, Alexander V, et al. The APPROACH study: a randomized, double-blind, placebo-controlled, phase 3 study of volanesorsen administered subcutaneously to patients with familial chylomicronemia syndrome (FCS). Atherosclerosis. 2017;263:e10.
  • Davidson M, Stevenson M, Hsieh A, et al. Examining the high disease burden and impact on quality of life in familial chylomicronemia syndrome - results from the IN-FOCUS study. Clin Cardiol. 2017;40:4–15.
  • Gaudet D, Signorovitch J, Swallow E, et al. Medical resource use and costs associated with chylomicronemia. J Med Econ. 2013;16:657-666.
  • Ž R. Developed with the special contribution of: European Association for Cardiovascular P, rehabilitation et al. ESC/EAS guidelines for the management of dyslipidaemias. The task force for the management of dyslipidaemias of the European Society of Cardiology (ESC) and the European Atherosclerosis Society (EAS). Eur Heart J. 2011;32(14):1769–1818.

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