Evinacumab for Homozygous Familial Hypercholesterolemia
| العنوان: | Evinacumab for Homozygous Familial Hypercholesterolemia |
|---|---|
| المؤلفون: | Gerald Watts, Farrah Deeba, David Sullivan, Paul Donald Bonnitcha, San San Min, Nimalie Jacintha Nanayakkara, Christopher Ebenbichler, Alexander Tschoner, Clemens Engler, Daniel Gaudet, Nathalie Roy, David Blackburn, Jean Bergeron, Hisee Villeneuve, Samir Saheb, Antonio Gallo, Sophia Beliard, Bertrand Dussol, Sophie Morange, Pascale Poullin, Rene Valero, Charalampos Milionis, Sebastian Filippas-Ntekouan, Eleftherios Klouras, Genovefa Kolovou, George Hatzigeorgiou, Spyridon Rammos, Paolo Rubba, Gabriella Iannuzzo, Mariko Harada-Shiba, Cheol Son, Hisashi Makino, Kiminori Hosoda, Kota Matsuki, Kyoko Kohmo, Masaki Matsubara, Masatsune Ogura, Michio Noguchio, Miki Matsuo, Ryo Koezuka, Tamiko Tamanaha, Tsutomu Tomita, Yoko Ohata, Yoshihiko Otsubo, Noriaki Koyanagi, Masaaki Kawashiri, Hayato Tada, Atsushi Nohara, Akihiro Nomura, Hirofumi Okada, Naohiko Fujii, Daisuke Hayashi, Sayoko Yonemoto, Shungo Fukuda, Koji Yanagi, Miwa Ryo, Masahiro Koseki, Makoto Nishida, Takeshi Okada, Erik Stroes, Laurens Reeskamp, Daniela Stols-Goncalves, Eva Hamulyak, Anho H Liem, Jurgen M Akkerhuis, Pieter R Nierop, Bastiaan M van Dalen, Sweder W E van de Poll, Adriana J M van Miltenburg-van Zijl, Marinus J Veerhoek, Frederick Raal, Sindeep Amrat Bhana, Olena Mitchenko, Vadym Romanov, Iryna Chulaevska, Leonid Rudenko, Olena Beregova, Igor Vakaliuk, Iryna Drapchak, Natalia Tymochko, Anna Isayeva, Olena Buriakovska, Maryna Vovchenko, Vira Tseluyko, Nataliya Matviychuk, Larysa Yakovleva, Paul Duell, Jonathan Barton Purnell, Robert Rosenson, Donald Smith, Theresa Halloran, Robert Gottlieb, Peter A McCullough, Zachary P Rosol, Andy Y Lee, Clay M Barbin, Seth J Baum, Reina Mendelson, Melissa Wright, Linda Hemphill, Akl Fahed, Traci Turner, Angela Ellard |
| المساهمون: | Gerald Watts, ., Deeba, Farrah, Sullivan, David, Donald Bonnitcha, Paul, San Min, San, Jacintha Nanayakkara, Nimalie, Ebenbichler, Christopher, Tschoner, Alexander, Engler, Clemen, Gaudet, Daniel, Roy, Nathalie, Blackburn, David, Bergeron, Jean, Villeneuve, Hisee, Saheb, Samir, Gallo, Antonio, Beliard, Sophia, Dussol, Bertrand, Morange, Sophie, Poullin, Pascale, Valero, Rene, Milionis, Charalampo, Filippas-Ntekouan, Sebastian, Klouras, Eleftherio, Kolovou, Genovefa, Hatzigeorgiou, George, Rammos, Spyridon, Rubba, PAOLO OSVALDO FEDERICO, Iannuzzo, Gabriella, Harada-Shiba, Mariko, Son, Cheol, Makino, Hisashi, Hosoda, Kiminori, Matsuki, Kota, Kohmo, Kyoko, Matsubara, Masaki, Ogura, Masatsune, Noguchio, Michio, Matsuo, Miki, Koezuka, Ryo, Tamanaha, Tamiko, Tomita, Tsutomu, Ohata, Yoko, Otsubo, Yoshihiko, Koyanagi, Noriaki, Kawashiri, Masaaki, Tada, Hayato, Nohara, Atsushi, Nomura, Akihiro, Okada, Hirofumi, Fujii, Naohiko, Hayashi, Daisuke, Yonemoto, Sayoko, Fukuda, Shungo, Yanagi, Koji, Ryo, Miwa, Koseki, Masahiro, Nishida, Makoto, Okada, Takeshi, Stroes, Erik, Reeskamp, Lauren, Stols-Goncalves, Daniela, Hamulyak, Eva, H Liem, Anho, M Akkerhuis, Jurgen, R Nierop, Pieter, M van Dalen, Bastiaan, E van de Poll, Sweder W, M van Miltenburg-van Zijl, Adriana J, J Veerhoek, Marinu, Raal, Frederick, Amrat Bhana, Sindeep, Mitchenko, Olena, Romanov, Vadym, Chulaevska, Iryna, Rudenko, Leonid, Beregova, Olena, Vakaliuk, Igor, Drapchak, Iryna, Tymochko, Natalia, Isayeva, Anna, Buriakovska, Olena, Vovchenko, Maryna, Tseluyko, Vira, Matviychuk, Nataliya, Yakovleva, Larysa, Duell, Paul, Barton Purnell, Jonathan, Rosenson, Robert, Smith, Donald, Halloran, Theresa, Gottlieb, Robert, A McCullough, Peter, P Rosol, Zachary, Y Lee, Andy, M Barbin, Clay, J Baum, Seth, Mendelson, Reina, Wright, Melissa, Hemphill, Linda, Fahed, Akl, Turner, Traci, Ellard, Angela, Graduate School, Vascular Medicine, ACS - Atherosclerosis & ischemic syndromes, Experimental Vascular Medicine, ACS - Pulmonary hypertension & thrombosis |
| المصدر: | New England journal of medicine, 383(8), 711-720. Massachussetts Medical Society |
| بيانات النشر: | Massachusetts Medical Society, 2020. |
| سنة النشر: | 2020 |
| مصطلحات موضوعية: | Adult, Male, medicine.medical_specialty, Adolescent, Evinacumab, Angiopoietin-like Protein, Disease, Familial hypercholesterolemia, 030204 cardiovascular system & hematology, Hyperlipoproteinemia Type II, Young Adult, 03 medical and health sciences, chemistry.chemical_compound, 0302 clinical medicine, Double-Blind Method, Internal medicine, Anticholesteremic Agent, Medicine, Infusions, Intravenou, 030212 general & internal medicine, Child, Aged, Least-Squares Analysi, business.industry, Cholesterol, Homozygote, Antibodies, Monoclonal, Cholesterol, LDL, General Medicine, Middle Aged, medicine.disease, Endocrinology, Receptors, LDL, Multicenter study, chemistry, Ldl metabolism, Mutation, Female, lipids (amino acids, peptides, and proteins), business, Human, Lipoprotein |
| الوصف: | BACKGROUND: Homozygous familial hypercholesterolemia is characterized by premature cardiovascular disease caused by markedly elevated levels of low-density lipoprotein (LDL) cholesterol. This disorder is associated with genetic variants that result in virtually absent (null-null) or impaired (non-null) LDL-receptor activity. Loss-of-function variants in the gene encoding angiopoietin-like 3 (ANGPTL3) are associated with hypolipidemia and protection against atherosclerotic cardiovascular disease. Evinacumab, a monoclonal antibody against ANGPTL3, has shown potential benefit in patients with homozygous familial hypercholesterolemia. METHODS: In this double-blind, placebo-controlled, phase 3 trial, we randomly assigned in a 2:1 ratio 65 patients with homozygous familial hypercholesterolemia who were receiving stable lipid-lowering therapy to receive an intravenous infusion of evinacumab (at a dose of 15 mg per kilogram of body weight) every 4 weeks or placebo. The primary outcome was the percent change from baseline in the LDL cholesterol level at week 24. RESULTS: The mean baseline LDL cholesterol level in the two groups was 255.1 mg per deciliter, despite the receipt of maximum doses of background lipid-lowering therapy. At week 24, patients in the evinacumab group had a relative reduction from baseline in the LDL cholesterol level of 47.1%, as compared with an increase of 1.9% in the placebo group, for a between-group least-squares mean difference of -49.0 percentage points (95% confidence interval [CI], -65.0 to -33.1; P |
| تدمد: | 1533-4406 0028-4793 |
| الوصول الحر: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::eb713acb8c8c2d3fe0124031a30d496aTest https://doi.org/10.1056/nejmoa2004215Test |
| حقوق: | OPEN |
| رقم الانضمام: | edsair.doi.dedup.....eb713acb8c8c2d3fe0124031a30d496a |
| قاعدة البيانات: | OpenAIRE |
| تدمد: | 15334406 00284793 |
|---|