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Disease control via intensified lipoprotein apheresis in three siblings with familial hypercholesterolemia.
Taylan, Christina; Schlune, Andrea; Meissner, Thomas; Azukaitis, Karolis; Udink Ten Cate, Floris E A; Weber, Lutz T.
Afiliación
  • Taylan C; Pediatric Nephrology, Children's and Adolescents' Hospital, University Hospital of Cologne, Cologne, Germany. Electronic address: christina.taylan@uk-koeln.de.
  • Schlune A; Department of General Pediatrics, Neonatology and Pediatric Cardiology, University Children's Hospital, Düsseldorf, Germany.
  • Meissner T; Department of General Pediatrics, Neonatology and Pediatric Cardiology, University Children's Hospital, Düsseldorf, Germany.
  • Azukaitis K; Pediatric Nephrology, Vilnius University-Hospital, Vilnius, Lithuania.
  • Udink Ten Cate FE; Department of Pediatric Cardiology, Heart Center Cologne, University Hospital of Cologne, Cologne, Germany.
  • Weber LT; Pediatric Nephrology, Children's and Adolescents' Hospital, University Hospital of Cologne, Cologne, Germany.
J Clin Lipidol ; 10(6): 1303-1310, 2016.
Article en En | MEDLINE | ID: mdl-27919346
BACKGROUND: Familial hypercholesterolemia (FH), the prevalent monogenic form of hypercholesterolemia, carries the risk of premature coronary heart disease. Lipoprotein-apheresis is established in children with severe dyslipidemia. We present 3 siblings with a negative/negative residual low-density lipoprotein (LDL) receptor mutation (p.Trp577Arg), unresponsive to drug treatment. OBJECTIVE: Intensified lipoprotein-apheresis is well tolerated and results in permanently low lipid values without harming the health-related quality of life in children. METHODS: Three homozygous FH siblings, aged 7-13 years, had been treated with statins and ezetimibe for 12 months but still showed highly elevated low-density lipoprotein cholesterol (LDL-C) plasma concentrations. They were started on double-filtration plasmapheresis that was subsequently intensified according to plasma lipid levels. RESULTS: Each lipoprotein apheresis session reduced LDL-C concentration by 66% to 70%. Treated plasma volume was doubled after 6 months due to a sustained rebound of LDL-C between sessions. However, the rebound remained unchanged. Only an increase in frequency of sessions to every 3 to 4 days resulted in acceptable pre-treatment LDL-C concentrations (Cmax). Neither cessation of statins nor reduction of plasma exchange volume to 1.5 fold in follow-up influenced Cmax. Intensified therapy did not harm health-related quality of life as assessed by PedsQL and was well tolerated. CONCLUSIONS: In pediatric FH patients unresponsive to drug treatment, intensified lipoprotein apheresis can normalize plasma lipid levels. Apparently, treatment frequency rather than volume has greater influence on its efficacy. The potential burden of intensified therapy to daily life has to be regarded. Serum lipid levels in FH should be normalized to minimize cardiovascular risk.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Eliminación de Componentes Sanguíneos / Hiperlipoproteinemia Tipo II Aspecto: Patient_preference Límite: Adolescent / Child / Humans Idioma: En Revista: J Clin Lipidol Asunto de la revista: BIOQUIMICA / METABOLISMO Año: 2016 Tipo del documento: Article Pais de publicación: Estados Unidos
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Eliminación de Componentes Sanguíneos / Hiperlipoproteinemia Tipo II Aspecto: Patient_preference Límite: Adolescent / Child / Humans Idioma: En Revista: J Clin Lipidol Asunto de la revista: BIOQUIMICA / METABOLISMO Año: 2016 Tipo del documento: Article Pais de publicación: Estados Unidos