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Effectiveness and safety of PCSK9 inhibitor therapy in patients with familial hypercholesterolemia within a therapeutic program in Poland: Preliminary multicenter data
Affiliations- 1st Department of Cardiology, Medical University of Gdansk, Poland
- National Center for Familial Hypercholesterolemia, University Clinical Center, Gdansk, Poland
- National Institute of Public Health – National Institute of Hyg iene, Warsaw, Poland
- Department of Hypertension, National Institute of Cardiology, Warsaw, Poland
- Department of Cardiac Diagnostics, Medical University of Gdansk, Poland
- Department of Metabolic Diseases, Jagiellonian University Medical College, Krakow, Poland
- Department of Cardiology and Cardiovascular Interventions, University Hospital, Krakow, Poland
- Department of Biology and Medical Genetics, Medical University of Gdansk, Poland
- Laboratory of Clinical Genetics, University Clinical Center, Gdansk, Poland
- Department of Preventive Medicine and Education, Medical University of Gdansk, Poland
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Abstract
Background: In Poland, treatment with proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors has become available free of charge in a therapeutic program. Assessed herein, is the efficacy and safety of alirocumab and evolocumab in patients with heterozygous familial hypercholesterolemia (FH).
Methods: Data of 55 adult FH patients who participated in the program were analyzed upon meeting the criteria established by the Ministry of Health (low density lipoprotein cholesterol [LDL-C] above 160 mg/dL on max. tolerated statin dose and ezetimib). The efficacy of PCSK9 inhibitors in reducing LDL-C with drug administration every 2 weeks was assessed after 3 months and 1 year of therapy. A safety profile evaluation was performed at each visit. 48 patients completed the 3-month and 21 for the 1-year observation periods (34 patients treated with alirokumab and 14 with evolocumab).
Results: The mean concentration of direct-measured LDL-C decreased from the initial level of 215.1 ± 74.5 mg/dL to 75.3 ± 64.1 mg/dL, i.e., by 65 ± 14% following 3 months of treatment. This effect was stable in 1-year observation (77.7 ± 72.8 mg/dL). Adverse effects were flu-like symptoms (13.0%), injection site reactions (11.1%), fatigue (5.6%) and musculoskeletal symptoms (5.6%). Seven patients failed to complete the 3-month treatment period due to side effects or non-compliance, and 1 patient failed to complete the 1-year treatment due to myalgia.
Conclusions: This study confirmed high effectiveness of PCSK9 inhibitors in reducing LDL-C levels in patients with FH. Due to restrictive inclusion criteria with LDL-C threshold level > 160 mg/dL (> 4.1 mmol/L) required for participation in the therapeutic program, a relatively small number of FH patients were eligible for treatment.
Abstract
Background: In Poland, treatment with proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors has become available free of charge in a therapeutic program. Assessed herein, is the efficacy and safety of alirocumab and evolocumab in patients with heterozygous familial hypercholesterolemia (FH).
Methods: Data of 55 adult FH patients who participated in the program were analyzed upon meeting the criteria established by the Ministry of Health (low density lipoprotein cholesterol [LDL-C] above 160 mg/dL on max. tolerated statin dose and ezetimib). The efficacy of PCSK9 inhibitors in reducing LDL-C with drug administration every 2 weeks was assessed after 3 months and 1 year of therapy. A safety profile evaluation was performed at each visit. 48 patients completed the 3-month and 21 for the 1-year observation periods (34 patients treated with alirokumab and 14 with evolocumab).
Results: The mean concentration of direct-measured LDL-C decreased from the initial level of 215.1 ± 74.5 mg/dL to 75.3 ± 64.1 mg/dL, i.e., by 65 ± 14% following 3 months of treatment. This effect was stable in 1-year observation (77.7 ± 72.8 mg/dL). Adverse effects were flu-like symptoms (13.0%), injection site reactions (11.1%), fatigue (5.6%) and musculoskeletal symptoms (5.6%). Seven patients failed to complete the 3-month treatment period due to side effects or non-compliance, and 1 patient failed to complete the 1-year treatment due to myalgia.
Conclusions: This study confirmed high effectiveness of PCSK9 inhibitors in reducing LDL-C levels in patients with FH. Due to restrictive inclusion criteria with LDL-C threshold level > 160 mg/dL (> 4.1 mmol/L) required for participation in the therapeutic program, a relatively small number of FH patients were eligible for treatment.
Keywords
familial hypercholesterolemia, PCSK9 inhibitors, alirocumab, evolocumab, LDL-cholesterol reduction, clinical side effects
About this articleTitle
Effectiveness and safety of PCSK9 inhibitor therapy in patients with familial hypercholesterolemia within a therapeutic program in Poland: Preliminary multicenter data
Journal
Issue
Article type
Original Article
Pages
62-71
Published online
2022-01-11
Page views
5622
Article views/downloads
881
DOI
Pubmed
Bibliographic record
Cardiol J 2022;29(1):62-71.
Keywords
familial hypercholesterolemia
PCSK9 inhibitors
alirocumab
evolocumab
LDL-cholesterol reduction
clinical side effects
Authors
Krzysztof Chlebus
Barbara Cybulska
Piotr Dobrowolski
Marzena Romanowska-Kocejko
Marta Żarczyńska-Buchowiecka
Natasza Gilis-Malinowska
Aneta Stróżyk
Justyna Borowiec-Wolna
Marcin Pajkowski
Beata Bobrowska
Renata Rajtar-Salwa
Aleksandra Kwapiszewska
Małgorzata Waluś-Miarka
Magdalena Chmara
Rafał Gałąska
Maciej Małecki
Tomasz Zdrojewski
Marcin Gruchała
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