open access

Vol 29, No 1 (2022)
Original Article
Submitted: 2021-10-13
Accepted: 2021-12-13
Published online: 2022-01-11
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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

Krzysztof Chlebus12, Barbara Cybulska3, Piotr Dobrowolski4, Marzena Romanowska-Kocejko25, Marta Żarczyńska-Buchowiecka25, Natasza Gilis-Malinowska12, Aneta Stróżyk12, Justyna Borowiec-Wolna12, Marcin Pajkowski25, Beata Bobrowska67, Renata Rajtar-Salwa67, Aleksandra Kwapiszewska4, Małgorzata Waluś-Miarka6, Magdalena Chmara89, Rafał Gałąska1, Maciej Małecki6, Tomasz Zdrojewski10, Marcin Gruchała12
DOI: 10.5603/CJ.a2022.0003
·
Pubmed: 35146730
·
Cardiol J 2022;29(1):62-71.
Affiliations
  1. 1st Department of Cardiology, Medical University of Gdansk, Poland
  2. National Center for Familial Hypercholesterolemia, University Clinical Center, Gdansk, Poland
  3. National Institute of Public Health – National Institute of Hyg iene, Warsaw, Poland
  4. Department of Hypertension, National Institute of Cardiology, Warsaw, Poland
  5. Department of Cardiac Diagnostics, Medical University of Gdansk, Poland
  6. Department of Metabolic Diseases, Jagiellonian University Medical College, Krakow, Poland
  7. Department of Cardiology and Cardiovascular Interventions, University Hospital, Krakow, Poland
  8. Department of Biology and Medical Genetics, Medical University of Gdansk, Poland
  9. Laboratory of Clinical Genetics, University Clinical Center, Gdansk, Poland
  10. Department of Preventive Medicine and Education, Medical University of Gdansk, Poland

open access

Vol 29, No 1 (2022)
Original articles — Clinical cardiology
Submitted: 2021-10-13
Accepted: 2021-12-13
Published online: 2022-01-11

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.

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Keywords

familial hypercholesterolemia, PCSK9 inhibitors, alirocumab, evolocumab, LDL-cholesterol reduction, clinical side effects

About this article
Title

Effectiveness and safety of PCSK9 inhibitor therapy in patients with familial hypercholesterolemia within a therapeutic program in Poland: Preliminary multicenter data

Journal

Cardiology Journal

Issue

Vol 29, No 1 (2022)

Article type

Original Article

Pages

62-71

Published online

2022-01-11

Page views

2991

Article views/downloads

632

DOI

10.5603/CJ.a2022.0003

Pubmed

35146730

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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