Parhofer Klaus G, Bramlage Peter, Gries Constanze, Harder Cornelia, Look Christiane, Paar W Dieter, Rauch-Kröhnert Ursula
Department of Medicine IV-Grosshadern, University Hospital, LMU Munich, Marchionistr. 15, München, 81377, Germany.
Institute for Pharmacology and Preventive Medicine, Bahnhofstrasse 20, 49661, Cloppenburg, Germany.
Drugs Real World Outcomes. 2025 Mar;12(1):63-74. doi: 10.1007/s40801-024-00471-w. Epub 2025 Jan 28.
Alirocumab is a fully human monoclonal antibody to proprotein convertase subtilisin kexin type 9 used for the reduction of low-density lipoprotein cholesterol (LDL-C) in high-risk patients not reaching their LDL-C target. Recently, a 2-mL prefilled autoinjector has been developed to support the monthly 300-mg dosing regimen with a single-injection administration.
Monthly application of 300 mg AlirRocumab (Praluent) using the 2-mL SYDNEY Device (MARS) is a non-interventional, open, prospective, multi-center cohort study conducted in Germany between 2021 and 2023 with an observational period of 12 weeks. Patients included had primary hypercholesterolemia (heterozygous familial or non-familial) or mixed dyslipidemia and confirmed vascular disease and other risk factors or confirmed familial heterozygous hypercholesterolemia. Primary objectives were to assess the effectiveness of the 2-mL SYDNEY autoinjector measured by the lipid-lowering effect of alirocumab and to document therapy satisfaction, patient adherence, and persistence. Secondary objectives were to assess safety (adverse events) and tolerability.
A total of 146 patients were analyzed: 110 (75.3%) patients were proprotein convertase subtilisin kexin type 9 inhibitor naïve and 36 (24.7%) were pre-treated with a proprotein convertase subtilisin kexin type 9 inhibitor. Patient mean age was 65.6 years with a preponderance of male gender (59.6%). At 12 weeks, the LDL-C value had decreased by a median of 59.5 mg/dL (1.5 mmol/L) in naïve patients (median relative decrease: - 52.0%). In the pre-treated group, the LDL-C value remained mainly unchanged (median slight numerical relative increase: 1.6%). Treatment satisfaction was rated similarly in both groups with most patients being satisfied/very satisfied and rating the injection as effective, safe, and easy to handle. Twenty-three adverse events in 13 patients (8.0%) were documented. Three patients experienced one serious adverse event each; for five patients, an adverse drug reaction was observed, although none was serious. The occurrence of adverse events was similar in both groups.
Alirocumab 300 mg administered with the 2-mL SYDNEY autoinjector was safe and effective in lowering LDL-C after 12 weeks in a routine clinical setting in Germany. The treatment schedule was perceived to be beneficial with excellent device acceptance and satisfaction, potentially increasing patient adherence.
Clinicaltrials.gov: NCT05129241.
阿利西尤单抗是一种完全人源化的抗前蛋白转化酶枯草溶菌素9单克隆抗体，用于降低未达到低密度脂蛋白胆固醇（LDL-C）目标的高危患者的LDL-C水平。最近，已开发出一种2毫升预填充自动注射器，以支持每月300毫克剂量方案的单次注射给药。
使用2毫升悉尼装置（MARS）每月应用300毫克阿利西尤单抗（波立达）是一项于2021年至2023年在德国进行的非干预性、开放性、前瞻性、多中心队列研究，观察期为12周。纳入的患者患有原发性高胆固醇血症（杂合子家族性或非家族性）或混合性血脂异常，并确诊有血管疾病和其他危险因素，或确诊为家族性杂合子高胆固醇血症。主要目的是通过阿利西尤单抗的降脂效果评估2毫升悉尼自动注射器的有效性，并记录治疗满意度、患者依从性和持续性。次要目的是评估安全性（不良事件）和耐受性。
共分析了146例患者：110例（75.3%）患者既往未使用过前蛋白转化酶枯草溶菌素9抑制剂，36例（24.7%）患者曾接受过前蛋白转化酶枯草溶菌素9抑制剂治疗。患者平均年龄为65.6岁，男性占多数（59.6%）。在12周时，初治患者的LDL-C值中位数下降了59.5毫克/分升（1.5毫摩尔/升）（中位数相对下降：-52.0%）。在预处理组中，LDL-C值基本保持不变（中位数轻微数值相对增加：1.6%）。两组的治疗满意度评分相似
