Center of Clinical Neuroscience, Neurological University Clinic Carl Gustav Carus, University of Technology, Dresden, Germany.
NeuroPoint Patient Academy and Neurological Practice, Ulm, Germany.
J Neurol. 2022 Jun;269(6):3276-3285. doi: 10.1007/s00415-021-10931-w. Epub 2022 Jan 4.
To evaluate the 5-year real-world benefit-risk profile of fingolimod in patients with relapsing-remitting MS (RRMS) in Germany.
Post-Authorization Non-interventional German sAfety study of GilEnyA (PANGAEA) is a non-interventional real-world study to prospectively assess the effectiveness and safety of fingolimod in routine clinical practice in Germany. The follow-up period comprised 5 years. Patients were included if they had been diagnosed with RRMS and had been prescribed fingolimod as part of clinical routine. There were no exclusion criteria except the contraindications for fingolimod as defined in the European label. The effectiveness and safety analysis set comprised 4032 and 4067 RRMS patients, respectively.
At the time of the 5-year follow-up of PANGAEA, 66.57% of patients still continued fingolimod therapy. Annualized relapse rates decreased from baseline 1.5 ± 1.15 to 0.42 ± 0.734 at year 1 and 0.21 ± 0.483 at year 5, and the disability status remained stable, as demonstrated by the Expanded Disability Status Scale mean change from baseline (0.1 ± 2.51), the decrease of the Multiple Sclerosis Severity Score from 5.1 ± 2.59 at baseline to 3.9 ± 2.31 at the 60-months follow-up, and the percentage of patients with 'no change' in the Clinical Global Impression scale at the 60-months follow-up (78.11%). Adverse events (AE) occurring in 75.04% of patients were in line with the known safety profile of fingolimod and were mostly non-serious AE (33.62%) and non-serious adverse drug reactions (50.59%; serious AE 4.98%; serious ADR 10.82%).
PANGAEA demonstrated the sustained beneficial effectiveness and safety of fingolimod in the long-term real-world treatment of patients with RRMS.
评估在德国，临床实际环境下，5 年时间内，那他珠单抗对复发缓解型多发性硬化症（RRMS）患者的获益-风险比。
在德国，一项名为吉尔尼娅（Gilenya）上市后授权非干预性安全性研究（PANGAEA）的观察性研究，前瞻性评估那他珠单抗在临床常规中的有效性和安全性。该研究的随访时间为 5 年。患者入组标准为诊断为 RRMS，并在临床常规中接受那他珠单抗治疗。无排除标准，除了那他珠单抗欧洲说明书中定义的禁忌症。有效性和安全性分析集分别纳入 4032 例和 4067 例 RRMS 患者。
在 PANGAEA 研究的 5 年随访时，66.57%的患者仍继续接受那他珠单抗治疗。年复发率从基线时的 1.5±1.15 降至第 1 年的 0.42±0.734 和第 5 年的 0.21±0.483，残疾状态保持稳定，扩展残疾状况量表评分基线时平均变化（0.1±2.51）、多发性硬化严重程度评分从基线时的 5.1±2.59降至第 60 个月随访时的 3.9±2.31 以及第 60 个月随访时临床总体印象量表评分中“无变化”的患者比例（78.11%）均证实了这一点。75.04%的患者发生的不良事件（AE）与那他珠单抗已知的安全性特征一致，且大多为非严重 AE（33.62%）和非严重药物不良反应（50.59%；严重 AE 4.98%；严重药物不良反应 10.82%）。
PANGAEA 研究表明，在 RRMS 患者的长期临床实际治疗中，那他珠单抗具有持续的获益-风险比。
