Achiron Anat, Aref Hany, Inshasi Jihad, Harb Mohamad, Alroughani Raed, Bijarnia Mahendra, Cooke Kathryn, Yuksel Ozgur
Multiple Sclerosis Center, Sheba Medical Center, 52621, Tel-Hashomer, Israel.
Ain Shams University, Cairo, Egypt.
BMC Neurol. 2017 Aug 7;17(1):150. doi: 10.1186/s12883-017-0913-3.
Evidence on the use of fingolimod in real-world clinical practice and data on patient-reported health-related quality of life (HRQoL) in countries such as the Middle East are sparse. The Prospective Evaluation of Treatment with Fingolimod for Multiple Sclerosis (PERFORMS) study assessed HRQoL and effectiveness and safety of fingolimod in patients with relapsing-remitting multiples sclerosis (RRMS), primarily in Middle Eastern countries.
This 12-month, observational, multicentre, prospective, real-world study was conducted in patients with RRMS who initiated fingolimod or another approved disease-modifying treatment (DMT) within 4 weeks before study entry. Patients were enrolled in a 2:1 ratio to obtain more data in fingolimod and parallel in other DMTs cohort by physicians during routine medical care. Key study outcomes included HRQoL assessed using MS International QoL (MusiQoL), MS relapses and disability. Safety was assessed throughout the study period. Due to the observational nature of the study, no neuroimaging assessments were mandated and central reading was not performed.
Of 249 enrolled patients, 247 were included in the analysis (fingolimod cohort 172; other DMTs cohort 75). Overall, the mean age of patients was 36.5 years, 64.4% were women and ~90% were Caucasians. At baseline, mean MS duration since diagnosis was 7.2 years in the fingolimod and 4.8 years in the other DMTs cohorts. Overall, mean changes in MusiQoL index scores were -2.1 in the fingolimod cohort and -0.7 in the other DMTs cohort at Month 12, but improvement was not significant vs. baseline in both cohorts. Proportion of relapse-free patients increased significantly during the study vs. 0-12 months before the study in the fingolimod cohort (80.2% vs. 24.4%; p < 0.0001). Proportion of patients free from disability progression was 86.5% in the fingolimod cohort. The incidences of AEs were 59.9% and 50.6% in the fingolimod and other DMTs cohorts, respectively. First-dose monitoring of fingolimod observed no cases of symptomatic bradyarrhythmia. Three cases of bradycardia were reported in the fingolimod cohort: one after the first dose and two during the study. No cases of macular oedema were observed during the study.
Fingolimod treatment maintained QoL over 12 months and was effective in reducing relapse rate and disability progression. No new safety findings were observed in this real-world observational study in Middle Eastern countries.
关于芬戈莫德在实际临床实践中的应用证据以及中东等国家患者报告的健康相关生活质量（HRQoL）数据较为匮乏。芬戈莫德治疗多发性硬化症的前瞻性评估（PERFORMS）研究评估了复发缓解型多发性硬化症（RRMS）患者中芬戈莫德的HRQoL、有效性和安全性，主要在中东国家进行。
这项为期12个月的观察性、多中心、前瞻性、真实世界研究纳入了在研究入组前4周内开始使用芬戈莫德或其他获批疾病修饰治疗（DMT）的RRMS患者。患者按2:1的比例入组，以便医生在常规医疗护理期间在芬戈莫德组获得更多数据，并在其他DMT组进行平行研究。主要研究结局包括使用MS国际生活质量量表（MusiQoL）评估的HRQoL、MS复发和残疾情况。在整个研究期间评估安全性。由于研究的观察性性质，未强制进行神经影像学评估且未进行中心阅片。
在249名入组患者中，247名被纳入分析（芬戈莫德组172名；其他DMT组75名）。总体而言，患者的平均年龄为36.5岁，64.4%为女性，约90%为白种人。基线时，芬戈莫德组自诊断以来的MS平均病程为7.2年，其他DMT组为4.8年。总体而言，在第12个月时，芬戈莫德组MusiQoL指数评分的平均变化为-2.1，其他DMT组为-0.7，但两组与基线相比改善均不显著。与研究前0 - 12个月相比，芬戈莫德组在研究期间无复发患者的比例显著增加（80.2%对24.4%；p < 0.0001）。芬戈莫德组无残疾进展的患者比例为86.5%。芬戈莫德组和其他DMT组的不良事件发生率分别为59.9%和50.6%。芬戈莫德的首剂监测未观察到有症状性缓慢性心律失常病例。芬戈莫德组报告了3例心动过缓病例：1例在首剂后，2例在研究期间。研究期间未观察到黄斑水肿病例。
芬戈莫德治疗在12个月内维持了生活质量，并且在降低复发率和残疾进展方面有效。在中东国家的这项真实世界观察性研究中未观察到新的安全性发现。
