Ferrazzano Gina, Fantozzi Roberta, Haggiag Shalom, Landi Doriana, Napoli Francesca, Buscarinu Maria Chiara, Malimpensa Leonardo, Bianco Assunta, Borriello Giovanna, Barbuti Elena, Marinelli Fabiana, Monteleone Fabrizia, Marchione Francesca, Falcone Nicola, Altieri Marta, Leodori Giorgio, Belvisi Daniele, Buttari Fabio, Pozzilli Valeria, Cicia Alessandra, Cortese Antonio, Sica Francesco, Landi Anna Chiara, Ferraro Elisabetta, Pozzilli Carlo, Mirabella Massimiliano, Tortorella Carla, Marfia Girolama Alessandra, Centonze Diego, Salvetti Marco, Conte Antonella
Department of Human Neurosciences, Sapienza University, Rome, Italy.
IRCCS Neuromed, Pozzilli, Italy.
Neurol Ther. 2025 Sep 19. doi: 10.1007/s40120-025-00818-7.
Ofatumumab (OFA) is a highly effective therapeutic option for multiple sclerosis (MS), but real-world data on its efficacy and safety remain limited. We evaluated the real-world efficacy and safety of OFA in patients with MS and explored the predictive value of frailty.
We retrospectively collected clinical and MRI data from 12 MS centers in Central Italy, including patients who initiated OFA between April 2022 and January 2024. We assessed annualized relapse rate (ARR), clinical relapses, radiological activity, and safety. Frailty, defined as increased vulnerability due to age-related health deficits, was measured using a frailty index (FI). The study was approved by the local Ethics Committee (No. 6357).
A total of 242 patients with MS were included (66.8% female and 33.2% male; mean age: 38.9 ± 10.3 years; disease duration: 7.7 ± 7.6 years). Of these, 95 (39.2%) were treatment-naïve, and 147 (60.8%) had switched from another therapy, mostly a first switch. The mean follow-up was 15.4 ± 5.4 months; all patients completed 12-month follow-up, and 103 completed 24 months. ARR dropped from 0.9 to 0.02 (p < 0.001). Only 4 patients (1.6%) had a clinical relapse, all within 6 months (mean time: 3.0 ± 1.8 months). Expanded Disability Status Scale (EDSS) scores remained stable (p > 0.05). MRI activity occurred in 10 patients (4.1%) at 6 months and 3 (1.2%) at 12 months; none at 24 months. Adverse events included flu-like symptoms (34.3%), injection site reactions (8.2%), and infections (18.5%). Among 239 patients assessed for frailty (mean FI: 0.06 ± 0.08), 187 were relatively fit (FI ≤ 0.10), 30 least fit, and 22 frail. FI predicted 24-month confirmed disability progression (p = 0.0068), with significant variation by frailty level (p = 0.0009).
This real-world study suggests that OFA is effective and safe for MS, offering rapid disease control. Lower frailty levels suggest preferential use in patients with lower baseline disability. Further large-scale, long-term studies are needed.
奥法妥木单抗（OFA）是治疗多发性硬化症（MS）的一种高效治疗选择，但关于其疗效和安全性的真实世界数据仍然有限。我们评估了OFA在MS患者中的真实世界疗效和安全性，并探讨了虚弱的预测价值。
我们回顾性收集了意大利中部12个MS中心的临床和MRI数据，包括2022年4月至2024年1月开始使用OFA的患者。我们评估了年化复发率（ARR）、临床复发、影像学活动和安全性。虚弱定义为由于与年龄相关的健康缺陷导致的易损性增加，使用虚弱指数（FI）进行测量。该研究获得了当地伦理委员会的批准（第6357号）。
共纳入242例MS患者（女性66.8%，男性33.2%；平均年龄：38.9±10.3岁；病程：7.7±7.6年）。其中，95例（39.2%）为初治患者，147例（60.8%）从另一种治疗方案转换而来，大多是首次转换。平均随访时间为15.4±5.4个月；所有患者均完成了12个月的随访，103例完成了24个月的随访。ARR从0.9降至0.02（p<0.001）。仅4例患者（1.6%）发生临床复发，均在6个月内（平均时间：3.0±1.8个月）。扩展残疾状态量表（EDSS）评分保持稳定（p>0.05）。6个月时10例患者（4.1%）出现MRI活动，12个月时3例（1.2%）出现；24个月时无。不良事件包括流感样症状（34.3%）、注射部位反应（8.2%）和感染（18.5%）。在239例评估虚弱的患者中（平均FI：0.06±0.08），187例相对健康（FI≤0.10），30例最不健康，22例虚弱。FI预测24个月确诊的残疾进展（p=0.0068），不同虚弱水平有显著差异（p=0.0009）。
这项真实世界研究表明，OFA对MS有效且安全，能快速控制疾病。较低的虚弱水平提示优先用于基线残疾较低的患者。需要进一步的大规模、长期研究。
