Effect of rituximab on disease activity in latin American patients with anti-aquaporin-4 (+) neuromyelitis optica spectrum disorder.
利妥昔单抗对拉丁美洲 anti-aquaporin-4 (+) 视神经脊髓炎谱系障碍患者疾病活动度的影响。
- 作者列表："Gomez-Figueroa E","Noriega-Morales G","Casallas-Vanegas A","Zabala-Angeles I","Garcia-Estrada C","Neri D","Sarachaga AJ","Rivas-Alonso V","Corona-Vazquez T","Flores-Rivera J
OBJECTIVES:The aim of the present study is to explore the efficacy of rituximab in patients with Neuromyelitis Optica spectrum disorders (NMOsd) with positive AQP4-IgG serostatus. PATIENTS AND METHODS:In this single center retrospective study, we recruited seropositive anti-AQP4 NMOsd patients who received treatment with Rituximab (RTX) for at least 2 years. Demographics were described and annualized relapse rate (AAR) and survival analysis were performed for time to relapse with Rituximab. All p values ≤0.05 we considered statistically significant. RESULTS:A total of 15 patients (100 % female) were identified. Mean age of disease onset was 34 ± 11 years, mean time of disease was 8.11 ± 4.04 years and the median number of relapses was 5 (2-16). Ten patients received an immunosuppressive agent before RTX. Mean age of RTX initiation was 37 ± 12 with a mean treatment duration of 52 ± 28 months. The median ARR before and after treatment with RTX was 2.08 vs 0.00, respectively, with a difference of -2.08 (p < 0.001) CONCLUSIONS: This study shows a statistically significant reduction in the ARR and an increase in the relapse-free rate in AQP4-IgG NMOsd patients treated with RTX. These findings support the use of rituximab in our population, and indirectly suggests that its prompt use could modify the course of the disease.
目的: 本研究的目的是探讨利妥昔单抗治疗血清 AQP4-IgG 阳性的视神经脊髓炎谱系疾病 (NMOsd) 患者的疗效。 患者和方法: 在这项单中心回顾性研究中，我们招募了接受利妥昔单抗 (RTX) 治疗至少 2 年的血清阳性 anti-AQP4 例 NMOsd 患者。描述人口统计学，并对利妥昔单抗复发时间进行年复发率 (AAR) 和生存分析。所有 p值 ≤ 0.05 我们认为有统计学意义。 结果: 共确定 15 例患者 (100% 为女性)。平均发病年龄 34 ± 11 岁，平均发病时间 8.11 ± 4.04 岁，中位复发次数 5 例 (2-16)。10 例患者在 RTX 前接受免疫抑制剂治疗。RTX 开始的平均年龄为 37 ± 12 岁，平均治疗持续时间为 52 ± 28 个月。RTX 治疗前后 ARR 中位数分别为 2.08 和 0.00，差异有-2.08 (p <0.001)。本研究显示，接受 RTX 治疗的 AQP4-IgG NMOsd 患者 ARR 显著降低，无复发率增加。这些发现支持利妥昔单抗在我们人群中的使用，并间接表明其及时使用可以改变疾病的进程。
METHODS:Objective To clarify the existence of monophasic neuromyelitis optica spectrum disorders (NMOSD) and to identify predictive factors of long-term relapse-free form. Methods We retrospectively analyzed 289 Chinese patients with NMOSD. Selected subjects were divided into three groups based on the time interval between disease onset and the first relapse, if any. Clinical and imaging data were acquired from each patient’s medical record and evaluated as predictive factors for NMOSD. Results In total, none of the participating patients exhibited a monophasic form of NMOSD. Rather, 241 patients were selected for relapse tendency analysis; 143 (59.3%) patients relapsed within the first year, 66 (27.4%) during 1–5 years, and 32 (13.3%) beyond 5 years. Such onset symptoms as optic neuritis (ON) and non-longitudinally extensive transverse myelitis (LETM) were independent prognostic factors for a prolonged remission interval.
METHODS::Neuromyelitis optica spectrum disorders (NMOSD) are an inflammation of the central nervous system associated with autoantibodies to aquaporin-4. We have undertaken a clinic-based survey of NMOSD in the Australia and New Zealand populations with the aim of characterising the clinical features and establishing the value of recently revised diagnostic criteria. Cases of possible NMOSD and age and sex-matched controls with multiple sclerosis (MS) were referred from centres across Australia and New Zealand. Cases were classified as NMOSD if they met the 2015 IPND criteria and remained as suspected NMOSD if they did not. Clinical and paraclinical data were compared across the three groups. NMOSD was confirmed in 75 cases and 89 had suspected NMOSD. There were 101 controls with MS. Age at onset, relapse rates and EDSS scores were significantly higher in NMOSD than in MS. Lesions and symptoms referable to the optic nerve were more common in NMOSD whereas brainstem, cerebellar and cerebral lesions were more common in MS. Longitudinally extensive spinal cord lesions were seen in 48/71 (68%) of cases with NMOSD. Elevations of CSF, white cell count and protein were more common in NMOSD. We have confirmed a clinical pattern of NMOSD that has been seen in several geographical regions. We have demonstrated the clinical utility of the current diagnostic criteria. Distinct patterns of disease are evident in NMOSD and MS, but there remains a large number of patients with NMOSD-like features who do not meet the current diagnostic criteria for NMOSD and remain a diagnostic challenge.
METHODS:Spinal cord injury (SCI) is a traumatic lesion that causes disability with temporary or permanent sensory and/or motor deficits. The pharmacological approach still in use for the treatment of SCI involves the employment of corticosteroid drugs. However, SCI remains a very complex disorder that needs future studies to find effective pharmacological treatments. SCI actives a strong inflammatory response that induces a loss of neurons followed by a cascade of events that lead to further spinal cord damage. Many experimental studies demonstrate the therapeutic effect of stem cells in SCI due to their capacity to differentiate into neuronal cells and by releasing neurotrophic factors. Therefore, they appear to be a valid strategy to use in the field of regenerative medicine. The purpose of this paper is to provide an overview of clinical trials, recorded in clinical trial.gov during 2005−2019, aimed to evaluate the use of stem cell-based therapy in SCI. The results available thus far show the safety and efficacy of stem cell therapy in patients with SCI. However, future trials are needed to investigate the safety and efficacy of stem cell transplantation.