Khandelwal Nikhil, Sanchirico Marie, Ajibade Ade, Munshi Kiraat, Vu Michelle, Engel-Nitz Nicole, Steiger Christina, Anderson Amy J, Karam Chafic
Takeda Pharmaceuticals USA, Lexington, Massachusetts.
Takeda Pharmaceuticals USA, Inc., Lexington, Massachusetts.
J Health Econ Outcomes Res. 2025 Jun 26;12(1):261-268. doi: 10.36469/001c.140817. eCollection 2025.
Multifocal motor neuropathy (MMN) is a rare, slowly progressive nerve disorder characterized by asymmetric limb weakness without sensory abnormalities. MMN is often misdiagnosed due to similarities in clinical symptoms with conditions including amyotrophic lateral sclerosis (ALS), making diagnosis and treatment challenging. This study assessed patient characteristics, treatment patterns, and the economic burden of MMN in the United States. Using the Optum Research Database, this retrospective analysis included patients with ≥1 diagnostic or nondiagnostic medical claim with an MMN diagnosis code between 2016 and 2020 (date of first diagnosis-related claim =index date), and continuous enrollment for 12 months preindex and postindex. Patients with MMN within this group were identified using more specific criteria; ≥2 MMN nondiagnostic claims separated by ≥30 days, with no subsequent ALS diagnosis during follow-up. All patients who did not meet these criteria were included in the MMN-mimic cohort. Outcomes included treatment patterns, differential diagnoses, healthcare utilization, and costs. Of 904 patients identified, 37% had MMN and 63% had an MMN-mimic condition. Patients with MMN were significantly younger than patients in the MMN-mimic cohort (mean, 64.9 vs 66.8 years; = .047). At preindex, significantly more patients with MMN received MMN-related medications than patients in the MMN-mimic cohort (20.5% vs 9.0%, respectively; < .001). Intravenous immunoglobulin (IVIG) was the most common MMN-related medication. At postindex, more patients with MMN used IVIG (28.0%) compared with preindex (16.4%). In the 12 months preindex and postindex, >70% of patients had ≥1 differential diagnosis. The MMN cohort had higher all-cause total costs than the MMN-mimic cohort (mean preindex, 48 132, respectively [ = .100]; mean postindex, 50 652 [P = .002]); they also had significantly higher MMN-related healthcare costs (mean preindex, 12 890 [ = .011]; mean postindex, 11 938 [ < .001]). This study showed that most patients with initial MMN diagnoses had an alternative disorder after subsequent evaluation/follow-up, and patients with MMN incurred higher costs. Many patients with MMN did not receive IVIG, suggesting that undertreatment or misattribution of diagnosis codes are common. Further education is needed regarding accurate diagnosis of MMN to ensure patient access to guideline-recommended treatment.
多灶性运动神经病(MMN)是一种罕见的、缓慢进展的神经疾病,其特征为肢体不对称无力且无感觉异常。由于MMN的临床症状与包括肌萎缩侧索硬化症(ALS)在内的疾病相似,常被误诊,这使得诊断和治疗颇具挑战性。本研究评估了美国MMN患者的特征、治疗模式及经济负担。利用Optum研究数据库,这项回顾性分析纳入了在2016年至2020年期间有≥1次诊断或未诊断的医疗索赔且带有MMN诊断代码的患者(首次诊断相关索赔日期=索引日期),并在索引日期前后连续登记12个月。该组内的MMN患者通过更具体的标准来确定;≥2次MMN未诊断索赔间隔≥30天,且随访期间无后续ALS诊断。所有不符合这些标准的患者被纳入MMN模拟队列。结果包括治疗模式、鉴别诊断、医疗保健利用情况和费用。在确定的904例患者中,37%患有MMN,63%患有MMN模拟疾病。MMN患者比MMN模拟队列中的患者显著年轻(平均年龄分别为64.9岁和66.8岁;P = 0.047)。在索引日期前,接受MMN相关药物治疗的MMN患者明显多于MMN模拟队列中的患者(分别为20.5%和9.0%;P < 0.001)。静脉注射免疫球蛋白(IVIG)是最常见的MMN相关药物。在索引日期后,使用IVIG的MMN患者比索引日期前更多(28.0%对比16.4%)。在索引日期前后的12个月里,>70%的患者有≥1次鉴别诊断。MMN队列的全因总成本高于MMN模拟队列(索引日期前平均分别为48132美元[P = 0.100];索引日期后平均为50652美元[P = 0.002]);他们的MMN相关医疗费用也显著更高(索引日期前平均为12890美元[P = 0.011];索引日期后平均为11938美元[P < 0.001])。这项研究表明,大多数最初被诊断为MMN的患者在后续评估/随访后患有其他疾病,且MMN患者产生的费用更高。许多MMN患者未接受IVIG治疗,这表明治疗不足或诊断代码错误归因很常见。需要进一步开展关于MMN准确诊断的教育,以确保患者能够获得指南推荐的治疗。