■视神经脊髓炎谱系障碍(NMOSD)是一种罕见且使人衰弱的疾病,在中国已得到越来越广泛的认可。政府已实施立法措施,以改善罕见疾病的治疗机会。
■要调查诊断过程,治疗状态,治疗可及性,中国NMOSD患者对疾病改良疗法(DMT)的治疗满意度。
■患者在线调查。
■这项横断面在线调查是在2022年11月至2023年1月之间进行的。包括18岁以上并被诊断患有NMOSD的患者。问卷由五个部分组成,涵盖人口统计学,诊断和治疗经验,DMT可用性,成本和负担能力,和治疗满意度使用治疗满意度问卷(第二版)。在调查结束时还收集了患者的意见和要求。
■共招募了375名诊断为NMOSD的患者,其中321名患者使用DMT。确定诊断需要1.22±3.22年和3.58±4.24次医院就诊。三分之一的患者仍然需要旅行超过2小时才能获得DMT。治疗总支出估计为每年59,827.00元(8315.95美元)。仅药品费用就占家庭年平均收入的52.22%。人们认为最常见的挑战是无力负担治疗费用和缺乏有效的选择。DMT的治疗满意度无显著差异,与其他DMT相比,利妥昔单抗的便利性得分最低。患者的年龄和获得药物所需的旅行时间与总体治疗满意度呈负相关。
■在中国,由于诊断困难,NMOSD患者在获得适当治疗方面面临挑战,遥远的药物访问,和高成本。政策应优先改善疾病教育,减轻患者的经济负担。
UNASSIGNED: Neuromyelitis optica spectrum disorder (NMOSD) is a rare and debilitating disease that has become more widely recognized in
China. Legislative measures have been implemented by the government to improve treatment access for rare diseases.
UNASSIGNED: To investigate the diagnostic journey, treatment status, treatment accessibility, and treatment satisfaction of the NMOSD patients on disease-modifying therapies (DMTs) in
China.
UNASSIGNED: A patient online survey.
UNASSIGNED: This cross-sectional online survey was conducted between November 2022 and January 2023. Patients over 18 years old and diagnosed with NMOSD were included. The questionnaire consisted of five sections covering demographics, diagnostic and treatment experiences, DMTs availability, cost and affordability, and treatment satisfaction using the Treatment Satisfaction Questionnaire for Medication (version II). Patient opinions and demands were also collected at the end of the survey.
UNASSIGNED: A total of 375 patients diagnosed with NMOSD were recruited, of which 321 patients used DMTs. It required 1.22 ± 3.22 years and 3.58 ± 4.24 hospital visits for a definitive diagnosis. One-third of the patients still needed to travel for over 2 h to access DMTs. The total treatment expenditure was estimated to be CNY 59,827.00 (USD 8315.95) a year. Drug expenses alone accounted for 52.22% of the average annual household income. The most common challenges perceived were the inability to afford treatment and a lack of effective options. No significant difference was found in treatment satisfaction among DMTs, except that rituximab scored lowest in convenience compared to other DMTs. Patients\' age and travel time required to obtain medications were negatively associated with global treatment satisfaction.
UNASSIGNED: In
China, patients with NMOSD face challenges in obtaining proper treatment due to diagnostic difficulties, distant medication access, and high costs. Policies should prioritize improving disease education and alleviating financial burdens for the patients.