Campbell Julie A, Henson Glen J, Ngwa Valery Fuh, Ahmad Hasnat, Taylor Bruce V, van der Mei Ingrid, Palmer Andrew J
Medical Sciences Precinct, Menzies Institute for Medical Research, University of Tasmania, 17 Liverpool Street, Hobart, TAS, 7000, Australia.
Pharmacoeconomics. 2025 Feb;43(2):223-239. doi: 10.1007/s40273-024-01417-4. Epub 2024 Aug 2.
Multiple sclerosis (MS) is a chronic autoimmune/neurodegenerative disease associated with progressing disability affecting mostly women. We aim to estimate transition probabilities describing MS-related disability progression from no disability to severe disability. Transition probabilities are a vital input for health economics models. In MS, this is particularly relevant for pharmaceutical agency reimbursement decisions for disease-modifying therapies (DMTs).
Data were obtained from Australian participants of the MSBase registry. We used a four-state continuous-time Markov model to describe how people with MS transition between disability milestones defined by the Expanded Disability Status Scale (scale 0-10): no disability (EDSS of 0.0), mild (EDSS of 1.0-3.5), moderate (EDSS of 4.0-6.0), and severe (EDSS of 6.5-9.5). Model covariates included sex, DMT usage, MS-phenotype, and disease duration, and analysis of covariate groups were also conducted. All data were recorded by the treating neurologist.
A total of N = 6369 participants (mean age 42.5 years, 75.00% female) with 38,837 person-years of follow-up and 54,570 clinical reviews were identified for the study. Annual transition probabilities included: remaining in the no, mild, moderate, and severe states (54.24%, 82.02%, 69.86%, 77.83% respectively) and transitioning from no to mild (42.31%), mild to moderate (11.38%), and moderate to severe (9.41%). Secondary-progressive MS was associated with a 150.9% increase in the hazard of disability progression versus relapsing-remitting MS.
People with MS have an approximately 45% probability of transitioning from the no disability state after one year, with people with progressive MS transitioning from this health state at a much higher rate. These transition probabilities will be applied in a publicly available health economics simulation model for Australia and similar populations, intended to support reimbursement of a plethora of existing and upcoming interventions including medications to reduce progression of MS.
多发性硬化症（MS）是一种慢性自身免疫性/神经退行性疾病，与残疾进展相关，主要影响女性。我们旨在估计描述MS相关残疾从无残疾进展到严重残疾的转移概率。转移概率是卫生经济学模型的重要输入。在MS中，这对于药物管理机构对疾病修正疗法（DMTs）的报销决策尤为重要。
数据来自MSBase注册库的澳大利亚参与者。我们使用四状态连续时间马尔可夫模型来描述MS患者如何在由扩展残疾状态量表（0 - 10级）定义的残疾里程碑之间转变：无残疾（EDSS为0.0）、轻度（EDSS为1.0 - 3.5）、中度（EDSS为4.0 - 6.0）和重度（EDSS为6.5 - 9.5）。模型协变量包括性别（sex）、DMT使用情况、MS表型和疾病持续时间，还对协变量组进行了分析。所有数据均由主治神经科医生记录。
本研究共纳入N = 6369名参与者（平均年龄42.5岁，75.00%为女性），随访时间共计38837人年，进行了54570次临床评估。年度转移概率包括：保持在无、轻度、中度和重度状态（分别为54.24%、82.02%、69.86%、77.83%）以及从无到轻度（42.31%）、从轻度到中度（11.38%）和从中度到重度（9.41%）。继发进展型MS与复发缓解型MS相比，残疾进展风险增加150.9%。
MS患者在一年后从无残疾状态转变的概率约为45%，进展型MS患者从该健康状态转变的概率要高得多。这些转移概率将应用于澳大利亚及类似人群的公开可用卫生经济学模拟模型中，旨在支持众多现有和即将出现的干预措施（包括减少MS进展的药物）的报销。
