Minden Sarah L, Kinkel R Philip, Machado Helene T, Levin Jonathan S, Rosenthal Meredith B, Iezzoni Lisa I
Department of Psychiatry, Brigham and Women's Hospital, USA.
Multiple Sclerosis Program, University of California San Diego, USA.
Mult Scler J Exp Transl Clin. 2019 Feb 20;5(1):2055217318820888. doi: 10.1177/2055217318820888. eCollection 2019 Jan-Mar.
Disease-modifying therapies benefit individuals with relapsing forms of multiple sclerosis, but their utility remains unclear for those without relapses.
To determine disease-modifying therapy use and costs in 2009, compare use in 2009 and 2000, and examine compliance with evidence-based guidelines.
We determined the extent and characteristics of disease-modifying therapy use by participants in the Sonya Slifka Longitudinal Multiple Sclerosis Study (Slifka) in 2000 (=2156) and 2009 (=2361) and estimated out-of-pocket and total (payer) costs for 2009. Two multivariable logistic regressions predicted disease-modifying therapy use.
Disease-modifying therapy use increased from 55.3% in 2000 to 61.5% in 2009. In 2009, disease-modifying therapy use was reported by 76.5% of participants with relapsing-remitting multiple sclerosis, 73.2% with progressive-relapsing multiple sclerosis, 62.5% with secondary progressive multiple sclerosis, and 41.8% with primary progressive multiple sclerosis. Use was significantly associated with relapsing-remitting multiple sclerosis, shorter duration of illness, one to two relapses per year, non-ambulatory symptoms, using a cane, younger age, higher family income, and having health insurance. Average annual costs in 2009 were US$939-3101 for patients and US$16,302-18,928 for payers.
Use rates were highest for individuals with relapsing-remitting multiple sclerosis, but substantial for those with progressive courses although clinical trials have not demonstrated significant benefits for them.
疾病修饰疗法对复发型多发性硬化症患者有益，但对无复发患者的效用仍不明确。
确定2009年疾病修饰疗法的使用情况和成本，比较2009年和2000年的使用情况，并检查对循证指南的依从性。
我们确定了2000年（=2156例）和2009年（=2361例）索尼娅·斯利夫卡纵向多发性硬化症研究（斯利夫卡研究）参与者使用疾病修饰疗法的程度和特征，并估计了2009年的自付费用和总（支付方）费用。两项多变量逻辑回归预测了疾病修饰疗法的使用情况。
疾病修饰疗法的使用率从2000年的55.3%增至2009年的61.5%。2009年，复发缓解型多发性硬化症患者中有76.5%报告使用了疾病修饰疗法，进展复发型多发性硬化症患者中有73.2%，继发进展型多发性硬化症患者中有62.5%，原发进展型多发性硬化症患者中有41.8%。使用情况与复发缓解型多发性硬化症、病程较短、每年复发一至两次、非行走性症状、使用手杖、年龄较小、家庭收入较高以及拥有医疗保险显著相关。2009年患者的年均费用为939 - 3101美元，支付方为16302 - 18928美元。
复发缓解型多发性硬化症患者的使用率最高，但进展型病程患者的使用率也较高，尽管临床试验尚未证明对他们有显著益处。
