Iaffaldano Pietro, Lucisano Giuseppe, Guerra Tommaso, Patti Francesco, Onofrj Marco, Brescia Morra Vincenzo, Zaffaroni Mauro, Pozzilli Carlo, Cocco Eleonora, Sola Patrizia, Salemi Giuseppe, Inglese Matilde, Bergamaschi Roberto, Gasperini Claudio, Conte Antonella, Salvetti Marco, Lus Giacomo, Maniscalco Giorgia Teresa, Totaro Rocco, Vianello Marika, Granella Franco, Ferraro Elisabetta, Aguglia Umberto, Gatto Maurizia, Sangalli Francesca, Chisari Clara Grazia, De Luca Giovanna, Carotenuto Antonio, Baroncini Damiano, Colombo Delia, Nica Mihaela, Paolicelli Damiano, Comi Giancarlo, Filippi Massimo, Amato Maria Pia, Trojano Maria
Department of Basic Medical Sciences, Neurosciences and Sense Organs, University of Bari 'Aldo Moro', Bari, Italy.
Department of Basic Medical Sciences, Neurosciences and Sense Organs, University of Bari 'Aldo Moro', Bari, Italy/Center for Outcomes Research and Clinical Epidemiology, Pescara, Italy.
Mult Scler. 2022 Dec;28(14):2243-2252. doi: 10.1177/13524585221114007. Epub 2022 Aug 15.
Definitions for reliable identification of transition from relapsing-remitting multiple sclerosis (MS) to secondary progressive (SP)MS in clinical cohorts are not available.
To compare diagnostic performances of two different data-driven SPMS definitions.
Data-driven SPMS definitions based on a version of Lorscheider's algorithm (DDA) and on the EXPAND trial inclusion criteria were compared, using the neurologist's definition (ND) as gold standard, in terms of sensitivity, specificity, positive predictive value (PPV), negative predictive value (NPV), Akaike information criterion (AIC) and area under the curve (AUC).
A cohort of 10,240 MS patients with ⩾5 years of follow-up was extracted from the Italian MS Registry; 880 (8.5%) patients were classified as SPMS according to the neurologist definition, 1806 (17.6%) applying the DDA and 1134 (11.0%) with the EXPAND definition. The DDA showed greater discrimination power (AUC: 0.8 vs 0.6) and a higher sensitivity (77.1% vs 38.0%) than the EXPAND definition, with similar specificity (88.0% vs 91.5%). PPV and NPV were higher using the DDA than considering EXPAND definition (37.5% vs 29.5%; 97.6% vs 94.0%).
Data-driven definitions demonstrated greater ability to capture SP transition than neurologist's definition and the global accuracy of DDA seems to be higher than the EXPAND definition.
目前尚无可靠识别临床队列中复发缓解型多发性硬化症（MS）向继发进展型（SP）MS转变的定义。
比较两种不同的数据驱动的SPMS定义的诊断性能。
以神经科医生的定义（ND）作为金标准，比较基于洛施奈德算法版本（DDA）和EXPAND试验纳入标准的数据驱动的SPMS定义在敏感性、特异性、阳性预测值（PPV）、阴性预测值（NPV）、赤池信息准则（AIC）和曲线下面积（AUC）方面的表现。
从意大利MS注册中心提取了10240例随访时间≥5年的MS患者队列；根据神经科医生的定义，880例（8.5%）患者被归类为SPMS，应用DDA定义的有1806例（17.6%），应用EXPAND定义的有1134例（11.0%）。与EXPAND定义相比，DDA显示出更大的辨别能力（AUC：0.8对0.6）和更高的敏感性（77.1%对38.0%），特异性相似（88.0%对91.5%）。使用DDA时的PPV和NPV高于考虑EXPAND定义时（37.5%对29.5%；97.6%对94.0%）。
数据驱动的定义在捕捉向SP转变方面比神经科医生的定义表现出更强的能力，且DDA的整体准确性似乎高于EXPAND定义。
