Causal effects of multiple sclerosis therapies in left-truncated registry data.
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Haile Dagmawi Chilot, Diouf Ibrahima, Ozakbas Serkan, Horakova Dana, Havrdova Eva Kubala, Patti Francesco, Eichau Sara, Alroughani Raed, Lugaresi Alessandra, Tomassini Valentina, Prat Alexandre, Girard Marc, Terzi Murat, Yamout Bassem, Khoury Samia J, Grammond Pierre, Blanco Yolanda, Shaygannejad Vahid, Foschi Matteo, Surcinelli Andrea, Neri Stefano, Weinstock-Guttman Bianca, Prevost Julie, Amato Maria Pia, Barnett Michael, Gerlach Oliver, John Nevin, Kermode Allan G, Fabis-Pedrini Marzena, Carroll William M, van der Walt Anneke, Butzkueven Helmut, van Pesch Vincent, Soysal Aysun, Gouider Riadh, Mrabet Saloua, Spitaleri Daniele, Cartechini Elisabetta, Maimone Davide, Ampapa Radek, Laureys Guy, Ramo-Tello Cristina, Di Gregorio Maria, Lapointe Emmanuelle, Slee Mark, Karabudak Rana, Garber Justin, Altintas Ayse, Hodgkinson Suzanne, Sanchez-Menoyo Jose Luis, Castillo-Triviño Tamara, Habek Mario, Al-Asmi Abdullah, Al-Harbi Talal, Csepany Tunde, Cárdenas-Robledo Simón, Taylor Bruce, Foong Yi Chao, Willekens Barbara, Shalaby Nevin, Moore Fraser, McGuigan Chris, Baghbanian Seyed Mohammad, Massey Jennifer, Hardy Todd A, Ramanathan Sudarshini, Gross-Paju Katrin, Gray Orla, Decoo Danny, Shaw Cameron, Simu Mihaela, Rozsa Csilla, Stuart Elizabeth A, Sharmin Sifat, Roos Izanne, Kalincik Tomas
Аннотация
OBJECTIVES: To examine whether causal effect estimates of DMTs over the full disease course can be reliably derived from left-truncated registry data.
METHODS: We analysed data from MSBase (144 centres, 41 countries) to assess the impact of left-truncation on causal treatment effect estimates. Cox marginal structural models (MSMs) estimated hazard ratios (HRs) for relapses, disability worsening and improvement, considering left-truncation at random and not-at-random. Fixed-time truncation and multivariable adjustment were applied to remediate bias.
RESULTS: The study included 5588 patients tracked from true MS onset. The null model, without left-truncation, estimated the DMT effect on relapse risk (HR = 0.64; 95% confidence interval (CI) = 0.54-0.77). Left-truncation inflated this estimate. Shorter random truncation (1 year) produced greater bias (HR = 0.34), decreasing with longer durations (3-year HR = 0.48). Truncation not-at-random biased relapse estimates (HR = 0.37). Disability outcomes were less sensitive.
CONCLUSION: MSMs can reliably estimate DMT effectiveness in left-truncated MS registry data, although accuracy depends on truncation mechanism and duration. Both random and not-at-random truncation impact relapse estimates. Disability outcomes appear less sensitive. Fixed-time truncation and covariate adjustment mitigated bias.