Target trial emulation to replicate randomised clinical trials using registry data in multiple sclerosis.
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Gavoille Antoine, Nourredine Mikail, Rollot Fabien, Casey Romain, Mathey Guillaume, Kerbrat Anne, Ciron Jonathan, De Sèze Jérôme, Stankoff Bruno, Maillart Elisabeth, Ruet Aurelie, Labauge Pierre, Kwiatkowski Arnaud, Zephir Helene, Papeix Caroline, Defer Gilles, Lebrun-Frenay Christine, Moreau Thibault, Laplaud David-Axel, Berger Eric, Clavelou Pierre, Thouvenot Eric, Heinzlef Olivier, Pelletier Jean, Al Khedr Abdullatif, Casez Olivier, Bourre Bertrand, Wahab Abir, Magy Laurent, Moulin Solène, Camdessanché Jean-Philippe, Doghri Ines, Sarov Mariana, Hankiewicz Karolina, Pottier Corinne, Dos Santos Amélie, Manchon Eric, Tchikviladze Maia, Rabilloud Muriel, Subtil Fabien, Vukusic Sandra
Аннотация
METHODS: This multicentre, retrospective, observational study was conducted using data extracted in December 2023 from the Observatoire Français de la Sclérose en Plaques (OFSEP) database. For each emulated trial, patients were included when they initiated one of the DMT evaluated in the corresponding RCT and met its inclusion criteria. Clinical outcomes were the annualised relapse rate and 3-month confirmed Expanded Disability Status Scale progression. Radiological outcomes were new/enlarged T2-lesions and new gadolinium-enhanced T1-lesions on a brain MRI. A targeted maximum likelihood estimator was used to estimate the treatment effect adjusted for confounding factors between groups and corrected for censoring and missing outcome assessment.
RESULTS: 14 111 patients were included in eight emulated trials: ASSESS (fingolimod vs glatiramer acetate), BEYOND (interferon beta vs glatiramer acetate), CONFIRM (dimethyl fumarate (DMF) vs glatiramer acetate), OPERA (ocrelizumab vs interferon beta), REGARD (interferon beta vs glatiramer acetate), RIFUND-MS (rituximab vs DMF), TENERE (teriflunomide vs interferon beta) and TRANSFORMS (fingolimod vs interferon beta). Treatment effects estimated in emulated trials were concordant with RCT findings in seven of eight trials for relapse rate, and in all six trials assessing disability progression. Radiological outcomes were more challenging to replicate; concordance was achieved in three of five trials for new T2-lesions, and one of four trials for new gadolinium-enhanced T1-lesions.
CONCLUSION: The combined use of a TTE methodology and high-quality registry data is a valid tool to evaluate treatment effectiveness in MS.