Real-World Effectiveness of Natalizumab Extended Interval Dosing in a French Cohort

Introduction Natalizumab, a therapy for relapsing–remitting multiple sclerosis (RRMS), is associated with a risk of progressive multifocal leukoencephalopathy (PML). Over the last several years, practitioners have used off-label extended interval dosing (EID) of natalizumab to reduce PML risk, despi...

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Published in:Neurology and therapy 2023-04, Vol.12 (2), p.529-542
Main Authors: Pelle, Juliette, Briant, Anais R., Branger, Pierre, Derache, Nathalie, Arnaud, Charlotte, Lebrun-Frenay, Christine, Cohen, Mikael, Mondot, Lydiane, De Seze, Jerome, Bigaut, Kevin, Collongues, Nicolas, Kremer, Laurent, Ricard, Damien, Bompaire, Flavie, Ohlmann, Charlotte, Sallansonnet-Froment, Magali, Ciron, Jonathan, Biotti, Damien, Pignolet, Beatrice, Parienti, Jean-Jacques, Defer, Gilles
Format: Article
Language:English
Subjects:
Extended interval dosing
Internal Medicine
Life Sciences
Medicine
Medicine & Public Health
Multiple sclerosis
Natalizumab
NCT
NCT04580381
Neurology
Original Research
Real-world evidence
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Summary:Introduction Natalizumab, a therapy for relapsing–remitting multiple sclerosis (RRMS), is associated with a risk of progressive multifocal leukoencephalopathy (PML). Over the last several years, practitioners have used off-label extended interval dosing (EID) of natalizumab to reduce PML risk, despite the absence of a large-scale efficacy evaluation. Methods We conducted a retrospective, multicenter cohort study among adults with RRMS receiving stable standard interval dosing (SID), defined as a ≥ 12-month consecutive period of ≥ 11 natalizumab infusions/year in France. We compared the 12-month risk difference of remaining relapse-free (primary endpoint) between patients who switched to EID (≤ 9 natalizumab infusions) and those who remained on SID, with a noninferiority margin of − 11%. We used propensity score methods such as inverse probability treatment weighting (IPTW) and 1:1 propensity score matching (PSM). Secondary endpoints were annualized relapse rate, disease progression, and safety. Results Baseline characteristics were similar between patients receiving EID ( n  = 147) and SID ( n  = 156). The proportion of relapse-free patients 12 months postbaseline was 142/147 in the EID (96.6%) and 144/156 in the SID group (92.3%); risk difference (95% CI) 4.3% (− 1.3 to 9.8%); p  
ISSN:2193-8253
2193-6536
DOI:10.1007/s40120-023-00440-5