Literature DB >> 18663181

Quantitative risk-benefit analysis of natalizumab.

J P Thompson1, K Noyes, E R Dorsey, S R Schwid, R G Holloway.   

Abstract

OBJECTIVE: To model the long-term risks and benefits of natalizumab in individuals with relapsing multiple sclerosis (MS).
METHODS: We created a Markov model to evaluate treatment effects on reducing relapses and slowing disease progression using published natural history data and clinical trial results. Health changes, measured in quality-adjusted life-years (QALYs), were based on patient health preferences. Patient cohorts treated with no disease-modifying treatment, natalizumab, subcutaneous interferon beta-1a, and a theoretical "perfect" MS treatment were modeled. Sensitivity analysis was used to explore model uncertainty, including varying risks of developing progressive multifocal leukoencephalopathy (PML).
RESULTS: Treatment with natalizumab resulted in 9.50 QALYs over a 20-year time horizon, a gain of 0.80 QALYs over the untreated cohort and 0.38 QALYs over interferon beta-1a. The health loss due to PML was small (-0.06 QALYs). To offset natalizumab's incremental health gain over interferon beta-1a, the risk had to increase from 1 to 7.6 PML per 1,000 patients treated over 17.9 months. The "perfect" MS treatment accumulated 10.59 QALYs over the 20-year time horizon, 1.89 QALYs above the untreated cohort. Interferon beta-1a resulted in greater QALY gains compared with natalizumab if natalizumab's relative relapse reduction was reduced from 68% to 35% or if interferon beta-1a's relative reduction was increased from 32% to 65%.
CONCLUSIONS: A more than sevenfold increase in actual risk of progressive multifocal leukoencephalopathy was required to decrease natalizumab's health gain below that of interferon beta-1a, and there remains considerable room for additional gains in health (>50%) beyond those already achieved with current therapies.

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Year:  2008        PMID: 18663181      PMCID: PMC2676947          DOI: 10.1212/01.wnl.0000319648.65173.7a

Source DB:  PubMed          Journal:  Neurology        ISSN: 0028-3878            Impact factor:   9.910


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  11 in total

1.  Natalizumab: benefit outweighs risk in selected patients with multiple sclerosis.

Authors:  J Theodore Phillips; Elliot M Frohman
Journal:  Ther Adv Neurol Disord       Date:  2008-11       Impact factor: 6.570

2.  Selection of first-line therapy in multiple sclerosis using risk-benefit decision analysis.

Authors:  David Bargiela; Matthew T Bianchi; M Brandon Westover; Lori B Chibnik; Brian C Healy; Philip L De Jager; Zongqi Xia
Journal:  Neurology       Date:  2017-01-13       Impact factor: 9.910

Review 3.  Integration of PKPD relationships into benefit-risk analysis.

Authors:  Francesco Bellanti; Rob C van Wijk; Meindert Danhof; Oscar Della Pasqua
Journal:  Br J Clin Pharmacol       Date:  2015-07-29       Impact factor: 4.335

4.  Modeling Approaches in Cost-Effectiveness Analysis of Disease-Modifying Therapies for Relapsing-Remitting Multiple Sclerosis: An Updated Systematic Review and Recommendations for Future Economic Evaluations.

Authors:  Luis Hernandez; Malinda O'Donnell; Maarten Postma
Journal:  Pharmacoeconomics       Date:  2018-10       Impact factor: 4.981

5.  Cost-effectiveness of injectable disease-modifying therapies for the treatment of relapsing forms of multiple sclerosis in Spain.

Authors:  Carole Dembek; Leigh Ann White; Jayson Quach; Andrea Szkurhan; Nazia Rashid; M R Blasco
Journal:  Eur J Health Econ       Date:  2013-04-25

6.  The present efficacy of multiple sclerosis therapeutics: Is the new 66% just the old 33%?

Authors:  Eric C Klawiter; Anne H Cross; Robert T Naismith
Journal:  Neurology       Date:  2009-09-22       Impact factor: 9.910

Review 7.  Modelling the cost effectiveness of disease-modifying treatments for multiple sclerosis: issues to consider.

Authors:  Joel P Thompson; Amir Abdolahi; Katia Noyes
Journal:  Pharmacoeconomics       Date:  2013-06       Impact factor: 4.981

8.  Risk-benefit considerations in the treatment of relapsing-remitting multiple sclerosis.

Authors:  Alessandra Lugaresi; Maria di Ioia; Daniela Travaglini; Erika Pietrolongo; Eugenio Pucci; Marco Onofrj
Journal:  Neuropsychiatr Dis Treat       Date:  2013-06-24       Impact factor: 2.570

9.  Clinical efficacy issues in the treatment of multiple sclerosis: update of natalizumab.

Authors:  Francesco Patti; Angelo Pappalardo
Journal:  Clinicoecon Outcomes Res       Date:  2009-08-21

Review 10.  Development of oral immunomodulatory agents in the management of multiple sclerosis.

Authors:  Richard Nicholas; Paolo Giannetti; Ali Alsanousi; Tim Friede; Paolo A Muraro
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