Unknown

Dataset Information

0

Trial of canakinumab, an IL-1? receptor antagonist, in patients with inclusion body myositis.


ABSTRACT: Objective:To assess whether canakinumab, a monoclonal antibody against IL-1? approved for autoinflammatory diseases, is effective as target-specific therapy in patients with sporadic inclusion body myositis (sIBM). Methods:Because in sIBM IL-1? colocalizes with amyloid precursor protein and upregulates amyloid aggregates enhancing degeneration, targeting IL-1? with canakinumab may arrest disease progression. On this basis, 5 ambulatory patients with sIBM participated in an institutional review board--approved open-labeled study with 150 mg canakinumab [4 bimonthly, then monthly subcutaneous injections] for a mean period of 15.8 months. Patients were assessed bimonthly with a manual dynamometer in 12 proximal and distal muscles and with grip force (GF) in both hands. Total muscle strength (TMS) was expressed in kilograms. Efficacy was defined as >15% increased strength after 12 months. Results:Patient 1 stopped at month 5 because of 23% loss in TMS and 32.35% in GF; patient 2 showed 37.1% increase in TMS and 13% in GF by month 9; patient 3 exhibited 26.7% reduction in TMS and 10% in GF at month 33; patient 4 showed 6.5% reduction in TMS and 1.6% in GF after 15 months, denoting relative stability; and patient 5 showed 30.4% loss in TMS and 20.8% in GF after 18 months. In patients 2 and 4, in whom 3-year longitudinal data were available, no effect on disease progression was noted. Conclusions:In this long-term, open-label study, canakinumab showed small, but not clinically appreciable, stabilizing benefits in 2 of 5 patients with sIBM over 1 year, was ineffective in 2 others, and might have worsened one. No patient improved. Classification of evidence:This study provides Class IV evidence that canakinumab was ineffective for patients with sIBM.

SUBMITTER: Kosmidis ML 

PROVIDER: S-EPMC6624107 | biostudies-literature | 2019 Jul

REPOSITORIES: biostudies-literature

altmetric image

Publications

Trial of canakinumab, an IL-1β receptor antagonist, in patients with inclusion body myositis.

Kosmidis Michalis L ML   Pikazis Dimitris D   Vlachoyiannopoulos Panayotis P   Tzioufas Athanasios G AG   Dalakas Marinos C MC  

Neurology(R) neuroimmunology & neuroinflammation 20190610 4


<h4>Objective</h4>To assess whether canakinumab, a monoclonal antibody against IL-1β approved for autoinflammatory diseases, is effective as target-specific therapy in patients with sporadic inclusion body myositis (sIBM).<h4>Methods</h4>Because in sIBM IL-1β colocalizes with amyloid precursor protein and upregulates amyloid aggregates enhancing degeneration, targeting IL-1β with canakinumab may arrest disease progression. On this basis, 5 ambulatory patients with sIBM participated in an institu  ...[more]

Similar Datasets

2014-02-02 | E-MTAB-2141 | biostudies-arrayexpress
| S-EPMC4196838 | biostudies-other
| S-EPMC2780610 | biostudies-literature
| S-EPMC4233319 | biostudies-literature
2021-07-01 | GSE151758 | GEO
| S-EPMC4590682 | biostudies-other
| S-EPMC5386438 | biostudies-literature
| S-EPMC5454400 | biostudies-literature
| S-EPMC6277289 | biostudies-literature
| S-EPMC4284131 | biostudies-literature