Sirolimus leads to rapid and sustained clinical improvement of motor deficits in a patient with inclusion body myositis.
flow cytometry
inclusion body myositis
mTOR inhibitor
rapamycin
sirolimus
Journal
European journal of neurology
ISSN: 1468-1331
Titre abrégé: Eur J Neurol
Pays: England
ID NLM: 9506311
Informations de publication
Date de publication:
04 2022
04 2022
Historique:
received:
27
11
2021
accepted:
21
12
2021
entrez:
7
3
2022
pubmed:
8
3
2022
medline:
5
4
2022
Statut:
ppublish
Résumé
To provide further evidence for sirolimus, a mammalian target of rapamycin inhibitor, as a treatment strategy for patients with inclusion body myositis (IBM). We acquired longitudinal clinical data and immunological assessments of CD8 Therapy with sirolimus 2 mg/day by mouth led to rapid and sustained clinical improvement of motor symptoms for an observation period of more than 1 year. Treatment was well tolerated, with no occurrence of adverse effects. We did not observe a meaningful alteration of CD8 The significant and persistent clinical improvement highlights the use of sirolimus as a potential treatment option in patients with IBM. In light of the lack of immunological treatment effects observed for cytotoxic CD8
Sections du résumé
BACKGROUND AND PURPOSE
To provide further evidence for sirolimus, a mammalian target of rapamycin inhibitor, as a treatment strategy for patients with inclusion body myositis (IBM).
METHODS
We acquired longitudinal clinical data and immunological assessments of CD8
RESULTS
Therapy with sirolimus 2 mg/day by mouth led to rapid and sustained clinical improvement of motor symptoms for an observation period of more than 1 year. Treatment was well tolerated, with no occurrence of adverse effects. We did not observe a meaningful alteration of CD8
CONCLUSIONS
The significant and persistent clinical improvement highlights the use of sirolimus as a potential treatment option in patients with IBM. In light of the lack of immunological treatment effects observed for cytotoxic CD8
Substances chimiques
Sirolimus
W36ZG6FT64
Types de publication
Case Reports
Langues
eng
Sous-ensembles de citation
IM
Pagination
1284-1287Informations de copyright
© 2022 The Authors. European Journal of Neurology published by John Wiley & Sons Ltd on behalf of European Academy of Neurology.
Références
Dimitri D, Benveniste O, Dubourg O, et al. Shared blood and muscle CD8+ T-cell expansions in inclusion body myositis. Brain. 2006;129:986-995.
Benveniste O, Hogrel J-Y, Belin L, et al. Sirolimus for treatment of patients with inclusion body myositis: a randomised, double-blind, placebo-controlled, proof-of-concept, phase 2b trial. Lancet Rheumatol. 2021;3:e40-e48.
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Benveniste O, Stenzel W, Hilton-Jones D, Sandri M, Boyer O, van Engelen BG. Amyloid deposits and inflammatory infiltrates in sporadic inclusion body myositis: the inflammatory egg comes before the degenerative chicken. Acta Neuropathol. 2015;129:611-624.
Glaubitz S, Zeng R, Schmidt J. New insights into the treatment of myositis. Ther Adv Musculoskelet Dis. 2020;12:1759720X1988649.
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