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Enzyme replacement therapy induces T-cell responses in late-onset Pompe disease

✍ Scribed by Miklos Banati; Zsolt Hosszu; Anita Trauninger; Laszlo Szereday; Zsolt Illes

Publisher
John Wiley and Sons
Year
2011
Tongue
English
Weight
330 KB
Volume
44
Category
Article
ISSN
0148-639X

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✦ Synopsis

Abstract

Introduction:

Enzyme replacement therapy (ERT) in ultra‐orphan Pompe disease generates anti‐rhGAA antibodies, which may interfere with efficacy.

Methods:

rhGAA‐specific T‐cell responses were examined at different time‐points in 6 Hungarian patients treated with rhGAA and compared with 1 untreated patient and 5 healthy controls.

Results:

The ex vivo percentage of activated T cells was increased in treated patients. rhGAA stimulation in vitro generated a dose‐dependent increase in intracellular interferon‐gamma (IFN‐γ) expression in CD4^+^ and CD8^+^ T cells. Isolated CD4^+^ and CD8^+^ T cells produced increased amounts of IFN‐γ and tumor necrosis factor‐alpha (TNF‐α) in half of the patients after in vitro stimulation with rhGAA, whereas interleukin (IL)‐4, IL‐6, and IL‐17 levels were not elevated. Expression of cytotoxic FasL and perforin molecules by natural killer (NK), NKT‐like, and CD8^+^ T cells were not increased ex vivo.

Conclusions:

We found that enzyme replacement therapy (ERT) induces pro‐inflammatory T‐cell responses in addition to the antibody response in Pompe disease. Muscle Nerve, 2011

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