Intractable Rare Dis Res. 2019;8(4):231-238. (DOI: 10.5582/irdr.2019.01121)

Quality of life in refractory generalized myasthenia gravis: A rapid review of the literature.

Garzón-Orjuela N, van der Werf L, Prieto-Pinto L, Lasalvia P, Castañeda-Cardona C, Rosselli D

Generalized myasthenia gravis (GMG) is a neuromuscular transmission disorder that creates a fluctuating weakness of the voluntary muscles. This study is aimed at understanding the effect that refractory GMG has on the quality of life of patients who suffer from it, and the effect of eculizumab on it. A systematic literature search was conducted in MEDLINE (Ovid), EMBASE and the Cochrane Database of Systematic Reviews (Ovid). Eligibility criteria were verified via the title and summary and afterward through the full text. The risk of bias of the included randomized clinical trials was evaluated and the data were synthesized in a descriptive manner. Nine studies were identified that evaluated the quality of life of patients with GMG. Regarding the effect of eculizumab, two studies were identified. The quality of life in patients with GMG is lower compared to ocular myasthenia gravis (MG) and MG in remission, especially in the domains of physical function, physical role, bodily pain, vitality, and social function. Patients treated with eculizumab had a better perception of their quality of life compared to those who received placebo. GMG affects the quality of life more than other types of MG. This outcome is of great importance for the choice of therapeutic options in patients with refractory GMG. Eculizumab generates improvements in the perception of patients' quality of life compared to placebo, making it a relevant therapeutic option in the management of refractory GMG.

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