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Effectiveness of Eculizumab Treatment for Generalized Myasthenia Gravis in Us Clinical Practice: GMG Registry Data
Journal of Neuromuscular Diseases ; 9:S239-S240, 2022.
Article in English | EMBASE | ID: covidwho-2043388
ABSTRACT

INTRODUCTION:

Clinical trial data have demonstrated that eculizumab improves clinical outcomes in individuals with refractory generalized myasthenia gravis (gMG). Data from clinical practice on treatment patterns and effectiveness of eculizumab in gMG are being collected by the Alexion-sponsored gMG Registry.

OBJECTIVE:

To describe treatment outcomes and safety (serious adverse events [SAEs]) in current gMG Registry participants during eculizumab therapy in clinical practice in the USA.

METHODS:

Starting in December 2019, adults with gMG who had ever received eculizumab enrolled in the gMG Registry (NCT04202341). After obtaining consent, demographic data, myasthenia gravis activities of daily living (MG-ADL) total score and Myasthenia Gravis Foundation of America (MGFA) classification were collected from medical records at two time points In the 6 months before eculizumab initiation and at first gMG Registry assessment after eculizumab treatment initiation (at Registry enrollment). SAEs in patients receiving eculizumab during Registry participation were recorded.

RESULTS:

As of November 29, 2021, in total, 111 adults with gMG had enrolled in the gMG Registry (male, 52.3%;mean [range] age at MG diagnosis, 56.1 [16.0-92.0] years). The mean (range) time from eculizumab initiation to gMG Registry enrollment was 2.0 (0.0-6.7) years. Mean (standard deviation) MG-ADL total score decreased from 8.3 (3.6) before eculizumab initiation to 3.1 (3.6) after eculizumab treatment. MGFA classification improved with eculizumab treatment Class I, 0.0% of patients before eculizumab initiation versus 28.9% after eculizumab treatment;class II, 36.8% versus 55.3%;class III, 52.6% versus 15.8%;class IV, 10.5% versus 0.0%. The median MGFA class was III before eculizumab initiation and II after eculizumab treatment. One SAE (invasive pulmonary aspergillosis) considered by the investigator to be related to eculizumab was reported in a patient who died, and two serious infections considered unrelated to eculizumab were reported (COVID-19/pneumonia and urinary tract infection);there were no meningococcal infections. Two patients died of causes considered to be unrelated to eculizumab (lung adenocarcinoma and acute congestive heart failure/myocardial infarction).

CONCLUSION:

These data from the gMG Registry provide evidence of the effectiveness of eculizumab for the treatment of gMG in clinical practice across the USA, demonstrating a benefit/risk profile consistent with that observed in clinical trials.
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Full text: Available Collection: Databases of international organizations Database: EMBASE Type of study: Experimental Studies / Prognostic study Language: English Journal: Journal of Neuromuscular Diseases Year: 2022 Document Type: Article

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Full text: Available Collection: Databases of international organizations Database: EMBASE Type of study: Experimental Studies / Prognostic study Language: English Journal: Journal of Neuromuscular Diseases Year: 2022 Document Type: Article