Academic and Employment Status in Patients With Generalized Myasthenia Gravis Treated With Eculizumab: A Case Series

Kang, Seung Ah; Sweeney, Megan M.; Govindarajan, Raghav

doi:10.1097/cnd.0000000000000391

Cited by 3 publications

(3 citation statements)

References 32 publications

(43 reference statements)

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“…It is a heterogeneous condition that may cause localized (most commonly ocular) muscle weakness, but is more frequently generalized (gMG), presenting with differing levels of weakness in muscle groups of the head, neck, trunk, and/or limbs [ 1 , 4 ]. The symptoms of gMG can substantially impact a patient’s daily personal and social functioning, work activities, economic well-being, and quality of life [ 1 , 6 – 14 ].…”

Section: Introductionmentioning

confidence: 99%

Ravulizumab pharmacokinetics and pharmacodynamics in patients with generalized myasthenia gravis

Ortiz

Katsuno

et al. 2023

J Neurol

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Introduction The terminal complement C5 inhibitor ravulizumab has a long elimination half-life, allowing maintenance dosing every 8 weeks. In the 26-week, double-blind, randomized, placebo-controlled period (RCP) of the CHAMPION MG study, ravulizumab provided rapid and sustained efficacy and was well tolerated in adults with anti-acetylcholine receptor antibody-positive (AChR Ab+) generalized myasthenia gravis (gMG). This analysis evaluated the pharmacokinetics (PK), pharmacodynamics (PD), and potential immunogenicity of ravulizumab in adults with AChR Ab+ gMG. Methods Data were analyzed from 86 patients who received ravulizumab in the CHAMPION MG RCP. Ravulizumab dosing was weight-based: initial loading dose of 2400/2700/3000 mg on Day 1 and maintenance doses of 3000/3300/3600 mg on Day 15 and then every 8 weeks. PK parameters were estimated from serum ravulizumab concentrations determined pre- and post-dose; PD effects of ravulizumab on serum free C5 concentrations were measured; and immunogenicity was assessed using anti-drug antibody and neutralizing-antibody assays. Results Target serum ravulizumab concentrations (> 175 µg/mL) were achieved immediately after the first ravulizumab dose (within 30 min of infusion completion) and maintained throughout the 26-week treatment period irrespective of patient body weight. Following the final maintenance dose, mean Cmax was 1548 µg/mL and Ctrough 587 µg/mL; no meaningful differences were noted among body-weight categories. Inhibition of serum free C5 was immediate, complete (< 0.5 μg/mL), and sustained throughout treatment in all patients. No treatment-emergent anti-drug antibodies were observed. Conclusions PK/PD evidence supports the use of ravulizumab every 8 weeks for immediate, complete, and sustained inhibition of terminal complement C5 in adults with AChR Ab+ gMG. Trial registration ClinicalTrials.gov ID: NCT03920293 (April 18, 2019).

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Section: Introductionmentioning

confidence: 99%

Ravulizumab pharmacokinetics and pharmacodynamics in patients with generalized myasthenia gravis

Ortiz

Katsuno

et al. 2023

J Neurol

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“…Among patients in Japan with MG, unwilling job transfers and decreased income were highlighted, 23 and seven US patients with MG reported altering their work/study pattern due to their condition, though they were able to resume the original pattern following treatment with eculizumab. 24 The lived experience of patients with MG may be comparable to that of patients with other diseases with similarly unpredictable and fluctuating cycles of relapsing and remitting symptoms. Our findings reveal a broadly similar degree of impairment among MG patients when compared with SLE, MS, myositis and chronic inflammatory demyelinating polyradiculoneuropathy.…”

Section: Discussionmentioning

confidence: 99%

“…This is in agreement with previous, single‐center studies. Among patients in Japan with MG, unwilling job transfers and decreased income were highlighted, 23 and seven US patients with MG reported altering their work/study pattern due to their condition, though they were able to resume the original pattern following treatment with eculizumab 24 …”

Section: Discussionmentioning

confidence: 99%

The impact of myasthenia gravis severity on work and daily activities

Pesa,

Choudhry,

de Courcy

et al. 2024

Muscle and Nerve

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Introduction/AimsPeople with myasthenia gravis (MG) experience impaired quality of life. However, the impact of MG symptoms on work productivity has not been well‐studied. We aimed to evaluate this impact and to examine associations between disease severity and the degree of impairment.MethodsData were drawn from the Adelphi MG Disease‐Specific Programme™, a multinational (USA, France, Germany, Italy, Spain, UK) survey completed by physicians and their patients with MG in 2020. Patient‐reported measures included the Work Productivity and Activity Impairment (WPAI): Specific Health Problem questionnaire.ResultsThe WPAI questionnaire was completed by 330 patients. Among those currently employed, the mean percentage of work time missed (absenteeism) was 13.3% (N = 116), percentage impairment of productivity at work (presenteeism) was 26.7% (N = 121), and overall work impairment was 30.0% (N = 110). Across all patients, impairment of non‐work‐related activities due to health problems (ADL impairment) was 39.2% (N = 330). Regression analysis indicated that impairment differed according to MG Foundation of America (MGFA) class (p = .0147, p < .0001, p < .0001 and p < .0001 for absenteeism, presenteeism, overall work impairment and ADL impairment, respectively). Being MGFA class III/IV was a predictor of presenteeism, overall work impairment and ADL impairment in a predictor model.DiscussionPatients with MG experience substantial work impairment particularly those with more severe symptoms, highlighting an important way in which patient quality of life is negatively affected. More effective treatment strategies would enable patients to lead more productive lives and could impact decisions relating to work and career.

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United States clinical practice experience with eculizumab in myasthenia gravis: symptoms, function, and immunosuppressant therapy use

Habib,

Klink,

Muppidi

et al. 2024

J Neurol

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Background/objectives The phase 3 REGAIN study and its open-label extension demonstrated the efficacy of the complement C5 inhibitor eculizumab in patients with treatment-refractory, acetylcholine receptor antibody–positive generalized myasthenia gravis (gMG). The aim of the ELEVATE study was to assess the effectiveness of eculizumab in clinical practice in adults with MG in the United States. Methods A retrospective chart review was conducted in adults with MG who initiated eculizumab treatment between October 23, 2017 and December 31, 2019. Outcomes assessed before and during eculizumab treatment using a pre- versus post-treatment study design included Myasthenia Gravis–Activities of Daily Living (MG-ADL) total scores; minimal symptom expression (MSE); physician impression of clinical change; minimal manifestation status (MMS); and concomitant medication use. Results In total, 119 patients were included in the study. A significant reduction was observed in mean MG-ADL total score, from 8.0 before eculizumab initiation to 5.4 at 3 months and to 4.7 at 24 months after eculizumab initiation (both p < 0.001). At 24 months after eculizumab initiation, MSE was achieved by 19% of patients. MMS or better was achieved by 30% of patients at 24 months. Additionally, 64% of patients receiving prednisone at eculizumab initiation had their prednisone dosage reduced during eculizumab treatment and 13% discontinued prednisone; 32% were able to discontinue nonsteroidal immunosuppressant therapy. Discussion Eculizumab treatment was associated with sustained improvements in MG-ADL total scores through 24 months in adults with MG. Prednisone dosage was reduced in approximately two-thirds of patients, suggesting a steroid-sparing effect for eculizumab.

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Academic and Employment Status in Patients With Generalized Myasthenia Gravis Treated With Eculizumab: A Case Series

Cited by 3 publications

References 32 publications

Ravulizumab pharmacokinetics and pharmacodynamics in patients with generalized myasthenia gravis

Ravulizumab pharmacokinetics and pharmacodynamics in patients with generalized myasthenia gravis

The impact of myasthenia gravis severity on work and daily activities

United States clinical practice experience with eculizumab in myasthenia gravis: symptoms, function, and immunosuppressant therapy use

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