This study aims to generate real world evidence (RWE) from Italian clinical practice on the impact of efgartigimod alfa in gMG patients encompassing clinical outcomes and patient reported experiences. The study population will consist in adult patients with a documented diagnosis of gMG who are AChR-antibody positive and for whom the decision of treatment with efgartigimod alfa for gMG has been made independently of study participation as part of routine clinical care. The total study duration will be up to 23 months
Primary data collection will be performed during the baseline visit and follow-up will be at month 6 and 12 after baseline. Secondary data collection from medical charts, information on myasthenia gravis (MG) related therapies (including rescue therapies), MG exacerbations, and MG-related hospitalizations will be collected for the 12 months preceding baseline. Additionally, the patient's MG history and overall medical history will be documented.
Study Type
OBSERVATIONAL
Enrollment
100
Ospedale Generale Regionale F. Miulli
Acquaviva delle Fonti, Italy
RECRUITINGAzienda Ospedaliero Universitaria delle Marche
Ancona, Italy
RECRUITINGA.O.U. Consorziale Policlinico
Bari, Italy
RECRUITINGASST Papa Giovanni XXIII
Bergamo, Italy
Changes in MG-ADL scores from baseline
The Myasthenia Gravis Activities of Living (MG-ADL) scale is an 8-item patient-reported scale that measures MG symptoms and functional status across 4 domains. The total score ranges from 0 to 24, with higher scores indicating greater disability.
Time frame: Up to 12 months
Percentage of patients achieving MSE
Minimal Symptom Expression (MSE) is defined as an MG-ADL score ≤ 1
Time frame: Up to 12 months
Changes in QMG scores over time
The quantitative myasthenia gravis (QMG) score is a commonly used scale for evaluating muscle weakness associated with myasthenia gravis. It consists in a 13-item direct physician assessment scoring system that quantifies disease severity, based on impairments of body functions and structures. The total QMG score ranges from 0 to 39, where higher scores indicated greater disease severity.
Time frame: Up to 12 months
Changes in MG-QoL-15r scores over time
Time frame: Up to 12 months
Changes in PROMIS Fatigue SF 4a scores over time
The PROMIS Fatigue Short Form 4a is a four-item questionnaire used to assess the experience of fatigue over the past seven days. The items are designed to capture different aspects of fatigue, including the impact of fatigue on physical and mental activities. The raw score can range from 4 to 20, with higher scores indicating more severe fatigue.
Time frame: Up to 12 months
Changes in FSS over time
The Fatigue Severity Scale (FSS) is a self-administered questionnaire with 9 items (questions) investigating the severity of fatigue in different situations during the past week. The final score ranges from 1 to 7.
Time frame: Up to 12 months
Changes in HADS-Anxiety scores over time
The Hospital Anxiety and Depression Scale (HADS) is a widely used screening tool that helps to identify possible or probable instances of anxiety and depression among patients in non-psychiatric hospital settings. It consists of 14 items, with 7 items related to anxiety (HADS-A) and 7 related to depression (HADS-D), collecting how the patient was feeling in the previous week. The score for each subscale ranges from 0 to 21.
Time frame: Up to 12 months
Changes in HADS-Depression scores over time
The Hospital Anxiety and Depression Scale (HADS) is a widely used screening tool that helps to identify possible or probable instances of anxiety and depression among patients in non-psychiatric hospital settings. It consists of 14 items, with 7 items related to anxiety (HADS-A) and 7 related to depression (HADS-D), collecting how the patient was feeling in the previous week. The score for each subscale ranges from 0 to 21.
Time frame: Up to 12 months
Changes in PSQI scores over time
The Pittsburgh Sleep Quality Index (PSQI) is a self-rated questionnaire which assesses sleep quality and disturbances over a 1-month time interval. Nineteen individual items generate seven "component" scores: subjective sleep quality, sleep latency, sleep duration, habitual sleep efficiency, sleep disturbances, use of sleeping medication, and daytime dysfunction. The sum of scores for these seven components yields one global score, ranging from 0 to 21, with higher scores indicating worse sleep quality.
Time frame: Up to 12 months
TANDEM - patient version scores over time
The Treatment Assessment in Dual Evaluation Model (TANDEM) - patient version is a non-validated, exploratory PRO designed to assess patient satisfaction with efgartigimod alfa treatment and symptom control in MG. It includes 8 items on therapeutic alignment, treatment experience, and symptom management.
Time frame: Up to 12 months
TANDEM - clinician version scores over time
The Treatment Assessment in Dual Evaluation Model (TANDEM) - clinician version is a non-validated, exploratory PRO designed to assess clinician satisfaction with the management of efgartigimod alfa treatment and symptom control in patients with MG. It includes 8 items on therapeutic personalization, treatment effectiveness, and symptom stability.
Time frame: Up to 12 months
CBI scores over time
The Caregiver Burden Inventory (CBI) is a 24-item questionnaire designed to assess the following different aspects of caregiver burden. Scores range from 0 to 96, with higher scores indicating greater feelings of burden.
Time frame: Up to 12 months
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Ospedale Bellaria
Bologna, Italy
RECRUITINGA.O.U. Renato Dulbecco
Catanzaro, Italy
RECRUITINGA.O.U. Careggi
Florence, Italy
RECRUITINGIRCCS Ospedale Policlinico San Martino
Genova, Italy
RECRUITINGA.O.U. Policlinico G. Martino
Messina, Italy
RECRUITINGIRCCS Cà Granda Ospedale Maggiore Policlinico
Milan, Italy
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