Generalized Myasthenia Gravis (gMG), gMG
Conditions
Brief summary
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
Detailed description
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.
Interventions
None listed
Sponsors
argenx
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* At least 18 years old at signing of informed Consent Form (ICF) and privacy form (PF) * Documented diagnosis of gMG * AChR-antibody positive * The treating physician has decided to initiate efgartigimod alfa as part of routine clinical care and in accordance with product labelling, independently from the study
Exclusion criteria
* Hypersensitivity to the active substance or to any of the excipients listed: sodium dihydrogen phosphate, monohydrate; disodium hydrogen phosphate, anhydrous; arginine hydrochloride; polysorbate 80, hyaluronidase, histidine, histidine hydrochloride monohydrate, methionine, polysorbate 20, sucrose. * Current or planned participation in an interventional clinical trial.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Changes in MG-ADL scores from baseline | Up to 12 months | 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. |
| Percentage of patients achieving MSE | Up to 12 months | Minimal Symptom Expression (MSE) is defined as an MG-ADL score ≤ 1 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Changes in QMG scores over time | Up to 12 months | 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. |
| Changes in MG-QoL-15r scores over time | Up to 12 months | — |
| Changes in PROMIS Fatigue SF 4a scores over time | Up to 12 months | 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. |
| Changes in FSS over time | Up to 12 months | 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. |
| Changes in HADS-Anxiety scores over time | Up to 12 months | 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. |
| Changes in HADS-Depression scores over time | Up to 12 months | 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. |
| Changes in PSQI scores over time | Up to 12 months | 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. |
| TANDEM - patient version scores over time | Up to 12 months | 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. |
| TANDEM - clinician version scores over time | Up to 12 months | 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. |
| CBI scores over time | Up to 12 months | 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. |
Countries
Italy
Contacts
CONTACTSabine Coppieters, MD
Outcome results
None listed