Generalized Myasthenia Gravis (gMG) is a rare autoimmune disease (a disease in which the body attacks its own tissues) that causes muscle weakness and significant fatigue.
Current treatments (corticosteroids, plasma exchange, intravenous immunoglobulin infusions) improve symptoms in many patients. However, many continue to suffer from fatigue and fatigability that are not well measured by standard tools. Moreover, these treatments can cause significant long-term side effects, reducing quality of life.
New treatments such as Rozanolixizumab (ROZ) are now available. They act rapidly and are well tolerated, allowing better symptom control while reducing the risks associated with conventional treatments.
To properly evaluate these new treatments, it is essential to understand patients' perspectives on their effectiveness. The scales used by physicians do not always capture all the symptoms experienced by patients, particularly fatigability. This is why a new tool has been developed: the MG symptoms PRO. This questionnaire allows patients to assess their own symptoms (fatigue, weakness of the eyes, mouth, breathing, muscle fatigability) in detail.
This research aims to better understand the effectiveness of treatments from the patients' perspective in order to improve their care.
The goal of the study is to evaluate the impact of Rozanolixizumab administration in real-world practice through the MG symptoms PRO questionnaire. This is an observational study, meaning that the medication is prescribed by the physician according to current regulations, and the study simply collects routine medical data during your follow-up, over a period of approximately 9 months.
Male or female subjects aged ≥18 years;
Diagnosed with MG with confirmed documentation and supported by a physical exam and confirmed seropositivity for anti AChR or anti MUSK antibodies;
Meets the clinical criteria as defined by the Myasthenia Gravis Foundation of America (MFGA) for generalized MG class IIa, IIb, IIIa, and IIIb;
Patient suitable for ROZ treatment (IgG ≥ 5,5 g/l);
Any current mental condition (psychiatric disorder, senility, or dementia) that, in the opinion of the investigator, may affect study compliance or prevent understanding of the aims, investigational procedures, or possible consequences of the study; and/or patient with a Mini-mental State evaluation (MMSE) of <25;
History of any illness or any clinical condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject;
History of ongoing malignancy (except basal cell and squamous cell carcinomas of the skin, or in situ carcinoma of the cervix uteri that have been completely excised and cured);
Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at inclusion;
Patients affected by generalized Myasthenia Gravis with anti-AChR and anti-MUSK and treated with ROZ
Cohort
Prospective
Patients diagnosed with generalized myasthenia gravis and treated with Rozanolixizumab
Other interventionPatients are evaluated using the MG Symptoms PRO questionnaire
To assess in real-life the impact of the ROZ on MG symptoms PRO score in generalized MG patients with anti-AChR or anti-MUSK antibodies
To monitor 7 days after the end of every cycle of treatment for 9 months the change from baseline of MG symptoms PRO score (frome non to severe)
Evaluate the impact of real life on MG symptoms PRO global sub-scores physical fatigue
To monitor 7 days after the end of every cycle of treatment for 9 months the change from baseline of MG symptoms PRO sub-score physical fatigue (from none of the time to all the time)
Evaluate the impact of real life on quality of life measured by MG-QOL15r
To monitor 7 days after the end of every cycle of treatment for 9 months the change from baseline of MG-QOL15r (MYASTHENIA GRAVIS QUALITY OF LIFE 15 REVISED) (from not at all to very much)
Evaluate the impact of real life on patient general state, measured by the Patient-Acceptable Symptom State (PASS)
To monitor 7 days after the end of every cycle of treatment for 9 months the change from baseline of PASS (PATIENT-ACCEPTABLE SYMPTOM STATE)
Evaluate the impact of real life on the evolution of gMG after each cycle measured by MG-ADL score
To monitor 7 days after the end of every cycle of treatment for 9 months the change from baseline of MG-ADL score (MYASTHENIA GRAVIS ACTIVITIES OF DAILY LIVING) (from normal to most severe)
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