Predictors and Prognostic Factors of Myasthenia Gravis Outcome

NCT05214612 | Recruiting DMC

• 1 other identifier: Myasthenia gravis Outcome
• Study Type: observational
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

This study aims to characterize the clinical features, frequency of different subgroups of MG, and identify predictors of treatment responsiveness among different subgroups of MG. The predictors are including primary outcome (percentage of changes in MG scales at baseline at time of enrollment and after 3 months) and secondary outcome (treatment-related adverse events). Also it aims to determine the frequency of patients with refractory MG. This information will be used to understand the trends and mechanisms of disease relapse, and optimal management strategies.

Conditions

Myasthenia Gravis; Autoimmune Diseases of the Nervous System; Neuromuscular Junction Diseases; Thymoma; Thymus Hyperplasia; Nervous System Diseases; Myasthenia Gravis, Generalized; Myasthenia Gravis, Ocular; Myasthenia Gravis Crisis; Myasthenia Gravis With Exacerbation (Disorder); Myasthenia Gravis, Adult Form; Myasthenia Gravis, Juvenile Form

Keywords

Predictors; Prognostic factors; Myasthenia Gravis; Outcome

Outcome Measures

Primary Outcomes (4)

• Change in MG-specific Activities of Daily Living scale (MG-ADL).

  The MG Activities of Daily Living (MG- ADL) Scale an easily administered, 8-item questionnaire. Each item is graded on a 4- point symptom severity scale (0 = normal, 3 = most severe), with the total score ranging from 0 to 24. Test items emphasize the functional impact of muscle weakness (eg, the ability to comb one's hair or brush one's teeth instead of hand grip or outstretched arm strength tests) rather than its quantitation. The MG-ADL requires no special equipment or training and can be administered in 10 minutes. The MG-ADL test domains include ocular (2 items), oropharyngeal (3 items), respiratory (1 item), and extremity/limb (2 items).

  The changes in points from baseline assessment score to 3 months follow up assessment score

• Change in MG quality of life 15 (MG-QOL15).

  Everyday clinical use led to the development of an abbreviated 15-item version, the MG-QOL15. These 15 items were derived from the mobility (9 items), symptoms (3 items), general contentment (1 item), and emotional well-being (2 items) domains of the 60-item version. Each of the items/statements (eg, "I have limited my social activity because of my condition") is scored by patients on a 5-point scale ranging from 0 ("not at all") to 4("very much") based on their experience over the previous 4 weeks; the item scores are summed to generate a total score ranging from 0 to 60.

  The changes in points from baseline assessment score to 3 months follow up assessment score

• Change in MG manual muscle testing (MG-MMT).

  The MG-MMT assesses the strength or function of 30 muscle groups typically affected by MG and includes a total of 18 items, with 12 assessed bilaterally, and 6 assessed as single items (lid closure, cheek puff, tongue protrusion, jaw closure, neck flexion, and neck extension). Each item is scored on a 5- point severity scale, based on the severity of muscle weakness \[0 = no weakness, 1 = weak/mild (25%) impairment, 2 = weak/moderate (50%) impairment, 3 = weak/severe (75%) impairment, 4 = paralyzed/unable to perform\]. Bilateral scores are summed for a total item score, and all item scores are summed for a total MG-MMT score. MG-MMT scores range from 0 to 120; domains include ocular (3 items, including eyelid closure), facial/oropharyngeal (3 items), axial strength (neck flexion/extension; 2 items), and limb strength (10 items).

  The changes in points from baseline assessment score to 3 months follow up assessment score

• Change in MG composite (MGC) Score.

  The MG Composite (MGC) Scale is considered a mixed outcome measure that incorporates both physician-evaluated and patient-reported outcome items. In creating this composite scale, the developers sought to minimize the number of items (and thus administration time), to maximize the sensitivity and clinical relevance of each item, and to weight items (as recommended by the Myasthenia Gravis Foundation of America MGFA task force) commensurate with their impact on functional status, QOL, overall health status, and prognosis.13 The MGC Scale is composed of individual items from outcome measures (including the quantitative myasthenia gravis score (QMG), the MG-ADL, and the MG-MMT). Total score spans from 0 to 50.

  The changes in points from baseline assessment score to 3 months follow up assessment score

Secondary Outcomes (2)

• The proportion of patients with treatment related adverse effects.

  At baseline and after 3 months

• The proportion of patients reaching minimal manifestations (MM) or better

  After 3 months

Interventions

Drug treatment of myasthenia gravis and treatment of crisis COMBINATION_PRODUCT

Patients with myasthenia gravis who are receiving medical treatment and treatment of crisis as plasmapheresis

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients diagnosed with MG either ocular disease or generalized disease associated with thymoma or not or post-thymectomy, from the outpatient clinics and inpatient wards will be eligible to participate.

You may qualify if:

• Age from 16 years and older.
• Gender: both sexes are included.
• Clinical Diagnosis of MG with supporting evidence as:
• unequivocal clinical response to pyridostigmine
• decrement \>10% in repetitive nerve stimulations study (RNS).
• Willingness to sample collection, imaging study and other disease-related examinations and assessments.

You may not qualify if:

• Age younger than 16 years.
• History of chronic psychiatric or neurological disorder other than MG that can produce weakness or fatigue.
• Severe systemic illness affecting life-expectancy.

Sponsors & Collaborators

• Assiut University: lead

Study Sites (1)

Assiut University

Asyut, 71515, Egypt

RECRUITING

Related Publications (9)

• Gilhus NE. Myasthenia Gravis. N Engl J Med. 2016 Dec 29;375(26):2570-2581. doi: 10.1056/NEJMra1602678. No abstract available.

  PMID: 28029925 BACKGROUND

• Sanders DB, Wolfe GI, Benatar M, Evoli A, Gilhus NE, Illa I, Kuntz N, Massey JM, Melms A, Murai H, Nicolle M, Palace J, Richman DP, Verschuuren J, Narayanaswami P. International consensus guidance for management of myasthenia gravis: Executive summary. Neurology. 2016 Jul 26;87(4):419-25. doi: 10.1212/WNL.0000000000002790. Epub 2016 Jun 29.

  PMID: 27358333 BACKGROUND

• Evoli A. Myasthenia gravis: new developments in research and treatment. Curr Opin Neurol. 2017 Oct;30(5):464-470. doi: 10.1097/WCO.0000000000000473.

  PMID: 28654435 BACKGROUND

• Gilhus NE, Tzartos S, Evoli A, Palace J, Burns TM, Verschuuren JJGM. Myasthenia gravis. Nat Rev Dis Primers. 2019 May 2;5(1):30. doi: 10.1038/s41572-019-0079-y.

  PMID: 31048702 BACKGROUND

• Lascano AM, Lalive PH. Update in immunosuppressive therapy of myasthenia gravis. Autoimmun Rev. 2021 Jan;20(1):102712. doi: 10.1016/j.autrev.2020.102712. Epub 2020 Nov 13.

  PMID: 33197578 BACKGROUND

• Suh J, Goldstein JM, Nowak RJ. Clinical characteristics of refractory myasthenia gravis patients. Yale J Biol Med. 2013 Jun 13;86(2):255-60. Print 2013 Jun.

  PMID: 23766745 BACKGROUND

• Silvestri NJ, Wolfe GI. Treatment-refractory myasthenia gravis. J Clin Neuromuscul Dis. 2014 Jun;15(4):167-78. doi: 10.1097/CND.0000000000000034.

  PMID: 24872217 BACKGROUND

• Anil R, Kumar A, Alaparthi S, Sharma A, Nye JL, Roy B, O'Connor KC, Nowak RJ. Exploring outcomes and characteristics of myasthenia gravis: Rationale, aims and design of registry - The EXPLORE-MG registry. J Neurol Sci. 2020 Jul 15;414:116830. doi: 10.1016/j.jns.2020.116830. Epub 2020 Apr 16.

  PMID: 32388060 BACKGROUND

• Jaretzki A 3rd, Barohn RJ, Ernstoff RM, Kaminski HJ, Keesey JC, Penn AS, Sanders DB. Myasthenia gravis: recommendations for clinical research standards. Task Force of the Medical Scientific Advisory Board of the Myasthenia Gravis Foundation of America. Ann Thorac Surg. 2000 Jul;70(1):327-34. doi: 10.1016/s0003-4975(00)01595-2. No abstract available.

  PMID: 10921745 BACKGROUND

MeSH Terms

Conditions

Myasthenia Gravis; Autoimmune Diseases of the Nervous System; Neuromuscular Junction Diseases; Thymoma; Thymus Hyperplasia; Nervous System Diseases

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Neurodegenerative Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Neoplasms, Complex and Mixed; Neoplasms by Histologic Type; Thymus Neoplasms; Thoracic Neoplasms; Lymphatic Diseases; Hemic and Lymphatic Diseases

Central Study Contacts

Nourelhoda AA Haridy, Lecturer

CONTACT

01063981139; nourelhodaahmed@aun.edu.eg

Eman MH Khedr, Professor

CONTACT

010058506632; Emankhedr99@yahoo.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal investigator, Lecturer of Neurology

Study Record Dates

• First Submitted: December 31, 2021
• First Posted: January 28, 2022
• Study Start: January 1, 2022
• Primary Completion: December 31, 2024
• Study Completion: March 31, 2025
• Last Updated: July 31, 2024

Record last verified: 2024-07

Locations

EG (1)