Overview

The MGFA Southeast Regional Conference provided in-depth education on myasthenia gravis (MG) management, recent therapeutic advances, seronegative MG challenges, safe exercise strategies, and available community support resources, engaging both patients and caregivers.

Conference Introduction & Logistics

• Three volunteer organizers and speakers were introduced; sponsors acknowledged.
• Attendees asked to use chat for Q&A; program agenda included expert talks and interactive sessions.

Pharmacological and Emergent Treatments for MG (Dr. Rosario)

• Explained neuromuscular junction physiology and how MG affects signal transmission.
• Outlined symptomatic treatment (e.g., Mestinon) and its mechanism—blocking acetylcholinesterase to increase acetylcholine.
• Discussed individualized dosing, short-term side effects, and potential Mestinon overdose risks.
• Reviewed immunosuppressants (e.g., prednisone, cellcept, imuran), their immune targets, and side effects.
• Described newer agents targeting complement and antibody recycling (e.g., Solaris, Ultomiris, Vyvgart).
• Covered emergent therapies: plasmapheresis (antibody removal via plasma exchange) and IVIG, emphasizing their short-term use.
• Highlighted treatment tailoring based on patient comorbidities and severity.

Exercise in MG (Dr. Plowman)

• Confirmed exercise is beneficial but requires special precautions for MG due to risk of muscle fatigue and crisis.
• Stressed importance of consulting healthcare providers, ideally a neurologically trained physical therapist.
• Advised low impact, low resistance activities; avoid repetitive or sustained contractions, overheating, and overexertion.
• Recommended gradual progression, frequent rest, and monitoring exertion levels (“stay in the green” of perceived exertion).
• Provided resources for safe MG-specific exercise and promoted supportive, individualized routines.

Advances in MG Therapies (Dr. Pulley)

• Reviewed traditional therapies (Mestinon, steroids, immunosuppressants) and when to escalate treatments.
• Presented on new drugs: fCRN inhibitors (e.g., Vyvgart), complement inhibitors (e.g., Solaris, Ultomiris, Zilucoplan), and their mechanisms.
• Compared efficacy and safety data between old and new agents using clinical trial endpoints.
• Addressed issues of drug access, real-world retreatment intervals, and considering patient preference for regimen selection.
• Discussed seronegative MG trial inclusion and the evolving use of advanced antibody testing.

Seronegative MG: Patient Perspective (Melissa Woolhurst)

• Shared a 16-year diagnostic journey marked by fluctuating symptoms, misdiagnoses, and eventual recognition by MG specialists.
• Emphasized unique challenges of seronegative MG: delayed diagnosis, lack of confirmed antibody, variance in provider awareness.
• Highlighted the importance of advocacy, support groups, specialist care, and educating one’s network.
• Noted ongoing research initiatives and new biomarker studies for seronegative MG.

Community Support Resources

• Health fairs and local/regional support groups introduced as avenues for patients, caregivers, and community education.
• Specialized groups (e.g., for caregivers, parents, young adults) and online community portal promoted.
• Caregiver support emphasized as vital, with group offerings tailored to their stress reduction and communication needs.

Decisions

• Health fairs to be expanded if demand persists.
• Encouraged pursuit of care from MG-expert providers for difficult or seronegative cases.

Action Items

• Ongoing – All: Seek or recommend expert MG care and participate in support groups or advocacy where possible.
• Ongoing – Conference Organizers: Gather participant feedback on desired educational topics for future sessions.
• As needed – Attendees: Utilize MGFA resources and engage in new online communities and support groups.

Questions / Follow-Ups

• Further research needed on MG exercise in remission and cold therapy.
• Attendees to suggest new support group topics or educational speakers.
• Continued advocacy for expanding clinical trial access for seronegative MG.

Certainly! Here is a comprehensive, in-depth summary and review of the MGFA Southeast Regional Conference lecture on myasthenia gravis (MG), covering all key points and details presented by the expert speakers and patient advocate:

Comprehensive Summary and Review of MGFA Southeast Regional Conference on Myasthenia Gravis

1. Conference Overview and Logistics

• The conference was organized by volunteer leaders and supported by multiple pharmaceutical sponsors.
• The format included expert presentations, Q&A via chat, and interactive discussions.
• Emphasis on muting during presentations and using chat for questions to facilitate smooth flow.
• The program aimed to educate patients, caregivers, and healthcare providers on MG management, new therapies, exercise, and community resources.

2. Pharmacological and Emergent Treatments for MG (Dr. Rosario)

Neuromuscular Junction Physiology and MG Pathophysiology

• MG is an autoimmune disorder where antibodies target acetylcholine receptors (AChR) at the neuromuscular junction.
• Normally, nerve signals release acetylcholine (ACh) vesicles that bind to muscle receptors, triggering contraction.
• In MG, antibodies block or destroy these receptors, impairing muscle contraction and causing weakness.
• Acetylcholinesterase enzyme breaks down ACh; blocking this enzyme increases ACh availability.

Symptomatic Treatment: Mestinon (Pyridostigmine)

• Mestinon blocks acetylcholinesterase, increasing ACh in the synaptic cleft.
• This temporarily improves muscle strength by overcoming antibody blockade.
• Available in oral tablets, syrup, and intramuscular injection (IV form discouraged due to cardiac risks).
• Onset: ~20 minutes; peak effect: 45-60 minutes; duration: 4-6 hours.
• Side effects: gastrointestinal upset (diarrhea, cramps), salivation, muscle twitching.
• Dose individualized; overdose can worsen weakness and cause bradycardia.
• Mestinon does not affect the immune system or antibody production.

Immunosuppressive and Immunomodulatory Therapies

• MG is driven by autoantibody production from B cells/plasma cells.
• Immunosuppressants reduce antibody production by targeting B cells/plasma cells.
• Common agents:
  • Prednisone: oral corticosteroid, effective but slow onset (weeks), with many side effects (insomnia, weight gain, osteoporosis).
  • Cellcept (Mycophenolate mofetil), Imuran (Azathioprine): oral immunosuppressants used as steroid-sparing agents.
  • Rituximab: monoclonal antibody targeting CD20 on B cells, effective especially in MuSK antibody-positive MG.
• Choice depends on disease severity, side effect profile, comorbidities, dosing convenience, and insurance coverage.

Complement Inhibitors

• Complement system activation by antibodies leads to destruction of muscle membrane.
• Drugs like Soliris (Eculizumab) and Ultomiris (Ravulizumab) block complement activation, preventing muscle damage.
• Both are IV infusions; Ultomiris has longer dosing interval (every 8 weeks vs. every 2 weeks for Soliris).
• Require meningococcal vaccination prior to treatment.
• Approved for adults with AChR antibody-positive generalized MG.

FcRn (Neonatal Fc Receptor) Inhibitors

• FcRn protects IgG antibodies from degradation, recycling them back into circulation.
• FcRn inhibitors (e.g., Vyvgart (Efgartigimod), Rozanolixizumab) block this recycling, increasing antibody degradation.
• Result: reduced levels of pathogenic IgG autoantibodies.
• Vyvgart is FDA-approved for AChR antibody-positive generalized MG; Rozanolixizumab is in late-stage trials.
• Side effects include headache and increased risk of infections.
• These drugs do not cause broad immunosuppression, only reduce IgG levels.

Emergent Treatments: Plasmapheresis and IVIG

• Plasmapheresis: mechanical removal of plasma containing antibodies; rapid but short-term effect.
• Typically done every other day for 5 sessions; used in MG crisis or pre-surgery.
• Risks: central line complications, hypotension, infection.
• IVIG: pooled immunoglobulins from donors; modulates immune response and blocks pathogenic antibodies.
• Used in crisis, exacerbations, or when plasmapheresis contraindicated.
• Side effects: headache, renal failure risk, thrombotic events.
• Not typically used for chronic management.

Surgical Treatment: Thymectomy

• Removal of thymus gland can improve MG, especially in younger patients with AChR antibody-positive generalized MG.
• Not typically recommended for ocular MG or well-controlled patients.
• Pathology may reveal thymoma or thymic hyperplasia.

3. Exercise in MG (Dr. Elizabeth Plowman)

Benefits of Exercise

• Prevents deconditioning, improves cardiopulmonary health, mobility, balance, metabolism, bone/joint health, and mood.
• Exercise is safe and beneficial in MG when done with appropriate precautions.

MG-Specific Exercise Concerns

• MG symptoms worsen with repeated or sustained muscle contractions.
• Traditional exercise mantras like “no pain no gain” or “push through fatigue” are contraindicated.
• Overexertion can trigger symptom exacerbation or crisis, especially respiratory muscle weakness.

Safe Exercise Guidelines

• Consult healthcare provider, ideally a neurologically trained physical therapist.
• Exercise when symptoms are well-controlled and at peak energy times (often morning).
• Avoid overheating; prefer cooler environments and water-based exercise.
• Avoid repetitive contractions of the same muscle group; use circuit training to alternate muscle groups.
• Avoid sustained muscle contractions; prefer flowing yoga over static holds.
• Start “ridiculously easy” and progress slowly with low resistance and low repetitions.
• Use frequent rest breaks; monitor exertion using Rate of Perceived Exertion (RPE) scale, staying in “green” zone (light to moderate exertion).
• Swimming/aquatic therapy is excellent but requires safety precautions (shallow water, flotation aids, not alone).
• Light weights or resistance bands are both acceptable; start with limb weight against gravity.
• Stop exercise before fatigue sets in; post-exercise fatigue lasting into next day indicates overexertion.
• Differentiate MG weakness (fluctuating, rapid onset with activity, improves with rest) from deconditioning (steady weakness).

Resources

• Texas Women’s University developed MG-specific exercise videos and safety guidelines.
• Support groups and physical therapists specializing in neuromuscular disorders are valuable resources.

4. Advances in MG Therapies (Dr. Fuller Pulley)

Traditional Treatment Recap

• Mestinon for symptomatic relief.
• Prednisone and non-steroidal immunosuppressants for immune modulation.
• IVIG and plasmapheresis for crisis or refractory cases.
• Rituximab preferred for MuSK antibody-positive MG.
• Thymectomy for selected AChR antibody-positive patients.

New Therapeutic Agents

FcRn Inhibitors

• Vyvgart (Efgartigimod): FDA-approved, IV weekly infusions, reduces IgG antibodies by blocking FcRn recycling.
• Rozanolixizumab: subcutaneous, similar mechanism, in late-stage trials.
• Both show significant improvement in MG-ADL and QMG scores.
• Side effects: headache, infections.
• Uncertainties remain about long-term immunoglobulin suppression and retreatment intervals.
• Potential future use in rescue therapy (hospitalized patients).

Complement Inhibitors

• Soliris (Eculizumab): first approved complement inhibitor for MG.
• Ultomiris (Ravulizumab): longer dosing interval, similar efficacy.
• Zilucoplan: subcutaneous complement inhibitor in trials.
• Require meningococcal vaccination.
• Show significant clinical improvement in MG-ADL and QMG scores.
• Choice among complement inhibitors may depend on dosing convenience, patient preference, and safety.

Seronegative MG and Clinical Trials

• Most trials focus on AChR antibody-positive patients.
• Seronegative patients often excluded due to diagnostic uncertainty.
• Cell-based assays can detect antibodies missed by standard tests, aiding diagnosis.
• Advocacy needed to expand trial inclusion and treatment access for seronegative MG.

Drug Access and Insurance

• High cost of new therapies leads to insurance barriers.
• Off-label use requires appeals and documentation.
• Rituximab coverage better due to cost-saving potential.
• Emergency or compassionate use possible in hospital settings.

5. Seronegative MG: Patient Perspective (Melissa Woolhurst)

Diagnostic Journey

• 16-year delay from symptom onset to diagnosis.
• Early symptoms: fluctuating voice changes, ptosis, muscle heaviness, clumsiness.
• Misdiagnoses: asthma, chronic fatigue syndrome, Lyme disease, anorexia.
• Symptoms worsened with infections and cold weather.
• Multiple doctors dismissed symptoms or misattributed them.
• Eventually diagnosed by MG specialist after trial of Mestinon and clinical exam.
• Seronegative MG patients face unique challenges: lack of antibody confirmation, provider awareness, and treatment options.

Living with Seronegative MG

• Importance of advocacy, specialist care, and support groups.
• Educating family, friends, and healthcare providers about MG.
• Recognizing triggers and managing symptoms proactively.
• Thymectomy helped in her case due to thymoma and hyperplasia.
• Emphasized never giving up despite diagnostic and treatment challenges.

6. Community Support Resources and Initiatives

MGFA Health Fairs

• New regional health fairs planned in multiple cities to connect patients, caregivers, and providers.
• Provide education, local resources, and experiential activities to understand MG.
• Encourage caregiver participation for better support.

Support Groups

• Numerous online and local support groups available, including specialized groups for caregivers, parents, young adults, and Spanish speakers.
• Groups provide education, emotional support, and community connection.
• Caregiver support groups address unique stressors and communication strategies.
• Speakers on topics like exercise, nutrition, stress management, and positive psychology are planned.

Online Community Portal

• MGFA launched an online community platform for ongoing peer support and information sharing.

7. Key Takeaways and Action Items

• MG treatment is highly individualized, involving symptomatic, immunosuppressive, complement, and FcRn-targeted therapies.
• Exercise is safe and beneficial with proper precautions and professional guidance.
• New therapies offer hope but require careful patient selection and monitoring.
• Seronegative MG remains a diagnostic and therapeutic challenge; ongoing research and advocacy are critical.
• Support groups and community resources are vital for patient and caregiver well-being.
• Patients should seek care from MG-expert providers and advocate for themselves.
• Health fairs and online communities enhance education and connection.

8. Frequently Asked Questions and Discussion Highlights

• Duration on immunosuppressants: Aim to taper to lowest effective dose; some may require lifelong therapy.
• Fatigue management: Rule out other causes; treat MG-related muscle fatigue with appropriate therapy.
• Treatment for seronegative MG: Most therapies except complement inhibitors can be used; clinical trials expanding.
• Cannabis research: No current evidence or research known.
• Thymectomy in seronegative MG: Considered in generalized disease; not for mild or ocular MG.
• Differentiating MG weakness vs. deconditioning: MG weakness fluctuates and worsens with activity but improves with rest.
• Exercise recommendations: Start low, go slow, avoid fatigue, use RPE scale.
• Support group topics: Exercise, nutrition, stress management, positive psychology, caregiver support.
• Finding MG-expert doctors: Use MGFA Partners in Care, support group recommendations.
• Insurance and drug access: Variable; appeals often needed for off-label or new therapies.
• Emergency drug use: Possible but case-dependent, often hospital decision.

Conclusion

The MGFA Southeast Regional Conference provided a thorough, multidisciplinary overview of myasthenia gravis, blending cutting-edge scientific advances with practical patient-centered advice. The integration of expert medical knowledge, patient experience, and community support resources offers a comprehensive roadmap for managing MG effectively. The conference underscored the importance of personalized care, ongoing research, and the power of community in navigating this complex autoimmune neuromuscular disease.

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