Traditional Medications & Treatments for Alpha1 Antitrypsin Deficiency

Alpha-1 Antitrypsin (AAT) augmentation therapy is the primary, FDA-approved treatment specifically for AATD. It involves regular infusions of purified human AAT protein to raise the level of circulating Alpha-1 Antitrypsin in the body, restoring the protease/antiprotease balance.

Think of it as:

• Replacing what your body can't produce enough of on its own
• Similar to insulin for diabetes—providing what's missing
• Protective therapy preventing tissue damage from uncontrolled proteases
• Long-term disease modification, not just symptom relief

Currently, there are four FDA-approved medications for AAT augmentation therapy:

1. Prolastin-C (Alpha-1 Proteinase Inhibitor [Human])

• Manufacturer: Grifols
• Formulation: Lyophilized (freeze-dried) powder for reconstitution
• Dosing: 60 mg/kg body weight once weekly
• Infusion Time: Approximately 15-30 minutes
• Storage: Refrigerated or room temperature
• Note: Most widely prescribed AAT therapy globally

2. Aralast NP (Alpha-1 Proteinase Inhibitor [Human])

• Manufacturer: Takeda (formerly Baxalta/Baxter)
• Formulation: Lyophilized powder for reconstitution
• Dosing: 60 mg/kg body weight once weekly
• Infusion Time: Approximately 15 minutes
• Storage: Room temperature (up to 25°C)
• Note: NP = "new process" with enhanced viral inactivation

3. Zemaira (Alpha-1 Proteinase Inhibitor [Human])

• Manufacturer: CSL Behring
• Formulation: Liquid solution (ready to use, no reconstitution)
• Dosing: 60 mg/kg body weight once weekly
• Infusion Time: Approximately 15 minutes or by rapid push (under 10 minutes)
• Storage: Refrigerated
• Note: Convenience of liquid formulation, no mixing required

4. Glassia (Alpha-1 Proteinase Inhibitor [Human])

• Manufacturer: Takeda
• Formulation: Liquid solution
• Dosing: 60 mg/kg body weight once weekly
• Infusion Time: Approximately 10-20 minutes
• Storage: Refrigerated
• Note: Designed for rapid infusion or home administration

The Science:

• Source: All FDA-approved products are derived from pooled human plasma from thousands of donors
• Purification: Extensive processing removes viruses, bacteria, and other proteins, leaving purified AAT
• Administration: Infused into bloodstream (IV infusion)
• Distribution: AAT circulates in blood and reaches lung tissue (primary target)
• Function: Inhibits neutrophil elastase and other proteases that damage tissue
• Duration: Levels remain protective for approximately one week
• Repeat: Weekly infusions maintain consistent protective levels

What It Does:

• Slows lung function decline
• Reduces frequency of COPD exacerbations
• Decreases lung inflammation
• May reduce mortality risk
• Potentially protects other organ systems
• May prevent disease progression if started early

What It Doesn't Do:

• Doesn't reverse existing lung damage
• Doesn't cure AATD (genetic condition remains)
• Doesn't eliminate need for other treatments (bronchodilators, etc.)
• Doesn't guarantee prevention of all complications

Current FDA Approval / Traditional Guidelines:

Approved For:

• Adults with AATD (specifically ZZ, SZ, or other rare phenotypes with very low AAT levels)
• Evidence of emphysema on imaging or pulmonary function testing
• Typically FEV1 between 35-60% predicted (though guidelines vary)
• Non-smokers or former smokers

Traditional Exclusion Criteria:

• Active smokers (must quit before therapy)
• No lung disease present
• Normal or near-normal lung function
• Liver disease as only manifestation
• MZ carriers (usually)

Evolving Perspective / Mark Egly Foundation Advocacy:

Arguments for Earlier/Broader Treatment:

Prevention vs. Waiting for Damage:

• Why wait for FEV1 to drop to 60% when tissue damage is preventable?
• Evidence suggests earlier treatment preserves function better
• Once lung tissue is destroyed, it can't be regenerated
• Prevention aligns with Hippocratic principle: "prevent disease whenever possible"

Systemic Benefits Beyond Lungs:

• AATD affects multiple organs, not just lungs
• AAT has anti-inflammatory effects systemically
• May prevent autoimmune disease development
• Potential cancer prevention through immune optimization
• Neurological protection (Alzheimer's prevention)
• Liver protection even without overt liver disease

Mark's Vision: "It is Mark and the Foundation's dream that Alpha1 Antitrypsin becomes the equivalent of the safest and healthiest 'vaccine type' treatment many individuals could receive throughout their entire lifetime."

Current Reality:

• Insurance typically requires significant lung damage before approval
• Standards of care are changing slowly
• Advocacy efforts underway to expand treatment criteria
• Some physicians prescribe "off-label" for preventive purposes

Infusion Center / Clinic Administration

Pros:

• Medical supervision during infusion
• Immediate help if adverse reaction
• No home setup required
• Social aspect (meet other patients)
• Vitals monitored

Cons:

• Travel to infusion center weekly
• Time commitment (infusion + travel)
• Less flexibility in scheduling
• Potential exposure to illnesses in clinic

Typical Process:

• Arrive at infusion center
• Vitals checked (blood pressure, pulse, temp, O2)
• IV line placed
• Infusion started (15-30 minutes)
• Monitored during and after infusion
• IV removed, vitals rechecked
• Home (total time: 1-2 hours typically)

Home Infusion

Pros:

• Convenience of home treatment
• Flexible scheduling
• No travel required
• Privacy and comfort
• Reduced infection exposure
• Time savings

Cons:

• Requires training and confidence
• Home storage of medication
• Self-management of equipment
• Need emergency plan if complications
• May feel isolated (vs. clinic social aspect)

Requirements:

• Physician approval
• Completion of training program
• Home health nurse visits initially
• Appropriate storage space (refrigerator)
• Emergency contact plan

Process:

• Medication delivered to home monthly
• Patient self-administers or has caregiver assist
• IV access (peripheral IV, port, or PICC line)
• Self-monitoring
• Coordination with home health agency

Subcutaneous Administration (Investigational)

Currently in Clinical Trials:

• Weekly or more frequent self-injections under skin (like insulin)
• Potentially higher dosing frequency with smaller volumes
• Early studies show promise
• May improve convenience and patient preference
• Not yet FDA approved

Common Side Effects (Generally Mild):

During/Immediately After Infusion:

• Headache (most common)
• Dizziness or lightheadedness
• Nausea
• Fatigue
• Chills or feeling cold
• Flushing or warmth
• Mild fever
• Muscle aches

Management:

• Slow infusion rate
• Pre-medication (acetaminophen, antihistamine)
• Hydration before/after
• Usually resolve within hours

Injection Site Issues:

• Bruising where IV placed
• Tenderness or soreness
• Mild swelling

Management:

• Rotate IV sites
• Apply warm compress
• Usually minor and temporary

Rare but Serious Side Effects:

Allergic Reactions:

• Hives, itching, rash
• Difficulty breathing, wheezing
• Chest tightness
• Swelling of face, lips, tongue
• Severe hypotension

Action: Stop infusion immediately, seek emergency care

Viral Transmission Risk:

• Extremely rare with modern purification
• All products undergo extensive viral inactivation
• Theoretical risk from plasma-derived product
• No documented HIV or hepatitis transmission with current products

IgA Deficiency Concern:

• Patients with IgA deficiency may have antibodies against IgA
• Risk of anaphylactic reaction
• Should be tested before starting therapy

Long-Term Considerations:

Ongoing Monitoring:

• Regular pulmonary function tests
• AAT trough levels (measure before next infusion)
• Liver function tests
• Monitoring for adverse effects

Vein Health:

• Repeated IV access can damage veins over time
• May need port or PICC line eventually
• Rotation of sites important
• Proper hydration helps vein health

The Financial Reality:

Annual Cost:

• Approximately $100,000 - $200,000+ per year
• Varies by product, dosage (weight-based), and pharmacy
• One of the most expensive chronic therapies

Why So Expensive:

• Plasma collection and testing from thousands of donors
• Extensive purification and viral inactivation processes
• Limited patient population (rare disease, limited market)
• Manufacturing complexity
• Distribution and storage requirements

Insurance Coverage:

Most insurance plans cover AAT therapy when:

• FDA-approved indication (emphysema with documented lung disease)
• Pre-authorization obtained
• Medical necessity documented
• Smoking cessation proven
• Proper diagnosis and testing

Challenges:

• Prior authorization can be lengthy and complex
• Criteria vary by insurer
• Denials common initially (appeals often successful)
• Documentation requirements extensive
• Some plans have step therapy requirements

Medicare Coverage:

• Part B covers IV infusions in clinic/hospital
• Part D may cover home infusions
• Supplemental coverage often needed for copays

Financial Assistance Programs:

Pharmaceutical Company Programs:

• CSL Behring Support: For Zemaira patients - copay assistance, reimbursement support
• Grifols Plasma Alliance: For Prolastin-C - patient assistance
• Takeda Patient Support: For Aralast NP and Glassia - copay cards, financial assistance
• Octapharma: Various support programs

General Assistance:

• HealthWell Foundation - Copay assistance for AATD
• Patient Advocate Foundation - Insurance navigation and copay relief
• AlphaNet - Case management, insurance coordination
• Alpha-1 Foundation - Resources and referrals
• Mark Egly Foundation - Resource connections

Getting Help:

• Contact AlphaNet coordinator (1-800-577-2586) - FREE service helping with insurance
• Specialty pharmacies often have financial coordinators
• Patient advocate services
• Hospital financial counselors

Purpose: Open airways, reduce breathlessness, improve lung function

Types:

Short-Acting Beta-Agonists (SABAs):

• Albuterol (ProAir, Ventolin, Proventil)
• Levalbuterol (Xopenex)
• Use: Quick relief for acute symptoms ("rescue inhaler")
• Duration: 4-6 hours

Long-Acting Beta-Agonists (LABAs):

• Salmeterol (Serevent)
• Formoterol (Foradil, Perforomist)
• Use: Maintenance, twice daily
• Duration: 12 hours
• Note: Should not be used alone (combine with corticosteroid)

Short-Acting Anticholinergics:

• Ipratropium (Atrovent)
• Use: Quick relief, maintenance
• Duration: 4-6 hours

Long-Acting Anticholinergics (LAMAs):

• Tiotropium (Spiriva)
• Aclidinium (Tudorza)
• Umeclidinium (Incruse)
• Glycopyrrolate (Seebri)
• Use: Once-daily maintenance
• Duration: 24 hours

Combination Inhalers:

• LABA + Corticosteroid: Advair, Symbicort, Breo, others
• LABA + LAMA: Anoro, Stiolto, Bevespi
• LABA + LAMA + Corticosteroid: Trelegy
• Advantage: Convenience, better adherence, synergistic effects

Purpose: Reduce airway inflammation

Common Options:

• Fluticasone (Flovent)
• Budesonide (Pulmicort)
• Mometasone (Asmanex)
• Ciclesonide (Alvesco)

Use: Maintenance therapy, typically twice daily

Note: Controversial in AATD—some evidence suggests less benefit than in other COPD, but individual response varies

Purpose: Reduce inflammation during exacerbations

Common: Prednisone, methylprednisolone

Use: Short courses (5-10 days) for acute worsening, NOT long-term due to side effects

Roflumilast (Daliresp)

• Reduces inflammation
• For severe COPD with chronic bronchitis and frequent exacerbations
• Oral medication, once daily
• Can have GI side effects

Purpose: Thin mucus, make it easier to cough up

Guaifenesin (Mucinex):

• Over-the-counter
• Helps loosen chest congestion
• Adequate hydration important

N-Acetylcysteine (NAC):

• Antioxidant properties
• Thins mucus
• Some evidence for reducing exacerbations
• Available as supplement or prescription

Purpose: Treat bacterial lung infections

When Used:

• Acute exacerbations with signs of infection
• Chronic lung infections (bronchiectasis)
• Prophylactic use in some cases of frequent infections

Common Options:

• Azithromycin (often used long-term for anti-inflammatory effects)
• Doxycycline
• Amoxicillin/clavulanate
• Fluoroquinolones (for serious infections)

Purpose: Maintain adequate blood oxygen levels

Indications:

• Resting O2 saturation ≤88%
• Exertional desaturation
• Sleep-related hypoxemia
• Improved survival with long-term oxygen when indicated

Delivery Systems:

• Compressed oxygen tanks (portable and stationary)
• Liquid oxygen systems
• Oxygen concentrators (home use)
• Portable oxygen concentrators (POCs) - battery powered, travel-friendly

Duration:

• Continuous (24/7)
• Only with exertion
• Only during sleep
• As prescribed based on individual needs

No Specific Medication for AATD Liver Disease:

• AAT augmentation does NOT treat liver manifestations
• Management is supportive and preventive

Strategies:

Lifestyle Modifications:

• Avoid alcohol completely
• Maintain healthy weight
• Avoid hepatotoxic medications when possible
• Vaccination (hepatitis A & B)
• Monitor liver function regularly

Treating Complications:

• Portal hypertension management
• Varices screening and treatment
• Ascites management (diuretics, paracentesis)
• Hepatic encephalopathy treatment (lactulose, rifaximin)
• Nutritional support

Liver Transplantation:

• Definitive treatment for end-stage AATD liver disease
• Excellent outcomes (80-90% 5-year survival)
• Corrects genetic defect in liver (but recipient still makes abnormal AAT from transplanted liver)
• Lungs not affected by liver transplant

Approach Depends on Specific Condition:

Rheumatoid Arthritis, Lupus, Other Autoimmune Diseases:

• Disease-modifying antirheumatic drugs (DMARDs)
• Biologic agents (TNF inhibitors, etc.)
• Immunosuppressants
• Corticosteroids (short-term)

AATD Consideration:

• AAT augmentation may help autoimmune symptoms (under investigation)
• Standard autoimmune treatments used
• Immunosuppression may increase infection risk (monitor closely with lung disease)

Anti-Inflammatory Strategies:

• Diet (Mediterranean, anti-inflammatory focus)
• Omega-3 fatty acids
• Exercise (appropriate level)
• Stress management
• Smoking cessation
• Limiting alcohol

What It Includes:

• Supervised exercise training
• Breathing techniques
• Nutritional counseling
• Energy conservation strategies
• Education about lung disease
• Psychosocial support
• Goal setting and self-management

Benefits:

• Improved exercise capacity
• Reduced breathlessness
• Better quality of life
• Fewer hospitalizations
• Increased confidence and independence
• Enhanced ability to perform daily activities

Duration: Typically 6-12 weeks, 2-3 sessions per week

Location: Hospital-based, outpatient centers, some home programs

Insurance: Usually covered with referral

For Severe Emphysema in Select Patients:

Surgical Lung Volume Reduction (LVRS)

• Removal of most damaged lung tissue
• Allows remaining healthier tissue to function better
• Major surgery with risks
• Benefit in carefully selected patients (particularly upper lobe predominant disease)

Bronchoscopic Lung Volume Reduction (BLVR)

• Endobronchial Valves: One-way valves placed in airways to collapse diseased areas
• Less invasive than surgery
• For heterogeneous emphysema
• Growing body of evidence for efficacy

Bullectomy

• Surgical removal of large bullae (air pockets)
• Can improve lung function if bullae compressing healthy tissue

When Considered:

• FEV1

Process:

• Referral to transplant center
• Extensive evaluation (medical, psychological, social)
• Listing with UNOS (United Network for Organ Sharing)
• Wait time varies (months to years)
• Lung allocation score determines priority

Types:

• Single lung transplant
• Double lung transplant (more common in AATD)

Outcomes:

• Good short-term survival (80-90% at 1 year)
• Median survival 5-6 years
• Some patients live 10, 15, 20+ years post-transplant
• Significantly improved quality of life
• Requires lifelong immunosuppression

Considerations:

• Major surgery with significant risks
• Lifelong anti-rejection medications
• Risk of infections, rejection, complications
• Extensive medical follow-up required
• Lifestyle changes necessary
• Genetic AATD not cured (but new lungs make normal AAT)

Gene Therapy

Goal: Correct the genetic defect causing AATD

Approaches:

• Gene Addition: Adding functional SERPINA1 gene
• Gene Editing: CRISPR or other technologies to fix mutation
• Delivery: Viral vectors, nanoparticles, direct injection

Status: Multiple clinical trials ongoing

• Some showing promise in early phases
• Years away from FDA approval
• Could potentially cure AATD with one-time treatment

Potential Benefits:

• Eliminate need for weekly infusions
• Correct underlying genetic cause
• Produce AAT naturally in body
• Prevent all organ manifestations

Challenges:

• Delivery to enough cells to make sufficient AAT
• Long-term expression and safety
• Immune response to vectors
• Cost and accessibility

Alternative AAT Production Methods

Recombinant AAT:

• Produced in cell cultures or transgenic animals rather than human plasma
• Unlimited supply potential
• No viral transmission risk
• Lower cost possible
• In development, not yet approved

Plant-Based Production:

• AAT produced in tobacco, corn, or other plants
• Scalable production
• Very low cost potential
• Early research phase

Transgenic Animal Production:

• Goats, cows producing human AAT in milk
• Proven feasible in research
• Regulatory pathway unclear

Inhaled AAT

Rationale: Deliver AAT directly to lungs where needed most

Status: Clinical trials with mixed results

• Some formulations showing promise
• May complement IV therapy
• Could be preventive in early disease

Advantages:

• Direct delivery to target organ
• Potentially more convenient
• Lower systemic dose needed

Oral AAT

Challenge: Protein degraded by digestive system

Approaches Being Investigated:

• Protected formulations
• Absorption enhancers
• Very early research

Small Molecule Drugs

Goal: Drugs that enhance AAT production or function

Approaches:

• Compounds that increase AAT gene expression
• Molecules that improve AAT folding and secretion
• Protease inhibitors (non-AAT based)

Status: Preclinical and early clinical research

Stem Cell Therapy

Goal: Replace AAT-deficient cells with normal cells

Approaches:

• Liver stem cells
• Induced pluripotent stem cells (iPSCs) corrected and reimplanted
• Very early research

Antisense Oligonucleotides (ASOs)

For Liver Disease Specifically:

• Reduce production of misfolded Z protein accumulating in liver
• Proof of concept in trials
• May prevent/treat liver disease manifestation

Important Note: These should complement, not replace, standard medical care. Discuss with your physician before starting any complementary therapy.

Anti-Inflammatory Diet:

• Mediterranean diet pattern
• Omega-3 rich foods (fatty fish, walnuts, flaxseed)
• Colorful fruits and vegetables
• Whole grains
• Limit processed foods, red meat, sugar

Adequate Protein:

• Important for muscle maintenance, immune function
• Lean proteins, plant proteins
• May need increased intake with lung disease

Antioxidants:

• Vitamin C, E, beta-carotene from food sources
• Colorful vegetables and fruits
• Some evidence for benefit in lung health

Hydration:

• Essential for mucus clearance
• 8+ glasses daily unless restricted
• Warm beverages may be soothing

N-Acetylcysteine (NAC):

• Antioxidant and mucolytic
• Some evidence for reducing exacerbations
• Generally safe
• Typical dose 600mg 1-2x daily

Vitamin D:

• Many COPD patients deficient
• Important for bone health, immune function
• Test levels, supplement if low

Omega-3 Fatty Acids:

• Anti-inflammatory
• May reduce systemic inflammation
• Fish oil or algae-based

Vitamin C and E:

• Antioxidants
• May help lung health
• Food sources preferred, supplements if needed

Coenzyme Q10 (CoQ10):

• Antioxidant
• May help with energy/fatigue
• Limited evidence but generally safe

Probiotics:

• May support immune function
• Gut health important for overall wellness

ALWAYS discuss supplements with physician—can interact with medications

Yoga:

• Gentle stretching, breathing focus
• Can improve flexibility, stress, breathing control
• Choose classes appropriate for limitations

Tai Chi:

• Gentle movement meditation
• Evidence for improving balance, strength, breathing in COPD
• Low impact, very safe

Meditation & Mindfulness:

• Stress reduction
• May improve coping, quality of life
• Pain management
• Many free apps and resources

Breathing Techniques:

• Pursed-lip breathing
• Diaphragmatic breathing
• Reduces breathlessness, improves efficiency
• Taught in pulmonary rehabilitation

• May help with breathlessness, stress
• Limited but some positive evidence
• Generally safe when performed by licensed acupuncturist

• Relaxation, stress relief
• May help with muscle tension
• Generally safe
• Choose therapist familiar with medical conditions

Important Questions to Ask:

About AAT Augmentation Therapy:

• Am I a candidate for augmentation therapy?
• What are the potential benefits in my case?
• What are the risks and side effects?
• How is it administered—clinic vs. home?
• How long will I need therapy?
• How will we monitor effectiveness?
• What if I can't get insurance approval?

About Other Treatments:

• Why are you recommending this medication?
• How does it work?
• What are alternatives?
• What side effects should I watch for?
• How will we know if it's working?
• Are there interactions with other medications?

General:

• What's my prognosis with treatment vs. without?
• When should I consider more aggressive therapies?
• Are there clinical trials I should consider?
• What lifestyle changes are most important?
• How often should I be monitored?

Traditional Approach:

• Wait for significant lung function decline (FEV1

Preventive Approach (Mark Egly Foundation Advocacy):

• Earlier intervention before damage occurs
• Consider AAT therapy even with normal lung function
• Focus on preventing complications, not just treating them
• Systemic benefits beyond lungs

Factors to Consider:

• Genotype (ZZ, SZ more aggressive)
• Age (younger = more years at risk)
• Family history (severe disease in relatives?)
• Baseline lung function trend
• Presence of symptoms
• Other organ involvement
• Personal values and preferences
• Insurance and financial considerations

Primary Goals:

• Slow disease progression
• Prevent or minimize lung function decline
• Reduce exacerbations
• Maintain quality of life
• Optimize functional capacity
• Prevent complications
• Maximize lifespan

Setting Realistic Expectations:

• Treatment slows decline, doesn't reverse damage
• Some decline may occur despite best therapy
• Individual responses vary
• Requires long-term commitment
• Combines medical treatment with lifestyle optimization

Why It Matters:

• Consistency is key to effectiveness
• Missing doses reduces protection
• Lifestyle factors impact outcomes as much as medication

Strategies for Adherence:

• Integrate treatment into routine
• Use reminders (phone alarms, apps)
• Prepare medications weekly
• Address barriers proactively (cost, side effects, access)
• Understand WHY you're taking each medication
• Track adherence to identify patterns
• Communicate with team about challenges

Regular Assessments:

• Pulmonary function tests (spirometry) - typically every 3-6 months
• AAT trough levels - annually or as needed
• Liver function tests - at least annually
• CT scans - as indicated
• Six-minute walk test - tracking exercise capacity
• Quality of life questionnaires

Self-Monitoring:

• Daily symptoms - cough, breathlessness, energy
• Pulse oximetry at home if appropriate
• Weight tracking
• Exacerbation frequency
• Functional abilities
• Overall well-being

Signs Your Treatment May Need Adjustment:

• Increasing breathlessness
• More frequent exacerbations
• Declining exercise tolerance
• Declining lung function on testing
• New symptoms
• Medication side effects affecting quality of life

Strategies:

• Work with AlphaNet Coordinator (FREE service) Expert insurance navigation
• Prior authorization assistance
• Appeals support
• Resource connections
• Apply for Patient Assistance ProgramsPharmaceutical company programs
• HealthWell Foundation
• Patient Advocate Foundation
• Specialty Pharmacy SelectionCompare costs and services
• Many offer financial coordinators
• Home delivery options
• Optimize InsuranceChoose plans with best chronic disease coverage
• Understand deductibles, out-of-pocket maximums
• Timing of care around calendar year
• Tax DeductionsMedical expenses >7.5% of AGI are deductible
• Keep meticulous records
• Consult tax professional
• Clinical TrialsInvestigational treatments provided free
• Close monitoring included
• Contributing to research

AATD Organizations:

• Alpha-1 Foundation: Treatment guidelines, specialist directory
• AlphaNet: Case management, treatment coordination (FREE)
• Mark Egly Foundation: Ask the MD, treatment decision support

Pharmacy Patient Support:

• Each manufacturer has patient support programs
• Financial assistance applications
• Copay cards and assistance

Clinical Trials:

• ClinicalTrials.gov - search "Alpha-1 Antitrypsin"
• Ask your AATD specialist about current trials
• Alpha-1 Foundation research updates

Treatment for AATD has never been more hopeful:

✅ Four FDA-approved augmentation therapies available now ✅ Gene therapy in clinical trials - potential cure on horizon ✅ Alternative AAT production methods in development ✅ Growing understanding of systemic benefits ✅ Advocacy for earlier treatment gaining traction ✅ Novel delivery methods being investigated ✅ Research into autoimmune and inflammatory connections ✅ Improved lung transplant outcomes

The standard of care is evolving rapidly.

The Mark Egly Foundation is at the forefront of advocating for:

• Earlier treatment initiation
• Expansion of treatment criteria beyond traditional lung-only focus
• Recognition of systemic benefits
• Preventive rather than reactive approach
• Access and affordability improvements

You are not a passive recipient of medical care—you are an active partner in your treatment decisions.

Empower yourself:

• 📚 Educate yourself about options
• 🤝 Build a strong healthcare team
• 💬 Ask questions until you understand
• 🎯 Set clear treatment goals
• 📊 Monitor your progress
• 🔄 Advocate for adjustments when needed
• 🌐 Connect with others on similar treatments
• 💪 Take charge of lifestyle factors you can control

Your treatment plan should reflect your values, goals, and circumstances—not just a one-size-fits-all protocol.

Questions about treatments? Need help navigating decisions?

Mark Egly Foundation Resources:

• 🩺 Ask the MD: Expert physician guidance - markeglyfoundation.com/ask-the-md
• 🤝 Peer Mentors: Connect with patients on similar treatments
• 📚 Educational Materials: Treatment decision guides
• 💬 Community Forums: Discuss treatment experiences with peers
• 📞 Phone: 1-800-MARK-EGLY
• ✉️ Email: treatments@markeglyfoundation.com

AlphaNet (FREE Case Management):

• 📞 1-800-577-2586
• 🌐 alphanet.org

Alpha-1 Foundation:

• 📞 1-800-GO-ALPHA-1
• 🌐 alpha1.org

Traditional treatments offer protection. Emerging therapies offer hope. Your active participation offers the best outcomes.

Together, we're not just treating AATD—we're transforming what's possible for everyone affected by Alpha-1 Antitrypsin Deficiency.

Last Updated: December 2025This information is for educational purposes and should not replace consultation with your healthcare providers.