What should be ordered for a patient with Alpha-1 (A1) antitrypsin deficiency?

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Last updated: May 4, 2025View editorial policy

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From the Guidelines

If someone is A1 anti-trypsin positive, no specific treatment is needed related to A1AT, as this indicates normal protein function. When considering the management of patients with normal A1AT levels, the focus should be on treating any underlying respiratory conditions, such as asthma or COPD, based on their specific symptoms. The treatment approach might include:

  • Bronchodilators (such as albuterol or tiotropium) 1
  • Inhaled corticosteroids (like fluticasone or budesonide) 1
  • Combination inhalers depending on symptom severity 1 It's essential to note that the presence of normal A1AT levels suggests sufficient protease inhibitor activity to protect the lungs from neutrophil elastase, the enzyme responsible for lung tissue damage when not properly regulated by A1AT. However, if the patient has A1AT deficiency, which would be indicated by a "negative" result rather than "positive", a different approach would be necessary, potentially including augmentation therapy, as suggested by the Canadian Thoracic Society meta-analysis and clinical practice guideline 1. This guideline conditionally recommends A1AT augmentation therapy for patients with COPD and documented A1AT deficiency, particularly those with specific clinical features and risk assessments, to preserve CT scan lung density and reduce mortality. Key points to consider in the management of A1AT deficiency include:
  • Clinical features suggestive of A1AT deficiency, such as early onset COPD, basal pan lobular emphysema, or family history of COPD or A1AT deficiency 1
  • Documented SERPINA1 genotypes associated with A1AT deficiency and severely reduced functional A1AT levels 1
  • Optimal pharmacological and non-pharmacological therapies for COPD, including inhaled bronchodilators, preventive vaccinations, and pulmonary rehabilitation 1

From the FDA Drug Label

GLASSIA is a sterile, ready to use, liquid preparation of purified human Alpha1-proteinase inhibitor (Alpha1-PI), also known as alpha1-antitrypsin (AAT). Augmenting the levels of functional protease inhibitor by intravenous infusion is an approach to therapy for patients with Alpha1-PI deficiency. Augmentation therapy with Alpha1-Proteinase Inhibitor (Human) is indicated only in patients with severe Alpha1-PI deficiency who have clinically evident emphysema

If someone is A1 anti-trypsin positive, alpha-1 antitrypsin (IV), such as GLASSIA, should be ordered for patients with severe Alpha1-PI deficiency who have clinically evident emphysema 2.

From the Research

Dietary Recommendations for A1 Anti-Trypsin Positive Individuals

There is no specific information in the provided studies regarding dietary recommendations for individuals with A1 anti-trypsin deficiency.

Treatment Options

  • Augmentation therapy with alpha-1 antitrypsin is the only specific therapy approved by the US Food and Drug Administration (FDA) for the treatment of A1 anti-trypsin deficiency 3, 4.
  • Intravenous infusion of A1-PI is the only therapy that directly addresses the underlying cause of AATD, and has demonstrated efficacy in a recent randomized, placebo-controlled trial 5.
  • Researchers are exploring alternative delivery routes, such as inhalation, and novel formulations, including recombinant AAT supplementation and PEGylation of AAT 6, 7.

Lifestyle Changes

  • Avoidance of environmental triggers, such as cigarette smoking, is a critical component of AAT deficiency treatment 3.

Testing and Diagnosis

  • A range of tests are available for confirming AATD, including serum alpha-1 proteinase inhibitor level measurement, phenotyping, genotyping, and gene sequencing 5.

References

Guideline

Guideline Directed Topic Overview

Dr.Oracle Medical Advisory Board & Editors, 2025

Research

Augmentation therapy in alpha-1 antitrypsin deficiency: advances and controversies.

Therapeutic advances in respiratory disease, 2010

Research

Suspecting and Testing for Alpha-1 Antitrypsin Deficiency-An Allergist's and/or Immunologist's Perspective.

The journal of allergy and clinical immunology. In practice, 2015

Research

Impact of administration route and PEGylation on alpha-1 antitrypsin augmentation therapy.

Journal of controlled release : official journal of the Controlled Release Society, 2025

Professional Medical Disclaimer

This information is intended for healthcare professionals. Any medical decision-making should rely on clinical judgment and independently verified information. The content provided herein does not replace professional discretion and should be considered supplementary to established clinical guidelines. Healthcare providers should verify all information against primary literature and current practice standards before application in patient care. Dr.Oracle assumes no liability for clinical decisions based on this content.

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