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Alpha-1 Antitrypsin Deficiency: Home Therapy.

Anna Annunziata1, Maurizia Lanza1, Antonietta Coppola1

  • 1Department of Respiratory Pathophysiology Monaldi Hospital, Naples, Italy.

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Summary

Home intravenous augmentation therapy is a viable treatment for alpha-1 antitrypsin deficiency, even during the SARS-CoV-2 pandemic. This approach demonstrated safety and improved patient quality of life during the emergency period.

Keywords:
CoV-2QOL scoreSARSalpha-1 antitrypsin (A1AT)augmentation therapyhome therapy

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Area of Science:

  • Pulmonology
  • Genetic Medicine
  • Infectious Disease Epidemiology

Background:

  • Alpha-1 antitrypsin deficiency (AATD) is a genetic disorder that can lead to lung and liver disease.
  • Home therapy offers a potential treatment strategy for AATD, though its availability is limited.
  • The SARS-CoV-2 pandemic presented challenges to healthcare delivery, necessitating adaptations in patient management.

Purpose of the Study:

  • To describe the experience of managing AATD patients with home care intravenous augmentation therapy.
  • To assess the safety and efficacy of this home treatment strategy during the COVID-19 emergency.
  • To evaluate the impact of home therapy on the quality of life for AATD patients.

Main Methods:

  • Retrospective analysis of AATD patients receiving home intravenous augmentation therapy.
  • Monitoring of treatment safety and adverse events during the SARS-CoV-2 pandemic.
  • Assessment of patient-reported quality of life using standardized questionnaires.

Main Results:

  • Home intravenous augmentation therapy was safely administered to AATD patients during the SARS-CoV-2 emergency period.
  • Patients receiving home therapy reported maintained or improved quality of life.
  • The home care model proved effective in ensuring continuity of treatment despite pandemic-related disruptions.

Conclusions:

  • Home intravenous augmentation therapy is a safe and effective strategy for AATD management, particularly during public health emergencies.
  • This approach can maintain treatment adherence and improve patient quality of life.
  • Expanding access to home therapy for AATD is recommended.