&lt;p&gt;Improving the Lives of Patients with Alpha-1 Antitrypsin Deficiency&lt;/p&gt;

Sandhaus, Robert A.; Strange, Charlie; Zanichelli, Andrea; Skålvoll, Karen; Koczulla, Andreas Rembert; Stockley, Robert A.

doi:10.2147/copd.s276773

Cited by 5 publications

(7 citation statements)

References 46 publications

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“…The only licensed treatment that has a disease-modifying effect on AATD progression is weekly IV infusion of plasma-derived AAT, which aims to delay the progression of emphysema, 19 , 20 reduce exacerbation frequency, 21 and improve patient quality of life. 5 , 22 Further information on the current treatment recommendations for AATD can be found within the chapter of this review series authored by Barjaktarevic and Campos. 4 However, not all countries have accepted AAT therapy.…”

Section: Current Treatment and Unmet Needsmentioning

confidence: 99%

“…At present, IV AAT therapy regimens can be inconvenient for patients due to the need for lifetime weekly infusions. 5 Moreover, this therapy addresses only AATD-associated lung disease and does not treat extra-pulmonary manifestations such as liver disease, which is discussed in detail within the chapter by Patel and Teckman. 26 Therefore, new cost-effective treatments are being sought that would not only be more convenient for patients, but also address the underlying genetic component of AATD, rather than simply slowing disease progression and reducing symptom severity.…”

Section: Current Treatment and Unmet Needsmentioning

confidence: 99%

“… 46 Since then, little has been reported on aerosolized AAT therapy in patients with AATD, and IV AAT therapy has been the standard method of administration. However, as we are now looking to further improve the lives of patients with AATD, 5 inhalation therapy is gaining more momentum.…”

Section: Research and Future Treatmentsmentioning

confidence: 99%

“…3 , 4 Therefore, patients with AATD have substantial unmet needs and several new on-going developments are focusing on reducing patient inconvenience, while providing efficacy in decreasing morbidity and mortality. 5 Following issues raised by patients on the need for a global registry, further information on comorbidities, natural history of the disease, risk factors for progression and the poor prognosis of lung disease, the European Alpha-1 Research Collaboration (EARCO) was created. 6 The EARCO is a Clinical Research Collaboration of the European Respiratory Society, which will collaborate with the European Reference Network (ERN) for respiratory diseases (ERN-LUNG), the ERN for hepatological diseases (ERN RARE-LIVER), and the registry of the Alpha-1 Liver Group.…”

Section: Introductionmentioning

confidence: 99%

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Alpha-1 antitrypsin deficiency research and emerging treatment strategies: what’s down the road?

Rahaghi

2021

Therapeutic Advances in Chronic Disease

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Intravenous infusion of alpha-1 antitrypsin (AAT) was approved by the United States Food and Drug Administration (FDA) to treat emphysema associated with AAT deficiency (AATD) in 1987 and there are now several FDA-approved therapy products on the market, all of which are derived from pooled human plasma. Intravenous AAT therapy has proven clinical efficacy in slowing the decline of lung function associated with AATD progression; however, it is only recommended for individuals with the most severe forms of AATD as there is a lack of evidence that this treatment is effective in treating wild-type heterozygotes (e.g., PI*MS and PI*MZ genotypes), for which the prevalence may be much higher than previously thought. There are large numbers of individuals that are currently left untreated despite displaying symptoms of AATD. Furthermore, not all countries offer AAT augmentation therapy due to its expense and inconvenience for patients. More cost-effective treatments are now being sought that show efficacy for less severe forms of AATD and many new therapeutic technologies are being investigated, such as gene repair and other interference strategies, as well as the use of chemical chaperones. New sources of AAT are also being investigated to ensure there are enough supplies to meet future demand, and new methods of assessing response to treatment are being evaluated. There is currently extensive research into AATD and its treatment, and this chapter aims to highlight important emerging treatment strategies that aim to improve the lives of patients with AATD.

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Section: Current Treatment and Unmet Needsmentioning

confidence: 99%

Section: Current Treatment and Unmet Needsmentioning

confidence: 99%

Section: Research and Future Treatmentsmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

See 2 more Smart Citations

Alpha-1 antitrypsin deficiency research and emerging treatment strategies: what’s down the road?

Rahaghi

2021

Therapeutic Advances in Chronic Disease

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show abstract

“…13 AAT therapy is administered as a weekly IV infusion by a healthcare professional (HCP) in an outpatient clinic or practice 14 and with appropriate training, is licensed for self-administration. 8,15 However, the need for weekly hospital visits may be a burden to some patients and has been made more challenging by the COVID-19 pandemic. The pandemic has challenged the effective management of patients with AATD in many countries across healthcare settings.…”

Section: Introductionmentioning

confidence: 99%