Long-term efficacy and safety of α1 proteinase inhibitor treatment for emphysema caused by severe α1 antitrypsin deficiency: an open-label extension trial (RAPID-OLE) -

Long-term efficacy and safety of α1 proteinase inhibitor treatment for emphysema caused by severe α1 antitrypsin deficiency: an open-label extension trial (RAPID-OLE)
Information type: Other primary research
Source: The Lancet Respiratory Medicine
Specialities: Respiratory disorders
Summary
2-year open-label extension of RAPID-RCT trial (n=140) supports continued efficacy of purified α1 proteinase inhibitor (Respreeza or Zemaira) in slowing disease progression over 4 years of treatment. Lost lung density was never recovered, highlighting need for early intervention.
UKMi comment
In UK, Respreeza is licensed for maintenance treatment, to slow the progression of emphysema in adults with documented severe alpha1-proteinase inhibitor deficiency (e.g. genotypes PiZZ, PiZ(null), Pi(null,null), PiSZ).
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