CHEST
Clinical InvestigationsLongitudinal Follow-up of Patients With α1-Protease Inhibitor Deficiency Before and During Therapy With IV α1-Protease Inhibitor
Section snippets
Materials and Methods
For this cohort study, we used one database that contained lungdata before the institution of α1-Pi therapyand a second database of a phase IV drug surveillance study.
Results
A total of 96 patients (62 males and 34 females, p = 0.004) wereavailable for analysis. The mean time of observation was 47.5 ± 28.1months (range, 12.2 to 148.3 months) before augmentation therapy and50.2 ± 30.2 months (range, 12 to 148.6 months) during the treatmentperiod. The mean total observation time was 98.9 ± 36.6 months(range, 24.9 to 196.3 months). Twenty-eight patients had an observationperiod > 10 years.
The majority of patients had PiZ phenotypes, and the frequencies of thephenotypes
Discussion
This study is the first intraindividual studycomparing lung function decline in 96 patients withα1-Pi deficiency before they receivedaugmentation therapy to a period of time in which they received weeklyIV augmentation therapy. There was a significantly lower decline inFEV1 during the period patients received IVaugmentation therapy compared to the period with out specific treatment. The overall reduction of the decline in FEV1 from49.2 to 34.3 mL/yr by 15 mL/yr is similar to the reduction
Additional Participants of the WATL Group for α1-PiDeficiency
R. Loddenkemper, N. Schönfeld, Berlin; E. Kaukel, R.D. Staud, Hamburg; G. Kanzow, Großhansdorf; K.J. Wiemann, Bad Schwartau; J. Braun, Lübeck; U. Lepp, Borstel; N. de Wall, Sande; K. Eberhardt, Bremen; H. Fabel, Hannover; G. Goeckenjan, Immenhausen; R. Kappes, Duesseldorf; E.W. Schmidt, Bochum; H. Steveling, Essen; J. Lorenz, J. Schlegel, W. Schmidt, Mainz; R. Buhl, J. Bargon, Frankfurt;V. Schulz, Heidelberg; R. Dierkesmann, Gerlingen; J.C. Virchow Jr.,Freiburg; M. Schwaiblmair, R.W. Hauck,
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Cited by (0)
Dr. Wencker was supported in part by Bayer Corporation, by aneducational grant given in conjunction with a clinical trial of inhaledα1-protease inhibitor and not related to this analysis.
Dr. Banik was employed by Bayer Vital as astatistician.
Significant portions of this article are part of a doctoral thesis byDr. Fuhrmann.
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A complete list of participants is located in the Appendix.