Lung Transplantation in Alpha-1-Antitrypsin Deficiency.
BACKGROUND
Lung transplantation is a therapeutic option for patients with end-stage lung disease and a survival benefit has been described in patients with alpha-1-antitrypsin deficiency (A1ATD). The aims of the current study were to determine the survival and health benefits of lung transplantation in UK patients with A1ATD compared to carefully matched non-transplant patients.


METHODS
Patients with the PiZZ (alpha-1-antitrypsin deficiency) genotype who had undergone lung transplantation between 1996 and 2011 were identified from the UK A1ATD registry. Lung physiology, health status and survival were compared pre- and post-transplant using carefully matched non-transplant patients.


RESULTS
Thirty-two A1ATD patients who had undergone lung transplant were identified. Lung function decline pre-transplant was not different to the closely matched non-transplanted cohort. The transplant group pre-transplant, although matched for FEV1, had lower gas transfer measurements, (mean KCO% predicted 41.0% SE ± 3.86 vs 55.6% SE ± 3.10 p < 0.001) and worse health status (SGRQ mean score 64.2 SE ± 2.5 vs 55.3 SE ± 2.0, p < 0.001). Post-transplant, physiology and health status improved significantly (p < 0.002). However, the post-operative mortality over 5 years was no better than for a second group of non-transplant patients further matched for gas transfer or a third group also matched for SGRQ.


CONCLUSIONS
Patients who underwent lung transplant had lower gas transfer and quality-of-life pre-transplant compared to non-transplant patients matched for FEV1, age and sex, suggesting that these parameters provide extra information helpful in decision making. Lung transplantation for A1ATD patients significantly improves quality-of-life but not survival.
a 1 ADAPT Project, Lung Function and Sleep Department, Queen Elizabeth Hospital Birmingham , Edgbaston , Birmingham , United Kingdom.|b 2 Heart and Lung Transplant Unit, Queen Elizabeth Hospital Birmingham , Edgbaston , Birmingham , United Kingdom.
Helen Marie
HM
Stone
a 1 ADAPT Project, Lung Function and Sleep Department, Queen Elizabeth Hospital Birmingham , Edgbaston , Birmingham , United Kingdom.|b 2 Heart and Lung Transplant Unit, Queen Elizabeth Hospital Birmingham , Edgbaston , Birmingham , United Kingdom.
Ross Gareth
RG
Edgar
b 2 Heart and Lung Transplant Unit, Queen Elizabeth Hospital Birmingham , Edgbaston , Birmingham , United Kingdom.
Richard Damian
RD
Thompson
a 1 ADAPT Project, Lung Function and Sleep Department, Queen Elizabeth Hospital Birmingham , Edgbaston , Birmingham , United Kingdom.
Robert Andrew
RA
Stockley
D003657:Decision Making; D005260:Female; D005500:Follow-Up Studies; D006304:Health Status; D006801:Humans; D016040:Lung Transplantation; D008297:Male; D008875:Middle Aged; D018579:Patient Selection; D011379:Prognosis; D011788:Quality of Life; D012042:Registries; D012129:Respiratory Function Tests; D012131:Respiratory Insufficiency / Q000209:etiology / Q000401:mortality / Q000601:surgery*; D012189:Retrospective Studies; D015996:Survival Rate / Q000639:trends; D006113:United Kingdom / Q000453:epidemiology; D019896:alpha 1-Antitrypsin Deficiency / Q000150:complications* / Q000401:mortality
Alpha-1-antitrypsin deficiency; health-related quality of life; lung transplant; survival