Alpha-1-antitrypsin augmentation therapy in deficient individuals enrolled in the Alpha-1 Foundation DNA and Tissue Bank

Figures & data

Figure 1 Flow diagram of study population analysis.

Table 1 Comparative table of the patients receiving versus not receiving augmentation therapy

	Augmented group	Non-augmented group	P
n (%)	124 (76%)	40 (24%)
Age in yearsa	61.3 (0.7)	56.1 (1.9)	0.01
Age at diagnosis of AATDa	46.4 (0.8)	44.5 (1.8)	0.32
% Female gender	52.4%	50%	0.26
% White race	95.2	90%	0.58
% Ex-smokers	84.7%	62.5%	<0.001
% Current smokers	0%	5%	0.06
Number of years of smokinga	19.4 (0.9)	17.3 (1.4)	0.2
Exposure to dust or fumes at work	56.5%	45%	0.21
Dyspnea	91.9%	75%	0.004
Asthma	25%	10%	0.04
COPD (emphysema)	37.1%	15%	0.009
Bronchiectasis	12.1%	5%	0.25
Pneumonia	21%	12.5%	0.23
Pneumothorax	3.2%	2.5%	1
Hepatitis	2.4%	7.5%	0.16
Cirrhosis	0%	5%	0.06
FHx of AATD	63.7%	67.5%	0.66
FHx of emphysema	40.3%	47.5%	0.42
Inhaled bronchodilator	86.3%	55%	<0.001
Inhaled corticosteroid	59.7%	45%	0.1
Inhaled anticholinergics	6.5%	0%	0.2
Oral bronchodilators	11.3%	5%	0.24
Systemic corticosteroids	7.3%	2.5%	0.3
Theophylline	10.5%	5%	0.3
Oxygen therapy	50.8%	12.5%	<0.001
Baseline FEV1 (L/m)a	1.4 (0.1)	2.4 (0.2)	<0.001
Baseline FEV1 (% of predicted)a	43 (2)	77 (5)	<0.001
Duration of follow-up (months)a	43.6 (3.2)	35.7 (3.2)	0.19
Number of spirometriesb	2	2	0.88
1st spirometry after 2004c	50%	57.5%	0.41
Deceased patients	8 (6.4%)	1 (2.5%)	0.46

Abbreviation: AATD, alpha-1 antitrypsin deficiency; FHx, family history.

Notes:

a Continuous values are provided as mean (± SE);

b Median number of spirometries;

c Percentage of patients in whom the first spirometry was performed after January 1, 2004.

Table 2 Annual ΔFEV₁ for the augmented and the non-augmented groups

Stratification	Groups				P-value
	Augmented		Non-augmented
	n	ΔFEV1 (mL/year)	n	ΔFEV1 (mL/year)
Initial FEV1
<30% pred	30	0.86 (17.63)	3	20.10 (31.06)	0.59
30%–65% pred	79	2.08 (24.03)	10	−51.92 (18.14)	0.07
>65% pred	15	−108.7 (17.29)	27	−29.24 (15.29)	0.0006
Initial FEV1
<50% pred	88	38.30 (33.65)	8	−86.73 (45.37)	0.026
≥50% pred	36	−49.46 (15.88)	32	−32.78 (13.15)	0.42
Smoking
Non-smokers	19	−25.06 (49.53)	15	−38.43 (12.12)	0.79
Ex-smokers	105	24.24 (25.72)	23	−41.19 (22.47)	0.05
Current smokers	0	–	2	–	–
Total	124	10.61 (21.37)	40	−36.96 (12.13)	0.05

Notes: Results shown in the parentheses are standard errors of the estimated change in FEV₁. A positive ΔFEV₁ represents an increase in FEV₁, and a negative ΔFEV₁ corresponds to a decrease in FEV₁.

Figure 2 ΔFEV₁ (mL/year) in augmented and non-augmented patients by initial FEV₁ % of predicted (>65%, 30% to 65% and <30%). *P = 0.0006, **P = 0.07.

Figure 3 ΔFEV₁ (mL/year) in augmented and non-augmented patients by initial FEV₁ % of predicted (<50% and ≥50%).*P = 0.026.

Figure 4 ΔFEV₁ (mL/year) in augmented and non-augmented patients by smoking status *P = 0.05.