Retrospective Database Analysis of Liver-Related Clinical Events in Adult and Pediatric Patients with Alpha-1 Antitrypsin Deficiency in the United States

Figures & data

Figure 1 Patient Disposition.

Abbreviations: AATD, alpha-1 antitrypsin deficiency; AEMR, Ambulatory Electronic Medical Records; ICD-9-CM, International Classification of Diseases, Ninth Revision, Clinical Modification; ICD-10-CM, International Classification of Diseases, Tenth Revision, Clinical Modification; Pi, protease inhibitor; SNOMED, Systematized Nomenclature of Medicine.

Table 1 Patient Demographics and Characteristics

	AATD with Liver Disease				AATD with Both Liver and Lung Disease
	Patients Aged <1 Year (n=41)	Patients Aged 1–5 Years (n=24)	Patients Aged 6–17 Years (n=58)	Patients Aged ≥18 Years (n=771)	Patients Aged ≥18 Years (n=541)
Age, years, median (IQR)	0 (0–0)	3 (2–5)	14 (10–16)	49 (38–57)	55 (46–61)
Sex, male, n (%)	25 (61)	13 (54)	36 (62)	451 (58)	275 (51)
Time in health state, months, median (IQR)^a	37 (22.6–55)	22 (9.0–36)	36 (15.0–60)	22 (10–40)	21 (10–38)
Most common comorbidities, n (%)^b	N/A^c
Asthma		2 (8)	8 (14)	62 (8)	90 (17)
Diabetes mellitus		0	2 (3)	107 (14)	107 (20)
Hyperlipidemia		0	3 (5)	208 (27)	166 (31)
Hypertension		2 (8)	2 (3)	221 (29)	217 (40)
Liver cirrhosis		0	0	15 (2)	60 (11)
Liver/gall bladder/pancreatic disease		1 (4)	0	80 (10)	132 (24)
Obesity		1 (4)	8 (14)	124 (16)	129 (24)
Sleep disorders		0	2 (3)	97 (13)	100 (18)
Study liver disease definition^d		0	0	0	93 (17)
Study lung disease definition^d		1 (4)	4 (7)	0	127 (23)
Charlson Comorbidity Index, n (%)	N/A^c
0		21 (88)	48 (83)	515 (67)	191 (35)
1		2 (8)	8 (14)	138 (18)	162 (30)
2		0	1 (2)	73 (10)	74 (14)
3		0	0	30 (4)	52 (10)
≥4		1 (4)	1 (2)	15 (2)	62 (11)
Payer type, n (%)
Commercial	25 (61)	12 (50)	42 (72)	511 (66)	353 (65)
Self-insured	12 (29)	9 (38)	13 (22)	224 (29)	151 (28)
Medicare risk	0	0	0	21 (3)	25 (5)
Medicaid	4 (10)	3 (13)	3 (5)	12 (2)	8 (1)
Other/unknown	0	0	0	3 (<1)	4 (<1)
Geographic region of the USA, n (%)
South	13 (32)	10 (42)	22 (38)	341 (44)	272 (50)
Midwest	13 (32)	10 (42)	16 (28)	187 (24)	129 (24)
Northeast	8 (20)	2 (8)	12 (21)	147 (19)	76 (14)
West	7 (17)	2 (8)	7 (12)	86 (11)	57 (11)
Unknown/missing	0	0	1 (2)	10 (1)	7 (1)

Notes: ^aDifference in time from first lung disease diagnosis (after liver disease diagnosis), or first liver disease diagnosis (after lung disease diagnosis), or first liver or first lung disease diagnosis (if within 90 days of each other) to last day of continuous enrollment period. ^bPresent in ≥10% of patients in at least one of the subgroups. ^cPatients <1 year were indexed at age 0 years and therefore did not have a 6-month baseline period for the collection of clinical characteristics. ^dClinician determined specific liver/lung disease diagnosis codes as AATD-specific; separately reported comorbidities may include broader definitions (eg alcoholic liver cirrhosis).

Abbreviations: AATD, alpha-1 antitrypsin deficiency; IQR, interquartile range; N/A, not available.

Figure 2 Liver-Related Clinical Event Rates per Person-Year in (A) Adult Patients (Aged ≥18 Years)^a and (B) Pediatric Patients^{b. a}The sum of patients in each health state is higher than the number of patients included in the study owing to patients contributing time in more than one health state during the study, according to their disease status. ^bData are reported for pediatric patients (stratified by age group [<1 year, 1–5 years and 6–17 years]) with AATD with liver disease. Number of events were calculated per person-year (365.25 days); composite data (≥1 liver-related clinical event) are shown for any patient with >1 clinical event.

Abbreviation: AATD, alpha-1 antitrypsin deficiency.

Figure 3 Median Time to First Liver-Related Clinical Event in (A) Adult Patients (Aged ≥18 Years) and (B) Pediatric Patients^{a. a}Data are reported for pediatric patients (stratified by age group [<1 year, 1–5 years and 6–17 years]) with AATD with liver disease. Time to first liver-related clinical event was reported from the index date. Patients with ≥1 clinical event are presented in the composite data.

Abbreviation: AATD, alpha-1 antitrypsin deficiency.

Data Sharing Statement

The original de-identified data used in this analysis were obtained from and are the property of IQVIA. IQVIA has restrictions prohibiting the authors from making the data set publicly available. Interested researchers may contact IQVIA to apply to gain access to the study’s data in the same way the authors obtained the data (see https://www.iqvia.com/contact/sf).