Efficacy and Safety of Omalizumab Treatment Over a 16-Year Follow-Up: When a Clinical Trial Meets Real-Life

Figures & data

Table 1 Demographics

No. of patients	8
Age at baseline, mean ± SD	42.6 ± 9.1
Males, n (%)	5 (62.5)
Former smokers, n (%)	1 (12.5)
Comorbidities, n (%)
Atopy	8 (100)
Multi-sensitivity	3 (37)
Rhinitis	5 (62)
Nasal polyposis	4 (50)
Dermatitis	3 (37)

Abbreviation: SD, standard deviation.

Table 2 Clinical, Functional and Laboratory Indexes Variations

	Baseline	32 Weeks	4 Years	9 Years	16 Years	p value
ACT, mean ± SD	12.6 ± 2.2	15.4 ± 3.1	17.5 ± 5	21.4 ± 3.1	21.5 ± 2.6	0.0002
AQLQ, mean ± SD	2.9 ± 0.5	4.2 ± 0.5	5.1 ± 0.9	5.8 ± 0.8	5.9 ± 0.6	<0.0001
Hospital admissions (n/patient/year), mean ± SD	0.3 ± 0.5	0 ± 0	0 ± 0	0 ± 0	0 ± 0	0.08
Severe exacerbations (n/patient/year), mean ± SD	3.6 ± 2.1	0.1 ± 0.4	0.1 ± 0.4	0.5 ± 1.1	0 ± 0	<0.0001
Mild-to-moderate exacerbations (n/patient/year), mean ± SD	1.8 ± 1.2	1.1 ± 1.1	0.6 ± 0.7	0.4 ± 0.7	0.4 ± 0.7	0.054
FEV1/FVC (%), mean ± SD	61.9 ± 10.4	67 ± 7.9	72.7 ± 11.1	70 ± 8.2	69.7 ± 7.2	0.24
FEV1 (%), mean ± SD	54.8 ± 13.5	72.8 ± 21.4	76.6 ± 13.3	83.6 ± 18.8	83.1 ± 14.9	0.02
Total IgE (IU/mL), mean ± SD	386.9 ± 196.5	285 ± 201.7	959.5 ± 1018	614.4 ± 448.5	641.3 ± 383.9	0.14

Abbreviations: ACT, asthma control test; SD, standard deviation; AQLQ, asthma quality of life questionnaire; FEV₁, forced expiratory volume in 1 second; FEV₁/FVC, forced expiratory volume in 1 second/forced vital capacity; IgE, immunoglobulin E.

Figure 1 (A) hospital admission variation during omalizumab therapy. (B) severe exacerbations variation during omalizumab therapy. (C) mild-to-moderate exacerbations variation during omalizumab therapy.

Figure 2 (A) FEV₁/FVC (%) variation during omalizumab therapy. (B FEV₁ (%) variation during omalizumab therapy. (C) total IgE levels (IU/mL) variation during omalizumab therapy.

Abbreviation: FEV₁, forced expiratory volume in 1 second; FEV₁/FVC, forced expiratory volume in 1 second/forced vital capacity; IgE, immunoglobulin E.

Figure 3 (A) ACT variation during omalizumab therapy. (B) AQLQ variation during omalizumab therapy.

Abbreviation: ACT, asthma control test; AQLQ, asthma quality of life questionnaire.

Table 3 Drugs Variation

Medication	Baseline	32 Weeks	4 Years	9 Years	16 Years	p value
ICS
High dose	8	2	2	1	4	0.031
Medium dose	0	6	5	2	4	0.018
Low dose	0	0	1	5	0	0.001
OCS	7	1	0	1	0	<0.0001
LABAs	8	8	8	7	7	0.53
LAMAs	5	2	2	2	2	0.39
LTRAs	4	1	0	2	3	0.15
Theophylline	1	0	0	0	0	0.39
SABAs	8	7	3	2	2	0.002

Abbreviations: ICS, inhaled corticosteroids; OCS, oral corticosteroids; LABAs, long-acting beta-agonists; LAMAs, long-acting muscarinic antagonists; LTRAs, leukotriene receptor antagonists; SABAs, short-acting beta agonists.

Table 4 Drugs Dosage Variation

Medication	Baseline	32 Weeks	4 Years	9 Years	16 Years	p value
ICS (mcg/day), mean ± SD	920 ± 425.5	780 ± 367.2	680 ± 175.6	710 ± 417.7	1100 ± 464	0.47
OCS (mg/day), mean ± SD	13.1 ± 10.9	1.3 ± 2.3	1.6 ± 1.8	0 ± 0	0 ± 0	0.003
SABAs (mcg/day), mean ± SD	137.5 ± 130.2	50 ± 75.6	0 ± 0	0 ± 0	0 ± 0	0.006
LTRAs (mg/day), mean ± SD	6.3 ± 7.4	2.5 ± 4.6	1.3 ± 3.5	3.1 ± 4.6	3.8 ± 5.2	0.52

Abbreviations: ICS, inhaled corticosteroids; SD, standard deviation; OCS, oral corticosteroids; SABAs, short-acting beta agonists; LTRAs, leukotriene receptor antagonists.

Figure 4 (A) ICS equivalent dosage variation during omalizumab therapy. (B) OCS equivalent dosage during omalizumab therapy.

Abbreviation: ICS, inhaled corticosteroids; OCS, oral corticosteroids.